Généthon is at the forefront of gene therapy research, particularly focusing on rare diseases such as Duchenne Muscular Dystrophy and various forms of Limb Girdle Muscular Dystrophy (LGMD). The company has developed innovative gene therapy products, including GNT0004 for Duchenne Muscular Dystrophy and GNT0006, GNT0007, and GNT0008 for different types of LGMD. These therapies utilize adeno-associated virus (AAV) vectors to deliver therapeutic genes, demonstrating efficacy in preclinical studies and progressing towards clinical trials. Généthon's research teams are actively engaged in refining these therapies, leveraging advanced methodologies such as artificial intelligence to enhance vector design and improve treatment precision while minimizing side effects.

In addition to its therapeutic development, Généthon is committed to addressing the challenges of bioproduction and immunogenicity associated with gene therapies. The organization collaborates with partners to develop cost-effective manufacturing processes and improve production efficiency, aiming to make these innovative treatments more accessible to patients. Généthon's immunology program focuses on understanding and managing the interactions between the immune system and gene therapy vectors, which is crucial for ensuring the safety and effectiveness of treatments. Through these efforts, Généthon strives to provide groundbreaking gene therapies that can significantly improve the quality of life for patients suffering from rare genetic disorders.

France

Created in 1990 and funded by AFM-TELETHON, Genethon, a pioneer in deciphering the human genome and identifying the genes associated with genetic diseases is today fully dedicated to the design and development of gene therapy treatments for rare diseases.

At Genethon more than 180 scientists, clinicians and engineers specialized in gene therapy drug development, from research to the clinic, have joined forces in order to provide these innovative treatments to patients affected with rare disorders. These treatments also pave the way for new therapeutic approaches for frequent disorders.

Généthon

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