Gamida Cell Announces Publication in Blood, the Journal of the American Society of Hematology, of the First Pivotal Trial to Evaluate a Cell Therapy…

BOSTON--(BUSINESS WIRE)--Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy company committed to cures for blood cancers and serious hematologic diseases, today announced that the results of a Phase 3 clinical study of omidubicel have been published in Blood, the official journal of the American Society of Hematology. Omidubicel is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell transplant solution for patients with hematologic malignancies.

The results demonstrate that transplantation with omidubicel leads to faster neutrophil and platelet recovery compared to a standard umbilical cord blood graft, and results in fewer early bacterial and viral infections and less time in the hospital.

We are pleased that the data from this well-conducted international Phase 3 trial have been published in Blood, the highly respected, peer-reviewed journal of the American Society of Hematology, said Ronit Simantov, M.D., chief medical officer of Gamida Cell. The robust results of this clinical trial have demonstrated that omidubicel could provide an important new option for patients with hematologic malignancies in need of a bone marrow transplant.

Data from this study were previously presented at the Transplantation & Cellular Therapy Meetings of the American Society of Transplantation and Cellular Therapy and Center for International Blood & Marrow Transplant Research, and most recently during the Presidential Symposium at the 47th Annual Meeting of the European Society for Blood and Marrow Transplantation. The pivotal study was an international, multi-center, randomized Phase 3 trial designed to compare the safety and efficacy of omidubicel to standard umbilical cord blood transplant in patients with high-risk hematologic malignancies undergoing a bone marrow transplant.

Previous studies have shown that engraftment with omidubicel is durable, with some patients in the Phase 1/2 study now a decade past their transplant. The Phase 3 data reinforce omidubicels potential to be a new standard of care for patients who are in need of stem cell transplantation but do not have access to an appropriate matched donor, said Mitchell Horwitz, M.D., lead author of the paper and a professor of medicine at the Duke Cancer Institute.

The full Blood manuscript is available here: https://ashpublications.org/blood/article/doi/10.1182/blood.2021011719/476235/Omidubicel-Versus-Standard-Myeloablative-Umbilical.

Details of Phase 3 Efficacy and Safety Results Shared in Blood

The intent-to-treat analysis included 125 patients aged 1365 years with a median age of 41. Forty-four percent of the patients treated on study were non-Caucasian, a population known to be underrepresented in adult bone marrow donor registries. Patient demographics and baseline characteristics were well-balanced across the two study groups. Patients with acute lymphoblastic leukemia, acute myelogenous leukemia, chronic myelogenous leukemia, myelodysplastic syndrome or lymphoma were enrolled at more than 30 clinical centers in the United States, Europe, Asia, and Latin America.

Gamida Cell previously reported in May 2020 that the study achieved its primary endpoint, showing that omidubicel demonstrated a statistically significant reduction in time to neutrophil engraftment, a measure of how quickly the stem cells a patient receives in a transplant are established and begin to make healthy new cells and a key milestone in a patients recovery from a bone marrow transplant. The median time to neutrophil engraftment was 12 days for patients randomized to omidubicel compared to 22 days for the comparator group (p<0.001).

All three secondary endpoints, details of which were first reported in December 2020, demonstrated a statistically significant improvement among patients who were randomized to omidubicel compared to patients randomized to standard cord blood graft. Platelet engraftment was significantly accelerated with omidubicel, with 55 percent of patients randomized to omidubicel achieving platelet engraftment at day 42, compared to 35 percent for the comparator (p = 0.028). Hospitalization in the first 100 days after transplant was also reduced in patients randomized to omidubicel, with a median number of days alive and out of hospital for patients randomized to omidubicel of 61 days, compared to 48 days for the comparator (p=0.005). The rate of infection was significantly reduced for patients randomized to omidubicel, with the cumulative incidence of first grade 2 or grade 3 bacterial or invasive fungal infection for patients randomized to omidubicel of 37 percent, compared to 57 percent for the comparator (p=0.027). Additional data reported in the manuscript included a comparison of infection density, or the number of infections during the first year following transplantation, which showed that the risk for grade 2 and grade 3 infections was significantly lower among recipients of omidubicel compared to control (risk ratio 0.5, p<0.001).

Data from the study relating to exploratory endpoints also support the clinical benefit demonstrated by the studys primary and secondary endpoints. There was no statistically significant difference between the two patient groups in incidence of grade 3/4 acute GvHD (14 percent for omidubicel, 21 percent for the comparator) or all grades chronic GvHD at one year (35 percent for omidubicel, 29 percent for the comparator). Non-relapse mortality was shown to be 11 percent for patients randomized to omidubicel and 24 percent for patients randomized to the comparator (p=0.09).

These clinical data results form the basis of a Biologics License Application (BLA) that Gamida Cell plans to submit to the U.S. Food and Drug Administration (FDA) in the fourth quarter of 2021.

About Omidubicel

Omidubicel is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplants for patients with hematologic malignancies (blood cancers), for which it has been granted Breakthrough Status by the FDA. Omidubicel is also being evaluated in a Phase 1/2 clinical study in patients with severe aplastic anemia (NCT03173937). The aplastic anemia investigational new drug application is currently filed with the FDA under the brand name CordIn, which is the same investigational development candidate as omidubicel. For more information on clinical trials of omidubicel, please visit http://www.clinicaltrials.gov.

Omidubicel is an investigational therapy, and its safety and efficacy have not been established by the FDA or any other health authority.

About Gamida Cell

Gamida Cell is an advanced cell therapy company committed to cures for patients with blood cancers and serious blood diseases. We harness our cell expansion platform to create therapies with the potential to redefine standards of care in areas of serious medical need. For additional information, please visit http://www.gamida-cell.com or follow Gamida Cell on LinkedIn or Twitter at @GamidaCellTx.

Cautionary Note Regarding Forward Looking Statements

This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995, including with respect to the potential for omidubicel to become a new standard of care and the anticipated submission of a BLA for omidubicel, which statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to Gamida Cells ability to prepare regulatory filings and the review process therefor; complications in Gamida Cells plans to manufacture its products for commercial distribution; and clinical, scientific, regulatory and technical developments. In light of these risks and uncertainties, and other risks and uncertainties that are described in the Risk Factors section and other sections of Gamida Cells Annual Report on Form 20-F, filed with the Securities and Exchange Commission (SEC) on March 9, 2021, as amended on March 22, 2021, and other filings that Gamida Cell makes with the SEC from time to time (which are available at http://www.sec.gov), the events and circumstances discussed in such forward-looking statements may not occur, and Gamida Cells actual results could differ materially and adversely from those anticipated or implied thereby. Any forward-looking statements speak only as of the date of this press release and are based on information available to Gamida Cell as of the date of this release.

