CNRS University

EVRY, France--( BUSINESS WIRE )-- Atamyo Therapeutics a clinical-stage biotechnology company focused on the development of new generation gene therapies targeting muscular dystrophies and cardiomyopathies, today announced that the U.S. Food & Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for ATA-200 to proceed in a Phase 1b/2b clinical trial. ATA-200 is a one-time gene therapy for the treatment of γ-sarcoglycan related limb-girdle muscular dystrophy Type 2C/R5 (LGMD2C/R5), a serious and debilitating condition that affects children and leads to loss of ambulation before adulthood. The multicenter Phase1b open-label dose escalation study ( NCT05973630 ) will evaluate safety, pharmacodynamics, efficacy, and immunogenicity in children receiving intravenous ATA-200, a single-dose Adeno-Associated Virus (AAV) vector carrying the human γ-sarcoglycan transgene. The deployment in the US of Atamyo’s clinical trial of ATA-200 is funded by The Dion Foundation for Children with Rare Diseases . This study has already received regulatory clearance in France and Italy. “This IND clearance in an important step to bring ATA-200 to US children suffering from this highly debilitating LGMD-2C/R5 disease,” said Sophie Olivier, MD, Chief Medical Officer of Atamyo. “We are proud to be the first treatment for LGMD-2C/R5 to enter into clinical development in the US and we are committed to opening the first US center before year-end,” added Stéphane Degove, Atamyo’s Chief Executive Officer. In addition, Atamyo announced the US FDA has awarded Orphan Drug Designation for ATA-200. Orphan Drug Designations by the FDA grants seven years of market exclusivity in the US. This Orphan Drug Designation for ATA-200 was granted a few weeks after ATA-200 received Rare Pediatric Disease Designation by the FDA. LGMD-2C/R5 is a rare genetic disease caused by mutations in the gene that produces γ-sarcoglycan, a transmembrane protein that is involved in the connection between muscle fibers and their environment. It affects an estimated 2,000 people in Europe and in the US. In the classical form, symptoms appear in early childhood and patients suffer from progressive muscular weakness leading to loss of ambulation before adulthood. Cardiac involvement, typically presenting as dilated cardiomyopathy, is reported in approximately half of patients and will eventually impact life expectancy. There is no curative treatment. The management of LGMD-2C/R5 is solely supportive. ATA-200, the gene therapy for LGMD-2C/R5, delivers a normal copy of the gene for production of γ-sarcoglycan. In preclinical mice models, ATA-200 demonstrated its tolerability and capability to correct symptoms and biomarkers of the pathology. Atamyo plans to initiate enrolling patients for this clinical trial in the 4 th quarter of 2024. The therapy is based on the research of Atamyo Chief Scientific Officer Isabelle Richard, Ph.D., Research Director at CNRS who heads the Progressive Muscular Dystrophies Laboratory at Genethon. About Atamyo Therapeutics Atamyo Therapeutics is a clinical-stage biopharma focused on the development of a new generation of effective and safe gene therapies for neuromuscular diseases. A spin-off of gene therapy pioneer Genethon, Atamyo leverages unique expertise in AAV-based gene therapy and muscular dystrophies from the Progressive Muscular Dystrophies Laboratory at Genethon. Atamyo’s most advanced programs address different forms of limb-girdle muscular dystrophies (LGMD), with two clinical-stage programs targeting respectively LGMD-R9 and LGMD-R5. The name of the company is derived from two words: Celtic Atao which means “Always” or “Forever” and Myo which is the Greek root for muscle. Atamyo conveys the spirit of its commitment to improve the life of patients affected by neuromuscular diseases with life-long efficient treatments. For more information visit www.atamyo.com

