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Meet the School of Medicine’s 2023 Distinguished Professors – Duke University School of Medicine

This spring, 27 faculty members in the Duke University School of Medicine have been awarded distinguished professorships. The new distinguished professors were honored at the annual distinguished professorship event on May 4.

In total, Duke University awarded distinguished professorships to 44 faculty members from seven Duke colleges and schools this year.

Distinguished professorships are awarded to faculty who have demonstrated extraordinary scholarship in advancing science and improving human health.

The 2023 recipients from the School of Medicine are:

Peter Allen, MD

Peter Allen, MD, is a professor of surgery. An accomplished surgeon who specializes in treating disorders of the pancreas, liver, bile ducts, stomach, and adrenal glands, he serves as chief of the Division of Surgical Oncology. Allen is known for his multidisciplinary approach to research and patient care, particularly for those with complex issues involving cancer. In addition to his patient care and research accomplishments, he is a superb medical educator and mentor. His reputation as a leader in his field serving as an author, editorial board member and visiting lecturer has elevated the Duke surgical oncology program.

Michael Bagnat, PhD

Michael Bagnat, PhD, is a professor of cell biology. He is a highly productive scientist who creatively combines tools and approaches from the fields of cell biology, developmental biology, physiology, genetics, biophysics, and computational modeling. His research has provided insight into the molecular physiology of fundamental processes in the gut and other organ systems. His studies of intestinal development and physiology have laid the groundwork for identifying modifiers of intestinal inflammation, and potential drivers of human inflammatory bowel disease. He has an exceptional reputation as a mentor and teacher.

Richard Bedlack, MD, PhD

Richard Bedlack, MD, PhD, is a professor of neurology. A leader in clinical care and research in the field of amyotrophic lateral sclerosis (ALS), he founded and developed the ALS clinic at Duke, which has become one of the nations largest and most comprehensive multi-disciplinary ALS care centers. He has spent nearly two decades studying and collaborating on epidemiologic, genetic, and interventional ALS research trials. He built an international research program called ALSU Entangled that uses social networking and crowd sourcing to review alternative therapies for ALS. He has also been an important advocate for military personnel with an increased risk of ALS, and he created a VA Cooperative Studies Program to study feasibility of a brain computer interface for veterans with ALS.

Michael Bolognesi, MD

Michael Bolognesi, MD, is a professor of orthopaedic surgery. As chief of the adult reconstruction service, he has used his academic and organizational leadership to make a significant impact on the field of orthopaedics and joint reconstruction in adults. His research is focused on improving clinical outcomes, implant survivorship, the biology of hip and knee arthritis, and cost-effectiveness. He leads the adult reconstruction fellowship program, and in 2019, he served as the 29th president of the American Association of Hip and Knee Surgeons.

Louis DeFrate, ScD

Louis DeFrate, ScD, is a professor of orthopaedic surgery and biomedical engineering. He studies anterior cruciate ligament (ACL) mechanics and has used advanced radiographic and MR imaging to investigate soft tissue structure, composition, and function, and to improve outcomes in ACL repair. DeFrate is vice chair for biomechanics, movement, and imaging research and the director of the K-lab. He employs innovative methods using motion capture, MRI, and biplanar radiography to study the knee. The American Academy of Orthopaedic Surgeons awarded DeFrate its Kappa Delta Young Investigator Award.

Cagla Eroglu, PhD

Cagla Eroglu, PhD, is a professor of cell biology and neurobiology. She studies the development of synaptic connectivity in the mammalian brain. Her focus has been on how glial cells called astrocytes affect synaptogenesis. As a postdoctoral fellow, she discovered the receptor for astrocyte-derived Thrombospondin on neurons, published in a landmark 2009 paper. The mechanisms her lab has uncovered for how specific molecules function have set new paradigms in the field. She is considered a leader in this rapidly growing area and has published numerous significant and high-impact papers.

Daniel George, MD

Daniel George, MD, is a professor of medicine and a professor in surgery. Since 2003 he has led the genitourinary section of the Duke Division of Medical Oncology, and he leads Dukes participation in the Department of Defense Prostate Cancer Clinical Trials Consortium. His research leading and collaborating on clinical trials of tyrosine kinase inhibitors, anti-androgen therapy, chemotherapy, and immunotherapy has led to advances in the treatment of patients with kidney cancer and prostate cancer. He is vice dean of diversity and equity in the Division of Medical Oncology, and he conducts clinical trials aimed at understanding and addressing the disproportionately poor outcome of Black men with prostate cancer.

Christopher Granger, MD

Christopher Granger, MD, is a professor of medicine and a professor in the School of Nursing. He has conducted practice-changing clinical research for over 30 years in cardiovascular medicine, leading a number of large international clinical studies of heart attacks, unstable angina, heart failure, and atrial fibrillation. He has also led clinical studies of blood thinners and coronary intervention for heart attacks, stroke prevention in atrial fibrillation, and prevention of heart attack for patients with coronary artery disease. He serves as the chairman of the American Heart Association Mission: Lifeline program to improve heart attack care nationally as well as the American College of Cardiology/American Heart Association guideline committee for heart attack care.

Gerald Grant, MD

Gerald Grant, MD, is a professor and chair of the Department of Neurosurgery and a professor in neurobiology. He is an internationally recognized pediatric neurosurgeon and surgeon-scientist who focuses on two critical areas: the biological function of the blood-brain barrier (BBB) and mechanisms involved in recovery from brain injury. He studies the unique features of the BBB surrounding brain tumors at the molecular and functional level. His research focuses on innovative ways to open the BBB to improve the delivery of novel drugs and immunotherapy to target brain tumors.

Matthias Gromeier, MD

Matthias Gromeier, MD, is a professor of neurosurgery. He has dedicated his career to unraveling RNA virus/host relations and devising methods of exploiting them for cancer immunotherapy and vaccine design. He has applied his discoveries to design an attenuated poliovirus to activate the immune system to target glioblastoma and other cancers. In addition, his lab has conducted mechanistic studies showing how the attenuated poliovirus stimulates presentation of tumor antigens and antigen-presenting cells.

Rana Gupta, PhD

Rana Gupta, PhD, is a professor in medicine in the Division of Endocrinology, Metabolism, and Nutrition, and a professor in cell biology. He is internationally known for his expertise in metabolic regulation and adipose tissue biology. His discoveries of genetic factors and specialized subpopulations of adipose tissue precursor cells open the door to translational implications in metabolic diseases. He has received honors including the Searle Scholar Award, the Hartwell Foundation Individual Biomedical Research Award, and the Richard E. Weitzman Outstanding Early Career Investigator Award from the Endocrine Society.

Susan Halabi, PhD

Susan Halabi, PhD, is a professor of biostatistics and bioinformatics and co-chief of the Division of Biostatistics in the Department of Biostatistics & Bioinformatics. She has been at the forefront of designing and analyzing clinical trials in oncology for over 25 years. She is focused on developing innovative variable selection methods for biomarkers and high dimensional data. Among her key contributions are building and validating prognostic models of outcomes for prostate cancer and identifying surrogate endpoints for overall survival. A past-president of the Society for Clinical Trials and the 2022 recipient of the Janet L. Norward Award, Dr. Halabi is a Fellow of the Society for Clinical Trials, the American Statistical Association, and the American Society for Clinical Oncology.

John Jelovsek, MD

John Jelovsek, MD, is a clinical researcher and professor of obstetrics and gynecology. He also serves as the director of data science for womens health in the Department of Obstetrics & Gynecology. He is an expert in developing and validating individualized patient-centered prediction tools that improve patient and clinician decision-making with contracting and possibly preventing pelvic floor disorders after childbirth. One such tool, which predicts urinary incontinence after pelvic organ prolapse surgery, has been incorporated into the American Urogynecologic Societys mobile app for clinical use and is now used by surgeons across the world.

Eric Laber, PhD

Eric Laber, PhD, is a professor of statistical science, biostatistics and bioinformatics, and a research professor of global health. He is an expert in data-driven decision making at the intersection of biostatistics, statistics, and machine learning, with applications in clinical trials, precision medicine, and experimental design. This field focuses on improving algorithms for solving sequential decision problems, with the ultimate goal of improving outcomes across diverse fields. He has also made significant contributions in the field of personalized medicine, contributing fundamental statistical methodology that has helped facilitate rapid advances in the field. He is the recipient of numerous prestigious awards and honors.

Seok-Yong Lee, PhD

Seok-Yong Lee, PhD, is a professor in biochemistry and cell biology. As a membrane structural biologist, he has developed advanced tools in X-ray crystallography, electron microscopy, cryogenic electron microscopy (cryo-EM), and electrophysiology and has applied these tools to solve challenging questions about membrane and protein structural biology. He has become a world leader in this field and has made major advancements in three different classes of membrane protein: active transporters, ion channels, and enzymes. He has provided a better understanding of the cold and menthol sensor in mammals and a pain sensor for noxious chemicals. His work has also led to structural drug designs that can improve pharmacological properties.

Jennifer Lodge, PhD

Jennifer Lodge, PhD, is a professor of molecular genetics and microbiology and serves as Duke Universitys vice president for research and innovation. She leads oversight of Dukes annual research portfolio. Lodges research focuses on the human pathogenic fungus Cryptococcus neoformans, exploring the biochemical processes by which this fungus builds its cell walls. Her work has brought the field of molecular pathogenesis for cryptococcosis from its infancy to one of the true model fungal pathogens for worldwide study. Lodge is a fellow of the American Academy of Microbiology, the American Association for the Advancement of Science, and the National Academy of Inventors.

Edward Miao, MD, PhD

Edward Miao, MD, PhD, is a professor of immunology, molecular genetics and microbiology, cell biology, and pathology. He also serves as vice chair for equity, diversity, and inclusion in the Department of Integrative Immunobiology. He studies mechanisms of cell death that play critical roles in the immune response to bacterial infection. He is expanding the medical knowledge base of the fundamental interactions between the immune system and a range of invaders. His lab has pioneered the use of environmental bacteria with pathogenic potential that have not adapted to evade mammalian immunity and comparing these to bona fide human pathogens that are able to hide from mammalian immune defenses.

David Page, PhD

David Page, PhD, is professor and chair of the Department of Biostatistics and Bioinformatics and a professor of computer science. A highly respected computer scientist whose work is primarily focused on artificial intelligence and machine learning, Page is recognized for his pioneering efforts to use machine learning in biomedical applications, including the first application of dynamic Bayesian network learning to time-series gene expression as a means to better learn how genes cause other gene expressions to change. Over his career, Page has received numerous awards and is a member of several NIH review committees. He was inducted into the American College of Medical Informatics in 2021.