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Gamida Cell Announces Publication in Blood, the Journal of the American Society of Hematology, of the First Pivotal Trial to Evaluate a Cell Therapy...

More than 800 medicines are in development for diseases that disproportionately affect racial and ethnic communities – PRNewswire

WASHINGTON, June 22, 2021 /PRNewswire/ -- We are in a new era of medicine where groundbreaking biopharmaceutical research and development is transforming medicine, but these innovations are meaningless if they don't reach patients, including those in underserved communities. Health disparities are not new, but the COVID-19 pandemic put a spotlight on long-standing health inequities that affect diverse racial and ethnic communities in America. Data shows these populations have been disproportionately impacted by COVID-19. In fact, American Indian/Alaskan Native, Hispanic, and Black populations are approximately twice as likely to die from COVID-19, as compared to non-Hispanic whites.

Researchers have found that people with certain health conditions, including chronic conditions such as Alzheimer's disease, certain cancers, chronic kidney disease, chronic lung diseases, type 2 diabetes, heart conditions, HIV infection, liver disease, obesity, sickle cell disease and stroke, are at higher risk of severe illness or death from COVID-19. Many of these conditions are tied to health disparities that disproportionality affect racial and ethnic communities for genetic and environmental reasons, or due to inequities in social and economic conditions.

Today, PhRMA released a new report exploring the 829 medicines in development that aim to address the diseases and conditions that affect racial and ethnic communities at a higher rate and are also associated with worse COVID-19 outcomes.

Among the medicines in development to improve management of these diseases are:

It is critical that all patients, including historically underserved racial and ethnic communities, have access to medicines. One way to reduce barriers to health care access and enable everyone to benefit from new medicines is to ensure that clinical trials are diverse and inclusive and include participants representative of the population the medicine intends (or aims) to treat. The biopharmaceutical industry has been working with patients, communities, regulatory authorities, health care practitioners, academics and policymakers to enhance diversity in clinical trials, so the clinical trial population testing medicines better reflect the patients that will use the new therapies and medicines should they are approved by the U.S. Food and Drug Administration.

To this end, PhRMA and its member companies have voluntarily adopted first-ever industry-wide principles on clinical trials diversity, adding a new chapter to the already existing Principles on Conduct Clinical Trials & Communication of Clinical Trial Results.The new clinical trial diversity principles are designed to build trust, reduce barriers to clinical trial access, enhance an understanding of drug effects in diverse patient populations, and promote the sharing of information on policies and practices to increase clinical trial diversity.

Equity is critical to the health and well-being of diverse racial and ethnic communities, and it remains essential to a robust ecosystem of innovation. America's biopharmaceutical companies are pushing for necessary systemic and long-term change to better meet the needs of underserved communities in America.

To learn more about the PhRMA Equity Initiative and PhRMA's commitment to inclusion, visit https://phrma.org/Equity and tune in to The Atlantic's Health Equity Summit where PhRMA's Chief Operating Officer, Lori Reilly, and Genentech's Chief Diversity Officer, Quita Highsmith, will have a conversation about building trust in clinical trials.

Learn more about the medicines in development to address health equity here.

This post originally appeared on the Catalyst blog.

CONTACT:Andrew Powaleny,[emailprotected], 202-835-3460

SOURCE Pharmaceutical Research and Manufacturers of America (PhRMA)

https://phrma.org/

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More than 800 medicines are in development for diseases that disproportionately affect racial and ethnic communities - PRNewswire

Andrs Garca Receives Distinguished Professor Award | Research – Research Horizons

Vision. Collaboration. Innovation. The qualities for which Georgia Tech has become so well-known were embodied in people like Bob Nerem, founding director of the Parker H. Petit Institute for Bioengineering and Bioscience (IBB) from 1995 to 2009, Parker H. Petit Distinguished Chair for Engineering in Medicine, and Institute professor emeritus until his death in March 2020.

In 1997 Nerem recruited Andrs Garca and his wife, Michelle LaPlaca, to join the pioneering IBB program at Tech after they completed their work as postdoctoral fellows at the University of Pennsylvania his in cell and molecular biology, hers in neurotrauma.

In 1998 when Garca and LaPlaca joined Tech, IBB launched its National Science Foundation Engineering Research Center in Tissue Engineering with Emory University, making it a strategic community for Garca to join to start his research program in an emerging field. Now as executive director of IBB and a Regents Professor in the Woodruff School of Mechanical Engineering, Garca is continuing Nerems legacy of vision, collaboration, and innovation in everything he does. In recognition of his work, he is the 2021 recipient of the Class of 1934 Distinguished Professor Award, the highest honor given to a Georgia Tech professor. The award is presented to a professor who has made significant, long-term contributions to teaching, research, and public service.

Known as a global pioneer in developing biomaterials systems for translational applications in regenerative medicine, Garca holds more than a dozen U.S. patents. Discoveries include the development of hydrogels for protein and cell delivery in regenerative medicine, engineering biofunctional materials to improve islet survival, and the design of infection-fighting materials. His research focuses on creating an engineered class of materials that can be used for applications to transplant a graft without immune-suppressive drugs. Human studies are planned to start next year. Researchers in his lab are developing new ways to treat Type 1 diabetes, eventually working with adult stem cells to reprogram them into insulin-producing cells. Future applications include addressing kidney failure and other diseases.

Creating Opportunities for Collisions

Garca is enthusiastic about his research, as well as all of the collaborative research in IBB. IBB is a fantastic community of faculty, trainees, and staff who come together in making discoveries and developing the technologies in bioengineering and bioscience that will change the world, he said. His goal is that IBB will continue to expand research and integrative opportunities to have a major economic impact, creating an environment to translate research into commercial products and therapies. With IBB we want to provide opportunities for collisions, unexpected interactions that lead to the discoveries. It was Bob Nerems vision to drive that sort of collaboration, he said.

Garca shared an example of one such collision: As part of a grant from the Juvenile Diabetes Research Foundation (JDRF), I was required to present unpublished research progress at a meeting with other researchers from throughout the country. After I made my presentation that morning, a JDRF director announced that for the next three-year cycle of funding we would need to collaborate with someone in the room on research. We went to lunch, and as I was building my sandwich, an immunologist introduced himself to me, complimented me on my presentation, and asked me if I thought I could develop a biomaterial to deliver the particular protein he was working with. You never ask an engineer if they think they can do something. Theyll find a way. I said I could, and we started working together.

An elected member of both the National Academy of Inventors and the National Academy of Engineering, Garca has established three startup companies in the past seven years. He has received numerous awards for his teaching and research and has published more than 230 peer-reviewed papers in prestigious journals.