EVerZom achieves first bioproduction of clinical grade stem cell-derived exosomes in large scale bioreactors in partnership with the French Blood Establishment (EFS) with unprecedented yields The disruptive, proprietary technology developed by EVerZom offers a yield 100 times higher than traditional methods. This innovation has just been tech-transferred and validated in a GMP-grade biomanufacturing environment. This first clinical batch will enable EVerZom to initiate regulatory non-clinical studies of its first drug candidate EVerGelTM. Paris-Créteil, October 2nd, 2024. EVerZom, a biopharmaceutical company developing exosome based biodrugs, announces that it has reached a key milestone in its development with the production of a first exosome GMP batch in a 10L bioreactor with unprecedented yields. Regulatory validation of the technology was carried out in GMP clean rooms of the Cellular Therapy and Engineering Unit of the French Blood Establishment (EFS) Ile-de-France in Créteil, a unit of reference for the bioproduction and quality control of Advanced Therapy Medicinal Product (ATMPs) and complex biologics. ‘We are extremely proud to have reached this key milestone in the biomanufacturing of our first batch of GMP-quality exosomes in collaboration with the EFS Créteil platform. This will enable us to accelerate the beginning of our clinical trials. Securing biomanufacturing is an essential technological building block for the progress of our lead programme EVerGelTM for the treatment of digestive fistulas in patients suffering from Crohn's disease. With this first batch, we will carry out the regulatory non-clinical studies, scheduled for early 2025, with the objective of the first-in-human early 2026,’ explains Jeanne Volatron, CEO of EVerZom ‘We are delighted to share our expertise to EVerZom for this first production of clinical-grade exosomes at yields unmatched to date. This technology will make exosomes available to patients as a therapeutic solution, enabling us to offer new disruptive biotherapies in the years to come’ explains Prof. Hélène Rouard, Head of the ATMP platform, from the EFS Créteil. The growing interest in extracellular vesicles in regenerative medicine Exosomes or extracellular vesicles are biological nanoparticles around 100 nm in size, found in all body fluids (blood, saliva, urine). They play a major role in inter-cell communication by transporting genetic information via RNA. Ten times more abundant than cells in the body, exosomes are the body's natural vectors. They are not immunogenic, have a strong capacity to penetrate cells and can be kept frozen on a long time. Thanks to these characteristics, they could revolutionize cell and gene therapies by opening the way to ‘off the shelf’ cell-free therapies. This is why research in this field is intensifying, with around 10,000 publications a year and numerous clinical trials confirming their safety and efficacy. Stem cell-derived exosomes, the main focus of EVerZom's research, have the capacity to regenerate tissues in a variety of pathologies such as osteoarthritis, tissue reconstruction after myocardial infarction and liver and kidney diseases. However, the clinical use of this new therapeutic modality is limited by the absence of a GMP-compliant bioproduction protocol and the very low yields of traditional production methods. This means very long research and preclinical testing times, as well as high costs. EVerZom, pioneering exosome-based therapies Since its inception in 2019, EVerZom has focused on developing innovative technological building blocks to make this therapeutic modality a reality for patients. The start-up developed and patented a bioproduction technology that enables reproducible, transferable and scalable production and is capable of multiplying yields by 100, thanks to mechanical stimulation of the cells based on the Kolmogorov length, backed up by a robust quality control system. Today, EVerZom is capable of producing clinical-grade exosomes and aims to develop its pipeline of proprietary therapeutic products for regenerative medicine or in co-development. The company has already forged more than twenty research partnerships worldwide, with biotechnology companies, pharmaceutical laboratories dedicated to human or animal health, and cosmetics companies. This industrial research program has been funded by the EIC Accelerator as part of the European Union's Horizon Europe program. About EVerZom EVerZom, a biopharmaceutical company developing exosomes based biodrugs. Exosomes are a new biological tool in regenerative medicine which are now seen as a promising alternative to cell-based therapies. A spin-off from the CNRS and the Université de Paris Cité, EVerZom initially developed an exclusive proprietary industrial process to produce exosomes on an industrial scale at high yield. EVerZom's ambition today is to build on this technological innovation platform to develop its therapeutic pipeline in three key therapeutic areas: digestive tissue healing, dermatology and liver regeneration. EVerZom has won numerous awards and programmes, including i-Lab and the prestigious EIC Accelerator, for its technological innovation platform. For more information: http://everzom.com Press contact : Florence Portejoie, FP2COM, 06 07 76 82 83, fportejoie@fp2com.fr About EFS The Établissement français du sang is France's public blood service. We are nearly 10,000 professionals, present at every stage of the healthcare chain, helping to treat over 1 million patients every year.We give to blood the power to heal and are a unique and essential player in the French healthcare system.Over the years, our establishment has built up an entire ecosystem of research and innovation. This activity gives rise to innovative therapies that we are able to develop and produce for the EFS and its partners.For over 10 years, EFS has capitalized on its expertise in cell bioproduction to offer its know-how in clinical batch biomanufacturing, developing innovative medical processes and treatments on its 4 pharmaceutical platforms (MTI) throughout France. Attachment EverZom_PR_First_GMP_Stem_Cell_Derived_exosomes_VDEF

PARIS--( BUSINESS WIRE )--Evexta Bio SA, a clinical-stage biotechnology company focused on developing first-in-class therapies in oncology, announces the appointment of Robert Doebele as CMO consultant. Dr. Doebele's extensive academic background includes a MD and PhD in Immunology from the University of Pennsylvania School of Medicine, residency and fellowship training in Medical Oncology and Internal Medicine at the University of Chicago Pritzker School of Medicine, and a Bachelor of Arts in Molecular Biology from Princeton University. Throughout his career, Dr. Doebele has made significant contributions to cancer research, focusing on molecular diagnostics, cancer biology, and the development of biomarkers. His previous roles include Associate Clinical Professor at the University of Colorado Anschutz Medical Campus, where he also directed the Thoracic Oncology Research Initiative. Before joining Evexta Bio, Robert was co-founder, Chief Scientific Officer and Chief Medical Officer of Rain Oncology. As CMO consultant, Robert will support the Company’s clinical development strategy, provide expert advice for protocols, processes and rupitasertib clinical plans and participate in scientific advisory committees. Scott Filosi, CEO of Evexta Bio said: “ I am delighted to welcome Robert as CMO consultant. We are confident that Robert’s expertise will greatly enhance our ongoing efforts in advancing clinical research and improving patient outcomes.” About Evexta Bio ( https://www.evextabio.com ) Evexta Bio is a biopharmaceutical company exploring the new frontiers of oncology in search of daring novel therapeutic approaches with the potential to save lives. Now, in the clinic, the company is currently developing two proprietary therapeutic assets with novel mechanisms of action across several indications: Rupitasertib, an optimized S6K inhibitor with efficient AKT1/AKT3 control of compensatory AKT feed-back loop. The oral anti-tumor agent is expected to enter phase 2 clinical trial in ESR1 mt ER+ HER2- advanced breast cancer. EVX020, a sole-in-class KIF20A kinesin inhibitor having shown potent nonclinical efficacy in hematological and solid tumor models. Two strategies are under assessment, development of EVX020 as a prodrug and as a payload for antibody-drug conjugate (ADC). Founded by Truffle Capital, supported by Merck KGaA (Darmstadt, Germany) as shareholder, Evexta Bio has forged alliances with leaders in academia and industry, including CNRS, Paoli-Calmettes Institute (Marseille, France) and Merck KGaA. The company is supported by seasoned management team, board of directors and medical advisory board.