CRISPR Therapeutics Provides Business Update and Reports First Quarter 2023 Financial Results – Yahoo Finance

CRISPR Therapeutics AG

-Regulatory submissions complete for exagamglogene autotemcel (exa-cel), formerly known as CTX001, in the U.S. for transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease (SCD)-

-EU and U.K. submissions validated by European Medicines Agency (EMA) and the Medicines and Healthcare products Regulatory Agency (MHRA); exa-cel has been granted an Innovation Passport under the Innovative Licensing and Access Pathway (ILAP) from the MHRA-

-Enrollment and dosing ongoing for CTX110, targeting CD19+ B-cell malignancies, and CTX130, targeting CD70 for the treatment of T cell lymphomas-

-Initiated clinical trials for next generation CAR T candidates, CTX112 targeting CD19+ B-cell malignancies and CTX131, targeting CD70+ solid tumors-

-Enrollment and dosing ongoing in a Phase 1/2 clinical trial of VCTX211 for the treatment of Type 1 Diabetes (T1D)-

-Continues to advance its lead in vivo program, CTX310, targeting angiopoietin-related protein 3 (ANGPTL3) into clinical trials this year-

ZUG, Switzerland and BOSTON, May 08, 2023 (GLOBE NEWSWIRE) -- CRISPR Therapeutics(Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today reported financial results for the first quarter ended March 31, 2023.

In the first quarter of 2023, we continued strong momentum across our portfolio. We and our partner Vertex have now completed regulatory submissions for exa-cel in the United States, European Union and United Kingdom, positioning exa-cel to potentially become the first approved CRISPR-based therapy in the world, a remarkable pace of progress considering the discovery of the CRISPR platform took place a little more than a decade ago, said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. In parallel, we continue to drive our portfolio programs, including the initiation of clinical trials for our next-generation CAR T candidates, CTX112 and CTX131. In addition, we expect to advance CTX310, our lead in vivo program targeting ANGPTL3, into clinical trials later this year. We are well-positioned to drive towards our mission of bringing transformative and potentially curative therapies to patients in need.

Story continues

Recent Highlights and Outlook

FirstQuarter 2023 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $1,889.5 million as of March 31, 2023, compared to $1,868.4 million as of December 31, 2022. The increase in cash of $21.1 million was primarily driven by the upfront payment received from Vertex in connection with a non-exclusive license agreement and a benefit from changes in net-working capital, offset by operating expenses.

Revenue: Total collaboration revenue was $100.0 million for the quarter ended March 31, 2023. Collaboration revenue for the first quarter of 2022 was not material. Collaboration revenue recognized in the first quarter of 2023 was primarily attributable to revenue recognized in connection with the upfront payment from Vertex.

R&D Expenses: R&D expenses were $99.9 million for the first quarter of 2023, compared to $118.2 million for the first quarter of 2022. The decrease in R&D expense was primarily driven by reduced variable external research and manufacturing costs.

G&A Expenses: General and administrative expenses were $22.4 million for the first quarter of 2023, compared to $28.0 million for the first quarter of 2022. The decrease in G&A expense was primarily driven by a decrease in external professional costs.

Collaboration Expense: Collaboration expense, net, was $42.2 million for the first quarter of 2023, compared to $30.6 million for the first quarter of 2022. The increase in collaboration expense, net, was primarily driven by an increase in manufacturing and pre-commercial costs associated with the exa-cel program.

Net Loss: Net loss was $53.1 million for the first quarter of 2023, compared to a net loss of $179.2 million for the first quarter of 2022.

About exagamglogene autotemcel (exa-cel)Exa-cel, formerly known as CTX001, is an investigational, autologous,ex vivoCRISPR/Cas9 gene-edited therapy that is being evaluated for patients with TDT or SCD characterized by recurrent vaso-occlusive crises (VOCs), in which a patients own hematopoietic stem cells are edited to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is the form of the oxygen-carrying hemoglobin that is naturally present during fetal development, which then switches to the adult form of hemoglobin after birth. The elevation of HbF by exa-cel has the potential to alleviate transfusion requirements for patients with TDT and reduce painful and debilitating sickle crises for patients with SCD. Earlier results from these ongoing trials were published inThe New England Journal of Medicinein January of 2021.

Based on progress in this program to date, exa-cel has been granted Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease designations from theFDA for both TDT and SCD. Exa-cel has also been granted Orphan Drug Designation from theEuropean Commission, as well as Priority Medicines (PRIME) designation from theEuropean Medicines Agency(EMA), for both TDT and SCD. In the U.K., exa-cel has been granted an Innovation Passport under the Innovative Licensing and Access Pathway (ILAP) from the MHRA.

About CLIMB-111 and CLIMB-121The ongoing Phase 1/2/3 open-label trials, CLIMB-111 and CLIMB-121, are designed to assess the safety and efficacy of a single dose of exa-cel in patients ages 12 to 35 years with TDT or with SCD, characterized by recurrent VOCs, respectively. The trials are now closed for enrollment. Patients will be followed for approximately two years after exa-cel infusion. Each patient will be asked to participate in CLIMB-131, a long-term follow-up trial.

About CLIMB-131This is a long-term, open-label trial to evaluate the safety and efficacy of exa-cel in patients who received exa-cel in CLIMB-111, CLIMB-121, CLIMB-141 or CLIMB-151. The trial is designed to follow participants for up to 15 years after exa-cel infusion.

About CLIMB-141 and CLIMB-151The ongoing Phase 3 open-label trials, CLIMB-141 and CLIMB-151, are designed to assess the safety and efficacy of a single dose of exa-cel in patients ages 2 to 11 years with TDT or with SCD, characterized by recurrent VOCs, respectively. The trials are now open for enrollment and currently enrolling patients ages 5 to 11 years of age and will plan to extend to patients 2 to less than 5 years of age at a later date. Each trial will enroll approximately 12 patients. Patients will be followed for approximately two years after infusion. Each patient will be asked to participate in CLIMB-131, a long-term follow-up- trial.

About the CRISPR-Vertex CollaborationCRISPR Therapeuticsand Vertex Pharmaceuticals entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. Exa-cel represents the first potential treatment to emerge from the joint research program. Under an amended collaboration agreement, Vertex now leads global development, manufacturing and commercialization of exa-cel and splits program costs and profits worldwide 60/40 withCRISPR Therapeutics.

About CTX110 and CTX112CTX110, a wholly owned program ofCRISPR Therapeutics, is a healthy donor-derived gene-edited allogeneic CAR T investigational therapy targeting cluster of differentiation 19, or CD19. CTX110 is being investigated in the ongoing CARBON clinical trial, which is designed to assess the safety and efficacy of CTX110 in adult patients with relapsed or refractory CD19-positive B-cell malignancies who have received at least two prior lines of therapy. CTX110 has been granted RMAT designation by the FDA. In addition, CTX112, a next-generation allogeneic CAR T cell therapy targeting CD19, is being investigated in a clinical trial. CTX112 includes two additional edits beyond CTX110 that are designed to enhance the potency of the CAR T cells.

About CTX130 and CTX131CTX130, a wholly owned program ofCRISPR Therapeutics, is a healthy donor-derived gene-edited allogeneic CAR T investigational therapy targeting cluster of differentiation 70, or CD70, an antigen expressed on various solid tumors and hematologic malignancies. CTX130 is being investigated for the treatment of relapsed or refractory T-cell hematologic malignancies in the COBALT-LYM trial and for renal cell carcinoma in the COBALT-RCC trial. CTX130 has been granted Orphan Drug designation for the treatment of T cell lymphoma by the FDA and RMAT designation for the treatment of relapsed or refractory Mycosis Fungoides and Szary Syndrome (MF/SS), types of cutaneous T cell lymphoma (CTCL). In addition, CTX131, a next-generation allogeneic CAR T cell therapy targeting CD70, is being assessed for safety and efficacy in a clinical trial investigating a basket of select solid tumors. CTX131 includes two additional edits beyond CTX130 that are designed to enhance the potency of the CAR T cells.

