At ASGCT 2026 in Boston, Genethon presented new clinical data from three of its gene therapy programs in Duchenne muscular dystrophy, gamma-sarcoglycanopathy (LGMD-R5), and Crigler-Najjar syndrome. In total, Genethon teams presented 8 oral presentations and 13 posters.

Atamyo Therapeutics presented the first safety, pharmacodynamics, and efficacy data from its ATA-200 gene therapy in LGMD-R5 at the ASGCT 2026 Annual Meeting. Results observed in the first treated patients showed high SGCG protein expression together with improvements in several biological and functional parameters

The discovery that the abnormal repetition of a short DNA sequence leads to the production of toxic proteins opens the way to new therapeutic approaches in oculopharyngodistal myopathy and oculopharyngeal myopathy with leukoencephalopathy.