Mentoring Students

Garca has supervised 15 postdoctoral researchers and advised/co-advised 37 Ph.D. students. He is known for his long-term commitment to his trainees, as well as mentoring students outside of his laboratory and classroom. While he has not taught for the past three years because of his responsibilities as IBB executive director, he still mentors students in his lab.

I take my responsibility as a mentor and supervisor seriously. It is important to have one-to-one interactions, Garca said. I take a practical approach and feel it is critical to explain why learning a topic is important, sharing practical applications, and offering experiential hands-on learning. I have had very supportive and engaged mentors and would like to pass that on to others.

Background

A native of Puerto Rico, Garca originally came to the states to study at Cornell University. He was very interested in the emerging field of biomedical engineering, but his father, an industrial engineer, advised him to major in another engineering discipline as a backup in case the biomedical field didnt develop as anticipated. Garca took his fathers advice, earning his bachelors degree in mechanical engineering and also taking biology and bioengineering classes.

During his senior year Garca participated in a project to design a structure to support fractured legs for horses. He worked to optimize the way a boot attached to the bone so that it wouldnt fracture again. He became interested in research, and his professors recommended that he go to graduate school. He earned his masters and Ph.D. in bioengineering from the University of Pennsylvania. Garca was the first person in his immediate family to earn a doctoral degree.

Garca and his family have embraced all things Georgia Tech. He and LaPlaca have two sons, Rafael, a Tech mechanical engineering (ME 2018) graduate working at GTRI, and Andrs, a fourth-year mechanical engineering student at Tech. They hold season basketball and football tickets. One of their dogs is named Buzz.

Garca said he was deeply honored, humbled, and shocked when Georgia Tech President ngel Cabrera called and told him he had been selected for this years Distinguished Professor Award. The award is special to me because it reflects the great contributions my friends, family, and peers have made in my life to get me to this point. I am grateful for my trainees, my collaborators, and colleagues, and for the support that Georgia Tech has provided in giving me the tools to succeed. Georgia Tech is the best, Garca said.

Quotes From Colleagues and Former Students

Professor Garca has been an integral part of growth of the international reputation of our bioengineering program and the Institute for Bioengineering and Bioscience. Having seen the sustained impact that he has had on students from K-12 (Project Engages) through graduate students, he is a remarkable educator who I feel is well deserving of this award.

Sam Graham Eugene C. Gwaltney Jr. School Chair in Mechanical Engineering Georgia Tech

He remains on my short list of speakers because I resonate so strongly with his approach very deep technical skills, outstanding problem definition, and tremendous colleague in service and collegiality. He is also a terrific mentor, and his former lab members are stars. He cares about doing great science and teaching people what he learned. Andrs Garca is a gem at Georgia Tech, and as an alum I hope you can keep him there he is doing some of the best biology on campus and is a superb attractor of the best students from MIT.

Linda G. Griffith S.E.T.I. Professor of Mechanical and Biological Engineering Director, MIT Center for Gynepathology Research Chair, MIT Biological Engineering Undergraduate Programs Committee

The lab around Professor Garca performs research at a unique broadness and depth. His remarkable combination of professional and personal skills is the key for his success and makes him a highly estimated collaboration partner for other scientists across disciplines and continents. He is the most invited American scientist at plenary lectures in European conferences on biomaterials. This is not only due to the high quality of his work, but also to his ability as a communicator and active discussion partner, his openness to address new topics in collaboration, and his passion for science and education that truly inspires and motivates young researchers.

Arnzazu del Campo Director INM-Leibniz Institute for New Materials Professor, Materials Synthesis, Saarland University

The five years that I spent in Andrs lab were transformative for me, and the influence of that experience is difficult to put into words. Andrs taught me many things how to be a scientist; how to develop creative and impactful ideas; how to execute on those ideas; how to write; how to present, etc. But more important than all the technical aspects of what I learned from Andrs, I learned from him who I wanted to be. Most of my professional life, and much of my personal life, is modeled after what I have learned from watching Andrs as a professor, colleague, friend, father, and husband.

Charles Gersbach Professor, Department of Biomedical Engineering Director, Center for Biomolecular and Tissue Engineering Director, Center for Advanced Genomic Technologies Duke University

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Andrs Garca Receives Distinguished Professor Award | Research - Research Horizons

Local foundation awards $1.25 million to MIND Institute to study rare genetic condition – UC Davis Health

The RDM Positive Impact Foundation is funding an ambitious $1.25 million research project at the UC Davis MIND Institute to study SYNGAP1. The rare genetic condition causes seizures (epilepsy), intellectual disability and developmental delays. It is also highly associated with autism; about half of all SYNGAP1 patients have an autism diagnosis.

A staff member conducts research in the Segal Lab.

Ron Mittelstaedt and his wife, Darin, who live in El Dorado Hills, run the foundation. Hes the executive chairman of Waste Connections, a solid waste and recycling company with 20,000 employees in North America, and his family operates Toogood Estate Winery in Somerset. The Mittelstaedts have donated millions of dollars to organizations that help children over the past 15 years.

This time, its personal.

About three and-a-half years ago, Ron Mittelstaedts best friend died, leaving behind three sons and their families. Ive become sort of a surrogate dad, and now a surrogate grandfather, explained Mittelstaedt. One of those grandsons was diagnosed with a SYNGAP1 mutation a year and-a-half ago. With the familys support, Mittelstaedt is providing meaningful funding to the MIND Institute to advance research about the syndrome.

The reality is, like many rare conditions, there arent a lot of great options. So, were trying to find potentially life-changing treatment that hopefully may impact the lives of people with SYNGAP1, he said.

Mittelstaedt was previously on the MIND Institutes inaugural National Council of Visitors (then called the MIND Institute Advisory Council), and funded a successful research project that developed a blood test for Tourette syndrome.

A staff member in the Silverman lab conducts behavioral neuroscience research.

We are grateful to the Mittelstaedts for their generosity, said MIND Institute Director Leonard Abbeduto. As a collaborative hub for preclinical and clinical research on neurodevelopmental disability, the MIND Institute is uniquely suited to build on past successes and tackle the complexities of SYNGAP1 to provide help for families.

The funding also supports UC Davis' $2 billion fundraising campaign, Expect Greater: From UC Davis, For the World, the largest philanthropic endeavor in university history. Together, donors and UC Davis are advancing work to prepare future leaders, sustain healthier communities, and bring innovative solutions to today's most urgent challenges.

SYNGAP1-related non-syndromic intellectual disability is a rare neurodevelopmental condition caused by a variation in one gene. The gene, SYNGAP1, contains instructions for making a protein (SynGAP). This protein is located at the junctions between nerve cells, called synapses, and helps regulate changes important for memory and learning. The protein also helps regulate communication between neurons.