Steven Patierno, PhD

Steven Patierno, PhD, is a professor of medicine and pharmacology and cancer biology. He is also a professor in family medicine and community health and deputy director of Duke Cancer Institute. Patierno is a renowned cancer researcher with training in molecular oncology and pharmacology and expertise in lung, breast, and prostate cancer. His work to develop innovative interventions for mitigating health disparities in the cancer patient population is well-recognized locally and nationally. His funding track record includes multiple R01 grants as principal investigator and co-principal investigator. Patiernos numerous awards and honors include the AACR Distinguished Science of Cancer Disparities Research Award and the Duke University Health System Diversity and Inclusion Award.

John Rawls, PhD

John Rawls, PhD, is a professor of molecular genetics and microbiology and cell biology. He also is a professor in medicine. Rawls studies the influence of the gut microbiome on vertebrate host physiology and is a world leader in using the tractable zebrafish model for such studies. He has used both zebrafish and mouse systems to yield insights about host-microbe interactions relevant to development, homeostasis, metabolism, and disease. The work impacts many areas, including gut motility, fat absorption, effects of diet, visceral adipose tissue, obesity, gut immune responses, diabetes, and neural development.

Danny Schust, MD

Danny Schust, MD, is a professor and vice chair for research in the Department of Obstetrics and Gynecology. He is a national and international leader and physician scientist in obstetrics and gynecology and is renowned in his field of reproductive endocrinology and infertility. He is widely recognized for his work in areas of early pregnancy, recurrent pregnancy loss, early placental development, reproductive infectious diseases, and the immunology of the human maternal-fetal interface. His research interests center on understanding human placental development, both normal and dysfunctional. His lab uses 2D and 3D stem cell-derived models to study human implantation and placentation.

Svati Shah, MD, MHS

Svati Shah, MD, MHS, is a professor of medicine and of biostatistics and bioinformatics. She is the associate dean of genomics and director of the Duke Precision Collaboratory, as well as vice chief of translational research and director of the Adult Cardiovascular Genetics Clinic in the Division of Cardiology. She brings expertise in biostatistics, bioinformatics, genetics, translational biology, and molecular discovery tools to her research into genetic and metabolic pathways in cardiovascular diseases. She has advanced both the understanding of underlying genetic risk for atherosclerotic disease and the biologic metabolic underpinnings of obesity and heart failure. Currently, her lab studies metabolic and genetic pathways of cardiometabolic diseases, integrating diverse genomic, metabolomic and proteomic techniques for identification of novel mechanisms of disease and biomarkers.

Beth Sullivan, PhD

Beth Sullivan, PhD, is a professor of molecular genetics and microbiology, professor of cell biology, and associate dean of research training. She studies epigenetic and genetic mechanisms of centromeres, specialized chromosomal sites involved in chromosome architecture and movement, kinetochore function, heterochromatin assembly, and sister chromatid cohesion. Dysregulation of chromosomal segregation underlies many human genetic disorders. Among Sullivans major accomplishments have been to define the functions and roles of telomeres. She has made important discoveries regarding the functions of centromeres, mechanisms of chromosomal segregation, and has contributed to the final full sequence of the human genome.

Shyni Varghese, PhD

Shyni Varghese, PhD, is a professor of orthopaedic surgery and a professor in the Departments of Biomedical Engineering and Mechanical Engineering and Materials Science. She is a biomedical engineer whose research addresses musculoskeletal tissue repair, disease biophysics, and organ-on-a-chip technologies. Her lab seeks to understand theeffect of physicochemical cues of the microenvironment on cellular behaviors leading to stem cell commitment, tissue repair and homeostasis, or disease progression. Her research has provided deep insights into how extracellular matrix interactions govern tissue regeneration in musculoskeletal systems and disease progression in the context of fibrosis.

Anthony Viera, MD

Anthony Viera, MD, is professor and chair of the Department of Family Medicine and Community Health and a professor in population health sciences. His focus is in the areas of cardiovascular disease prevention research, in particular improving detection and control of hypertension, ambulatory blood pressure monitoring, and obesity prevention. His most influential work on masked hypertension and blood pressure monitoring addresses a huge public health problem and has the potential to prevent cardiovascular disease in those that are not currently diagnosed by traditional methods. Much of his scholarly work has focused on using rigorous scientific methods to address fundamental questions in primary care practice as well as educating and mentoring students and trainees to bring data science and clinical investigation to primary care.

Kevin Weinfurt, PhD

Kevin Weinfurt, PhD, is professor and vice chair of research in the Department of Population Health Sciences and a faculty member of the Duke Clinical Research Institute. He is also a professor of psychology and neuroscience, psychiatry and behavioral health, and biostatistics and informatics. He is co-director of the Center for Health Measurement and of the Clinical Research Training Program. He conducts research on measuring patient-reported outcomes, medical decision-making, and bioethics.

Christopher Woods, MD

Christopher Woods, MD, is a professor of medicine, pathology, and global health. He is the executive director of the Hubert/Yeargan Center for Global Health and associate director of the Duke Center for Applied Genomics and Precision Medicine. His research crosses multiple disciplines, including innovative diagnostic platforms, clinical trials, and clinical testing in infectious disease diagnosis. He has used genomic approaches of the host response for diagnosis of infectious disease, which has established him as a leader in diagnostic innovation. His research impact spans the globe and has facilitated projects in 33 countries and studentled projects in 17 countries.

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Meet the School of Medicine's 2023 Distinguished Professors - Duke University School of Medicine

The first fecal transplant pill is heading to pharmacies – Freethink

This article is an installment of Future Explored, a weekly guide to world-changing technology. You can get stories like this one straight to your inbox every Thursday morning bysubscribing here.

The FDA has approved a first-of-its-kind pill, made from human fecal matter, to treat bacterial infections in the gut potentially kicking off an era in which we target the microbiome to treat many other diseases.

This approval is the tipping point for the field, Eric Shaff, president and CEO of Seres Therapeutics, the company that created the pill, told TIME Magazine.

The gut ecosystem: Your digestive tract contains trillions of microbes, known collectively as your gut microbiome. While that might sound creepy, the tiny hitchhikers are mostly harmless and often helpful they help digest food, produce vitamins, aid the immune system, and more.

The composition of the gut microbiome has been linked to everything from depression to cancer.

Your diet, ancestry, medications, and even your social network help determine the composition of your gut microbiome, and if this ecosystem becomes unbalanced theres not enough diversity, or too much/too little of some kind of microbe you can experience health problems.

These problems probably arent just limited to the GI tract, either the gut microbiome has been linked to everything from depression to cancer.

The challenge: Repeated infections with the bacteria C. difficile (C. diff) are one of the most well-known examples of how an imbalanced gut microbiome can affect health.

C. diff infections typically occur in patients whove recently been prescribed antibiotics, because antibiotics can kill off too many bacteria that compete with C. diff for resources. This allows the microbe to overpopulate the gut, causing diarrhea, fever, and nausea.

In severe cases, a C. diff infection can be fatal an estimated 15,000 people die from them in the US every year.

In 2019, a patient died because his fecal transplant contained E. coli.

While another round of different antibiotics may clear up a C. diff infection, the bacteria is very persistent about one in six people diagnosed with an infection will get another one within 2 to 8 weeks.

To help restore balance in the gut, people with severe or recurrent C. diff can undergo a therapy called a fecal transplant, which is exactly what it sounds like: fecal matter donated by someone with a healthy gut is transplanted (usually rectally) into the patient. The idea is that the microbes from the healthy donor can repopulate and restore balance to the patients microbiome, preventing C. diff from growing out of control.

While the fecal matter used for these transplants is typically screened for harmful bacteria and viruses prior to use, some have slipped through the cracks in the past people have gotten sick from fecal transplants, and in 2019, a patient died because his donor stool contained E. coli.

The availability of a fecal microbiota product that can be taken orally is a significant step forward.

In November 2022, the FDA approved the first fecal transplant therapy for C. diff infections that therapy, Rebyota, is sourced from qualified donors and carefully screened, giving patients a safer way to treat their infections.

What Rebyota couldnt give them was an easier way they would still need to receive the fecal transplant rectally in a doctors office.

Whats new? Now, the FDA has approved VOWST, a fecal transplant pill to treat recurrent C. diff infections.

Seres Therapeutics manufactures the pill using screened fecal matter, and the treatment is four capsules once a day for three days in adults who have already received antibiotics for recurrent C. diff infections.

Credit: Seres Therapeutics

In a phase 3 study, 88% of patients given the fecal transplant pill went 8 weeks without a recurrent C. diff infection compared to 60% of those who received a placebo. Six months after the therapy, 79% of the treatment group was still infection-free compared to just 53% of the placebo group.

The availability of a fecal microbiota product that can be taken orally is a significant step forward in advancing patient care and accessibility for individuals who have experienced this disease that can be potentially life-threatening, said Peter Marks, director of the FDAs Center for Biologics Evaluation and Research.

Looking ahead: Seres is already in the middle of a phase 1b study for its next microbiome-targeting pill, SER-155, which is being developed for people whove had organ or stem cell transplants.

It contains a mix of lab-grown bacteria designed to inhibit the proliferation of pathogens, reduce gut inflammation, and modulate the immune system. The hope is that it will prevent antibiotic-resistant infections and graft-versus-host disease (GvHD), a potentially fatal complication in which donated cells attack a recipients body.

If we see traction [with transplant patients], then we think there are opportunities in treating cirrhosis, cancer neutropenia, and other conditions where antimicrobial resistance is a problem, Shaff told TIME.

Well be able to look at a persons microbiome and tell a patient their risk of developing a disease.

Meanwhile, other research groups are developing microbiome-targeting therapies to treat cancer, multiple sclerosis, and even aging itself, while still others are trying to identify biomarkers in the microbiome that could help with disease diagnosis and prevention.

In the future, well be able to look at a persons microbiome and tell a patient their risk of developing a disease, much like we do now with commercially available human gene panels, predicts Purna Kashyap, co-associate director of the Microbiome Program in the Mayo Clinic Center for Individualized Medicine.

This holds promise as a preventive strategy because, unlike our genes, the microbiome can be changed, he continued.

The bottom line: Even before fecal transplants became the standard of care for recurrent C. diff, people were trying to improve the health of their microbiomes with probiotics, gut healthy diets, and more but the efficacy of those approaches has always been questionable.

Only thanks to a recent wave of scientific interest are we now seeing just how important the gut microbiome is to our health and how we can precisely manipulate it to achieve real results the kind good enough to secure FDA approval.

Wed love to hear from you! If you have a comment about this article or if you have a tip for a future Freethink story, please email us attips@freethink.com.

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The first fecal transplant pill is heading to pharmacies - Freethink

A Lifetime of Achievement for an Oncology Nurse – Curetoday.com

Mary Colasuonno, B.S.N., RN, BMTCN, started her career at the City of Hope National Medical Center (COHNMC) more than 30 years ago. COHNMC is a National Cancer Institute-designated health care facility providing care for patients with cancer in Duarte, California. Mary has worked on a bone marrow transplant inpatient unit for all of her nursing career.

