SGCG

Atamyo has received approval to initiate a clinical trial of ATA-200 gene therapy in France and Italy ATA-200 is a single-injection gene therapy aimed to treat LGMD2C/R5 caused by mutations in the γ-sarcoglycan gene The phase 1b, dose-escalation study will evaluate the safety and efficacy of ATA-200 in children 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 the first authorization of a Clinical Trial Application (CTA) in Europe for ATA-200, its gene therapy for the treatment of the γ-sarcoglycan related limb-girdle muscular dystrophy Type 2C/R5 (LGMD2C/R5). This authorization was first granted by the Italian Medicines Agency (AIFA), then by the French Medicines Agency (ANSM). “We are thrilled to obtain our CTA approval in France and Italy for the devastating LGMD2C/R5 disease affecting primarily a pediatric population and for which there is no approved treatment,” said Dr Sophie Olivier, Chief Medical Officer of Atamyo. “Atamyo plans to initiate dosing in patients for ATA-200 in third quarter 2024.” “LGMD 2C/R5 is a severe muscular dystrophy with an onset in early childhood and loss of ambulation generally occurring before adolescence,” said Pr. Giacomo Comi, Full Professor of Neurology at the University of Milan (Italy), and principal investigator of this trial. “It is a great motivation to know that the work we are doing has the potential to make a life-changing difference for the patients affected by this disease.” “This is a significant milestone for LGMD-2C/R5 patients and for Atamyo as ATA-200 is the first treatment targeting LGMD-2C/R5 to enter clinical trials,” said Stéphane Degove, CEO of Atamyo Therapeutics. “With the ongoing clinical trial of ATA-100 in LGMD2I/R9, the initiation of the clinical program for ATA-200 confirms our unique capabilities in bringing to patients suffering from limb-girdle muscular dystrophies a new generation of safe and effective gene therapies.” This clinical trial (NCT05973630) is a multicenter, Phase 1b, open-label, dose escalation study evaluating safety, pharmacodynamic, efficacy, and immunogenicity of intravenous ATA-200, a single-dose Adeno-Associated Virus (AAV) vector carrying the human γ-sarcoglycan transgene. LGMD2C/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. 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. There is no curative treatment. The management of LGMD2C/R5 is solely supportive. ATA-200, a gene therapy candidate for LGMD2C/R5, delivers a normal copy of the gene for production of γ-sarcoglycan protein. In preclinical models, a single systemic injection of ATA-200 demonstrated its tolerability and capability to correct symptoms and biomarkers of the pathology. ATA-200 has been granted Orphan Medicinal Product Designation by the European Medicines Agency (EMA). The therapy is based on the research of Atamyo Chief Scientific Officer Isabelle Richard, Ph.D., Research Director at National Center of Scientific Research (CNRS) in France and head of the Progressive Muscular Dystrophies Laboratory at Genethon. In addition to its LGMD2C/R5 gene therapy, Atamyo is developing a clinical trial with ATA-100 gene therapy for LGMD2I/R9, related to deficiencies in FKRP; and is in IND-enabling studies for LGMD2A/R1, related to deficiencies in calpain protein. 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 LGMD2I/R9 and LGMD2C/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 . View source version on businesswire.com: Contacts U.S. Contact: Charles Craig, Opus Biotech Communications Charles.s.craig@gmail.com, 404-245-0591 European Contact: contact@atamyo.com Source: Atamyo Therapeutics View this news release online at:

A multicenter phase 1b study will evaluate safety, pharmacodynamic and efficacy of ATA-200 ATA-200 is the second Atamyo’s next generation gene replacement therapy entering the clinic ATA-200 is eligible to receive up to €8m ($8.6m) of non-dilutive financing from France 2030 program EVRY, France--(BUSINESS WIRE)-- Atamyo Therapeutics, a biotechnology company focused on the development of new-generation gene therapies targeting neuromuscular diseases, today announced the filing of a Clinical Trial Application (CTA) in Europe for ATA-200, its gene therapy targeting γ-sarcoglycan (SGCG) related limb-girdle muscular dystrophy Type 2C/R5 (LGMD2C/R5). The company also announced that ATA-200 has been granted a non-dilutive financing up to €8m ($8.6m) from France 2030 public program managed by Bpifrance, to support its clinical trials and manufacturing development programs. This clinical trial (EUCT 2023-506440-16-00) is a multicenter, Phase 1b, open-label, dose escalation study evaluating safety, pharmacodynamic, efficacy, and immunogenicity of intravenous ATA-200, a single-dose Adeno-Associated Virus (AAV) vector carrying the human γ-sarcoglycan transgene. “LGMD-R5 is the most rapidly progressive and debilitating form of LGMDs with onset of symptoms in early childhood, a loss of ambulation before adulthood, and frequent cardiac impairment. No curative treatment exists for this disease,” said Dr Sophie Olivier, Chief Medical Officer of Atamyo. “ATA-200 incorporates a new promoter that enhances the liver and cardiac safety of gene therapy,” said Isabelle Richard, Ph.D., Co-founder and Chief Scientific Officer of Atamyo. “This first-in-class experimental treatment represents a new hope for the patients.” “We are thrilled to European Clinical trial and to receive such a strong public financing for the devastating LGMD2C/R5 disease,” said Stephane Degove, Co-Founder and Chief Executive Officer of Atamyo. “With the ongoing clinical trial in LGMD-R9, the initiation of the clinical program for LGMD-R5/2C confirms our unique capabilities in bringing to patients suffering from limb-girdle muscular dystrophies a new generation of safe and effective gene therapies.” LGMD2C/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 only. 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. There is no curative treatment. The management of LGMDR5 is solely supportive. ATA-200, the gene therapy for LGMD2C/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 dosing in patients for ATA-200 in the first half of 2024. In addition to its LGMD2C/R5 gene therapy, Atamyo is developing a clinical trial with ATA-100 gene therapy for LGMD2I/R9, related to deficiencies in FKRP; and is in IND-enabling studies for LGMD2A/R1, related to deficiencies in calpain protein. 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 About France 2030 Launched by the President of France Republic in October 2021, France 2030 is a €54 ($58) billion program which aims to accelerate the transformation of key sectors of the French economy through innovation. From fundamental research, emergence of an idea, and up to the production of a new product or service, France 2030 supports the entire life cycle of innovation until its industrialization. More information on : france2030.gouv.fr View source version on businesswire.com: Contacts U.S. Contact: Charles Craig, Opus Biotech Communications charles.s.craig@gmail.com, 404-245-0591 European contact: contact@atamyo.com Source: Atamyo Therapeutics View this news release online at:

ROCKVILLE, Md., April 27, 2021 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that four oral and nine poster presentations will be presented at the American Society of Gene and Cell Therapy's 24th Annual Meeting taking place from May 11 to 14, 2021, in virtual format. Presentations and posters will be available at . Oral presentations include: Abstract Title: Effects of Sexual Dimorphism and Genetic Background on AAV Tissue Transduction in Mice Following Intravenous Administration of a Diverse Capsid Pool (abstract #94) Presenter: Elad Firnberg, Ph.D., Scientist II, Gene Transfer Technologies, REGENXBIO Session Title: Novel Factors in AAV Transduction and AAV Genomes Date/Time: Wednesday, May 12, 2021 from 6:00 to 6:15 p.m. ET Abstract Title: Intravenous Administration of a Barcoded and Pooled AAV Library for the Comprehensive Characterization and Comparison of Capsid Tropisms (abstract #189) Presenter: April Giles, Ph.D., Scientist II, Gene Transfer Technologies, REGENXBIO Session Title: Novel AAV Biology and Platform Technologies Date/Time: Thursday, May 13, 2021 from 6:00 to 6:15 p.m. ET Abstract Title: High-Throughput Screening of AAV Productivity to Enable Rapid Capsid Characterization (abstract #192) Presenter: Jenny Egley, Scientist, Gene Transfer Technologies, REGENXBIO Session Title: Novel AAV Biology and Platform Technologies Date/Time: Thursday, May 13, 2021 from 6:45 to 7:00 p.m. ET Abstract Title: RGX-121 Gene Therapy for the Treatment of Severe Mucopolysaccharidosis Type II: Interim Analysis of the First in Human Study (abstract #215) Presenter: Roberto Giugliani, M.D., Ph.D., Professor, Department of Genetics, UFRGS, Medical Genetics Service, HCPA, Porto Alegre, Brazil Session Title: Gene Therapy for Lysosomal Storage Disorders Date/Time: Friday, May 14, 2021 from 12:15 to 12:30 p.m. ET Poster presentations include: Abstract Title: Identification of Transcriptional Start Sites in a Tandem Promoter by 5'-RACE and NGS (abstract #317) Presenter: Kirk Elliot, Senior Associate Scientist, REGENXBIO Session Title: AAV Vectors - Virology and Vectorology Date/Time: Tuesday, May 11, 2021 from 8:00 to 10:00 a.m. ET Abstract Title: Comparative Cardiac, Neurologic, and Skeletal Effectiveness of Intravenous and Intrathecal AAV9-IDUA Delivered Individually or Combined in a Murine Model of Mucopolysaccharidosis Type I (abstract #490) Presenter: Lalitha Belur, Ph.D., Assistant Professor Genetics, Cell Bio at University of Minnesota Session Title: Metabolic, Storage, Endocrine, Liver and Gastrointestinal Diseases Date/Time: Tuesday, May 11, 2021 from 8:00 to 10:00 a.m. ET Abstract Title: Comprehensive Evaluation of an AAV Library in the Mice and Nonhuman Primate Ocular Tissues Following Intravitreal Delivery (abstract #568) Presenter: Wei-Hua Lee, Ph.D., Scientist II, Research and Early Development, Target Discovery, REGENXBIO Session Title: Neurologic Diseases Date/Time: Tuesday, May 11, 2021 from 8:00 to 10:00 a.m. ET Abstract Title: RGX-202, an AAV8 Vector Encoding an Extended Microdystrophin Efficiently Restores Dystrophin Associated Protein Complex and Corrects Satellite Cells in a Dystrophic Mouse Model (abstract #597) Presenter: Chunping Qiao, Ph.D., Principal Scientist, REGENXBIO Session Title: Musculo-Skeletal Diseases Date/Time: Tuesday, May 11, 2021 from 8:00 to 10:00 a.m. ET Abstract Title: A Novel AAV8 Vector for Microdystrophin Gene Therapy of Duchenne Muscular Dystrophy: Preclinical Studies in the MDX Mouse (abstract #614) Presenter: SunJung Kim, Ph.D., Senior Scientist, Pharmacology and Toxicology, Preclinical Development, REGENXBIO Session Title: Musculo-Skeletal Diseases Date/Time: Tuesday, May 11, 2021 from 8:00 to 10:00 a.m. ET Abstract Title: A Case Study on Viral Clearance for a Downstream AAV Process using a Model Virus Panel and Non-Infectious MVM Surrogate (abstract #877) Presenter: Shaojie Weng, Scientist II Session Title: Pharmacology/Toxicology Studies or Assay Development Date/Time: Tuesday, May 11, 2021 from 8:00 to 10:00 a.m. ET Abstract Title: Assessment of Inflammatory and Neurodegenerative Biomarkers in NHPs with DRG Toxicity (abstract #889) Presenter: Ryan Reddinger, Ph.D., Scientist I, Preclinical Development, REGENXBIO Session Title: Pharmacology/Toxicology Studies or Assay Development Date/Time: Tuesday, May 11, 2021 from 8:00 to 10:00 a.m. ET Abstract Title: Quantification of Double-Stranded RNAs Produced by AAV Vectors: A Methodological Reassessment (abstract #891) Presenter: Chunping Qiao, Ph.D., Principal Scientist, REGENXBIO Session Title: Pharmacology/Toxicology Studies or Assay Development Date/Time: Tuesday, May 11, 2021 from 8:00 to 10:00 a.m. ET Abstract Title: Identification of a Kidney Specific AAV Following Insertion of Tissue Homing Peptides in Variable Region IV (abstract #292) Presenter: Samantha A. Yost, Ph.D., Scientist II, Gene Transfer Technologies, REGENXBIO Session Title: AAV Vectors - Virology and Vectorology Date/Time: Tuesday, May 11, 2021 from 8:00 to 10:00 a.m. ET About REGENXBIO Inc. REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO's NAV Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates in multiple therapeutic areas. Contacts: Tricia Truehart Investor Relations and Corporate Communications 347-926-7709 ttruehart@regenxbio.com Investors: Brendan Burns, 212-600-1902 brendan@argotpartners.com Media: David Rosen, 212-600-1902 david.rosen@argotpartners.com Company Codes: NASDAQ-NMS:RGNX