When the variation is present, the SYNGAP1 protein in cells is reduced which causes an increase in the excitability in the synapses. This makes it difficult for neurons to communicate and increases the likelihood of seizure events. This can lead to a variety of symptoms:

Jill Silverman

SYNGAP1 syndrome affects 1-4 out of 10,000 people. The first patient was identified in 2009.

The MIND Institutes interventional genetics team includes faculty who specialize in multiple research areas.

Ron Mittelstaedt

Each of us is a world expert in our particular discipline, so bringing us all together means the chances of success are much more likely, said Jill Silverman, associate professor in the Department of Psychiatry and Behavioral Sciences and an internationally recognized expert in the use of rodent models for therapeutic development. Silvermans Lab is known for its expertise in behavioral neuroscience research.

In addition to Silverman, the SYNGAP1 team includes three other MIND Institute faculty members:

The sum of the group is going to be much greater than anything we could have done alone, said Fink, whose lab focuses on therapeutic development for neurodevelopmental conditions and neurodegenerative diseases. The fact that the foundation has funded us as a team, across multiple centers and programs is really unique. This funding brings us all together for an important project.

Kyle Fink in his lab, which focuses on therapeutic development for neurodevelopmental conditions and neurodegenerative diseases.

The researchers will work on parallel tracks, each contributing a piece of the puzzle.

Silverman will conduct specialized behavioral tests on mouse models of SYNGAP1, using tools with corresponding metrics in humans, such as EEGs (a type of brain scan) to determine clinically relevant outcomes.

Nord and Fink will create a new mouse model that contains the mutated human SYNGAP1 gene, while Segal and Fink will create new molecular therapies to counter that mutated gene. Theyll also figure out how to deliver those therapies to the brain.

Were not just trying to treat the symptoms of the disease with a drug, explained Segal, whose lab specializes in molecular analysis. We are trying to change the underlying genetic condition, and our particular approach is to do that in a way that does not change the DNA sequence. We use tools to change the gene expression instead, which we think will make safer therapies. Its really a state-of-the-art approach. Its molecular therapy.

David Segal working in his lab, which specializes in molecular analysis.

The collaborative approach, often called team science, coupled with the RDM Positive Impact Foundations support, allows for an ambitious, fast-tracked research program. The $1.25 million frees the researchers from the need to apply for multiple federal grants and enables them to focus immediately on SYNGAP1.

The team excels in whats often called bench to bedside research, translating results from the lab directly into therapies for patients.

We see these patients, we meet with them, were on Zoom calls with them and I want to find something that works for them. I want to change their lives. Thats what Im driven by, Silverman said.

Silverman, Fink and Segal have had previous success with their work on another rare genetic condition, Angelman syndrome, which causes developmental delay, speech and balance challenges and intellectual disability.

David Segal

Their labs helped to create and characterize the first rat model of Angelman syndrome last year. The Segal lab also created a protein therapeutic that could increase the level of the affected gene in mouse models of Angelman syndrome, a major discovery.

All three labs are still working on a wide range of therapeutics for Angelman, including molecular therapies delivered with viruses or stem cells and novel small molecule compounds.

Ron Mittelstaedt is hoping for another success story, this time with SYNGAP1, but hes also realistic about the research process.

We are all very aware that going down this path doesnt guarantee anything except the ability to get up to bat, and we could get a hit or strike out. But doing nothing guarantees you dont get a hit, so its important for us to take action, and were hopeful well hit a home run.

UC Davis researchers get $3 million FAST grant to find treatment for Angelman syndrome

The UC Davis MIND Institute in Sacramento, Calif. was founded in 1998 as a unique interdisciplinary research center where families, community leaders, researchers, clinicians and volunteers work together toward a common goal: researching causes, treatments and potential prevention of neurodevelopmental disabilities. The institute has major research efforts in autism, fragile X syndrome, chromosome 22q11.2 deletion syndrome, attention-deficit/hyperactivity disorder (ADHD) and Down syndrome. More information about the institute and its Distinguished Lecturer Series, including previous presentations in this series, is available on the Web at mindinstitute.ucdavis.edu.

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Local foundation awards $1.25 million to MIND Institute to study rare genetic condition - UC Davis Health

MeCP2 is a microsatellite binding protein that protects CA repeats from nucleosome invasion – Science Magazine

MeCP2 binds hydroxymethylated CA repeats

Despites of decades of research on the Rett syndrome protein MeCP2, its function remains unclear. Ibrahim et al. show that MeCP2 is a hydroxymethylated cytosine-adenosine (CA) repeat-binding protein that modulates chromatin architecture at a distance from the transcription start site (see the Perspective by Zhou and Zoghbi). MeCP2 accumulates and spreads around modified CA repeats and competes for nucleosome occupancy. Loss of MeCP2 results in a widespread increase in nucleosome density inside lamina-associated domains and transcriptional dysregulation of genes enriched in CA repeats. These results shed light on the underlying molecular mechanism of Rett syndrome, a severe disease associated with mutations in MeCP2.

Science, abd5581, this issue p. eabd5581; see also abj5027, p. 1390

Rett syndrome is a severe neurodevelopmental disorder that is mainly caused by mutations in the methyl-CpG-binding protein 2 gene (MeCP2). Initially, MeCP2 was identified as an essential brain protein that binds to methylated CpG (mCG) via its methyl-binding domain (MBD) and acts as transcriptional repressor. However, during early brain development, the postnatal accumulation of MeCP2 parallels the genome-wide high-level accumulation of hydroxymethylcytosine (hmC) and methylated CpA (mCA), suggesting that MeCP2 may also recognize and bind to DNA sequences that contain these modified nucleotides.

The ability of MeCP2 to recognize both mCA and hmC as well as mCG has led to conflicting conclusions regarding its function in transcriptional regulation, because these cytosine modifications are associated with either repression (mCA and mCG) or activation (hmC) of transcription. The unambiguous identification of the MeCP2 target sequence(s) would help to clarify this issue.

CA repeats (CAn) represent ~1% of the mouse genome and belong to the microsatellite family. They are widely distributed throughout the genome and have been shown to affect transcription of nearby genes. Our recent data reveal that CAn are methylated (mCAn) or hydroxymethylated (hmCAn) in various cell types. In a search for proteins that could specifically recognize and bind these CA repeats, we identified MeCP2 as a specific reader of CA repeats. We hypothesized that the methylation status of CAn is essential for the recognition and binding of MeCP2, possibly through recognition of the modified nucleotides in CA repeats with distinct affinities, relevant for its neuronal function in transcriptional regulation.

