Atamyo Therapeutics Presents Promising Results in the First Patients Treated With Its ATA-200 Gene Therapy in the Clinical Trial Targeting LGMD-R5 Limb-girdle Muscular Dystrophy
Atamyo Therapeutics, a biotechnology company specializing in the development of next-generation gene therapies for limb-girdle muscular dystrophy (LGMD), announced at the MDA (Muscular Dystrophy Association) Conference 2026 the first safety, pharmacodynamics, and efficacy results for its ATA-200 gene therapy in LGMD-2C/R5 limb-girdle muscular dystrophy associated with γ-sarcoglycan deficiency (SGCG, gamma-sarcoglycanopathy). The results are from the first patients treated in the clinical trial conducted at the Powell Gene Therapy Center at the University of Florida by Dr. Barry Byrne and supported by The Dion Foundation for Children with Rare Diseases.
IND for ATA-200, a Gene Therapy for the Treatment of Limb-Girdle Muscular Dystrophy Type 2C/R5 (LGMD2C/R5), cleared to proceed by FDA
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.
Atamyo Therapeutics Observes LGMD Awareness Day with Updates on Key Milestones in its Clinical Development of Gene Therapies for Patients Suffering from Limb-Girdle Muscular Dystrophies
In recognition of LGMD Awareness Day today, Atamyo Therapeutics a clinical-stage biotechnology company focused on the development of new generation gene therapies targeting muscular dystrophies and cardiomyopathies, announced the enrollment of the last patient in the dose-escalation phase of its Phase 1b clinical trial of ATA-100, a gene therapy for patients with limb-girdle muscular dystrophy Type 2I/R9 (LGMD-2I/R9, protocol code ATA-001-FKRP).
Atamyo Therapeutics to Provide Corporate Overviews and Updates on Its Pipeline Progress at 6 Upcoming Conferences in the US and Europe
Atamyo Therapeutics, a biotechnology company focused on the development of new-generation gene therapies targeting neuromuscular disease, today announced its participation in six upcoming conferences in the US and Europe to highlight recent developments in its programs targeting limb-girdle muscular dystrophies (LGMDs).
The Dion Foundation and Atamyo Therapeutics Announce a Partnership to Expand into the US Atamyo’s Clinical Trial of ATA-200 Gene Therapy to Treat Limb-Girdle Muscular Dystrophy Type 2C/R5
Atamyo Therapeutics and The Dion Foundation for Children with Rare Diseases, today announced a key partnership to expand into the US a first-in-human clinical trial of ATA-200, Atamyo’s gene therapy for the treatment of the γ-sarcoglycan related limb-girdle muscular dystrophy Type 2C/R5 (LGMD2C/R5).
Atamyo Therapeutics Obtains Regulatory Authorization in Europe to Initiate a Clinical Trial for ATA-200, its Gene Therapy to Treat Limb-Girdle Muscular Dystrophy Type 2C/R5
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).
First Clinical Results of ATA-100, a Gene Therapy for the Treatment of Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9), Presented at ESGCT
Atamyo Therapeutics, a biotechnology company focused on the development of new-generation gene therapies targeting muscular dystrophies and cardiomyopathies, today announced the presentation of the first clinical results obtained with ATA-100 in the on-going ATA-001 Phase 1b/2b clinical trial. ATA-100 is a one-time gene therapy for the treatment of fukutin-related protein (FKRP) limb-girdle muscular dystrophy Type 2I/R9 (LGMD2I/R9). Atamyo also announced today that Data Safety Monitoring Board (DSMB) authorized the enrollment of the second dose cohort of the ATA-001 clinical trial.
Atamyo Therapeutics Announces participation in Conferences and Key Scientific Communications on its LGMD programs
Atamyo Therapeutics, a biotechnology company focused on the development of new-generation gene therapies targeting neuromuscular disease, today announced its participation to forthcoming conferences and scientific communications on its programs targeting limb-girdle muscular dystrophies (LGMDs).
ATA-200, Atamyo Therapeutics’ Gene Therapy to Treat Limb-Girdle Muscular Dystrophy Type 2C/R5, Reaches Key Milestones with the Filing of a Clinical Trial Application in Europe and a Non-Dilutive Financing from France 2030 Program
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).
IND for ATA-100, a Gene Therapy for the Treatment of Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9), cleared to proceed by FDA
Atamyo Therapeutics, a 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-100 to proceed in a Phase 1b/2b clinical trial. ATA-100 is a one-time gene therapy for the treatment of fukutin-related protein (FKRP) limb-girdle muscular dystrophy Type 2I/R9 (LGMD2I/R9).
Gene Therapy Pioneer Roger Hajjar, MD, Joins Atamyo Therapeutics Board of Directors
Atamyo Therapeutics, a biotechnology company focused on the development of new-generation gene therapies targeting muscular dystrophies and cardiomyopathies, today announced the appointment of Roger J. Hajjar, MD, Director of the Gene and Cell Therapy Institute at Mass General Brigham in Boston, to its Board of Directors.
Atamyo Therapeutics Announces First Patient Dosed with ATA-100 Gene Therapy in LGMD-R9 Clinical Trial
Atamyo Therapeutics, a biotechnology company focused on the development of new-generation gene therapies targeting neuromuscular diseases, today announced the dosing with ATA-100 of a first patient in a phase 1/2 clinical study in FRKP-related limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9).
Atamyo Therapeutics Announces Significant Milestones for ATA-100 and ATA-200, its Gene Therapy Programs to Treat Limb-Girdle Muscular Dystrophy 2I/R9 and 2C/R5
Atamyo Therapeutics, a biotechnology company focused on the development of new-generation gene therapies targeting neuromuscular diseases, today announced multiple major milestones for ATA-100 and ATA-200, its one-time gene-replacement therapies for the treatment of limb-girdle muscular dystrophy types 2I/R9 and 2C/R5 (LGMD2I/R9 and LGMD2C/R5 respectively), as well as the reinforcement of its management team.
Atamyo Therapeutics Reaches Significant Regulatory and Financial Milestones for ATA-100, its Gene Therapy to Treat Limb-Girdle Muscular Dystrophy Type 2I/R9
Atamyo Therapeutics, a biotechnology company focused on the development of new-generation gene therapies targeting neuromuscular diseases, today announced multiple major regulatory and financial milestones for ATA-100, a one-time gene therapy for the treatment of fukutin-related protein (FKRP) limb-girdle muscular dystrophy Type 2I/R9 (LGMD2I/R9).
Atamyo Therapeutics Obtains First Regulatory Authorization in Europe to Initiate a Clinical Trial for ATA-100, its Gene Therapy to Treat Limb-Girdle Muscular Dystrophy Type 2I/R9
Atamyo Therapeutics, a biotechnology company focused on the development of new-generation gene therapies targeting neuromuscular diseases, today announced the first authorization of a Clinical Trial Application (CTA) in Europe for ATA-100, its gene therapy for the treatment of the fukutin-related protein (FKRP) limb-girdle muscular dystrophy Type 2I/R9 (LGMD2I/R9). This authorization was granted by the United Kingdom Medicines & Healthcare products Regulatory Agency (MHRA). Additional CTAs were filed in France and Denmark.
Atamyo Therapeutics Submits CTA in Europe for ATA-100, Its Gene Therapy to Treat Limb-Girdle Muscular Dystrophy Type 2I/R9
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-100, its gene therapy targeting fukutin-related protein (FKRP) limb-girdle muscular dystrophy Type 2I/R9 (LGMD2I/R9).