Recently she was asked to step in as an interim nurse manager for another hematology inpatient unit. She wholeheartedly accepted and developed close relationships with the nursing staff there over the span of three months.

In her career as a nurse, she has always made a conscious effort to make every interaction count. Mary is always determined to provide positivity to others. In her own words, positivity fosters healing. She is a firm believer that whatever light you can provide to a patient will be what is needed in that moment.

Mary has positively affected patients in so many ways. She has inspired her patients to want to do better not only for themselves but for her as well. Mary recalled a patient she took care of 30 years ago. This patient returns to COHNMC each year for the Bone Marrow Transplant Reunion. At this event, patients celebrate their survivor- ship after having a stem cell transplant at COHNMC. Every year, this patient seeks Mary out to cherish Marys impact on her healing and conquering of cancer.

Marys patients have described her as an angel who was especially appointed to care for them. One patient even recognized her voice in a dark room during a nurse shift handoff. This patient was overwhelmed with sadness when she learned that her cancer was no longer treatable. Mary helped this patient cope with the emotional toll that cancer takes on its victims. Even though Mary had been her nurse years prior, she recognized her voice immediately. Mary has a genuine and authentic approach with patients that makes her unforgettable. She prayed with the patient who was faced with the life-wrenching reality of a terminal diagnosis.

Mary goes out of her way to care for patients, even when they arent assigned to her or even on her unit. Her sister-in-laws husband received a diagnosis of diffuse large B-cell lymphoma. She took it upon herself to discuss the treatment course with her sister-in-law and her husband.

Mary believes strongly in the value of education and knowledge. She feels compelled to teach each patient about the treatment process and side effects so that patients are not surprised by what occurs. According to Mary, knowledge provides power for patients. In her perspective, nurses can become accustomed to the treatments they provide and they often forget that the experience is extremely new to patients. She goes out of her way to make sure all treatments and interventions are explained to and understood by patients.

Along with patient care, Mary has been a lifetime volunteer. Even during her break from nursing to raise her children, Mary volunteered with the international evangelical Christian nonprofit Awana and her local church. She raised wonderful children who are now her legacy and continue her spirit of volunteerism. Mary is filled with pride when she describes how her son raised more than $100,000 to build a medical clinic in Zimbabwe.

Her influence has a profound effect on all of those around her. In the past year, she became the propelling force behind a blood pressure clinic at the Duarte Senior Center. She gets to know the senior citizens who come to the clinic and really listens to them. One lady purchased the same blood pressure cuff that Mary uses and came back to show Mary how she was following Marys advice and tracking her vital signs.

In her interim position as nurse manager over an 18-bed inpatient hematology oncology unit, Mary has led the team to outstanding patient outcomes. Central line bloodstream infections are an ongoing concern at COHNMC. Mary collaborated with infection prevention to ensure that current policies were upheld. She reviewed the policy and expectations for appropriate central line care with every nurse in the unit. Since she started the reinforce- ment of the policies, their unit has not had one line infection.

Not only is Mary a phenomenal healer for her patients, but she also leads her team to seek out excellence in patient outcomes. In everything Mary does, she does so with empathy and an innate understanding of how others feel. It is my honor to nominate Mary Colasuonno for the Extraordinary Healer Award for Oncology Nursing. In any and all definitions of a healer, Mary exceeds. Thank you for considering Mary for this distinct honor.

For more news on cancer updates, research and education, dont forget tosubscribe to CUREs newsletters here.

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A Lifetime of Achievement for an Oncology Nurse - Curetoday.com

FATE THERAPEUTICS INC Management’s Discussion and Analysis of Financial Condition and Results of Operations (form 10-Q) – Marketscreener.com

The following discussion and analysis should be read in conjunction with ourfinancial statements and accompanying notes included in this Quarterly Report onForm 10-Q and the financial statements and accompanying notes thereto for thefiscal year ended December 31, 2022 and the related Management's Discussion andAnalysis of Financial Condition and Results of Operations, which are containedin our Annual Report on Form 10-K filed with the Securities and ExchangeCommission on February 28, 2023.

This Quarterly Report on Form 10-Q contains "forward-looking statements" withinthe meaning of Section 27A of the Securities Act and Section 21E of theSecurities Exchange Act of 1934, as amended (the Exchange Act). Suchforward-looking statements, which represent our intent, belief, or currentexpectations, involve risks and uncertainties and other factors that could causeactual results and the timing of certain events to differ materially from futureresults expressed or implied by such forward-looking statements. In some casesyou can identify forward-looking statements by terms such as "may," "will,""expect," "anticipate," "estimate," "intend," "plan," "predict," "potential,""believe," "should" and similar expressions. Factors that could cause orcontribute to differences in results include, but are not limited to, those setforth under "Risk Factors" under Item 1A of Part II below. Except as required bylaw, we undertake no obligation to update these forward-looking statements toreflect events or circumstances after the date of this report or to reflectactual outcomes.

Overview

We are a clinical-stage biopharmaceutical company dedicated to bringing afirst-in-class pipeline of programmed cellular immunotherapies to patients withcancer and autoimmune disorders. Our development of first-in-class cell therapyproduct candidates is based on a simple notion: we believe that better celltherapies start with better cells.

To create better cell therapies, we have pioneered a therapeutic approach thatwe generally refer to as cell programming: we create and engineer human inducedpluripotent stem cells (iPSCs) to incorporate novel synthetic controls of cellfunction; we generate a clonal master iPSC line for use as a renewable source ofcell manufacture; and we direct the fate of the clonal master iPSC line toproduce our first-in-class cell therapy product candidate. Analogous to mastercell lines used to manufacture biopharmaceutical drug products such asmonoclonal antibodies, we believe clonal master iPSC lines can be used to massproduce multiplexed-engineered cellular immunotherapies which are well-definedand uniform in composition, can be stored in inventory for off-the-shelfavailability, can be combined and administered with other therapies, and canhave broader patient reach.

Utilizing this therapeutic approach, we are advancing a cell therapy pipelinecomprised of off-the-shelf, multiplexed-engineered, iPSC-derived natural killer(NK) and T-cell product candidates that are selectively designed, incorporatenovel synthetic controls of cell function, and are intended to deliver multiplemechanisms of therapeutic importance to patients for the treatment of cancer andautoimmune diseases.

We have entered into a research collaboration and license agreement with theRegents of the University of Minnesota to develop off-the-shelf, engineeredNK-cell cancer immunotherapies derived from clonal master iPSC lines.Additionally, we have entered into a research collaboration and licenseagreement with Memorial Sloan Kettering Cancer Center (MSK) to developoff-the-shelf, engineered T-cell cancer immunotherapies derived from clonalmaster iPSC lines.

In September 2018, we entered into a collaboration and option agreement (OnoAgreement) with Ono Pharmaceutical Co. Ltd. (Ono) for the joint development andcommercialization of off-the-shelf, iPSC-derived CAR T-cell product candidatesfor the treatment of cancer. In June 2022, we entered into an amendment (OnoAmendment) to the Ono Agreement to expand the collaboration to include theresearch and development of off-the-shelf, iPSC-derived CAR NK-cell productcandidates, and pursuant to the Ono Agreement, Ono agreed to provide novelbinding domains targeting a second solid tumor antigen under the collaboration.

In April 2020, we entered into a collaboration and option agreement with JanssenBiotech, Inc. (Janssen), part of the Janssen Pharmaceutical Companies of Johnson& Johnson (Janssen Agreement), for the development and commercialization ofoff-the-shelf, iPSC-derived CAR NK and CAR T-cell product candidates for thetreatment of cancer. Through the period ending December 31, 2022, Janssen hadexercised a commercial option for two collaboration candidates: an iPSC-derived,CAR-targeted NK cell product candidate for the treatment of B-cell lymphoma, forwhich the U.S. Food and Drug Administration (FDA) allowed an Investigational NewDrug (IND) application in December 2022; and an iPSC-derived, CAR-targeted NKcell product candidate for the treatment of multiple myeloma, for which thecompanies were preparing to submit an IND application to the FDA in early 2023.On January 3, 2023, we received notice of termination from Janssen of theJanssen Agreement. The termination of the Janssen Agreement took effect on April3, 2023, and during the three months ended March 31, 2023, we performed winddown activities, including discontinuing development of all collaborationproduct candidates, including two product candidates that were expected to enterthe clinic in 2023.

In January 2023, we announced the discontinuation of our FT516, FT596, FT538,and FT536 NK cell programs to focus our resources on advancing our mostinnovative and differentiated programs.

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We were incorporated in Delaware in 2007, and are headquartered in San Diego,CA. Since our inception in 2007, we have devoted substantially all of ourresources to our cell programming approach and the research and development ofour product candidates, the creation, licensing and protection of relatedintellectual property, and the provision of general and administrative supportfor these activities. To date, we have funded our operations primarily throughthe public and private sale of common stock, the private placement of preferredstock and convertible notes, commercial bank debt and revenues fromcollaboration activities and grants.

We have never been profitable and have incurred net losses in each year sinceinception. Substantially all of our net losses resulted from costs incurred inconnection with our research and development programs and from general andadministrative costs associated with our operations. We expect to continue toincur operating losses for at least the foreseeable future. Our net losses mayfluctuate significantly from quarter to quarter and year to year. We expect ourexpenses will increase substantially in connection with our ongoing and plannedactivities as we:

conduct our ongoing and planned clinical trials of our product candidates, whichmay include higher clinical trial expenses associated with arrangements we mayenter into with clinical research organizations (CROs) for the execution andmanagement of certain clinical trials, including trials outside of the UnitedStates;

conduct Good Manufacturing Practice (GMP) production, including through the useof contract manufacturing organizations (CMOs) for the conduct of some or all ofthe activities required for manufacturing our iPSC-derived cell productcandidates, process and scale-up development and technology transfer activitiesfor the manufacture of our product candidates, including those undergoingclinical investigation and IND-enabling preclinical development;

procure laboratory equipment, materials and supplies for the manufacture of ourproduct candidates and the conduct of our research activities;

conduct preclinical and clinical research to investigate the therapeuticactivity of our product candidates;

continue our research, development and manufacturing activities, including underour sponsored research and collaboration agreement with Ono;

maintain, prosecute, protect, expand and enforce our intellectual propertyportfolio;

engage with regulatory authorities for the development of, and seek regulatoryapprovals for, our product candidates;

build out business operations at our corporate headquarters, including internalGMP production capabilities;

continue to implement the corporate restructuring and reduction in force that weannounced in January 2023; and

continue operating as a public company and support our operations and developcommercial infrastructure for potential commercialization of our productcandidates.