About VCTX210 and VCTX211VCTX210 is an investigational, allogeneic, gene-edited, immune-evasive, stem cell-derived investigational therapy for the treatment of T1D. VCTX210 is being developed under a co-development and co-commercialization agreement betweenCRISPR TherapeuticsandViaCyte, Inc. VCTX211 is an allogeneic, gene-edited, stem cell-derived investigational therapy for the treatment of T1D, which incorporates additional gene edits that aim to further enhance cell fitness. This immune-evasive cell replacement therapy is designed to enable patients to produce their own insulin in response to glucose.

AboutCRISPR TherapeuticsCRISPR Therapeuticsis a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA.CRISPR Therapeuticshas established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts,CRISPR Therapeuticshas established strategic partnerships with leading companies includingBayer, Vertex PharmaceuticalsandViaCyte, Inc.CRISPR Therapeutics AGis headquartered inZug, Switzerland, with its wholly-ownedU.S.subsidiary,CRISPR Therapeutics, Inc., and R&D operations based inBoston, MassachusettsandSan Francisco, California, and business offices inLondon, United Kingdom. For more information, please visitwww.crisprtx.com.

CRISPR THERAPEUTICS standard character mark and design logo,COBALT, CTX001, CTX110, CTX112, CTX130, CTX131, CTX310, VCTX210, VCTX211 are trademarks and registered trademarks ofCRISPR Therapeutics AG. All other trademarks and registered trademarks are the property of their respective owners.

CRISPR Therapeutics Forward-Looking StatementThis press release may contain a number of forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements made byDr. Kulkarniin this press release, as well as statements regarding CRISPR Therapeutics expectations about any or all of the following: (i) its preclinical studies, clinical trials and pipeline products and programs, including, without limitation, the status of such studies and trials, regulatory filings for exa-cel and timing of data releases and regulatory submissions; (ii) potential benefits of exa-cel and the FDAs review of the BLAs and impact of Priority Review on such timing; (iii) benefits of Dr. Prasads employment; (iv) the sufficiency of its cash resources; (v) benefits of its collaborations, including potential milestone payments and royalties on future products under the non-exclusive license agreement; and (vi) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies. Without limiting the foregoing, the words believes, anticipates, plans, expects and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. AlthoughCRISPR Therapeuticsbelieves that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: the potential for preliminary data from any clinical trial not to be indicative of final trial results; the potential that clinical trial results may not be favorable; that one or more of its internal or external product candidate programs will not proceed as planned for technical, scientific or commercial reasons; that future competitive or other market factors may adversely affect the commercial potential for its product candidates; uncertainties inherent in the initiation and completion of preclinical studies for its product candidates and whether results from such studies will be predictive of future results of future studies or clinical trials; uncertainties about regulatory approvals to conduct trials or to market products; it may not realize the potential benefits of its collaborations; uncertainties regarding the intellectual property protection for its technology and intellectual property belonging to third parties, and the outcome of proceedings (such as an interference, an opposition or a similar proceeding) involving all or any portion of such intellectual property; and those risks and uncertainties described under the heading "Risk Factors" in CRISPR Therapeutics most recent annual report on Form 10-K, quarterly report on Form 10-Q and in any other subsequent filings made byCRISPR Therapeuticswith theU.S. Securities and Exchange Commission, which are available on theSEC'swebsite atwww.sec.gov. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made.CRISPR Therapeuticsdisclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

Investor Contact:Susan Kim+1-617-307-7503susan.kim@crisprtx.com

Media Contact:Rachel Eides+1-617-315-4493rachel.eides@crisprtx.com

CRISPR Therapeutics AGCondensed Consolidated Statements of Operations(Unaudited, In thousands except share data and per share data)

Three Months Ended March 31,

2023

2022

Revenue:

Collaboration revenue

$

100,000

$

178

Grant revenue

762

Total revenue

$

100,000

$

940

Operating expenses:

Research and development

99,935

118,245

General and administrative

22,360

See the rest here:
CRISPR Therapeutics Provides Business Update and Reports First Quarter 2023 Financial Results - Yahoo Finance

Board of Regents medals awarded for teaching excellence … – University of Hawaii

The Regents Medal for Excellence in Teaching is awarded by the Board of Regents as tribute to faculty members who exhibit an extraordinary level of subject mastery and scholarship, teaching effectiveness and creativity and personal values that benefit students.

Rosanna Alegado is an associate professor of oceanography in the University of Hawaii at Mnoa School of Ocean and Earth Science and Technology (SOEST). Her work involves meaningful academic collaborations and partnerships with Indigenous communities.

She led SOESTs significant curriculum revision toward a required immersive course to ground all incoming graduate students in an understanding of working as marine biologists within Hawaiian culture. Its success has been recognized by the National Science Foundation with multi-year funding to foreground Indigenous knowledge, practices and values, and to transform and Indigenize higher education in STEM.

Alegado is regarded as an influential educator for other teaching faculty, as well as her students. She said, By challenging my students to integrate multiple didactic frameworks, one can achieve the most comprehensive understanding of a subject.

Her colleagues say that Rosie is not popular by being easy, and that her efforts are the epitome of teaching exceptionalism.

Tammy Hailipua Baker is an associate professor of theatre and dance in the University of Hawaii at Mnoa College of Arts, Languages & Letters. As a steward of Indigenous knowledge, she fulfilled that kuleana (responsibility) by building the Hawaiian Theatre Program, the only one of its kind focused primarily on performance.

A colleague, who was also her student in Hawaiian language, views the experience of acting in her productions as a master class in pedagogy. Professor Baker is continually supporting students and others in the production of 40 performers in speaking and singing lines individually and collectively. [She was] clearly the director throughout, nevertheless each actor (students) and production staff (teachers) were all made to feel their work was necessary and appreciated.

Baker is internationally recognized, the first from Hawaii to receive the Kennedy Centers Medallion of Excellence. A reviewer of her plays describes them as guides to restoring language and reclaiming the stories of generations of Indigenous populations; gifts to a culture whose language and history have been suppressed. Her transformative work shines through the passion, voice and aloha spirit of her students.

Richard C. Chen is an associate professor at the William S. Richardson School of Law at the University of Hawaii at Mnoa. He brings patience and empathy to all his interactions with students, never assuming the problem is with the students. This tenet is a teaching practice that extends into a way of modeling for the students as they enter the profession of law, as lawyers who seek to listen, learn and improve throughout their careers.

A cohort of 19 evening students for four straight semesters signed an enthusiastic letter of support for Chen, stating they collectively hope that our endorsement can begin to illuminate our appreciation of his talents as an educator and the positive impact he made during the formative stages of our legal education.

A colleague stated, Who wouldnt want to be in his classes? He is a professor whose empathy, kindness, brilliance and skill shine through in everything he does and it is elevating, inspirational and contagious.

Lincoln A. Gotshalk is a professor of kinesiology and exercise science in the University of Hawaii at Hilos College of Natural and Health Sciences. He is a musculoskeletal physiologist, anatomist and exercise physiologist with a strong background in muscular strength and power training and total body systemic response to exercise and stress.

He advises students, and teaches anatomy and physiology, research methods, nutrition and the science of diet and weight control, basic and advanced kinesiology courses, physiology of exercise and the science behind athletic training programs. Gotshalk is the director of the Laboratory for Exercise Sciences, which manages concurrent research projects.

Dr. Gotshalk most definitely has the ability to make every student feel appreciated and an important part of both the classroom and the lab group, noted a nominator. The experiences I have gained are ones I will never forget and I am thankful for all he has done to help me find my place in the UH Hilo community.

Karadeen Kam-Kalani is a professor of speech at Honolulu Community College. Her teaching philosophy recognizes that positive encouragement goes a long way in helping students gain the confidence they need to become better public speakers.

She is an inspiring and motivating instructor who strives to provide an environment for her students to foster self-discovery, steady improvement and growing confidence.

One student, self-conscious about his stutter, was nervous about taking a speech class. In Kam-Kalanis course, however, he learned to take a breath between sentences, use hand gestures to complement his talking points, and engage his audience with thoughtful questions. Her positive feedback helped him to improve his speaking capabilities.

The highlight of his learning journey came when one of his speeches was chosen as an example for other students to emulate. When he was asked how this recognition made him feel, his face lit up and he said, I felt awesome!