Here we show that within the MBD family, MeCP2 is the only protein that specifically recognizes and binds to CA repeats, with much stronger affinity than mCG and mCA. MeCP2 selectively recognizes CA repeat DNA in a strand-specific manner and requires at least five consecutive CA dinucleotides to optimally bind DNA. While MeCP2 can bind in vitro to modified and nonmodified CA repeats, it exhibits impressive selectivity toward hydroxymethylated CA repeats, which are modified by DNA (cytosine-5)-methyltransferase 3A. The modified cytosine, only when located within a CA repeat, serves as a nucleation point for both MeCP2 accumulation and spreading around the repeat, which, in turn, correlates with nucleosome exclusion. In addition, loss of MeCP2 results in widespread increase in nucleosome density within lamina-associated domains (LADs) and transcriptional dysregulation of CA repeatenriched genes located outside LADs.

We have also dissected the molecular basis of the MeCP2 hydroxymethylated CA repeat recognition by solving the crystal structure of MeCP2 in complex with hmCAn. The CA repeat creates a well-defined DNA shape, with a considerably modified geometry, including a widened major groove and negative roll parameters, located precisely at the modification site. We show that the molecular recognition of the hydroxymethylated CA repeat specifically occurs through Arg133, a key MeCP2 residue whose mutation causes Rett syndrome.

Our work identifies MeCP2 as a hydroxymethylated CA repeat DNA binding protein that targets the 5hmC-CA-rich sequence, which are specifically located on one strand. Our data provide insights into the origin of Rett syndrome at the molecular level and suggest that this neurodevelopmental disorder could be viewed as a chromatin disease, originating from the inability of mutant MeCP2 to bind and protect the CA repeats from nucleosome invasion. Our results open a previously unexplored area of research focused on understanding the role of specific protein binding to microsatellites and other repeats in neurological diseases of unknown etiology.

MeCP2 is a microsatellite binding protein that specifically recognizes hydroxymethylated CA repeats. Depletion of MeCP2 alters the chromatin organization of CA repeats and LADs and results in nucleosome accumulation on CA repeats and genome-wide transcriptional dysregulation. WT, wild-type; KO, knockout.

The Rett syndrome protein MeCP2 was described as a methyl-CpG-binding protein, but its exact function remains unknown. Here we show that mouse MeCP2 is a microsatellite binding protein that specifically recognizes hydroxymethylated CA repeats. Depletion of MeCP2 alters chromatin organization of CA repeats and lamina-associated domains and results in nucleosome accumulation on CA repeats and genome-wide transcriptional dysregulation. The structure of MeCP2 in complex with a hydroxymethylated CA repeat reveals a characteristic DNA shape, with considerably modified geometry at the 5-hydroxymethylcytosine, which is recognized specifically by Arg133, a key residue whose mutation causes Rett syndrome. Our work identifies MeCP2 as a microsatellite DNA binding protein that targets the 5hmC-modified CA-rich strand and maintains genome regions nucleosome-free, suggesting a role for MeCP2 dysfunction in Rett syndrome.

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MeCP2 is a microsatellite binding protein that protects CA repeats from nucleosome invasion - Science Magazine

The Stem Cell Manufacturing market is projected to reach USD 18.0 billion by 2026 from USD – GlobeNewswire

New York, May 26, 2021 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Stem Cell Manufacturing Market by Product, End User - Global Forecast to 2026" - https://www.reportlinker.com/p05361410/?utm_source=GNW

By product, the consumables segment accounted for the largest share of the Stem Cell Manufacturing market The Stem Cell Manufacturing market by product is categorized into consumables, instruments, and stem cell lines.The consumables segment dominated the market in 2020.

The large share of this segment can be attributed to the frequent purchase of consumables, rising stem cell research, and increasing demand for stem cell therapies.

Asia Pacific: The fastest-growing region in the Stem Cell Manufacturing market.

The Asia Pacific market is projected to grow at the highest CAGR during the forecast period, mainly due to supportive regulatory framework and increasing public-private initiatives to encourage public awareness about stem cell-based treatments.

North America: the largest share of the Stem Cell Manufacturing market North America accounted for the largest share of the Stem Cell Manufacturing market. Factors such as private funding and grants to support the clinical evaluation of stem cells for various applications are the major factors driving the market growth.

Breakdown of primaries The study contains insights from various industry experts, ranging from component suppliers to Tier 1 companies and OEMs. The break-up of the primaries is as follows: By Respondent Supply Side- 63%, Demand Side- 37% By Designation Executives- 25%, CXOs, Directors--30%, Managers - 45% By Region North America - 40%, Europe - 25%, APAC 20%, LATAM- 10%, MEA- 5%

The Stem Cell Manufacturing market is dominated by a few globally established players such as Thermo Fisher Scientific (US), Merck Millipore (Germany), Lonza Group AG (Switzerland), Danaher Corporation (US), Sartorius AG (Germany), Bio-Rad Laboratories (US), Becton, Dickinson and Company (US), Stemcell Technologies (Canada), Fujifilm Holdings Corporation (Japan), Miltenyi Biotec (Germany), Terumo BCT Inc. (US), Corning Inc. (US), Bio-Techne Corporation (US), Takara Bio Group (Japan), Eppendorf AG (Germany), Getinge (Sweden), Himedia Laboratories (India), Anterogen (South Korea), Cellgenix GMBH (Germany) and Promocell (Germany).

Research Coverage: The report segments the Stem Cell Manufacturing market-based on region (North America, Europe, Asia Pacific, Latin America and Middle East & Africa), Product [Consumables (Culture Media and other consumables), Instruments (Bioreactors & Incubators, Cell Sorters and other instruments) and Stem Cell Lines (Hematopoietic stem cells, Mesenchymal stem cells, Induced Pluripotent Stem cells, Embryonic stem cells, Neural Stem cells and Multipotent adult progenitor stem cells)], Application [Research (Life science research and Drug discovery and development), Clinical (Allogenic stem cell therapy and Autologous stem cell therapy) and Cell & Tissue Banking Applications], End User (Pharmaceutical & Biotechnology Companies, Academic institutes, Research laboratories & contract research organisations, Hospitals and surgical centres, Cell & tissue banks and Other End Users).

The report also provides a comprehensive review of market drivers, challenges, and opportunities in the Stem Cell Manufacturing market

Key Benefits of Buying the Report: The report will help the leaders/new entrants in this market with information on the closest approximations of the revenue numbers for the overall market and the sub-segments.This report will help stakeholders understand the competitive landscape and gain more insights to better position their businesses and plan suitable go-to-market strategies.