We do not expect to generate any meaningful revenues from product sales,royalties, or sales milestones unless and until we successfully completedevelopment and obtain regulatory approval for one or more of our productcandidates, which we expect will take a number of years. If we obtain regulatoryapproval for any of our product candidates, we expect to incur significantcommercialization expenses related to product sales, marketing, manufacturingand distribution. Accordingly, we will seek to fund our operations throughpublic or private equity or debt financings, collaboration arrangements, orother sources. However, we may be unable to raise additional funds or enter intosuch other arrangements when needed on favorable terms or at all. Our failure toraise capital or enter into such other arrangements when needed would have anegative effect on our financial condition and ability to develop our productcandidates.

Financial Operations Overview

We conduct substantially all of our activities through Fate Therapeutics, Inc.,a Delaware corporation, at our facilities headquartered in San Diego,California. The results of operations include the operations of the Company andits subsidiaries. To date, the aggregate operations of our subsidiaries have notbeen significant and all intercompany transactions and balances have beeneliminated in consolidation.

Collaboration Revenue

To date, we have not generated any revenues from therapeutic product sales orroyalties. Our revenues have been derived from collaboration agreements andgovernment grants.

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Agreement with Janssen Biotech, Inc.

On April 2, 2020 (the Janssen Agreement Effective Date), we entered into aCollaboration and Option Agreement (the Janssen Agreement) with Janssen Biotech,Inc. (Janssen), part of the Janssen Pharmaceutical Companies of Johnson &Johnson. Additionally, on the Janssen Agreement Effective Date, we entered intoa Stock Purchase Agreement (the Stock Purchase Agreement) with Johnson & JohnsonInnovation - JJDC, Inc. (JJDC). Under the terms of the Janssen Agreement and theStock Purchase Agreement taken together, we received $100.0 million, of which$50.0 million was an upfront cash payment and $50.0 million was in the form ofan equity investment by JJDC. Additionally, we are entitled to receive fees forthe conduct of all research, preclinical development and IND-enabling activitiesperformed by us under the Janssen Agreement.

We determined the common stock purchase by JJDC represented a premium of $9.93per share, or $16.0 million in aggregate (the Equity Premium), and the remaining$34.0 million was recorded as issuance of common stock in shareholders' equity.

On January 3, 2023, we received notice of termination from Janssen of theJanssen Agreement. The termination will take effect on April 3, 2023, and duringthe three months ending March 31, 2023, we performed wind down activities,including discontinuing development of all collaboration product candidatesunder the Janssen Agreement. We expect to be reimbursed for all wind downactivities associated with the termination of the Janssen Agreement in thesecond quarter of this year. Under the terms of the Janssen Agreement, inconnection with the termination, (i) all licenses and other rights granted toeither party pursuant to the Janssen Agreement have terminated, subject tolimited exceptions set forth in the Janssen Agreement; (ii) both parties havewound down all development, commercialization and manufacturing activities underthe Janssen Agreement; (iii) neither party has any right to continue to develop,manufacture or commercialize any collaboration candidate or collaborationproduct or use the other party's materials; and (iv) neither party is restrictedfrom independently developing, manufacturing, or commercializing any product,including any products directed to the same antigens as those of anycollaboration candidate or collaboration product.

During the three months ended March 31, 2023, we recognized $52.3 million ofcollaboration revenue under the Janssen Agreement, of which $41.2 million waspreviously deferred. During the three months ended March 31, 2022, we recognized$15.9 million of collaboration revenue under the Janssen Agreement.

Agreement with Ono Pharmaceutical Co., Ltd.

On September 14, 2018, we entered into a Collaboration and Option Agreement (theOno Agreement) with Ono for the joint development and commercialization of twooff-the-shelf iPSC-derived CAR T-cell product candidates (Candidate 1 andCandidate 2). Pursuant to the terms of the Ono Agreement, we received anupfront, non-refundable and non-creditable payment of $10.0 million.Additionally, we are entitled to receive fees for the conduct of research anddevelopment under a joint development plan, which fees were estimated to be$20.0 million in aggregate.

We concluded that certain units of account within the Ono Agreement representeda customer relationship and in accordance with ASC 606, we determined that theinitial transaction price under the Ono Agreement equals $30.0 million,consisting of the upfront, non-refundable and non-creditable payment of $10.0million and the aggregate estimated research and development fees of $20.0million. In addition, we identified our performance obligations under the OnoAgreement, including our grant to Ono of a license to certain of ourintellectual property subject to certain conditions, our conduct of researchservices, and our participation in a joint steering committee. We determinedthat all performance obligations should be accounted for as one combinedperformance obligation since no individual performance obligation is distinct,and that the combined performance obligation is transferred over the expectedterm of the conduct of the research services, which is estimated to be fouryears.

In December 2020, we entered into a letter agreement with Ono pursuant to whichOno delivered proprietary antigen binding domains targeting an antigen expressedon certain solid tumors for incorporation into Candidate 2 and paid the Companya milestone fee of $10.0 million for further research and development ofCandidate 2. In addition, Ono terminated all further research and developmentwith respect to Candidate 1, and we retained all rights to research, develop andcommercialize Candidate 1 throughout the world without any obligation to Ono.

In June 2022, we entered into an amendment with Ono to the Ono Agreement (theOno Amendment). Pursuant to the Ono Amendment, the companies agreed to designatean additional antigen expressed on certain solid tumors for research andpreclinical development, and Ono agreed to contribute proprietary antigenbinding domains targeting such additional solid tumor antigen (Candidate 3). Inaddition, for both Candidate 2 and Candidate 3, the companies expanded the scopeof the collaboration to include the research and development of iPSC-derived CARNK cell product candidates (in addition to iPSC-derived CAR T-cell productcandidates) targeting the designated solid tumor antigens. Similar to Candidate2, we granted to Ono, during a specified period of time, a preclinical option toobtain an exclusive license under certain intellectual property rights, subjectto payment of an option exercise fee to us by Ono, to develop and commercializeCandidate 3 in all territories of the world, where we retain rights toco-develop and co-commercialize Candidate 3 in the United States and Europeunder a joint arrangement with Ono under which we are eligible to share at least50% of the profits and losses. We maintained worldwide rights of manufacture forCandidate 3. The preclinical option expires upon the earlier of: (a) September30, 2024, or (b) the achievement of the pre-defined preclinical milestone underthe joint development plan for Candidate 3. Subject to payment of an extensionfee by Ono, Ono may choose to defer its

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decision to exercise the preclinical option until no later than June 2026. Underthe Ono Amendment, aggregate estimated research and development fees have beenincreased by approximately $9.3 million, for a total estimated $29.3 million inaggregate research and development fees over the course of the joint developmentplan.

In November 2022, Ono exercised its preclinical option to Candidate 2, and weexercised our preclinical option to co-develop and co-commercialize (CDCCOption) in the United States and Europe under a joint arrangement with Ono. As aresult, we received an option exercise fee of $12.5 million from Ono.

During the three months ended March 31, 2023, we recognized $6.7 million ofcollaboration revenue and $1.0 million of contra-research and developmentexpense under the Ono Agreement. During the three months ended March 31, 2022,we recognized $2.5 million of collaboration revenue under the Ono Agreement.

Research and Development Expenses

Research and development expenses consist of costs associated with the research,preclinical development, process and scale-up development, manufacture andclinical development of our product candidates, the research and development ofour cell programming technology including our iPSC product platform, and theperformance of research and development activities under our collaborationagreements. These costs are expensed as incurred and include:

salaries and employee-related costs, including stock-based compensation;

costs incurred under clinical trial agreements with investigative sites;

costs to acquire, develop and manufacture preclinical study and clinical trialmaterials, including our product candidates;

costs associated with conducting our preclinical, process and scale-updevelopment, manufacturing, clinical and regulatory activities, including feespaid to third-party professional consultants, service providers and suppliers;

costs incurred for our research, development and manufacturing activities,including under our collaboration agreements;

costs for laboratory equipment, materials and supplies for the manufacture ofour product candidates and the conduct of our research activities;

costs incurred to license and maintain intellectual property; and

facilities, depreciation and other expenses including allocated expenses forrent and maintenance of facilities.

We plan to increase our current level of research and development expenses forthe foreseeable future as we continue the clinical and preclinical developmentand manufacture of our product candidates, research and develop our iPSC productplatform, and perform our obligations under collaboration agreements includingunder our agreements with Ono, University of Minnesota and MSK. Our currentplanned research and development activities over the next twelve months consistprimarily of the following:

conducting clinical trials of our product candidates, including through theengagement of CROs to manage various aspects of our clinical trials;

conducting GMP production, including through the use of CMOs for the conduct ofsome or all of the activities required for manufacturing our iPSC-derived cellproduct candidates, process and scale-up development and technology transferactivities for the manufacture of our product candidates, including thoseundergoing clinical investigation and IND-enabling preclinical development;

procuring laboratory equipment, materials and supplies for the manufacture ofour product candidates and the conduct of our research activities;

conducting preclinical and clinical research to investigate the therapeuticactivity of our product candidates; and

conducting research, development and manufacturing activities, including underour sponsored research and collaboration agreement with Ono.

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Due to the inherently unpredictable nature of preclinical and clinicaldevelopment and manufacture, and given our novel therapeutic approach and thecurrent stage of development of our product candidates, we cannot determine andare unable to estimate with certainty the timelines we will require and thecosts we will incur for the development and manufacture of our productcandidates. Clinical and preclinical development and manufacturing timelines andcosts, and the potential of development and manufacturing success, can differmaterially from expectations. In addition, we cannot forecast which productcandidates may be subject to future collaborations, when such arrangements willbe secured, if at all, and to what degree such arrangements would affect ourdevelopment and manufacturing plans and capital requirements. We cannot predictthe effects of the impact of global economic and market conditions, the COVID-19pandemic and the ongoing conflict in Ukraine on our business and operations, andour expenditures may be increased by delays or disruptions due to these or otherfactors, including as a result of actions we take in the near term to ensurebusiness continuity and protect against possible supply chain shortages.

General and Administrative Expenses

General and administrative expenses consist primarily of salaries andemployee-related costs, including stock-based compensation, for our employees inexecutive, operational, finance and human resource functions; professional feesfor accounting, legal and tax services; costs for obtaining, prosecuting,maintaining, and enforcing our intellectual property; and other costs and fees,including director and officer insurance premiums, to support our operations asa public company. We anticipate that our general and administrative expenseswill increase in the future as we increase our research and developmentactivities, maintain compliance with exchange listing and SEC requirements,protect and enforce our intellectual property, and continue to operate as apublic company.

Other Income (Expense)

Other income (expense) consists of changes in the fair value of stock priceappreciation milestones associated with the Amended and Restated ExclusiveLicense Agreement dated May 15, 2018 (Amended MSK License) with Memorial SloanKettering Cancer Center (MSK), interest income earned on cash and cashequivalents and interest income from investments (including the amortization ofdiscounts and premiums).