Tiffany-Joy Kawaguchi, serves as the program director and interim academic fieldwork coordinator in the occupational therapy assistant (OTA) program at Kapiolani Community College. Kawaguchi is an occupational therapist (OT) with more than 22 years of experience.

In 2015, Kawaguchi started a federally funded pro-bono clinic for the OTA program based on her belief that through doing, students become what they have the capacity to be. She utilizes meaningful experiences and intentional practice opportunities to help students access and then apply critical pieces of information to the OT process.

Dr. Tiff is undeniably dedicated to enabling her students to succeed, said an OTA program student. She accommodates numerous learning styles, grades fairly and offers detailed feedback so we know how to improve. Despite the endless list of things she has to do, she makes each one of us feel valued.

In 2016, Kawaguchi received the Laura N. Dowsett OT of the Year Award from the OT Association of Hawaii and was selected to represent Kapiolani CC in the inaugural Hawaii Association for Career and Technical Education Emerging CTE Leader Program in 2018. In 2021, Kawaguchi was awarded the Francis Davis Award for Excellence in Undergraduate Teaching.

Kamuela Kimokeo is the director of the Hawaii Music Institute and head of the music program at Windward Community College, where he teaches ukulele and slack key guitar. He created the groundbreaking Kaohekani Hawaiian music certificatea series of 8-week online classes taught by some of Hawaiis legendary artists.

Kimokeo shares his passion for music and instills in his students the joy of learning.

Ive come away from his courses a better musician and have a much better understanding and appreciation for the music of Hawaii, said one student. I am very proud to say that I have composed my own song.

The American Educational Research Association recently recognized Kimokeo for his research on song composition and performance as educational tools of personal empowerment. He earned his PhD in curriculum and instruction with a music emphasis, and his MEdT from UH Mnoa.

Kimokeo performs with Jerry Santos and his own N Hk Hanohano award-winning group Hiikua.

Monica LaBriola is an assistant professor of history in the University of Hawaii at Mnoa College of Arts, Languages & Letters. Her work focuses on engaging, yet challenging approaches to the area of Pacific studies, at the forefront of instructional excellence at UH Mnoa, while touching lives beyond the academic community.

At public forums and conferences, LaBriola draws diverse cultural workers passionate about the Pacific region as well as academics. Her guidance and vision on the development of resources in this area is praised by a colleague, who said that LaBriolas editorship of Teaching Oceania has impacted education across Hawaii, the Pacific, nationally and internationally.

She initiated and led two cohorts of Women in Pacific Studies, and is lauded by colleagues and students for successfully supporting the education of the student community experiencing the least educational equity at UH Mnoa and across the UH System.

A cohort member wrote, Professor LaBriola acknowledges the complexity of the university and encourages us to continue in academia while also dreaming of alternatives to knowledge production and dissemination.

Donald K. Maruyama is a culinary arts professor at Leeward Community College. Prior to joining Leeward CC as a chef instructor in 2007, he spent more than 20 years in the food and beverage industry.

He served as the culinary arts program coordinator from 2016 to 2020. For the past three years, Maruyama has been the professional arts and technology division chair, overseeing the automotive technology, culinary arts and digital media programs.

Dons strength as an instructor is his enthusiasm to share his personal experiences to his students about how true and real it is working in the industry as he does not sugarcoat, said Ron Umehira, dean of career and technical education. His strengths as a program and division colleague are his patience to listen, gather the facts, analyze and then support the best course of action.

Maruyama attended Kapiolani Community College, the University of Hawaii at Mnoa and Grinnell College. He currently serves as a Hawaii Culinary Education Foundation advisory board member, Hawaii Food & Wine Festival committee member, and on the board of directors for Hawaii Restaurant Association Education Foundation.

Summer Maunakea is an assistant professor in curriculum studies in the University of Hawaii at Mnoa College of Education. She grounds her teaching practices in academic rigor, agency and aloha. A colleague described observing her as expertly weaving place-based teaching and learning, ina (land)-based education and stewardship and Indigenous epistemology and practice.

She holds herself to high expectations as a teacher, knowing her instruction must have a positive intergenerational impact for students to grow holistically into healthy individuals capable of making pono (righteous) decisions and contributing to their communities.

For me, this is what love looks like in education, said a graduate student. The love and community that Professor Maunakea cultivates in the classroom supports immense intellectual experimentation and risk taking. I am immensely grateful for her teaching.

To a senior colleague, her teaching, research and service are considered to be visionary, meaningfully advocating for Indigenous education, sustainability, eco-justice, inclusive outdoor education and school-community partnerships.

Alexander Stokes is an assistant professor of cell and molecular biology at the John A. Burns School of Medicine at the University of Hawaii at Mnoa. They developed practices to create inclusive, rigorous classroom settings with each student fully engaged. One method, Problem-Based Learning, values students directing their own learning, developing team-learning skills and assuming very active roles in their education.

Stokes developed a tool kit for inclusive pedagogy reflecting under-represented, predominantly female, low-income, first-generation students in undergraduate classes. A student said, Professor Stokes utilizes a cutting-edge hybrid teaching style that unlocks students intellectual potential by acting as a conductor of a symphony in a collaborative learning orchestra. I was imbued with a passion and was inspired to further academic pursuits.

A colleague said, Alex is that professor, the one who transports students to a new view of themselves. Stokes is a leader in pedagogical innovation at the interface between biology, biomedicine and data science education in Hawaii.

Shawn Sumiki is an instructor of culinary arts at Hawaii Community College. Known for his outstanding work ethic, calm demeanor and generosity, Sumiki has taught at Hawaii CC since 2008 and is an alumnus of the program he now leads.

Culinary Arts students value his talent and experience, and appreciate the positive environment he creates in the program.

Chef Shawn is an incredible teacher, and I am so grateful to have him as my culinary instructor, one student wrote in support of his nomination. He creates a friendly environment around him that encourages learning and growth.

Sumiki is very supportive of campus events and collaborates frequently with community partners on opportunities that provide students with real-world experience and networking in the food and hospitality industry. He has donated his time and talent preparing meals to support disaster relief efforts on Hawaii Island. In 2019, Sumiki was honored with the Hawaii Community College Outstanding Service Award.

Maureen Mo Tabura is an assistant professor in the nursing program at Kauai Community College and has been teaching for more than 17 years. She has been the nursing program coordinator since 2016. She and Division Chair Tammie Napoleon are the face of the Kauai CC nursing program.

Professor Maureen Mo Tabura is one of a kind. Her commitment to teaching is immeasurable, said nursing student Ma Suerte Rebucal. She is not only excellent in imparting her knowledge through her life-changing lectures, but she brings out the best in us. I have encountered great lecturers as well as teachers who bring out the best in their students, but I have never seen someone who does both except for Professor Mo.

Tabura earned her BS of Nursing at the College of New Jersey, and her Masters in Nursing Education from UH Mnoa. She was a UH Community Colleges Leadership Champion from 2011 to 2012. She has been a board member of Kauai United Way since 1996, receiving the Founders Award in 2008. Tabura also served on the Kauai County Board of Ethics from 2014 to 2018.

Eli Tsukayama is an associate professor of marketing at UH West Oahu. His research focuses on understanding individual differences (e.g., personality traits) that can be used to segment and understand target markets. He has an extensive background in statistics as well as seven years of experience working in the corporate world as an Information Technology consultant.

One of Tsukayamas students said, Although I was nervous in the beginning with his reminder of how hard the course was, I decided to stick it out and Im glad I did because I learned a lot of life lessons from himto take criticism as a lesson, or how to properly ask qualitative and quantitative questions.

Tsukayama was among the authors of A megastudy of text-based nudges encouraging patients to get vaccinated at an upcoming doctors appointment. The paper was published on April 29 in the Proceedings of the National Academy of Sciences, the official journal of the National Academy of Sciences and one of the worlds most-cited and comprehensive multidisciplinary scientific journals.

Rosemary Rosie Vierra is a professor and the coordinator for UH Maui Colleges dental hygiene program. She has taught in the program since 2008. Many students attest to Vierras dedication and passion for the success of her students as evident in the hours she spends teaching, coordinating and striving to elevate the program.

