Month: February 2026

Atamyo Therapeutics Announces the Dosing of a Fourth Patient with ATA-200 in an Ongoing LGMD-R5 Clinical Trial, Supported by CureSCG

Posted on by admin_ata

Atamyo Therapeutics announces continued progress in its ongoing clinical trial of ATA-200, with the recent dosing of a fourth patient, marking an important milestone in the development of its gene therapy for γ-sarcoglycan related limb girdle muscular dystrophy Type 2C/R5 (LGMD2C/R5).

Atamyo acknowledges the support of CureSCG, whose contribution has played an important role in supporting the ongoing clinical trial. CureSCG is a patient advocacy organization committed to accelerating research efforts in LGMD sarcoglycanopathies and supporting families affected by the disease. Their involvement reflects a shared commitment to advancing therapeutic innovation for the patient community. 

 “This new step reflects the coordinated efforts of clinical teams, the trust of patients, and the commitment of our partners. The support from CureSCG demonstrates the strength of a community working together to advance therapeutic options for individuals living with γSGC.” — Angela Columbano, CEO, Atamyo Therapeutics.

“We are proud to contribute to the advancement of this important clinical program and deeply grateful to the Dion Foundation for initiating this effort in collaboration with Atamyo. We also thank Atamyo and the clinical teams for their continued dedication. Supporting the dosing of the fourth patient represents a meaningful step forward for the γ-SGC community, and we remain committed to helping move this research forward for families who are waiting for treatment options.” — Rana Abu Khadra, Co-Founder, CureSCG.

CureSCG intends to continue supporting the program as it advances, both within and outside the United States, in alignment with its long term mission to advance scientific and clinical efforts for individuals living with LGMD sarcoglycanopathies.

Atamyo remains firmly committed to advancing safe and innovative gene therapies for rare neuromuscular diseases and looks forward to the continued progression of the γSGC clinical program. 

Atamyo Therapeutics Announces First Three Patients Dosed with ATA-200 Gene Therapy in on-going LGMD-R5 Clinical Trial, and Extension of Partnership with the Dion Foundation

Posted on by admin_ata
  • First three patients dosed with ATA-200 gene replacement therapy 
  • Extension of Partnership with The Dion Foundation for Children with Rare Diseases to support financing of the Phase 1b, dose-escalation study evaluating the safety and efficacy of ATA-200 in children with LGMD-R5
  • First clinical results from the ongoing study conducted by Dr. B. Byrne (U. Florida) expected to be published in the coming months

Evry, France and Boston, MA (February 3, 2026) – Atamyo Therapeutics and The Dion Foundation for Children with Rare Diseases, today announced the expansion of their partnership to support financing of the 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), and the dosing of a third US pediatric patient.

The Phase1b/2 open-label dose escalation study (NCT05973630) evaluates safety, pharmacodynamics, efficacy, and immunogenicity in children receiving intravenous ATA-200, a single-dose Adeno-Associated Virus (AAV) gene therapy carrying the human γ-sarcoglycan transgene. The study is being conducted at the Powell Gene Therapy Center, University of Florida, by Dr. Barry Byrne.

Additional results from this ongoing study are expected to be published in the coming months as further follow-up data become available.

“We are delighted to expand our partnership with Atamyo to treat more U.S. children in the very first clinical trial for children with LGMD2C/R5,” said Courtney Dion, Co-Founder and President of the Dion Foundation. “The Dion Foundation fully supports Atamyo’s ATA-200 program and the promise it holds to bring real, lasting impact to children living with LGMD2C and their families.”

“We are deeply grateful of the collaboration with The Dion Foundation and with Dr. Barry Byrne at the Powell Gene Therapy Center.” said Angela Columbano, Chief Executive Officer of Atamyo Therapeutics. With the support of Atamyo’s shareholder, Genethon, we are delighted of the progress of this first-in-human trial in LGMD-R5. Beyond the children currently enrolled, this study contributes important scientific and clinical knowledge that helps advance the field of therapies for LGMD for patients around the world, and we are proud of giving children affected by LGMD-R5 the opportunity to receive a treatment that could be life-changing.”

“We have been delighted to work with Atamyo and the Dion Foundation in the design and conduct of the ATA-200 study.” said Dr. Barry Byrne, Associate Chair of Pediatrics and Director of the Powell Gene Therapy Center, University of Florida, in Gainesville, Florida, where the first three patients were dosed, and principal investigator of this trial. “We look forward to continuing assessing the potential benefit of ATA-200 in the first children to receive the product.”

About the ATA-200 program in LGMD-2C/R5

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 presented 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, Atamyo’s 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. 

ATA-200 has been awarded Orphan Drug Designation in the US and Europe, and Rare Pediatric Disease Designation by the US FDA.

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.

US & Europe contacts: contact@atamyo.com
Stephanie Bardon – communication@genethon.fr /+33 (0)6 45 15 95 87
Daniel Eramian Opus Biotech Communications – danieleramian@comcast.net