California Institute for Regenerative Medicine Award

On April 5, 2018, we executed an award agreement with the California Institutefor Regenerative Medicine (CIRM) pursuant to which CIRM awarded us $4.0 millionto advance our FT516 product candidate into a first-in-human clinical trial (theAward). In November 2019, we submitted an IND application for FT516 in advancedsolid tumors. As of March 31, 2023, we have received aggregate disbursementsunder the Award in the amount of $4.0 million.

Pursuant to the terms of the Award, we, in our sole discretion, have the optionto treat the Award either as a loan or as a grant. In connection with ourdecision to discontinue our FT516 program during the first quarter of 2023, wereversed the liability associated with the Award, and recorded such amount inother income during the three months ended March 31, 2023.

Critical Accounting Policies and Significant Judgments and Estimates

Our management's discussion and analysis of our financial condition and resultsof operations are based on our unaudited condensed consolidated financialstatements, which have been prepared in accordance with United States generallyaccepted accounting principles. The preparation of these unaudited condensedconsolidated financial statements requires us to make estimates and judgmentsthat affect the reported amounts of assets, liabilities, revenues, and expensesand the disclosure of contingent assets and liabilities in our financialstatements. On an ongoing basis, we evaluate our estimates and judgments,including those related to the fair value of the stock price appreciationmilestones for the Amended MSK License, contracts containing leases, accruedexpenses, stock-based compensation, and the estimated total costs expected to beincurred under our collaboration agreements. We base our estimates on historicalexperience, known trends and events, financial models, and various other factorsthat are believed to be reasonable under the circumstances, the results of whichform the basis for making judgments about the carrying values of assets andliabilities that are not readily apparent from other sources. Actual results maydiffer from these estimates under different assumptions or conditions.

The estimates and judgments involved in our accounting policies as described inItem 7 of our Annual Report on Form 10-K for the year ended December 31, 2022,continue to be our critical accounting policies and there have been no othermaterial changes to our critical accounting policies during the three monthsended March 31, 2023.

See Note 1 to the unaudited condensed consolidated financial statements for asummary of critical accounting policies and information related to recentaccounting pronouncements.

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Results of Operations

Comparison of the Three Months Ended March 31, 2023 and 2022

The following table summarizes the results of our operations for the threemonths ended March 31, 2023 and 2022 (in thousands):

Collaboration Revenue. During the three months ended March 31, 2023 and 2022, werecognized revenue of $59.0 million and $18.4 million, respectively, under ourcollaboration agreements with Janssen and Ono. The increase in collaborationrevenue was attributable to recognition of deferred revenue balances associatedto the Janssen contract termination.

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FATE THERAPEUTICS INC Management's Discussion and Analysis of Financial Condition and Results of Operations (form 10-Q) - Marketscreener.com

Ocean Biomedical (NASDAQ: OCEA) Announces 70% Increase in … – InvestorsObserver

Ocean Biomedical (NASDAQ: OCEA) Announces 70% Increase in Price Target by EF Hutton from $10 to $17 on New Glioblastoma Results Validating Profound Tumor Suppression with Anti-Chi3L1 Antibody

Providence, RI, May 01, 2023 (GLOBE NEWSWIRE) -- Ocean Biomedical, Inc. ( NASDAQ: OCEA ), a biopharma company working to accelerate the development and commercialization of scientifically compelling assets from research universities and medical centers, announced today that an equity analyst coverage report issued by EF Hutton has increased that firms price target by 70%, now targeting $17 per share, up from their initial target of $10 per share. This increase was attributed in part to the publication in Cancer Research of findings suggesting compelling activity of Chi3L1 in animal models of glioblastoma.

The animal models cited in the studies used mice implanted with human glioblastoma to test the efficacy of Ocean Biomedicals anti-Chi3L1 therapeutic candidate, and showed tumor reduction of greater than 60% in 2 different study approaches. The groundbreaking research uncovers in detail how the suppression of Chi3L1 works to keep glioma stem cells from differentiating into the most aggressive forms of glioblastoma. This data provides further evidence of the therapeutic potential of Oceans anti-Chi3L1 for solid tumors.

From the EF Hutton The publication elucidates the mechanism by which Chi3L1 licenses the proliferation of GBM in animal models, and the role by which antibodies against Chi3L1 play in limiting their growth, the EF Hutton report notes, Further, the publication speaks to the effect of OCEA's antibodies on the GBM stem cell niche. Dr. Elias' previous work has demonstrated the multifaceted role that Chi3L1 plays in other solid tumor types such as melanoma and lung cancer.

Dr. Elias work is the foundation of both the oncology and fibrosis programs at Ocean, the report additionally notes, adding that, the key takeaway message from Dr. Tapinos work is that if the reversion of GBM cells to a less mature state (also known as a mesenchymal phenotype) is prevented, one may be able to provide meaningful benefit to patients with this challenging condition, which is uniformly fatal.

From Ocean Biomedical We are honored to see independent institutional research analyst coverage recognizing our core programs in oncology, fibrosis, and infectious diseases that we believe have the potential to save thousands of lives, commented Dr. Chirinjeev Kathuria co-founder and Chairman of OCEA.

We appreciate EF Huttons close attention to our research news and long-term analytics, said Gurinder Kalra, Oceans Chief Financial Officer,

Our team of experienced biopharma executives and top-tier scientists are working to move our research programs forward step by step towards IND filings on each of them as efficiently as possible, said Elizabeth Ng, CEO of Ocean Biomedical.

Oceans core assets in oncology, fibrosis, and infectious diseases, all based on new target discoveries enabling first-in-class drug and vaccine candidates, were developed through past and ongoing grants totaling $123.9 million.

A copy of EF Huttons full analyst report can be obtained directly from EFHutton.

All reports on OCEA prepared by analysts represent the views of such analysts and are not necessarily those of OCEA. OCEA is not responsible for the content, accuracy, or timelines provided by analysts. OCEA does not expressly or by implication warrant or assume any legal liability or responsibility for the accuracy, completeness, or usefulness of any information, assumption, data, forecast, price target, estimate, or projection contained in the reports or industry notes provided by analysts, and the dissemination of such reports or industry notes does not necessarily constitute or imply OCEAs endorsement or recommendation.

About Ocean Biomedical

Ocean Biomedical, Inc. (Ocean Biomedical or the Company) is a Providence, Rhode Island-based biopharma company with an innovative business model that accelerates the development and commercialization of scientifically compelling assets from research universities and medical centers. Ocean Biomedical deploys the funding and expertise to move new therapeutic candidates efficiently from the laboratory to the clinic, to the world. Ocean Biomedical is currently developing five promising discoveries that have the potential to achieve life-changing outcomes in lung cancer, brain cancer, pulmonary fibrosis, and the prevention and treatment of malaria. The Ocean Biomedical team is working on solving some of the worlds toughest problems, for the people who need it most.

To learn more, visit http://www.oceanbiomedical.com .

Forward-Looking Statements

The information included herein and in any oral statements made in connection herewith include forward-looking statements within the meaning of the safe harbor provisions of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements may be identified by the use of words such as estimate, plan, project, forecast, intend, will, expect, anticipate, believe, seek, target or other similar expressions that predict or indicate future events or trends or that are not statements of historical matters, although not all forward-looking statements contain such identifying words. These forward-looking statements include, but are not limited to, statements regarding estimates and forecasts of financial and performance metrics and expectations. These statements are based on various assumptions, whether or not identified herein, and on the current expectations of the Companys management and are not predictions of actual performance. These forward-looking statements are provided for illustrative purposes only and are not intended to serve as, and must not be relied on by any investor as, a guarantee, an assurance, a prediction or a definitive statement of fact or probability. Actual events and circumstances are difficult or impossible to predict and will differ from assumptions.

Any discoveries announced by the Company are based solely on laboratory and animal studies. Ocean Biomedical has not conducted any studies that show similar efficacy or safety in humans. There can be no assurances that this treatment will prove safe or effective in humans, and any clinical benefits of this treatment is subject to clinical trials and ultimate approval of its use in patients by the FDA. Such approval, if granted, could be years away.

Forward-looking statements are predictions, projections and other statements about future events that are based on current expectations and assumptions and, as a result, are subject to risks and uncertainties. These forward-looking statements are not guarantees of future performance, conditions or results, and involve a number of known and unknown risks, uncertainties, assumptions and other important factors, many of which are outside the control of the Company that could cause actual results or outcomes to differ materially from those discussed in the forward-looking statements. Important factors, among others, that may affect actual results or outcomes include but are not limited to: (i) the outcome of any legal proceedings that may be instituted against the Company; (ii) changes in the markets in which the Company competes, including with respect to its competitive landscape, technology evolution, or regulatory changes; (iii) changes in domestic and global general economic conditions; (iv) the risk that the Company may not be able to execute its growth strategies; (v) risks related to the ongoing COVID-19 pandemic and response, including supply chain disruptions; (vi) the risk that the Company may not be able to develop and maintain effective internal controls; (vii) the risk that the Company may fail to keep pace with rapid technological developments to provide new and innovative products and services or make substantial investments in unsuccessful new products and services; (viii) the ability to develop, license or acquire new therapeutics; (ix) the risk that the Company will need to raise additional capital to execute its business plan, which may not be available on acceptable terms or at all; (x) the risk that the Company experiences difficulties in managing its growth and expanding operations; (xi) the risk of product liability or regulatory lawsuits or proceedings relating to the Companys business; (xii) the risk of cyber security or foreign exchange losses; or (xiii) the risk that the Company is unable to secure or protect its intellectual property.

The foregoing list of factors is not exhaustive. You should carefully consider the foregoing factors and the other risks and uncertainties that are described in the Companys Annual Report on Form 10-K for the year ended December 31, 2021 and its Quarterly Report on Form 10-Q for the quarter ended September 30, 2022, and which are described in the Risk Factors section of the Companys definitive proxy statement filed by the Company on January 12, 2023, and other documents to be filed by the Company from time to time with the SEC and which are and will be available at http://www.sec.gov . These filings identify and address other important risks and uncertainties that could cause actual events and results to differ materially from those contained in the forward-looking statements. Forward-looking statements speak only as of the date they are made. Readers are cautioned not to put undue reliance on forward-looking statements. These forward-looking statements should not be relied upon as representing the Companys assessments as of any date subsequent to the date of this filing. Accordingly, undue reliance should not be placed upon the forward-looking statements.

Ocean Biomedical Investor Relations OCEANIR@westwicke.com

Ocean Biomedical Media Relations OCEANPR@westwicke.com

Kevin Kertscher Communications Director

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VERTEX PHARMACEUTICALS INC / MA Management’s Discussion and Analysis of Financial Condition and Results of Operations (form 10-Q) – Marketscreener.com

OVERVIEW

Financial Highlights

Revenues In the first quarter of 2023, our net product revenues increased to $2.4 billion

primarily due to the

internationally and

Expenses Our total research and development ("R&D"), acquired in-process research and

administrative ("SG&A") expenses

compared to $818.3

primarily due to increased

in mid- to late-stage

net product revenues in

increased to $11.5

as of December 31, 2022

cash flows partially

Inc. ("Entrada") and

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TRIKAFTA/KAFTRIO is approved and reimbursed or accessible in more than 30countries outside the U.S.