One student described Vierras authentic concern for student well-being and success: She always puts the students first and makes us feel like our voices matter. She not only cares about our success but also our personal well-being. She is a big advocate for mental health, which I appreciate very much.

Vierras energy and connection with the community enable her to create enriching learning opportunities such as service learning, outreach to public high schools and partnerships with businesses and organizations that provide students valuable experiences in the field.

One student said, Since the beginning of our cohort in the fall of 2021, she has gone above and beyond for us students to succeed. Her priority is always to help us succeed, whether its volunteering to help us meet our clinical requirements to finding us patients.

Read this article:
Board of Regents medals awarded for teaching excellence ... - University of Hawaii

Advances in Cell Therapy – Technology Networks

Several cell therapies are now approved for treating human disease and many more are being developed. In this article, we review the latest research in cell therapy development, including advances made in the lab that show promise for translation to the clinic, to improvements in developing, testing and manufacturing.

Cell therapy is the transfer of cells into a patient, either cells from the patient themselves (autologous) or from a donor (allogeneic), for therapeutic purposes. Researchers have been attempting to use cells as treatments for hundreds of years, but for several decades, the only successful application was the use of bone marrow transplantation for leukemia.1 Today, key advances in understanding and genetic engineering of stem- and non-stem (somatic) cells have led to a series of cell therapy approvals. The first stem cell therapy was approved in 2014 for a rare condition called severe limbal stem cell deficiency (LSCD) caused by burns to the eye,2 and in 2017, the first chimeric antigen receptor (CAR) T-cell treatment, Kymriah (tisagenlecleucel), was approved for children and young adults with B-cell leukemia.3

Following these authorizations, the global market for cell-based therapies was valued at USD 21.6 billion in 2022 and is expected to expand to USD 62.4 billion in 2030.4 Although the largest market share is oncology, cell therapies are being explored for a wide range of other indications including central nervous system (CNS) and neurodegenerative diseases, autoimmune disease, cardiovascular disease and rare, orphan diseases.1

Cell therapies are either based on stem or somatic cells. In this article, we explore some examples highlighting both approaches, and the challenges of translating their promise in the clinic.

Do you want to discover more about CAR T-cell bioprocessing and CAR molecule design for novel therapies?Download this case study to learn about different types of cell development pipelines, key bioprocessing steps to optimize therapy development, and advances in CAR T bioprocessing and CAR molecule design.View Case Study

One of the fastest-moving areas of cell therapy is the development of CAR T cells in oncology. In this approach, T cells collected from patients or donors are engineered to express a chimeric antigen receptor that recognizes unique tumor antigens, grown in large numbers, and then administered back to patients. Despite the excitement in the field since the first approvals, there remain considerable challenges. These include the difficulty of reproducing the success seen in hematological cancers to solid tumors, and optimizing patient response in terms of the amount and duration of anti-tumor immunity. 1

For a long time, a major challenge for many of these therapies was manufacturing the cells, says David Zhang, a member of the Mooney lab at the Wyss Institute for Biologically Inspired Engineering, Harvard University. Then people figured out better ways of making lots of CAR T cells from a process perspective, with different bioreactor systems and other technological advancements. However, although it became possible to make large numbers of T cells from patient samples, there was still huge variation in individual patient responses to treatment. This led to retrospective analyses of attributes of the cells and factors during the manufacturing process that influence patient response. But the one thing that no one seemed to be looking at was how to optimize the way the T cells were getting activated in the first place, says Zhang. We knew that how we activated the cells was really important to the response we got, so we developed a system to fine tune activation parameters, and see how this affected their function.

The system is designed to mimic the function of the T-cell zone in human lymph nodes, by comprising biodegradable mesoporous silica rods that can be loaded with immune-stimulatory molecules called cytokines. The rods are coated with a lipid bilayer, to which you can attach different activating antibodies. T cells require three signals to be able to proliferate and differentiate: a T-cell receptor stimulation signal, costimulatory signals and a mitogenic growth signal, says Zhang. Our system allows T cells to naturally rearrange the artificial membranes to form an immunological synapse while supporting paracrine signaling, mimicking two important parts of physiological T-cell activation.

Zhang used this system to fine tune all the different parameters that could influence the ultimate T-cell product. But the one thing that really influenced the resulting cell phenotype and function was changing the amount or dose of T-cell stimulation the cells received.

We found that theres a quantitative relationship between your starting cell population, the amount of stimulation you provide, and the phenotype and function of the CAR T cells, says Zhang. We were able to fine-tune the levels of T-cell stimulation to match different phenotypes of the T cells obtained from patients with leukemia, which allowed us to produce CAR T cells with significantly enhanced tumor-clearing activity in a patient-specific manner.

The team used a simple machine learning model to find a pattern between these parameters, which Zhang says can easily be adapted to any starting cell population and any desired CAR T product characteristics, such as enhanced tumor memory, rapid proliferation or a durable response.5 This means that it becomes possible to fine-tune the product based on the patients original T-cell sample.

Given that T-cell samples are always phenotyped, and sometimes genotyped, at the start of manufacturing, similar strategies could be used to personalize the therapy using our approach, says Zhang. It means you can start with one donor sample, and potentially generate, for example, three different CAR T products that are better for different use cases or even different stages of the disease.

Active cell therapy drugs represent the latest in cancer treatment. Of these, chimeric antigen receptor (CAR) cell modalities are considered the most successful.Download this whitepaper to learn more about robust and reproducible methods for CAR expression detection, choosing the best reagents for your assay requirements, and next-generation CAR constructs and the future of cancer treatment.

View Whitepaper

An alternative to harvesting mature cells like T cells is to differentiate or engineer immature stem cells into your desired cell therapeutic product. One of the most active areas of research using this approach is in restoring or protecting the function of nerve cells. In Justin Ichidas lab at the University of Southern California, they are growing stem cells into different types of nerve cells to be used as treatments and for screening drugs. One of their projects, in collaboration with Jon-Paul Pepper, a specialist in facial reconstructive surgery, aimed to help people who sustained facial nerve injuries after an accident.6 These injuries often affect motor neurons in the face muscles, and although the nerve can regenerate it does so very slowly, by the time the muscle has already atrophied, explains Ichida. Because we can grow motor neurons from stem cells, we thought perhaps these cultivated cells could babysit the muscle, until the real nerve regenerates. To test this, they cut the sciatic nerve in mice and introduced motor neurons grown from induced pluripotent stem cells. The home-grown stem cells successfully extended their axons and innervated the muscle. After a couple of months, you could see there was a huge difference in preserved muscle mass, whereas the leg muscles in mice that did not receive the motor neuron therapy, completely atrophied away, says Ichida. What was really interesting, is that the nerve cells didnt require any additional stimulation to innervate the muscle in the way that a natural nerve would do.

Now, Ichida is turning his attention to another type of cell that can be derived from pluripotent stem cells, called microglia. There is growing evidence that microglia, which are the primary immune cells in the brain, play a critical role in the duration and progression of neurodegenerative diseases such as motor neuron disease (also known as amyotrophic lateral sclerosis; ALS).7 We studied stem cells derived from patients with C9orf72 ALS, the most common form, and found a neuroprotective population of microglia, says Ichida.

Although many of the microglial cells that we derived from patients are actually neurotoxic, we found that when they were grown in close proximity to a C9orf72 motor neuron, they switch into a protective state. These protective microglia do exist in the brains of ALS patients, but they are in the minority. Having made this discovery, Ichidas team transplanted the protective microglia into mice harboring a mutation that causes ALS and identified the signaling pathway that switches the microglia from a neurotoxic to a neuroprotective state. Now, they plan to engineer the cells to lock them into a neuroprotective state so they can be transplanted into patients. One hurdle is to see whether stem cells can be transplanted into the human brain, says Ichida. Its been recently shown in rodents that you can transplant microglia and that the grafts extend broadly throughout the brain, so we have some proof of concept already there.

Further proof of concept comes from another study where engineered stem cells were transplanted into the CNS and showed early clinical promise. In the first clinical trial of its kind, a team of investigators from Cedars-Sinai Medical Center demonstrated that it was possible to deliver a combined stem cell and gene therapy into the spinal cord of patients with ALS.8 The stem cells produce a protein called glial cell line-derived neurotrophic factor (GDNF) which can promote the survival of motor neurons. The trial has proved the short-term safety of the approach, and now larger numbers of patients are needed to test its efficacy.