The report also helps stakeholders understand the pulse of the Stem Cell Manufacturing market and provides them information on key market drivers, challenges, and opportunities. Read the full report: https://www.reportlinker.com/p05361410/?utm_source=GNW

About Reportlinker ReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

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The Stem Cell Manufacturing market is projected to reach USD 18.0 billion by 2026 from USD - GlobeNewswire

Jasper Therapeutics Announces New Clinical Trial with the National Institute of Allergy and Infectious Diseases to Evaluate JSP191 in Chronic…

REDWOOD CITY, Calif.--(BUSINESS WIRE)--Jasper Therapeutics, Inc., a biotechnology company focused on hematopoietic cell transplant therapies, today announced the initiation of a Phase 1/2 clinical trial to evaluate JSP191, the companys first-in-class anti-CD117 monoclonal antibody, as a targeted, non-toxic conditioning regimen prior to allogeneic transplant for chronic granulomatous disease (CGD). Jasper Therapeutics and the National Institute of Allergy and Infectious Diseases (NIAID) have entered into a clinical trial agreement in which NIAID will serve as the Investigational New Drug (IND) sponsor for this study.

CGD is a rare, inherited disease of the immune system that develops in infancy or early childhood and results in severe and sometimes life-threatening infections. Allogeneic hematopoietic stem cell transplant is a proven cure for CGD. However, its use is limited because current conditioning agents used to deplete stem cells in preparation for transplantation are genotoxic and associated with limited efficacy and serious adverse effects, including veno-occlusive disease, long-term infertility and secondary malignancies.

We look forward to collaborating with NIAID on this Phase 1/2 clinical trial, which should provide important information about the potential of JSP191 as a safer and more effective conditioning agent for patients with CGD undergoing hematopoietic stem cell transplant, said Kevin N. Heller, M.D., Executive Vice President, Research and Development, of Jasper Therapeutics. Through this clinical trial agreement with NIAID, as well as others with the National Institutes of Health and academic centers, we are continuing to develop JSP191 for additional pretransplant conditioning regimens beyond severe combined immunodeficiency and acute myeloid leukemia/myelodysplastic syndromes, which have demonstrated safety and efficacy in early-stage clinical trials to date.

About JSP191

JSP191 is a first-in-class humanized monoclonal antibody in clinical development as a conditioning agent that clears hematopoietic stem cells from bone marrow, creating an empty space for donor or gene-corrected transplanted stem cells to engraft. While hematopoietic cell transplantation can be curative for patients, its use is limited because standard high dose myeloablative conditioning is associated with severe toxicities and standard low dose conditioning has limited efficacy. To date, JSP191 has been evaluated in more than 90 healthy volunteers and patients. It is currently enrolling in two clinical trials for acute myeloid leukemia (AML)/myelodysplastic syndromes (MDS) and severe combined immunodeficiency (SCID) and is scheduled to begin enrollment in three additional studies in 2021 for severe autoimmune disease, sickle cell disease and Fanconi anemia patients undergoing hematopoietic cell transplantation.

About Jasper Therapeutics

Jasper Therapeutics is a biotechnology company focused on the development of novel curative therapies based on the biology of the hematopoietic stem cell. The company is advancing two potentially groundbreaking programs. JSP191, a first-in-class anti-CD117 monoclonal antibody, is in clinical development as a conditioning agent that clears hematopoietic stem cells from bone marrow in patients undergoing a hematopoietic cell transplantation. It is designed to enable safer and more effective curative allogeneic and autologous hematopoietic cell transplants and gene therapies. In parallel, Jasper Therapeutics is advancing its preclinical engineered hematopoietic stem cell (eHSC) platform, which is designed to overcome key limitations of allogeneic and autologous gene-edited stem cell grafts. Both innovative programs have the potential to transform the field and expand hematopoietic stem cell therapy cures to a greater number of patients with life-threatening cancers, genetic diseases and autoimmune diseases than is possible today. For more information, please visit us at jaspertherapeutics.com.

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Jasper Therapeutics Announces New Clinical Trial with the National Institute of Allergy and Infectious Diseases to Evaluate JSP191 in Chronic...

Meso-Brain project explores 3D printed stem cells to treat neurological conditions – 3D Printing Industry

A stem cell research project headed up by Aston University is developing 3D nanoprinting techniques that they claim could revolutionize neuroscience and the treatment of diseases such as Parkinsons and dementia.

The EU-funded Meso-Brain project aims to generate customizable 3D printed networks of stem-cell-derived neurons to produce a new generation of accurate modeling and testing tools.

The project hopes to address the limitations of current neuronal culturing techniques by combining cutting-edge research within human stem cell biology, nanoscale 3D printing, computational network modeling and light sheet microscopy to discover novel treatment options for the long-term alleviation of brain dysfunction.

Meso-Brain hopes to unlock meaningful and practicable insights into the functioning of the brain, and will eventually allow researchers to accurately model brain networks more realistically than ever before.

Cell 3D printing

Stem cells generally serve as a repair system for the body, and, being unspecialized, are able to develop into a variety of different types of cells. As such, stem cells can develop into specialized cells such as blood, muscle, and brain cells, when required.

Cell 3D printing is an area that is receiving growing interest from researchers and 3D printing firms alike as a means of harnessing these desirable properties, particularly for regenerative medicine and bioprinting applications.

For instance, 3D printer OEM 3D systems announced a breakthrough in its Print to Perfusion bioprinting platform earlier this year, which can now rapidly produce full-size human lung scaffolds that can be perfused with living cells to create tissues. Meanwhile, scientists at the University of Buffalo have developed a new 3D bioprinting method that reduces the time needed to create cell-laden hydrogel structures, potentially bringing 3D printed organs closer to reality.

Elsewhere, researchers from the University of New South Wales have developed a novel technique to 3D print bone-mimicking structures containing living cells with potential uses for bone tissue engineering and disease modeling, and a new bioink created by Lund University is capable of supporting new cell and blood vessel growth once transplanted into new material.

In a similar vein to the Meso-Brain project, 3D bioprinting has previously been deployed by medical tech company Fluicell, R&D firm Cellectricon, and the Karolinska Institutet university to arrange neural brain cells into complex patterns in order to model the progress of neurological diseases.

The Meso-Brain project

The project was first launched by Aston University in 2016 with the goal of using nanoscale 3D printing to replicate the brains neural networks. The project has since received further funding from the EUs Horizon 2020 FET-Open program to accelerate the pace of neuroscience research and pharmaceutical drug discovery.

Coordinated by Aston University, the project is made up of six partners from three countries, including human cell culture specialist Axol Bioscience, digital service provider Kite Innovation, the Institute of Photonic Sciences (ICFO), the University of Barcelona, and LZH Laser Zentrum Hannover E.V.

Meso-Brain combines revolutionary tools for micro-fabrication, neuronal network development and monitoring, and functional analysis to bring to light 3D human neuronal networks with tailored characteristics.

