SGCG

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

-- Protein expression in muscle was sustained for two years following treatment in the low dose cohort, with mean beta-sarcoglycan expression of 54% at 24 months, compared to 36% at Day 60, as measured by western blot -- -- Mean NSAD score improvement of 5.7 points from baseline was sustained through 24 months in low-dose cohort, and mean NSAD score improvement of 4.0 points from baseline at one year in high-dose cohort -- -- Results in both cohorts continue to reinforce the safety and tolerability profile of SRP-9003 -- CAMBRIDGE, Mass., March 18, 2021 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today shared new results from the ongoing study of SRP-9003 (rAAVrh74.MHCK7.hSGCB), the Company’s investigational gene therapy for limb-girdle muscular dystrophy Type 2E (LGMD2E). In the first look at expression data from biopsies taken two years after a single administration of SRP-9003, results found sustained protein expression in muscle tissue. In functional outcomes assessments taken two years following treatment in Cohort 1 (low-dose cohort) and one year after treatment in Cohort 2 (high-dose cohort), patients continued to demonstrate stability in their NSAD (North Star Assessment for Dysferlinopathies) total score and improvements on timed function tests. Results are being presented today at the 2021 Muscular Dystrophy Association (MDA) Annual Clinical and Scientific Conference. SRP-9003 is in development for the treatment of LGMD2E (also known as beta-sarcoglycanopathy and LGMDR4), a devastating monogenic neuromuscular disease caused by a lack of beta-sarcoglycan (beta-SG) proteins. SRP-9003 is a gene therapy construct that transduces skeletal and cardiac muscle, delivering a gene that codes for the full-length beta-SG protein, the absence of which is the sole cause of the progressive degeneration and a shortened lifespan characterized by the disease. “This data is the first look at longer-term expression data with any gene therapy for muscular dystrophy. The meaningful and sustained levels of beta-sarcoglycan protein expression at two years and continued strength of the functional outcomes measured are tremendously positive and support continued advancement of this investigational treatment for patients,” said Louise Rodino-Klapac, Ph.D., executive vice president and chief scientific officer, Sarepta Therapeutics. “In Cohort 2, we also saw strong expression of delta-sarcoglycan and gamma-sarcoglycan proteins in addition to beta-sarcoglycan, which suggests that SRP-9003 is working to restore the dystrophin associated protein complex, or DAPC, which provides biological support for the sustained functional benefits observed in both cohorts. LGMD2E is one of the most severe forms of LGMD and causes significant disability in children while frequently leading to early mortality and the data continue to suggest this treatment could bring much needed hope to these patients.” Efficient transduction in skeletal muscle and robust beta-sarcoglycan protein expression were seen in both dose cohorts following infusion with SRP-9003, and significant creatine kinase (CK) reductions were observed. Cohort 1 (Dosed at 1.85×1013 vg/kg), 24 months following treatment: As measured by western blot, mean beta-SG of 54% at 24 months of normal control, compared to 36% measured at Day 60. Percent immunofluorescent (IF) positive fibers was 48% compared to normal control, compared to 51% at Day 60. Participants showed a mean intensity of 35% of transduced beta-SG, properly localized to the muscle sarcolemma, as measured by IF, compared to 47% at Day 60. The mean NSAD improvement from baseline of 5.7 points at 18 months was sustained through 24 months. All three participants demonstrated continued improvements from baseline across all functional measures, including time-to-rise, four-stair climb, 100-meter walk test and 10-meter walk test. Cohort 2 (Dosed at 7.41×1013 vg/kg), 12 months following treatment: At Day 60, the expression of beta-SG (72% mean positive fibers and 73% mean intensity) resulted in increased expression of delta-sarcoglycan, with 65% mean positive fibers and 103% intensity, and gamma-sarcoglycan, 60% mean positive fibers and 97% intensity. These results suggest treatment with SRP-9003 demonstrates reconstitution of the DAPC, which could lead to improved membrane integrity and thus improved clinical motor outcomes measures. All three participants demonstrated improvements from baseline across all functional measures, including the NSAD, time-to-rise, four-stair climb, 100-meter walk test and 10-meter walk test. The mean NSAD improvement from baseline was 4.0 points at one year, compared to 3.7 at 6 months. In an exploratory evaluation of all SRP-9003 treated patients compared to a natural history cohort; patients treated with SRP-9003 demonstrated significant improvements in functional outcomes after 24 months. The mean decline in total NSAD score for patients in the natural history cohort was 4.6 points while SRP-9003 treated patients demonstrated a mean improvement of 4.6 points for a clinically meaningful difference of 9.2 points. Since the last update from this study in October 2020, there have been no new drug-related safety signals observed, and no decreases in platelet counts outside of the normal range and no evidence of clinical complement activation observed in either dose cohort. About SRP-9003 and the Study SRP-9003 uses the AAVrh74 vector, which is designed to be systemically and robustly