The key in ALS, and its very similar in Parkinsons disease and Alzheimers disease, is that these are very genetically diverse diseases, and so for most patients we dont have a full understanding of the genetic causes behind each persons condition, says Ichida. What we can do is take these patients stem cells and remake their neurons in a dish. Then, because these neurons in the lab mimic the same disease process in the patient, we can reprogram them, or we can use them to test new drugs that will work. In this way, were going to find treatments that are more likely to work across the whole breadth of the ALS population.

References (Click to expand)

1.Bashor CJ, Hilton IB, Bandukwala H, Smith DM, Veiseh O. Engineering the next generation of cell-based therapeutics.Nat Rev Drug Discov. 2022;21(9):655-675. doi: 10.1038/s41573-022-00476-6

2.First stem-cell therapy recommended for approval in EU. European Medicines Agency. https://www.ema.europa.eu/en/news/first-stem-cell-therapy-recommended-approval-eu. Published September 17, 2018. Accessed March 9, 2023.

3.FDA approval brings first gene therapy to the United States. FDA. https://www.fda.gov/news-events/press-announcements/fda-approval-brings-first-gene-therapy-united-states. Published March 24, 2020. Accessed March 9, 2023.

4.Cell therapy market size & trends analysis report, 2030. Grand View Research. https://www.grandviewresearch.com/industry-analysis/cell-therapy-market. Accessed March 9, 2023.

5.Zhang DKY, Adu-Berchie K, Iyer S, et al. Enhancing CAR-T cell functionality in a patient-specific manner.Nat Commun. 2023;14(1):506. doi: 10.1038/s41467-023-36126-7

6.Pepper JP, Wang TV, Hennes V, Sun SY, Ichida JK. Human induced pluripotent stem cell-derived motor neuron transplant for neuromuscular atrophy in a mouse model of sciatic nerve injury. JAMA Facial Plast Surg. 2017;19(3):197-205. doi: 10.1001/jamafacial.2016.1544

7.Clarke BE, Patani R. The microglial component of amyotrophic lateral sclerosis. Brain. 2020;143(12):3526-3539. doi: 10.1093/brain/awaa309

8. Baloh RH, Johnson JP, Avalos P, et al. Transplantation of human neural progenitor cells secreting GDNF into the spinal cord of patients with ALS: a phase 1/2a trial. Nat Med. 2022;28(9):1813-1822. doi: 10.1038/s41591-022-01956-3

Read more:
Advances in Cell Therapy - Technology Networks

Restoring vision through retinal ganglion cell repopulation – Ophthalmology Times Europe

Ophthalmology is pioneering the field with clinical trials of stem cellderived retinal pigment epithelium and photoreceptor transplantation to potentially restore vision in diseases such as age-related macular degeneration and macular dystrophies. (Image credit freshidea / stock.adobe.com)

Ophthalmologists are all too familiar with the heartbreak that accompanies a new diagnosis of advanced optic neuropathy and the inability to offer patients treatment that can restore their vision. The unifying feature of all optic neuropathies is the death of retinal ganglion cells (RGCs), which are the projection neurons that transmit visual information from the retina to the brain via axons running through the optic nerve. Optic nerve diseases are both prevalent and irreversible; glaucoma alone affects more than 80 million individuals worldwide.

RGCs are central nervous system neurons and, like brain and spinal cord neurons, are not spontaneously regenerated following injury or insult in mammals. However, regenerative medicine advances are opening new pathways to restoring function in historically incurable neurodegenerative diseases, and ophthalmology is pioneering the field with clinical trials of stem cellderived retinal pigment epithelium and photoreceptor transplantation to potentially restore vision in diseases such as age-related macular degeneration and macular dystrophies.

Unfortunately, RGC replacement represents a more formidable challenge. Unlike photoreceptors, which comprise only four major subtypes, are intrinsically light responsive and make only a single synaptic connection to an adjacent retinal bipolar cell, RGCs are much more complex. Primates possess more than a dozen molecularly, functionally and topographically unique subtypes of RGCs that receive afferent input from complex inner retinal circuits that can include dozens of presynaptic bipolar and amacrine cells, and that must extend lengthy axons through the optic nerve and into one of several visual centres in the brain.

The list of challenges in making functional RGC replacement a reality is daunting and will require therapeutic manipulation of cellular pathways involving neuronal survival, migration, dendritogenesis and axogenesis, pathfinding, synaptogenesis and myelination. However, there is reason to be hopeful. Although the concept of optic nerve regeneration has long been the subject of fantasy, recent advances in neuroscience have converged to a point where functional RGC replacement may now be feasible.

Generation of new RGCs

The generation of induced pluripotent stem cells (iPSCs) from individual patients is now commonplace. By obtaining a skin biopsy, blood sample, or even urine specimen, it is possible to create patient-specific cell lines capable of nearly infinite expansion and to differentiate those iPSCs into a variety of specialised cells that are lost in various disease states. Indeed, scientists have developed several methodologies to create immature RGCs or even entire neural retinas (retinal organoids) from stem cells.

These advances have provided the tools necessary to begin preclinical experimentation in transplanting new RGCs into eyes with optic neuropathy. Separately, investigators have made significant advances in understanding the molecular pathways that enable lower vertebrates, like teleost fish, to regenerate their optic nerves following injury. By activating several proregenerative genes in the retinal Mller glia of adult mice, investigators have successfully coaxed these cells to proliferate and then transdifferentiate into RGC-like cells. Therefore, viable techniques to regenerate RGCs from both exogenous and intrinsic sources are available.

Integration into the retina

Placing new RGCs into the diseased retina is only the first step in functional vision restoration. Once present, RGCs must elaborate dendrites within the inner plexiform layer (IPL) and generate synapses with bipolar and amacrine cells to receive visual information that will be processed and relayed to the brain. Recent work in our laboratory has demonstrated that this does not occur spontaneously after RGCs are transplanted into the eye.

The internal limiting membrane (ILM; the basement membrane that separates the neural retina from the vitreous cavity) constitutes a barrier to the retinal transit of drugs and gene vectors, and we have shown that the ILM also inhibits the retinal integration of RGCs introduced into the vitreous cavity. By disrupting the ILM, we have been able to achieve not only migration of transplanted RGCs into the neuroanatomically relevant layer of the retina but also extension of dendrites into the IPL, where synaptogenesis can occur.

Because removal of the ILM through surgical peeling is an established technique used to treat macular holes and other vitreoretinal disorders in human patients, this finding provides a directly translatable approach to achieving retinal engraftment of transplanted neurons. Research is now poised to leverage synergy between RGC transplantation approaches and recent advances in neuroprotection to achieve greater and more widespread survival of donor RGCs across the retina, and to begin promoting dendrite extension and patterning, synaptogenesis, and integration into specific inner retinal circuits.

Eye-brain connectivity

Perhaps the most arduous challenge to RGC replacement will be promoting and guiding donor RGC axons from the retina to relevant visual processing centres in the brain, such as the lateral geniculate nucleus. Fortunately, great strides in the neuroregeneration field have been made in this area over the past 15 years. By studying endogenous RGCs injured by direct optic nerve trauma, investigators have identified several molecular pathways that can be modulated to unleash a latent regenerative capacity of mammalian RGCs. With therapeutic manipulation, mouse RGCs can regenerate axons for several millimeters past the site of an optic nerve crush.

Although many regenerating axons meander in a random fashionsometimes growing into ectopic locations such as the contralateral optic nervein some instances, these axons reach relevant visual centres in the brain and even restore rudimentary visually guided behaviors. Ongoing work to provide chemotactic guidance cues is increasing the efficiency of targeted axon regeneration, and applying these techniques to repopulated RGCs provides a promising means to regenerate the optic nerve de novoin cases of advanced optic neuropathy.