Potential Near-Term Launch Opportunities

We are preparing for the following near-term launches of potential new products:

Exa-cel in SCD and TDT

We recently completed rolling submissions of our biologics licensingapplications ("BLAs") for exa-cel in the U.S. Exa-cel has been granted FastTrack, Regenerative Medicine Advanced Therapy, Orphan Drug and Rare PediatricDisease designations in the U.S.

Vanzacaftor/tezacaftor/deutivacaftor in CF

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Pipeline

Cystic Fibrosis

Beta Thalassemia and Sickle Cell Disease

We are evaluating the use of exa-cel, a non-viral ex vivo CRISPR gene-editingtherapy, for the treatment of SCD and TDT.

Dosing in the Phase 3 CLIMB-111 and CLIMB 121 clinical trials evaluatingexa-cel continues, as does the CLIMB 131 long-term follow-up clinical trial inpatients 12 years of age and older.

Two additional Phase 3 clinical trials evaluating exa-cel in children with SCDor TDT 5 to 11 years of age are ongoing.

Neuropathic Pain

APOL1-Mediated Kidney Disease

The FDA granted inaxaplin Breakthrough Therapy designation for APOL1-mediatedFSGS and the EMA granted inaxaplin Orphan Drug and PRIME designations for AMKD.

Type 1 Diabetes

Our hypoimmune cell research program continues to progress.

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Alpha-1 Antitrypsin Deficiency

Additional Earlier Stage R&D Programs

Investments in External Innovation

Recent investments in external innovation are included below:

We announced a new licensing agreement for the use of CRISPR's gene-editingtechnology, known as CRISPR/Cas9, to accelerate the development of ourhypoimmune cell therapies for T1D.

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Sales of our products depend, to a large degree, on the extent to which ourproducts are reimbursed by third-party payors, such as government healthprograms, commercial insurance and managed health care organizations.Reimbursement for our products, including our potential pipeline therapies,cannot be assured and may take significant periods of time to obtain. Wededicate substantial management and other resources to obtain and maintainappropriate levels of reimbursement for our products from third-party payors,including governmental organizations in the U.S. and ex-U.S. markets.

We expect to continue to identify and evaluate potential acquisitions and mayinclude larger transactions or later-stage assets.

Collaboration and In-Licensing Arrangements

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In February 2023, we closed our strategic collaboration and licensing agreementwith Entrada. Upon closing, we made an upfront payment of $225.1 million toEntrada, and purchased $24.9 million of Entrada's common stock.

Acquired In-Process Research and Development Expenses

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millions, except percentages and per share amounts)Product revenues, net

millions, except percentages)

millions, except percentages)

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Research and Development Expenses

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Our research expenses have been increasing over the last several years as wehave invested in our pipeline and expanded our cell and genetic therapycapabilities, resulting in increased headcount, outside services and otherdirect expenses and infrastructure costs associated with our researchfacilities. We expect to continue to invest in our research programs with afocus on creating transformative medicines for serious diseases.

Our development expenses increased by $118.5 million, or 26%, in the firstquarter of 2023 as compared to the first quarter of 2022, primarily due toincreased costs to support clinical trials associated with our advancingpipeline programs, including pain, our CF triple combination ofvanzacaftor/tezacaftor/deutivacaftor, exa-cel and T1D. We are significantlyinvesting in internal headcount, leveraging outsourced services, and investingin infrastructure to support these programs.

Acquired In-process Research and Development Expenses

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to another due to upfront, contingent milestone, and other payments pursuant toour existing and future business development transactions, includingcollaborations, licenses of third-party technologies, and asset acquisitions.

Selling, General and Administrative Expenses

Selling, general and administrative expenses increased by 12% in the firstquarter of 2023 as compared to the first quarter of 2022, primarily due to thecontinued investment to support the commercialization of our medicines andincreased support for our pipeline product candidates.

Contingent Consideration

The fair value of our contingent consideration decreased by $1.9 million and$7.5 million in the first quarter of 2023 and 2022, respectively.

Other Non-Operating Income (Expense), Net

Interest Income

Interest Expense

Interest expense was $11.4 million and $14.9 million in the first quarter of2023 and 2022, respectively. The majority of our interest expense in theseperiods was related to imputed interest expense associated with our leasedcorporate headquarters in Boston.

Other Income (Expense), Net

Income Taxes

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LIQUIDITY AND CAPITAL RESOURCES

The following table summarizes the components of our financial condition as ofMarch 31, 2023 and December 31, 2022:

Net cash provided by (used in):

Investing Activities

Financing Activities

Sources and Uses of Liquidity

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Table of Contents

Credit Facilities & Financing Strategy

Future Capital Requirements

We have significant future capital requirements, including:

Facility and finance lease obligations.

Royalties we pay to the Cystic Fibrosis Foundation on sales of our CF products.

Cash paid for income taxes.

In addition, we have significant potential future capital requirementsincluding:

To the extent we borrow amounts under our existing credit agreement, we wouldbe required to repay any outstanding principal amounts in 2027.

As of March 31, 2023, we had $2.9 billion remaining authorization availableunder our Share Repurchase Program.

There have not been any material changes to our future capital requirementsdisclosed in our Annual Report on Form 10-K for the year ended December 31,2022, which was filed with the Securities and Exchange Commission, or SEC, onFebruary 10, 2023.

CRITICAL ACCOUNTING POLICIES AND ESTIMATES

Our discussion and analysis of our financial condition and results of operationsare based upon our condensed consolidated financial statements prepared inaccordance with generally accepted accounting principles in the U.S. The

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Table of Contents

RECENT ACCOUNTING PRONOUNCEMENTS

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Editas Medicine Announces First Quarter 2023 Results and … – GlobeNewswire

Company to provide a clinical update on the EDIT-301 Phase 1/2 RUBY trial for SCD in June at the European Hematology Association Congress (EHA) and in a Company-sponsored webinar

On track to dose 20 total patients by year-end in the RUBY trial

First patient in EDIT-301 EDITHAL trial for TDT dosed with successful neutrophil and platelet engraftment; Company on track to provide clinical update by year-end

Appointed Emma Reeve as Chair of the Board, effective at Annual Meeting of Stockholders, and Elliott Levy, M.D. as an Independent Director

CAMBRIDGE, Mass., May 05, 2023 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a clinical stage genome editing company, today reported business highlights and financial results for the first quarter 2023.

I am energized by our strong start to the year. We entered 2023 with the objective of accelerating the development of EDIT-301 and positioning Editas as a leader in programable in vivo gene editing. Following on our December EDIT-301 clinical data showing a competitive and potentially differentiated product, we have built considerable momentum with our EDIT-301 program, including dosing and engraftment of the first patient in our EDITHAL trial. We look forward to disclosing the clinical progress of EDIT-301 when we provide a RUBY trial update with safety and efficacy data from multiple patients next month in an oral presentation at the European Hematology Association Congress and in a Company-sponsored webinar. I am pleased with the progress weve made against our strategic plan, commented Gilmore ONeill, M.B., M.M.Sc., President and Chief Executive Officer, Editas Medicine. Alongside our newly sharpened strategic focus are our world-class scientists and employees who are committed to our strategic direction and are building on the momentum from our clinical milestones to date and driving execution towards our goals.

Recent Achievements and Outlook

Ex Vivo Hemoglobinopathies

Business Development & Other Corporate Highlights

Elliott Levy, M.D., appointed to the Editas Board of Directors as an independent directorDr. Levy is an accomplished biopharmaceutical executive with more than 20 years of global research and development expertise, including leading clinical strategy and development for multiple programs at all stages of development at global biopharmaceutical companies Amgen and Bristol Myers Squibb.

Linea Aspesi joined Editas as Chief People OfficerMs. Aspesi brings to Editas more than 25 years experience, including 15 years in the life sciences sector, aligning talent plans to company vision, mission, and values, and partnering with senior leaders to define and drive cultural transformation strategies.

First Quarter 2023 Financial Results

Cash, cash equivalents, and marketable securities as of March 31, 2023, were $401.8 million compared to $437.4 million as of December 31, 2022. The Company expects existing cash, cash equivalents and marketable securities to fund operating expenses and capital expenditures into 2025.

Upcoming Events

Editas Medicine plans to participate in the following scientific and medical conference:

Editas Medicine plans to participate in the following investor events:

Conference CallThe Editas Medicine management team will host a conference call and webcast today at 8:00 a.m. ET to provide and discuss a corporate update and financial results for the first quarter of 2023. To access the call, please dial 1-877-407-0989 (domestic) or 1-201-389-0921 (international) and ask for the Editas Medicine earnings call. A live webcast of the call will also be available on the Investors section of the Editas Medicine website at http://www.editasmedicine.com, and a replay will be available approximately two hours after its completion.

AboutEditas MedicineAs a clinical stage genome editing company, Editas Medicine is focused on translating the power and potential of the CRISPR/Cas9 and CRISPR/Cas12a genome editing systems into a robust pipeline of treatments for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision genomic medicines for a broad class of diseases. Editas Medicine is the exclusive licensee of Broad Institute and Harvard Universitys Cas9 patent estates and Broad Institutes Cas12a patent estate for human medicines. For the latest information and scientific presentations, please visit http://www.editasmedicine.com.

Forward-Looking StatementsThis press release contains forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995. The words anticipate, believe, continue, could, estimate, expect, intend, may, plan, potential, predict, project, target, should, would, and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Forward-looking statements in this press release include statements regarding the initiation, timing, progress and results of the Companys preclinical and clinical studies and its research and development programs, including dosing 20 total patients by year-end in the RUBY trial, the timing for the Companys receipt and presentation of data from its clinical trials and preclinical studies, including a clinical update for the RUBY trial in June 2023 and an additional clinical update by year-end and a clinical update from the EDITHAL trial by year-end, potential of, and expectations for, the Companys product candidates, the timing or likelihood of regulatory filings and approvals, and the Companys expectations regarding cash runway. The Company may not actually achieve the plans, intentions, or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various important factors, including: uncertainties inherent in the initiation and completion of pre-clinical studies and clinical trials, including the RUBY and EDITHAL trials, and clinical development of the Companys product candidates, including EDIT-301; availability and timing of results from pre-clinical studies and clinical trials; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products and availability of funding sufficient for the Companys foreseeable and unforeseeable operating expenses and capital expenditure requirements. These and other risks are described in greater detail under the caption Risk Factors included in the Companys most recent Annual Report on Form 10-K, which is on file with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this press release represent the Companys views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. Except as required by law, the Company explicitly disclaims any obligation to update any forward-looking statements.

