Gene Therapies targeting neuromuscular diseases

Atamyo Therapeutics is dedicated to developing innovative gene therapies specifically targeting neuromuscular diseases, particularly various forms of limb-girdle muscular dystrophies (LGMD). The company employs advanced techniques in gene therapy, utilizing modified adeno-associated viruses (AAV) to create effective delivery systems for gene replacement. Their approach focuses on designing tailored vectors that enhance the specificity, safety, and efficacy of treatments, thereby minimizing side effects and restoring normal physiological functions in patients. Atamyo's pipeline includes clinical-stage programs for LGMD-R9 and LGMD-R5, with ongoing studies aimed at evaluating the safety and efficacy of their gene therapy candidates, ATA-100 and ATA-200.

The gene therapy candidates ATA-100 and ATA-200 are designed to address specific genetic mutations associated with LGMD-R9 and LGMD-R5, respectively. ATA-100 targets the fukutin-related protein (FKRP) gene, while ATA-200 focuses on the gamma-sarcoglycan gene, both of which are crucial for muscle function. These therapies aim to deliver normal copies of the respective genes to correct the underlying genetic defects. Preclinical studies have shown promising results, indicating that a single systemic injection of these therapies can effectively alleviate symptoms and improve biomarkers associated with these conditions. Atamyo's commitment to advancing these therapies reflects their mission to provide long-lasting and impactful treatments for patients suffering from debilitating neuromuscular disorders.

France

Atamyo Therapeutics is focused on the development of a new generation of safe and effective 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). 

Atamyo Therapeutics

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