Efforts toward RGC repopulation

Although significant headway has been made to generate, protect, enhance or regenerate portions of RGCs in specific model systems, a lot of work clearly remains before a comprehensive treatment paradigm will be capable of replacing RGCs throughout the entirety of their contribution to the visual pathway. A pressing challenge is to begin consolidating the most efficacious approaches to each step of the RGC replacement process into a unified approach. Stem cell biologists are engineering RGCs with enhanced survival and integration capacity. Developmental biologists are detailing the molecular cues that drive patterning of RGC connectivity in the retina and the brain.

Cellular neuroscientists are curating the specific transcriptional and wiring patterns of RGCs and their changes in optic neuropathic disease states. Biomedical engineers are developing methods for transplanting delicate neurons on protective scaffolds and providing long-term trophic support and guidance cues to grafted cells.

Given the complexity of the task at hand, collaborative efforts among scientists with diverse expertise will be necessary to generate clinically viable methods for RGC repopulation. A group of leading investigators in the fields of ophthalmology and neuroscience has organised the Retinal ganglion cell (RGC) Repopulation, Stem cell Transplantation, and Optic nerve Regeneration consortium, which is bringing together more than 200 investigators worldwide. By fostering in-depth discussions regarding the most important obstacles to be overcome and organizing sustained collaborative research endeavors, we hope major headway will be made in the coming years to bring RGC repopulation toward the stage of clinical trial.

Read the rest here:
Restoring vision through retinal ganglion cell repopulation - Ophthalmology Times Europe

Thomas Hospital stem cells headed to International Space Station – NBC 15 WPMI

International Space Station (Photo: NASA) Thomas Hospital stem cells headed to International Space Station

Infirmary Health has announced that, through an ongoing partnership with LifeSouth Community Blood Centers, CD-34 cells (stem cells) collected from umbilical cord blood, including those collected by Megan Dimoff, M.D., at Thomas Hospital, will soon travel to the International Space Station.

LifeSouth was selected to provide these cells to Abba Zubair, M.D., Ph.D., of the Mayo Clinic for his research study aboard the Space Station. Dr. Zubair and others are studying how these stem cells will divide or expand in space versus how the same cells expand on Earth, with the ultimate goal that these cells will be used for cancer treatment.

At Infirmary Health, our mission is LIFE. We are honored to fulfill our commitment to our mission through this unique opportunity granted to Thomas Hospital and Dr. Dimoff by Dr. Zubair and his team. We are incredibly proud of Dr. Dimoff, her representation of Thomas Hospital and her passion for advancing the practice of medicine., said Ormand Thompson, president of Thomas Hospital.

LifeSouths Cord Blood Bank provides products used for this study. At Thomas Hospital and Mobile Infirmary, mothers can donate their newborns umbilical cord blood, which is made available for patients needing a stem cell transplant. If the cord blood is not viable for transplant, it can be used for medical research aimed at curing cancers, diseases and genetic disorders.

A spacecraft is scheduled to launch this month from Kennedy Space Center and will carry these cryogenically-preserved cells to the International Space Station. Crew members on board will thaw the cells and seed them into specialized hardware to allow them to expand.

For more information about the Cord Blood Bank and other birth services at Infirmary Health, visit https://www.infirmaryhealth.org/services/womens-health/birth-services/.

To view a video interview of Dr. Megan Dimoff, visit https://youtu.be/U0VogIUsXsc.

Continue reading here:
Thomas Hospital stem cells headed to International Space Station - NBC 15 WPMI

New events including ‘Be the Match’ and ‘Teddy Bear Clinic’ at … – Waukon Standard

The annual Family Wellness Fair will be held Wednesday, May 10 from 3:30-6 p.m. at Veterans Memorial Hospital (VMH) in Waukon. Health and wellness testing, exhibits, activities and refreshments will all be providing a complete evening of entertainment and education for families of all ages. This year, masks will not be required and the fair will be back to a full-size event with refreshments and taste testing. Everyone in the area communities is welcome to attend this free event.

Be the MatchNew this year, visitors to the fair can participate in the Be the Match bone marrow sampling which will be provided by the hospitals Laboratory. This is the same program that Robyn Roberts from Good Morning America endorses following her own successful bone marrow transplant match 10 years ago. A blood stem cell donation can be a cure for blood cancer, sickle cell, and other deadly diseases. Many patients and their families are counting on drives like this to find their match. The process is simple and painless and will be available for all visitors to the fair.

ExhbitorsMany exhibitors will have display tables with information, demonstrations, and activities for the entire family. To date, the exhibitors include: Allamakee County Emergency Management, Allamakee Substance Abuse Prevention, Gundersen Medical and Vision Clinic, Helping Services for Northeast Iowa, Northeast Iowa Agency on Aging, Northeast Iowa Behavioral Health, Northeast Iowa Community Action (NEICAC), Northeast Iowa Recreation Center, TASC, Village Creek Bible Camp, Waukon Good Samaritan Center, Waukon Lions Club For Kids Sight, Waukon Park and Recreation/Waukon Aquatic Center and the Waukon Wellness Center.EntertainmentEntertainment for the evening will include special appearances by Klyde Thinger the Clown and Mike Chapman, Balloon Artist. In addition, a Waukon Fire Department fire truck and Veterans Memorial Hospital ambulance will be parked outside the entrance to the fair for children to view. Face painting will again be offered this year, as well as a medical temporary tattoo table and health care career photo booth, plus many other youth games such as a bean bag toss and Operation game, among others. The Gundersen Air helicopter is also scheduled to be on-site for outdoor tours on the way to the newly remodeled Gundersen Medical Clinic where the teddy bear clinic will be held that evening, offering youth free teddy bears as they experience a medical office visit.

Health FairVMH staff will be on hand to perform a number of free health tests and share health information such as blood pressure testing, grip strength testing and childrens reach game, O2 saturation and carbon monoxide testing, the effects of smoking, diabetes and blood sugar testing, chair massages, gut health, Vitamin D and blood type testing, surgical services, correct hand washing technique and cell phone sanitation, physical therapy, safe drug disposal, advanced directives and patient portal registration, among many other topics. Again this year, Veterans Memorial Hospital staff will be demonstrating and instructing Hands Only CPR that can effectively be used by anyone, on anyone who is experiencing cardiac arrest. Information on how to Stop The Bleed with hands-on practice will also be highlighted. Hospital staff will also be offering tours of the CT machine and new Mammogram machine as well as Infusion and Outpatient area.Door PrizesOver 100 door prizes will be given away that night. All visitors to the Family Wellness Fair are eligible to win just by signing in at the registration table as they enter the event. Names will be drawn throughout the evening with all winners having their choice of door prizes.

All visitors to the Family Wellness Fair are asked to enter the fair through the Medical Clinic main entrance. The fair route will proceed through the building into the hospital, out through the ER entrance to the newly remodeled Gundersen Clinic building, back through the hospital lower level, exiting out the main entrance of the hospital. Parking will be available at both the clinic and hospital lots, as well as all street parking.

The annual Family Wellness Fair, held Wednesday, May 10 from 3:30-6 p.m., is a community wide event offering families of all ages a night of entertainment and education about the resources available to them. This fun-filled family event is free and open to all members of the community of any age. For any additional information, feel free to contact Erin Berns, Public Relations Director at Veterans Memorial Hospital at 563-568-3411.

More:
New events including 'Be the Match' and 'Teddy Bear Clinic' at ... - Waukon Standard

UFC champion Aljamain Sterling opens up about his last-ditch therapy to save title bout – 7NEWS

UFC Bantamweight Champion Aljamain Sterling has turned to unconventional medicine to help ensure that he was defending his title against rival Henry Cejudo at UFC 288 on Sunday.

The New York native underwent stem cell therapy to help fix a torn bicep, which he had battled through two title bouts with.

WATCH THE EXCLUSIVE INTERVIEW: Sterling discusses his stem cell therapy.

Stream ONE Fight Night, ONE Friday Fights events and get access to highlights and ONEs library archive of more than 100 past events for free on 7plus >>

The Funkmaster says the move was crucial in allowing him to fight this weekend after already having to delay the fight which was earmarked for March.

It was very important helping me with my injury. It was either that or get surgery, he told 7NEWS.com.au.

I thought the bicep was going to heal up on its own, but it didnt.