Through Meso-Brain, the consortium is working on developing a new type of neural culture and interacting interface system integrated with conductive polymers, that will facilitate electrical stimulation and recording of individual cells.

In naturally developing circuits in the brain, neurons and connections are first generally configured and then gradually refined over time in response to chemical and electrical activity. To replicate this process in the researchers own 3D printed scaffolds, neurons and astrocytes derived from stem cells are developed at specific cytophilic points through the use of chemical messages and electrical activity to promote and drive functional network development.

After this process, functional connectivity maps are drawn using newly-developed mathematical formulations to verify the function of the 3D printed neural network structure.

The customizable properties of the 3D printed scaffolds enable fluorescence imaging and interrogation with photonic and optical approaches, therefore making it possible to see how the neurons interact with each other in real-time.

Future impacts of Meso-Brain

It is hoped that the research developments within Meso-Brain will allow researchers to accurately and dynamically model brain networks to identify neurons in various states of dysfunction and test their reaction to new medicines and other treatments.

According to the project partners, the development of human 3D neuronal networks that exhibit physiologically relevant and reproducible architecture and activity could be foundational to the scientific community, enabling large-scale scientific investigation of human brain network function.

The projects results also hope to facilitate large-scale pharmaceutical testing on human cells and human disease models with stem cells derived from patients, eventually leading to advances in neural transplantations for central nervous system therapy and repair.

Ultimately, the researchers believe Meso-Brain can aid the understanding and treatment of a range of neurological conditions such as dementia, Parkinsons, and brain trauma.

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Featured image shows a CT brain scan of a cranium with TBI. Image via Qrons.

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Meso-Brain project explores 3D printed stem cells to treat neurological conditions - 3D Printing Industry

Global Flow Cytometry Market (2020 to 2026) – by Technology, Product and Service, Application, End-user and Geography – ResearchAndMarkets.com -…

DUBLIN--(BUSINESS WIRE)--The "Flow Cytometry Market Based on Technology, Product and Service, Application, End-User, and Geography - Global Forecast up to 2026" report has been added to ResearchAndMarkets.com's offering.

The Flow Cytometry Market is projected to grow at the rate of 8.7% CAGR by 2026. Flow cytometry is a biophysical, laser-based analytical technology that calculates and analyzes various physical features of cells or particles present in a fluid when passed through a laser beam. Flow cytometry has several benefits over conventional analytical techniques such as ELISA, including its capability to provide accurate results within less time at about similar prices.

The key factors propelling the flow cytometry market include the emergence and commercial application of advanced technologies in flow cytometry and the increase in the adoption of flow cytometry in stem cell research and clinical research. Apart from that, the inadequate purchasing power of end-users in emerging countries and high product costs are the factors to impede the growth of the flow cytometry market.

Companies Mentioned

The cell-based flow cytometry technology leads the overall market to maintain its dominance over the forecast period. Growing demand for early diagnosis and increasing consciousness regarding the advantages of cell-based flow cytometry are the factors contributing to its dominance. Moreover, technology has enormous applications in many research domains. The cell-based flow cytometry technology is mainly used in drug discovery for the physiological significance of the results.

In the market for products & services, the reagents & consumables segment has the most significant market share. This is due to the high penetration along with the benefits such as precise outcomes and user-friendly. Moreover, the introduction of new technologies and their detection capability also boost the demand for the products and services.

As per the application segmentation, the research segment has held a significant share in the market. This segment's major share is attributed to the rising number of research and development activities concerning cancer and other diseases. This technology is mostly used in pharmaceutical research and also has application in various researches.

The commercial organization segment has acquired the maximum share in the market due to the vast application of this technology in various verticals. The emergence of several probes and reagents for particular applications in diagnostics and drug discovery is predicted to generate numerous growth opportunities for the segment by serving the users in research and small peripheral laboratories.

North America has occupied the highest share in the flow cytometry market. This is due to the early adoption of advanced technology, high investments in research and developments, and widespread flow cytometry application in diagnostic activities.

High precision of measurement performance by using flow cytometers and the related valuable outcomes accelerates the global demand for flow cytometers among clinicians, drug developers, and food safety technicians. Moreover, higher sensitivity, easy-to-use analysis, and quick outcomes associated with flow cytometry will also likely drive the flow cytometers' global market growth soon.

Key Topics Covered:

1. Executive Summary

2. Industry Outlook

3. Market Snapshot

4. Market characteristics

4.1. Market Overview

4.2. Market Segmentation

4.3. Market Dynamics

4.3.1. Drivers

4.3.2. Restraints

4.3.3. Opportunities

4.4. DRO - Impact Analysis

5. Technology: Market Size & Analysis

6. PRODUCT & SERVICE: Market Size & Analysis

7. Application: Market Size & Analysis

8. END-USER: Market Size & Analysis

9. Geography: Market Size & Analysis

10. Competitive Landscape

10.1. Competitor Comparison Analysis

10.2. Market Developments

10.2.1. Mergers and Acquisitions, Legal, Awards, Partnerships

10.2.2. Product Launches and execution

11. Vendor Profiles

12. Companies to Watch

13. Analyst Opinion

14. Annexure

For more information about this report visit https://www.researchandmarkets.com/r/qr18f6

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Global Flow Cytometry Market (2020 to 2026) - by Technology, Product and Service, Application, End-user and Geography - ResearchAndMarkets.com -...

Moderna Highlights Advances in Platform Science and Innovative Vaccine Research at Fourth Annual Science Day – Business Wire

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Moderna, Inc., (Nasdaq: MRNA) a biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines, today announced new research being highlighted as part of the Companys fourth annual Science Day. Modernas Science Day is designed to provide insight into the continued diverse efforts underway at Moderna to better understand how to use mRNA as medicines and vaccines and underscores the Companys continued commitment to basic science and innovation.

Science Day gives us an opportunity to provide insights into the advancements in our platform science and our further understanding of how to use mRNA as both a vaccine and a medicine. Our investments in basic science continue to result in major steps forward in our platforms capabilities, and these have allowed us to open new therapeutic areas and new scientific directions, said Stephen Hoge, M.D., President of Moderna. Our investments in platform research along with our digital backbone and manufacturing plants have enabled us to create first-in-class mRNA medicines and vaccines. Today, we're excited to highlight our work to identify and address SARS-CoV-2 variants of concern, optimize our proprietary lipid nanoparticle (LNP) technology, and deliver mRNA to hematopoietic stem cells.

Moderna currently has 24 mRNA development programs in its portfolio with 14 having entered clinical studies. The Companys updated pipeline can be found at http://www.modernatx.com/pipeline. Moderna and collaborators have published more than 65 peer-reviewed papers.