delivered to skeletal, diaphragm and cardiac muscle, making it an ideal candidate to treat peripheral neuromuscular diseases. AAVrh74 has lower immunogenicity rates than reported with other human AAV vectors. The MHCK7 promoter has been chosen for its ability to robustly express in the heart, which is critically important for patients with limb-girdle muscular dystrophy Type 2E (LGMD2E), also known as beta-sarcoglycanopathy and LGMDR4, many of whom die from pulmonary or cardiac complications. This open label, first-in-human study is evaluating a single intravenous infusion of SRP-9003 among children with LGMD2E between the ages of 4 and 15 years with significant symptoms of disease. The SRP-9003 study has two cohorts, each studying a different dose-per-kilogram based on the weight of the patient. Three participants in the low-dose cohort (Cohort 1) were treated with a one-time infusion of SRP-9003 dosed at 1.85×1013 vg/kg and an additional three participants in the high-dose cohort (Cohort 2) received a one-time infusion dosed at 7.41×1013 vg/kg based on linear standard qPCR titer method. The six participants were between the ages of 4 and 13. Post-treatment biopsies were taken at 60 days. Sarepta has exclusive rights to the LGMD2E gene therapy program initially developed at the Abigail Wexner Research Institute at Nationwide Children’s Hospital. About Limb-girdle Muscular Dystrophy Limb-girdle muscular dystrophies are genetic diseases that cause progressive, debilitating weakness and wasting that begin in muscles around the hips and shoulders before progressing to muscles in the arms and legs. Patients with limb-girdle muscular dystrophy Type 2E (LGMD2E) begin showing neuromuscular symptoms such as difficulty running, jumping and climbing stairs before age 10. The disease, which is an autosomal recessive subtype of LGMD, progresses to loss of ambulation in the teen years and often leads to early mortality. There is currently no treatment or cure for LGMD2E. Sarepta has five LGMD gene therapy programs in development, including subtypes for LGMD2E, LGMD2D, LGMD2C, LGMD2B and LGMD2L, and holds an option for a sixth program for LGMD2A. About Sarepta Therapeutics At Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophies (LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth (CMT), and other CNS-related disorders, with more than 40 programs in various stages of development. The Company’s programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. For more information, please visit or follow us on Twitter, LinkedIn, Instagram and Facebook. Forward-Looking Statements This press release contains "forward-looking statements." Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding, SRP-9003 being the ideal candidate to treat peripheral neuromuscular diseases; the potential benefits of SRP-9003, including its potential to restore the dystrophin associated protein complex (DAPC); the potential benefits of MHCK7 and the AAVrh74 vector, including its potential to be systemically and robustly delivered to skeletal, diaphragm and cardiac muscle; and potential market opportunities. These forward-looking statements involve risks and uncertainties, many of which are beyond our control. Known risk factors include, among others: success in preclinical trials and clinical trials, especially if based on a small patient sample, does not ensure that later clinical trials will be successful; the data presented in this release may not be consistent with the final data set and analysis thereof or result in a safe or effective treatment benefit; different methodologies, assumptions and applications we utilize to assess particular safety or efficacy parameters may yield different statistical results, and even if we believe the data collected from clinical trials of our product candidates are positive, these data may not be sufficient to support approval by the FDA or foreign regulatory authorities; if the actual number of patients suffering from LGMD is smaller than estimated, our revenue and ability to achieve profitability may be adversely affected; we may not be able to execute on our business plans and goals, including meeting our expected or planned regulatory milestones and timelines, clinical development plans, and bringing our product candidates to market, due to a variety of reasons, some of which may be outside of our control, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover our product candidates and the COVID-19 pandemic; and even if Sarepta’s programs result in new commercialized products, Sarepta may not achieve the expected revenues from the sale of such products; and those risks identified under the heading “Risk Factors” in Sarepta’s most recent Annual Report on Form 10-K for the year ended December 31, 2020 filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company which you are encouraged to review. Any of the foregoing risks could materially and adversely affect the Company’s business, results of operations and the trading price of Sarepta’s common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof. Internet Posting of Information We routinely post information that may be important to investors in the 'For Investors' section of our website at . We encourage investors and potential investors to consult our website regularly for important information about us. Source: Sarepta Therapeutics, Inc. Sarepta Therapeutics, Inc. Investors: Ian Estepan, 617-274-4052 iestepan@sarepta.com Media: Tracy Sorrentino, 617-301-8566 tsorrentino@sarepta.com