We tried the PT (physical therapy) route, but we were trying PT for about a year, and it didnt heal up in time.

Staring down a potential surgery that could delay his timeline even further, Sterling scrambled to get a booking.

Stopping short of going under the knife, he decided that a stem cell therapy clinic in Colombia was his best bet.

With only a few months notice, the tight turnaround made it difficult for the American to get a booking.

The earliest they could have gotten me in was like late March, Aljamain Sterling said.

If that was the case, I thought I might as well get the surgery because it was going to too late (to fight in May).

Thankfully, we had some people from the UFCPI pull some strings.

Shout out to Heather, she was able to move some things around with the coordinators over there and get me in earlier.

There he underwent state-of-the-art stem cell procedures for his bicep, along with other niggling injuries.

I think it has helped a ton. Im feeling good. Im using it pretty well, he said.

Theres still some pain here and there, but its way better than before, and hopefully it is enough to get me through this fight and get my hand raised.

After his success with the stem cell therapy, the UFC champion believes that it should be more widely available throughout the US and other western countries.

In particular, Sterling lauded Bioxcellerator and clinics like it around the world.

Oh, 100 per cent, Sterling replied.

We have it here (in the US), but its nowhere near the same level as they have in Colombia.

The culturing, the way they do it. Its just the next level over there at Bioxcellerator, and they do a good job.

I think thats why theyre one of the best locations to go to.

When you go in the approach of the facility, the hospitality, the cleanliness of the place, its a really top-notch and I look forward to going back.

READ MORE

Read more from the original source:
UFC champion Aljamain Sterling opens up about his last-ditch therapy to save title bout - 7NEWS

On the horizon: Treatments for both forms of AMD – Ophthalmology Times Europe

More therapies are available for wet age-related macular degeneration, but research also is focused on the dry form of the disease.

Once there were none, but now there are many treatments for age-related macular degeneration (AMD). By far, more therapies are available for wet AMD, but research also is focused on dry AMD. The goal is to effectively reduce the treatment burden in addition to improving and preserving vision.

Dr Katherine Talcott highlighted the more recent therapeutic additions to the market. She is a staff surgeon at the Cleveland Clinic Cole Eye Institute and an assistant professor of ophthalmology at the Cleveland Clinic Lerner College of Medicine, Cleveland, Ohio, US.

Neovascular AMD therapies

Anti-vascular endothelial growth factor (VEGF) drugs are the first-line treatments for neovascular AMD, which have been effective in improving vision in randomised clinical trials.

However, Dr Talcott noted data from real-world studies illustrate that it is difficult to achieve good long-term outcomes. This can happen because of undertreatment resulting from the treatment burden on patients, caregivers and clinical practices.

The hope for the FDA-approved port delivery system (Susvimo implant, ranibizumab injection; Genentech), a permanent, refillable, surgically implanted device that continuously releases treatment, was to alleviate the treatment burden of patients with neovascular AMD. Susvimo is under a temporary recall because of dislodgement of the internal structures of the device.

Faricimab (Vabysmo; Genentech) is a bispecific antibody that is injected intravitreally and inhibits both VEGF-A and ANG2. In clinical trial results, the visual acuity gains with faricimab were equivalent to those achieved with aflibercept (Eylea; Regeneron Pharmaceuticals). One distinct advantage is that fewer injections are needed with faricimab compared with aflibercept, a finding that suggests the drug may be more durable.

The holy grail in the treatment of neovascular AMD is a one-and-done treatment for the disease and this is where gene therapy may play a role. RGX-314 (ReGenXBio) is a one-time surgically implanted subretinal gene therapy to encode for an anti-VEGF protein. The phase 1/2 trial results were promising, Dr Talcott reported, and the implant is being evaluated in two phase 3 trials, ATMOSPHERE (NCT04704921) and ASCENT (NCT05407636). This is a promising option, she said, and noted the importance of the availability of longer-acting options on the horizon.

Dry AMD therapies

Geographic atrophy (GA), the end stage of dry AMD, has become a major research target for which, until recently, no treatments were available. Most of the current therapies being evaluated are complement inhibitors of C3 and C5, which are important factors in development of inflammation in the eye and ultimately cell death.

Pegcetacoplan (Syfovre; Apellis), a complement C3 inhibitor, was approved in February 2023 by the US Food and Drug Administration to treat GA. A phase 2 study showed that the drug slowed progression of GA, which led to the DERBY (NCT03525600) and OAKS (NCT03525613)studies, the primary end point of which is the change in the total area of the GA lesions at 12 months. At the 18-month time point, data from both studies showed significant changes in the rate of growth of the GA in the patients receiving active treatment. There were no functional improvements in vision seen with the therapy.

Another drug under study is avacincaptad pegol (Zimura; Iveric Bio), a C5 inhibitor in the GATHER 1 and 2 studies. The end point of both studies was the rate of growth of GA, which was shown to slow with treatment.

With both investigational drugs, there is a higher rate of conversion to neovascular AMD compared with sham treatment.

Gene therapies also may have a role in controlling GA. Gyroscope Therapeutics is looking at a complement factor I, GT005, in the FOCUS study (NCT03846193), in which the gene therapy is delivered into a bleb created in the subretinal space.

Neovascular AMD and GA both result in irreversible vision loss that require effective treatments to reduce individual and societal treatment burdens. Many studies are ongoing and investigating a number of therapeutic options. There also may be a role in the future for potential surgical innovations that may help address the treatment burden, Dr Talcott concluded.

Originally posted here:
On the horizon: Treatments for both forms of AMD - Ophthalmology Times Europe

The miracle and gift of organ donation – The Tryon Daily Bulletin – Tryon Daily Bulletin

Published 11:36 am Monday, May 1, 2023

Ive worked in healthcare my entire adult life and remain amazed by advances in medical technology, new medicines, and contemporary surgical procedures.

I served as a dialysis nurse at a hemodialysis clinic early in my nursing career. Caring for my patients three times a week for several hours of treatment, I often became close to them. One of the most exciting times during this position was when a patient on the transplant list had a beeper go off! It was a huge celebration and a rush to quickly remove them from dialysis and get them to the transplant center!

In April, we celebrated National Donate Life Month. Through this article, I hope to catch your attention on the life-saving possibilities of organ transplantation and the dire need for organ donors.

At any given time, more than 105,000 people in the U.S. are on a transplant list. The need is great, yet the pool of available healthy organs is small. Every nine minutes, another person is added to the list, while seventeen people die each day waiting for an organ match. For each organ/eye/tissue donor, we can heal or save more than seventy-five lives.

GAME-CHANGING ADVANCEMENTS

Post-Circulatory Death Donation:Historically, donated organs came from donors who died from brain death while the heart continued to beat. Medical advances now allow the transplantation of organs where the heart has stopped beating. As a result, up to thirty percent of all organ donations now come from such donors.

Organ Perfusion Systems:Mechanical devices called organ perfusion systems help organs remain viable at body temperature outside the body. For example, the heart in a box is a technology that resuscitates a stopped heart and can keep it beating for up to eight hours before its transplanted. There are similar devices that preserve lungs and livers outside the body. The new perfusion technology has doubled the transplant window.

Organs from Hepatitis C-positive Donors:Organs from hepatitis C-positive donors were not considered viable. But because of a new generation of antiviral medications, these organs are now safely transplanted into patients. After the organs are transplanted, patients begin antiviral therapy that typically eliminates the virus from the body within seven days.

Stem Cell Therapy:Since its inception in the 1980s, stem cell research has advanced significantly, and today we are on the cusp of miraculous developments. The patients stem cells divide into daughter cells, which can become new stem cells or evolve into any specialized cell in the body. Under the right conditions, stem cells can replace damaged cells. Researchers now believe stem cells can grow new organs.Think about the magnitude of this promise!

In theory, organs grown from the patients stem cells would be accepted by the body, eliminating the need for anti-rejection medication and reducing the overall risk of transplantation. Other applications of stem cell technology include treating disease and genetic conditions. For example, weve learned from one study that manipulated bone marrow cells transplanted into two young boys halted the growth of a fatal brain disease called adrenoleukodystrophy. Countless promising applications suggest stem cell therapy is the future of medicine.