At this years Science Day, Moderna will present new platform science and preclinical research, including:

mRNA Delivery to Hematopoietic Stem and Progenitor Cells (HSPC)

Of the nearly 30 trillion cells in the body, approximately 90% are of hematopoietic origin. Hematopoietic lineages originating in the bone marrow are intimately involved in maintaining homeostasis and human health. Nonetheless, there are hundreds of hematologic or immune-related disorders caused or exacerbated by cells of the hematopoietic lineage. In a disease setting, cells of hematopoietic origin interact with host tissues to drive chronic inflammatory and immune disorders. Because some hematopoietic stem and progenitor cells (HSPC) have self-renewal and pluripotent properties, targeting HSPC has the potential to modulate underlying chronic inflammation and immune-related disorders.

Advances in lipid nanoparticle (LNP) technology has allowed for delivery to the bone marrow following systemic LNP administration in vivo. This achievement has enabled Moderna scientists to deliver mRNA directly to bone marrow HSPC in vivo, leading to HSPC transfection and long-term modulation of all hematopoietic lineages. This represents a major milestone in impacting chronic inflammatory and immune related disease.

mRNA Engineering: Optimizing Ribosome Load

The ribosome plays a central role in orchestrating the life of mRNA inside the cell. Understanding how to modulate translation by changing mRNA sequence could enable a powerful lever to control the performance of an mRNA drug. To develop such a lever, the Moderna's scientists strove to characterize mechanistically how differences in translation drive differences in protein expression. In this research, an unexpected relationship emerged where mRNAs with low translation initiation rates conferred the highest and most durable levels of protein expression. By understanding the mechanisms that drive this behavior, the Company is taking mRNA design from a guess-and-check discipline into an engineering discipline. With tools in place, and more under development, Moderna is improving its ability to make an mRNA that generates the right amount of the right protein, for the right amount of time, in the right cell type. As Moderna builds these levers into its mRNA drugs, the Company believes it will be able to target more indications with more precision.

Addressing SARS-CoV-2 Variants of Concern

One part of the Companys strategy to remain ahead of the SARS-CoV-2 virus is to closely monitor and address emerging variants of concern and waning immunity. Moderna is using artificial intelligence (AI) and machine learning to predict escape risk. This involves developing statistical models to understand and predict escape risk, including identifying breakthrough sequences from clinical trials and real-world data, examining spike protein biophysical and pseudovirus neutralization data, studying spike mutations and lineage info, and time tracking.

Science Day Webcast Information

Moderna will host its annual Science Day for analysts and investors at 8:00 a.m. ET on Thursday, May 27. A live webcast will be available under Events and Presentations in the Investors section of the Moderna website at investors.modernatx.com. A replay of the webcast will be archived on Modernas website for one year following the presentation.

About Moderna

In 10 years since its inception, Moderna has transformed from a science research-stage company advancing programs in the field of messenger RNA (mRNA), to an enterprise with a diverse clinical portfolio of vaccines and therapeutics across six modalities, a broad intellectual property portfolio in areas including mRNA and lipid nanoparticle formulation, and an integrated manufacturing plant that allows for both clinical and commercial production at scale and at unprecedented speed. Moderna maintains alliances with a broad range of domestic and overseas government and commercial collaborators, which has allowed for the pursuit of both groundbreaking science and rapid scaling of manufacturing. Most recently, Modernas capabilities have come together to allow the authorized use of one of the earliest and most-effective vaccines against the COVID-19 pandemic.

Modernas mRNA platform builds on continuous advances in basic and applied mRNA science, delivery technology and manufacturing, and has allowed the development of therapeutics and vaccines for infectious diseases, immuno-oncology, rare diseases, cardiovascular diseases and auto-immune diseases. Today, 24 development programs are underway across these therapeutic areas, with 14 programs having entered the clinic. Moderna has been named a top biopharmaceutical employer by Science for the past six years. To learn more, visit http://www.modernatx.com.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding: the potential for delivery of mRNA to hematopoietic stem and progenitor cells (HSPC) in vivo; methods of detecting and interrogating HSPC in vivo; the potential for delivering LNPs to hematopoietic stem cells in vivo; the ability to optimize codons and mRNA structures to increase total protein outputs; the potential for the Company to develop processes for controlling protein expression by modifying ribosomal loads; the Companys ability to engineer LNPs capable of accessing difficult-to-transfect primary cells with efficient endosomal escape and high functional mRNA delivery; the Companys strategy for combatting COVID-19, including processes for monitoring emerging variants and waning immunity; and strategies for modeling viral escape. In some cases, forward-looking statements can be identified by terminology such as will, may, should, could, expects, intends, plans, aims, anticipates, believes, estimates, predicts, potential, continue, or the negative of these terms or other comparable terminology, although not all forward-looking statements contain these words. The forward-looking statements in this press release are neither promises nor guarantees, and you should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties, and other factors, many of which are beyond Modernas control and which could cause actual results to differ materially from those expressed or implied by these forward-looking statements. These risks, uncertainties, and other factors include, among others: the fact that there has never been a commercial product utilizing mRNA technology approved for use; the fact that the rapid response technology in use by Moderna is still being developed and implemented; the safety, tolerability and efficacy profile of the Moderna COVID-19 Vaccine observed to date may change adversely in ongoing analyses of trial data or subsequent to commercialization; the Moderna COVID-19 Vaccine may prove less effective against variants of the SARS-CoV-2 virus, or the Company may be unsuccessful in developing future versions of its vaccine against these variants; despite having ongoing interactions with the FDA or other regulatory agencies, the FDA or such other regulatory agencies may not agree with the Companys regulatory approval strategies, components of our filings, such as clinical trial designs, conduct and methodologies, or the sufficiency of data submitted; Moderna may encounter delays in meeting manufacturing or supply timelines or disruptions in its distribution plans for the Moderna COVID-19 Vaccine; whether and when any biologics license applications and/or additional emergency use authorization applications may be filed in various jurisdictions and ultimately approved by regulatory authorities; potential adverse impacts due to the global COVID-19 pandemic such as delays in regulatory review, manufacturing and clinical trials, supply chain interruptions, adverse effects on healthcare systems and disruption of the global economy; and those other risks and uncertainties described under the heading Risk Factors in Modernas most recent Annual Report on Form 10-K filed with the U.S. Securities and Exchange Commission (SEC) and in subsequent filings made by Moderna with the SEC, which are available on the SECs website at http://www.sec.gov. Except as required by law, Moderna disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this press release in the event of new information, future developments or otherwise. These forward-looking statements are based on Modernas current expectations and speak only as of the date hereof.

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Moderna Highlights Advances in Platform Science and Innovative Vaccine Research at Fourth Annual Science Day - Business Wire