EDITAS MEDICINE, INC.Consolidated Statement of Operations(amounts in thousands, except share and per share data)(Unaudited)

EDITAS MEDICINE, INC.Selected Consolidated Balance Sheet Items(amounts in thousands)(Unaudited)

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Editas Medicine Announces First Quarter 2023 Results and ... - GlobeNewswire

Global Cell and Tissue Analysis Market Research Report 2023 – Benzinga

DUBLIN, May 4, 2023 /PRNewswire/ --The "Global Markets and Technologies for Cell and Tissue Analysis" report has been added to ResearchAndMarkets.com's offering.

The global market for cell and tissue analysis was estimated to be $16.7 billion in 2021 and is expected to increase to $28.9 billion in 2027, growing at a CAGR of 9.6% during the forecast period. In this report the cell and tissue analysis market is segmented based on technology type, end user and region.

This report focuses on the global market of cell and tissue analysis (CTA) products and provides an updated review, including basic design and applications in various arenas of biomedical and life science research. The report deals with CTA products covering the total market, which includes three main areas of applications.

Cell and tissue analysis products are playing pivotal roles in biomedical research. In the expanding field of biomedical research and development researchers and scientists employ a variety of cell and tissue analysis products. These products play important roles in the diagnosis of various forms of cancer and other diseases and also assume crucial roles when it comes to understanding diverse cellular activities. With the rise of chronic and infectious diseases, there is an urgent need for efficient diagnostics and better healthcare conditions to treat diseases. Along with the recent intensification of research in academia and the biomedical industry, demand is increasing significantly for advanced technology for drug development and screening.

Personalized drug treatment is becoming a reality. In response to the rise in the incidence of a number of diseases and an ageing population, the drug discovery industry is developing new and more efficacious drugs based on specific biomarker signatures and hence cell and tissue analysis technologies will also help in personalized drug development.

Apart from this the increasing demand for single-cell analysis and the extensive research ongoing in this area will further contribute to the market. Other drivers contributing to the market include advances in technologies and product launches, increasing prevalence of chronic diseases, an ageing population, and rising investments and funding. However, the market is facing some challenges such as the high cost of instruments, lack of skilled labor and stringent regulations.

Major players in the market are Thermo Fisher Scientific, Danaher, Becton, Dickinson & Co., Illumina, and Agilent Technologies. Technology types covered are biospecimen technology, cell separation, and cell and tissue characterization. Based on type, the cell and tissue characterization segment has the highest share. Each type is further categorized into subtypes.

North America has the highest share of the market by region, followed by Europe. Extensive R&D activities take place in the region, there are major players present, there is plentiful funding, and there is an increasing prevalence of chronic diseases. All these factors will contribute to growth in the market

Report Includes

Key Topics Covered:

Chapter 1 Introduction

Chapter 2 Summary and Highlights

Chapter 3 Market and Technology Background3.1 Introduction3.1.1 History3.1.2 Cells3.1.3 Animal Tissue3.2 Classification of Techniques Used in Cell and Tissue Analysis3.3 Biospecimen Techniques3.3.1 Microarray3.3.2 Tissue Microarrays3.3.3 Cellular Microarray3.3.4 Dna Microarray or Dna Chip3.3.5 Protein Microarray or Peptide Chip3.4 Cell-Separation Techniques3.4.1 Mechanical and Physical Dissociation3.4.2 Based on Adherence3.4.3 Based on Size and Density3.4.4 Based on Affinity3.4.5 Lab-On-Chip Techniques3.5 Cell and Tissue Characterization3.5.1 Cell-Based Assays3.5.2 Histology and Immunohistochemistry3.5.3 Flow Cytometry3.5.4 Genotyping and Expression Analysis3.5.5 Western Analysis3.5.6 Components of Cell-Based Assays

Chapter 4 Market Dynamics4.1 Factors Affecting the Market4.1.1 Market Drivers4.1.2 Challenges for the Market for Cell and Tissue Analysis4.1.3 Impact of the Covid-19 Pandemic

Chapter 5 Emerging Technologies

Chapter 6 Market Breakdown by Technology6.1 Cell and Tissue Analysis Technologies6.1.1 Biospecimen Technology6.1.2 Cell Separation Technology6.1.3 Cell and Tissue Characterization

Chapter 7 Market Breakdown by End-user7.1 End-users7.1.1 Biopharmaceutical Companies7.1.2 Academic and Research Institutes7.1.3 Healthcare and Clinical

Chapter 8 Market Breakdown by Region8.1 North America8.2 Europe8.3 Emerging Markets

Chapter 9 Regulatory Aspects9.1 New Approvals of Cell and Tissue Analysis Products9.2 Recalls and Safety Alerts

Chapter 10 Patent Analysis10.1 Patent Activity on Cell and Tissue Analysis10.1.1 Patent Review by Year10.1.2 Patent Review by Country10.1.3 Patent Review by Company, University and Institute

Chapter 11 Competitive Landscape11.1 Mergers and Acquisitions11.2 Competitive Analysis11.2.1 Biospecimen Technology11.2.2 Cell Separation Technology11.2.3 Cell and Tissue Characterization Technology

Chapter 12 Company Profiles

For more information about this report visit https://www.researchandmarkets.com/r/l2g1qi

About ResearchAndMarkets.comResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.

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Global Cell and Tissue Analysis Market Research Report 2023 - Benzinga

Promising Future In Live Cell Imaging Market, Analysis By … – Digital Journal

PRESS RELEASE

Published May 3, 2023

The research elaborates on the growth rate of the Live Cell Imaging Market and examines it using a complete and trustworthy examination of the company profile. The research offers a thorough analysis, market size, share, insights, evaluation for developing segments, and many other important market aspects.

The global live cell imaging market was worth USD 2.35 billion in 2021 and is expected growing at a 9.2% CAGR from 2021 to 2030.

Live cell imaging is a technique used to observe and study biological processes at the cellular level in real-time using microscopy. It allows scientists to visualize and monitor dynamic cellular events such as cell division, migration, and death. The global Holotomography (HT)market is expected to grow significantly in the coming years due to the increasing demand for high-resolution imaging techniques in life sciences research and the development of new drugs.

The live cell imaging market is primarily driven by the increasing demand for advanced imaging techniques in the life sciences industry. The growing need for high-resolution imaging techniques to understand the complex biological processes at the cellular level is expected to boost the market growth. In addition, the development of new drugs and therapies for various diseases, such as cancer, is also driving the demand for Holotomography techniques.

The market is also expected to benefit from technological advancements in live cell imaging systems. The introduction of new imaging techniques such as confocal microscopy, two-photon microscopy, and super-resolution microscopy is expected to increase the adoption of Holotomography systems in research and development activities. However, the high cost of live cell imaging systems and the lack of skilled professionals to operate the systems are expected to restrain the market growth to some extent.

Read Report here: https://www.factualmarketresearch.com/Reports/Live-Cell-Imaging-Market

Key Players

Market Segmentation

Live Cell Imaging Market, Based onProduct

Live Cell Imaging Market, Based onApplication

Live Cell Imaging Market, Based onTechnology

Live Cell Imaging Market, Based on Regions

Report customized for your company: https://www.factualmarketresearch.com/Reports/Live-Cell-Imaging-Market

Brief Table of Contents: Live Cell Imaging Market

Introduction

Market Overview

Technology Overview

Market Segmentation

Competitive Landscape

Future Outlook

Conclusion

Read Detailed TOC here: https://www.factualmarketresearch.com/Reports/Live-Cell-Imaging-Market

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Factual Market Research is a one-stop shop for relevant information and market research. Through our specialised and syndicated research services, we assist our clients in finding answers to their research needs. The number of our industry specialties exceeds 20. Read the study description and get in touch with us on our website to learn more in-depth details about your industry.

Highest calibre Market Research Reports and Business Intelligence Solutions are provided by Factual Market Research. To assist our clients in making informed business decisions and achieving their goals for success, FMR wishes to provide business analytics and consulting.

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Cellectis Provides Business Update and Reports Financial Results … – GlobeNewswire

NEW YORK, May 04, 2023 (GLOBE NEWSWIRE) -- Cellectis (the Company) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today provided a business update and announced its results for the three-month period ending March 31, 2023.

Cellectis took a notable step forward this quarter with the first patient being dosed in France with our in-house manufactured product candidate UCART22 in the BALLI-01 clinical study. UCART22 is currently the most advanced allogeneic CAR T-cell product in development for relapsed or refractory B-cell acute lymphoblastic leukemia. We believe that our off-the-shelf treatment approach, coupled with our ability to manufacture UCART product candidates entirely in-house, gives us a main advantage on the market: it potentially maximizes the chances for eligible patients to be treated without delay, said Andr Choulika, Ph.D., CEO of Cellectis.

Cellectis also announced last month that it implemented the use of Sanofis alemtuzumab as a Cellectis Investigational Medicinal Product, coded as CLLS52, as part of the lymphodepletion regimen for UCART22 in the BALLI-01 clinical trial, for UCART123 in the AMELI-01 clinical trial, and for UCART20x22 in the NATHALI-01 clinical trial. This follows the partnership and supply agreements we entered with Sanofi regarding alemtuzumab.

This quarter, Cellectis announced the closing of the global offering of 25 million dollars of its Depository Shares, launched in February the net proceeds of the global offering and option of the Company is 22.8 million dollars and in April, the drawdown of the 20 million euros under the Finance Contract for up to 40 million euros credit facility made with the European Investment Bank in December 2022. Cellectis plans to use the net proceeds of the funds to focus on the development of its pipeline of allogeneic CAR T-cell product candidates UCART22, UCART20x22 and UCART123, the Company decided to stop enrollment and treatment of patients with UCARTCS1. Indeed, to accelerate the speed of enrollment of patients in the MELANI-01 study, evaluating UCARTCS1, the Company would have had to invest meaningful amount of resources. To optimize its resources, Cellectis decided to focus its development efforts on the BALLI-01, AMELI-01 and NATHALI-01 studies.

We are excited about the drive in our clinical trials, building on the momentum of our lead product candidates in our pipeline, and the upcoming milestones for 2023.

Pipeline Highlights

UCART Clinical Developments Programs

BALLI-01 (evaluating UCART22) in relapsed or refractory B-cell acute lymphoblastic leukemia (r/r B-ALL)

NATHALI-01 (evaluating UCART20x22) in relapsed or refractory B-cell non-Hodgkin lymphoma (r/r NHL)

AMELI-01 (evaluating UCART123) in relapsed or refractory acute myeloid leukemia (r/r AML)

MELANI-01 (evaluating UCARTCS1) in relapsed or refractory multiple myeloma (r/r MM)

Research Data & Preclinical Programs

TALEN-edited MUC1 CAR T-cells

Multiplex engineering for superior generation of efficient CAR T-cells

Licensed Allogeneic CAR T-cell Development Programs

Servier and Allogene: anti-CD19 programs

Allogene continues to enroll patients in the industrys first potentially pivotal Phase 2 allogeneic CAR T clinical trial with ALLO-501A. Allogene announced that the single-arm ALPHA2 trial will enroll approximately 100 r/r large B cell lymphoma (LBCL) patients who have received at least two prior lines of therapy and have not received prior anti-CD19 therapy. Allogene expects to complete enrollment in H1 2024.After the close of the quarter, Allogene announced that pooled data from the Phase 1 ALPHA/ALPHA2 trials of ALLO-501/501A, in r/r LBCL would be presented at the American Society of Clinical Oncology (ASCO) Annual Meeting June 2 6, 2023 in Chicago, Illinois.