HOW YOU CAN HELP

Locally, we work with LifeShare Carolinas. Their position statement is Honoring the wishes of organ, eye, and tissue donorsgiving hope, life, and healing. I share this information to ask that you consider becoming an organ donor and communicating those wishes to your family.

Your donation can help save lives. And your financial gifts help fund public education to inspire more people to give the gift of life and to support donor families through the Bridge to Healing program.

If you have a healthcare topic of interest or want to learn more about St. Lukes Hospital, send me a note at Michelle.Fortune@slhnc.org. Also, please follow us on Facebook, Twitter, and LinkedIn, or visit our website at StLukesNC.org.

Excerpt from:
The miracle and gift of organ donation - The Tryon Daily Bulletin - Tryon Daily Bulletin

As Precision Medicine is critical, we cannot think about one size fits … – BSA bureau

South Korea-based GPCR Therapeutics is a venture-backed, clinical-stage international biopharmaceutical company with an innovative approach to developing therapeutics built on its proprietary G protein-coupled receptors (GPCRs) data. The companys proprietary data-driven approach has identified over 1,000 GPCR pairs upon which drug screening campaigns can be pursued. In an email interview with BioSpectrum Asia, Dr Pina Cardarelli, Chief Scientific Officer, GPCR Therapeutics explains more about their unique approach and how GPCR-targeted therapies may change the cancer treatment landscape.

Can you explain what GPCR heteromers are and why they are a promising target for cancer therapeutics?

Before I answer your question about heteromers, let me explain what GPCRs are first. GPCRs represent the largest protein family that consists of 7 transmembrane proteins located on the cell membrane. They transmit chemical signals from extracellular stimuli resulting in key physiological effects. Their endogenous ligands include hormones, neurotransmitters and chemokines.

GPCRs have been implicated in many diseases, such as type 2 diabetes mellitus (T2DM), Alzheimers disease, and cancer as well as many others. As of 2020, 142 compounds were in clinical trials targeting 83 different GPCRs (19 are novel GPCRs with no previous FDA-approved drugs).

Based on technology from Seoul National University that has been pursued at GPCR Therapeutics, Inc. we know distinct GPCRs can associate with other GPCRs forming heteromers. When both ligands are present, they amplify the signal induced by each ligand-receptor pair alone. Using a bimolecular fluorescence complementation (BiFC) technique, the team discovered over 1000 interactors of two distinct GPCRs coming together to form a heteromer.

The team then focused on one GPCR called CXCR4, which plays a critical role in cancer progression. CXCR4 drives cell migration, metastasis, angiogenesis, proliferation and survival. Multiple CXCR4 inhibitors have failed in the clinic; however, we believe a blockade of multiple pathways could be the key to getting a sustained therapeutic benefit.

Can you walk us through your drug discovery and development process, from identifying potential targets to clinical trials?

We identified over 40 GPCRs that associate with CXCR4. We then prioritised the list to GPCRs where the ligand was known. This allowed us to perform functional assays like calcium flux assay to determine if we see additive or synergistic effects. The top selected GPCR that was selected was the beta 2 adrenergic receptor (2AR).

So why 2AR? Many similar qualities are shared with CXCR4. Interestingly, this target also plays an important role in cancer. Firstly, the ligands for this target, Epinephrine (Epi) and Norepinephrine (NE) are induced under stress. Patients diagnosed with cancer experience significant stress, and this stress promotes tumorigenesis, proliferation, angiogenesis and immunosuppression. For their immunosuppressive role, Epi and NE induce suppressive immune cells, such as M2 or MDSCs in tumour microenvironments. In addition, a number of studies have shown that cancer patients on non-selective beta-blockers, such as propranolol, have improved PFS and OS.

While propranolol was commercially available, it was critical that we acquire a CXCR4 inhibitor and preferably one that had been tested clinically. We acquired Burixafor, now known as GPC-100, from a company called Taigen. A review of their clinical data showed exciting safety and efficacy in stem cell mobilisation. From our in vivo stem cell mobilisation studies, we found the addition of propranolol improved the mobilisation of cells driven by GPC-100. Concurrently, we embarked on the synthesis of a GMP-grade new drug product, identified a clinical CRO and initiated a clinical trial design.

What specific types of cancers are you currently targeting with your therapeutics, and what progress have you made in these areas?

Because the US clinical study previously conducted in stem cell mobilisation in a number of hematologic malignancies showed promising data, we decided to focus on Multiple Myeloma. Currently, the trial is a two-arm study with each arm having 20 patients. In the first arm, patients will be treated with a low dose of propranolol and GPC-100. The second arm is a triple combination which includes GPC-100, propranolol and G-CSF. We are excited to report that our first clinical site was activated on February 14, 2023. With respect to future trials, GPC-100 plus propranolol can be extended to stem cell mobilisation in NHL or used as a chemosensitiser in AML. Finally, if our arm that omits G-CSF is successful, this gives us an opportunity to work on diseases where G-CSF is contraindicated, like Sickle Cell Disease.

How does your company plan to address potential challenges or roadblocks in bringing GPCR heteromer-targeted therapeutics to market?

We have recently published a paper in scientific reports, meaning that our co-targeting drug development strategy has been peer-reviewed and validated in academia. We plan to continue our work and expand our pipeline by targeting the interaction of CXCR4 and other GPCRs as well.

If our Phase 2 study is successful, it will increase the likelihood of finding a partner to collaborate with us in further developing this programme and bringing our therapeutics to market.

With whom have you partnered to further your mission and research?

A recent partnership between AdAlta and GPCR Therapeutics was established to evaluate a new cancer treatment approach combining beta blockers plus AdAltas CXCR4-inhibiting i-bodies. AdAlta is a clinical-stage drug discovery company developing novel therapeutic products from its i-body platform and we will evaluate AdAltas CXCR4 inhibiting ibodies as cancer therapeutics, using GPCR Therapeutics proprietary combination inhibition approach.

Can you speak to any recent milestones or successes that your company has achieved in its mission to develop cancer therapeutics targeting GPCR heteromers?

In the past seven months, we have opened up our own independent US laboratory site in the San Francisco Bay area. As mentioned above, we have recently published one paper which supports our heteromer hypothesis with another GPCR known as histamine receptor H1 (HRH1). HRH1 is widely expressed in various cancers, and, like ADRB2, the level of co-expression of CXCR4 and HRH1 is related to poor prognosis, in this case, in breast cancer patients. The simultaneous expression of both receptors leads to the formation of the CXCR4-HRH1 heteromer, and this complex demonstrates enhanced signalling and migration capabilities. We have also submitted an additional manuscript that provides evidence for ADRB2 and heteromer formation in cancer cell lines endogenously expressing each receptor using a time-resolved fluorescence spectroscopy technique. Of course, our biggest milestone was conducting the numerous steps that are required to initiate a clinical trial. The tremendous work that ultimately led to proceeding into the clinic with our first site activation in February, was incredibly rewarding.

How do you envision the future of cancer treatment and the role that GPCR-targeted therapies may play in this landscape?

As we progress forward, we cannot think about one size fits all. Precision medicine is critical. With the developments in AI, I believe that we will be able to genetically determine the profile of the tumour, and integrate that information with multiple databases leading to more precisely treating the patients with the correct drugs. What is also critical is to determine what combinations are appropriate. Since I was intimately involved in the rise of checkpoint inhibitors, I see their success but also am clear that not all tumours respond so we need to determine what is the best approach for these patients. I also suspect that CAR-T will make great advances eventually leading to more cures. Currently, there is a long delay to produce the cells and the side effect profile is not ideal. Hopefully, we will see advancement in allogeneic CAR-T or CAR-NK.

Because I have spent most of my career working on biologics, one area that I am particularly interested in is leading the company into this space. We only have one approved antibody drug targeting GPCR for cancer treatment. I see significant opportunities for us to consider bispecific antibodies that target two GPCRs or one antibody that can block the formation of the two GPCRs and thereby block signal crosstalk. To demonstrate our commitment to this area, we have recently hired an expert antibody engineer to move us quickly into this space.

Ayesha Siddiqui

Visit link:
As Precision Medicine is critical, we cannot think about one size fits ... - BSA bureau