Allogene: anti-BCMA and anti-CD70 programs

Partnerships

Cytovia Therapeutics, Inc. (Cytovia)

Corporate Updates

Global offering and American Depositary Shares (ADS)

Calyxt and Cibus Merger Agreement

Warrant agreement with the European Investment Bank

Financial results

The interim condensed consolidated financial statements of Cellectis, which consolidate the results of Calyxt, Inc. of which Cellectis owned approximately 48.2% of outstanding shares of common stock (as of March 31, 2023), have been prepared in accordance with International Financial Reporting Standards, as issued by the International Accounting Standards Board (IFRS).

We present certain financial metrics broken out between our two reportable segments Therapeutics and Plants in the appendices of this Q1 2023 financial results press release.

On January 13, 2023, Calyxt, Cibus Global LLC (Cibus) and certain other parties named therein, entered into an Agreement and Plan of Merger (the Merger Agreement), pursuant to which, subject to the terms and conditions thereof, Calyxt and Cibus will merge in an all-stock transaction (the Calyxt Merger). As a consequence of the foregoing, Calyxt meets the held-for-sale" criteria specified in IFRS 5 and has been classified as a discontinued operation.

Cash: As of March 31, 2023, Cellectis, excluding Calyxt, had $88 million in consolidated cash, cash equivalents, and restricted cash. This compares to $95 million in consolidated cash, cash equivalents and restricted cash as of December 31, 2022. This difference mainly reflects $30 million of cash out, which include $6 million of payments for R&D expenses, $4 million for SG&A suppliers, $15 million for staff costs, $4 million for rents and taxes, $1 million of reimbursement of the PGE loan and a $23 million net cash inflow from the capital raise closed in February.

Based on the current operating plan, Cellectis (excluding Calyxt) anticipates that the cash and cash equivalents as of March 31, 2023 will fund Cellectis operations into the third quarter of 2024.

Revenues and Other Income: Consolidated revenues and other income were $3.6 million for the three months ended March 31, 2023 compared to $3.8 million for the three months ended March 31, 2022. The slight decrease of $0.2 million between the three months ended March 31, 2023, and 2022 reflects the recognition of two milestones related to Cellectis agreement with Cytovia for $1.5million in 2022 while recognition of revenues in 2023 is not material and was almost fully offset by an increase of the research tax credit for $1.0 million in addition to the recognition of a BPI R&D grant of $0.3 million.

R&D Expenses: Consolidated R&D expenses were $21.1 million three months ended March 31, 2023 compared to $26.6 million for the three months ended March 31, 2022. The $5.5 million decrease was primarily attributable to (i) a $2.6 million decrease in personal expenses due to departures not replaced (ii) a $3.0 million decrease in purchases, external expenses and other (from $13.8 million in 2022 to $10.8 million in 2023) mainly explained by internalization of our manufacturing and quality activities to support our R&D pipeline.

SG&A Expenses: Consolidated SG&A expenses were $5.0 million for the three months ended March 31, 2023 compared to $6.1 million for the three months ended March 31, 2022. The $1.1 million decrease primarily reflects (i) a $0.9 million decrease in purchases, external expenses and other (from $3.7 million in 2022 to $2.9 million in 2023) mainly explained by the implementation of our new enterprise resource planning (ERP) software in 2022 (ii) a $0.2 million decrease in personal expenses.

Net income (loss) from discontinued operations: The $1.7 million decrease of net loss from discontinued operations between the three-month period ended March 31, 2022 and 2023 is primarily driven by (i) the decrease of $2.6 million of R&D expenses (from $3.2 million in 2022 to $1.3 in 2023) and SG&A expenses (from $2.9 million in 2022 to $2.2 million in 2023) partially offset by (i) the increase of $0.7 million of net financial loss and (ii) the increase of $0.2 million of other operating expenses.

Net Income (loss) Attributable to Shareholders of Cellectis including Calyxt: The consolidated net loss attributable to shareholders of Cellectis was $30.1 million (or $0.58 per share) for the three months ended March 31, 2023, of which $27.8 million was attributed to Cellectis continuing operations, compared to $31.9 million (or $0.70 per share) for the three months ended March 31, 2022, of which $28.3 million was attributed to Cellectis continuing operations. This $1.8 million decrease in net loss between the three months of 2023 and 2022 was primarily driven by (i) a $5.3 million decrease of research and development, (ii) a decrease of $1.7 million of loss from discontinued operations, (iii) a $1.3 million decrease of SG&A expenses partially offset by (i) an increase in net financial loss of $5.3 million primarily due to the decrease of the fair value of Cytovias convertible note on March 31, 2023 of $4.6 million compared to a $7.9 million on December 31, 2022, (ii) a decrease of $0.2 million of revenues and other income, (iii) an increase of other operating expenses of $0.6 million, (iv) a decrease of $0.4 million in loss attributable to non-controlling interests due to the decrease in Calyxts net loss.

Adjusted Net Income (Loss) Attributable to Shareholders of Cellectis: The consolidated adjusted net loss attributable to shareholders of Cellectis was $28.1 million (or $0.55 per share) for the three months ended March 31, 2023, of which $26.2 million is attributed to Cellectis, compared to a net loss of $29.3 million (or $0.64 per share) for the three months ended March 31, 2022, of which $26.0 million was attributed to Cellectis.

Please see Note Regarding Use of Non-IFRS Financial Measures for reconciliation of GAAP net income (loss) attributable to shareholders of Cellectis to adjusted net income (loss) attributable to shareholders of Cellectis.

We currently foresee focusing our cash spending at Cellectis for 2023 in the following areas:

* These amounts reflect adjustments made in connection with the presentation of the discontinued operation

Note Regarding Use of Non-IFRS Financial Measures

Cellectis S.A. presents adjusted net income (loss) attributable to shareholders of Cellectis in this press release. Adjusted net income (loss) attributable to shareholders of Cellectis is not a measure calculated in accordance with IFRS. We have included in this press release a reconciliation of this figure to net income (loss) attributable to shareholders of Cellectis, which is the most directly comparable financial measure calculated in accordance with IFRS. Because adjusted net income (loss) attributable to shareholders of Cellectis excludes Non-cash stock-based compensation expensea non-cash expense, we believe that this financial measure, when considered together with our IFRS financial statements, can enhance an overall understanding of Cellectis financial performance. Moreover, our management views the Companys operations, and manages its business, based, in part, on this financial measure. In particular, we believe that the elimination of Non-cash stock-based expenses from Net income (loss) attributable to shareholders of Cellectis can provide a useful measure for period-to-period comparisons of our core businesses. Our use of adjusted net income (loss) attributable to shareholders of Cellectis has limitations as an analytical tool, and you should not consider it in isolation or as a substitute for analysis of our financial results as reported under IFRS. Some of these limitations are: (a) other companies, including companies in our industry which use similar stock-based compensation, may address the impact of Non-cash stock- based compensation expense differently; and (b) other companies may report adjusted net income (loss) attributable to shareholders or similarly titled measures but calculate them differently, which reduces their usefulness as a comparative measure. Because of these and other limitations, you should consider adjusted net income (loss) attributable to shareholders of Cellectis alongside our IFRS financial results, including Net income (loss) attributable to shareholders of Cellectis.

*These amounts reflect adjustments made in connection with the presentation of the discontinued operation

About Cellectis

Cellectis is a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies. Cellectis utilizes an allogeneic approach for CAR-T immunotherapies in oncology, pioneering the concept of off-the-shelf and ready-to-use gene-edited CAR T-cells to treat cancer patients, and a platform to make therapeutic gene editing in hemopoietic stem cells for various diseases. As a clinical-stage biopharmaceutical company with over 23 years of experience and expertise in gene editing, Cellectis is developing life-changing product candidates utilizing TALEN, its gene editing technology, and PulseAgile, its pioneering electroporation system to harness the power of the immune system in order to treat diseases with unmet medical needs. Cellectis headquarters are in Paris, France, with locations in New York, New York and Raleigh, North Carolina. Cellectis is listed on the Nasdaq Global Market (ticker: CLLS) and on Euronext Growth (ticker: ALCLS). For more information, visit http://www.cellectis.com. Follow Cellectis on social media: @cellectis, LinkedIn and YouTube

Forward-looking Statements

This press release contains forward-looking statements within the meaning of applicable securities laws, including the Private Securities Litigation Reform Act of 1995. Forward-looking statements may be identified by words such as anticipate, believe, intend, expect, plan, scheduled, could, would and will, or the negative of these and similar expressions. These forward-looking statements, which are based on our managements current expectations and assumptions and on information currently available to management, including information provided or otherwise publicly reported by our licensed partners. Forward-looking statements include statements about advancement, timing and progress of clinical trials (including with respect to patient enrollment and follow-up), the timing of our presentation of data and submission of regulatory filings, the adequacy of our supply of clinical vials, the operational capabilities at our manufacturing facilities, the sufficiency of cash to fund operations, the adequacy and continuity of supply of clinical supply and alemtuzumab, the ability of an anti-CD52 as alemtuzumab to improve any efficacy and the potential benefit of UCART product candidates. These forward-looking statements are made in light of information currently available to us and are subject to numerous risks and uncertainties, including with respect to the numerous risks associated with biopharmaceutical product candidate development. With respect to our cash runway, our operating plans, including product development plans, may change as a result of various factors, including factors currently unknown to us. Furthermore, many other important factors, including those described in our Annual Report on Form 20-F and the financial report (including the management report) for the year ended December 31, 2022 and subsequent filings Cellectis makes with the Securities Exchange Commission from time to time, as well as other known and unknown risks and uncertainties may adversely affect such forward-looking statements and cause our actual results, performance or achievements to be materially different from those expressed or implied by the forward-looking statements. Except as required by law, we assume no obligation to update these forward-looking statements publicly, or to update the reasons why actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future.

For further information, please contact:

Media contact:

Pascalyne Wilson, Director, Communications, +33 (0)7 76 99 14 33, media@cellectis.com

Investor Relation contacts:

Arthur Stril, Chief Business Officer, +1 (347) 809 5980, investors@cellectis.com

Ashley R. Robinson, LifeSci Advisors, +1 617 430 7577

1 Cash position includes cash, cash equivalents and restricted cash. Restricted cash was $5 million as of March 31, 2023.

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