Genethon, a leading gene therapy research facility, has significantly progressed in developing a new generation of adeno-associated virus (AAV) capsids using artificial intelligence (AI). These capsids enhance gene therapies for muscle diseases by targeting muscles more effectively and avoiding the liver.
This breakthrough, published in Nature Communications, utilizes AI to design capsids that selectively bind to integrin alpha V beta 6, a molecule on muscle cell surfaces. By precisely targeting this receptor, the new capsids deliver genetic material directly to muscle tissue, reducing the need for high doses that could cause adverse effects.
Isabelle Richard, head of Genethon’s Progressive Muscular Dystrophies Team indicated that Genethon’s AI-designed gene therapy vectors represent a paradigm shift in neuromuscular disease treatment and that they exhibit increased efficacy and safety, opening new avenues for treating various neuromuscular diseases.
The efficacy of the LICA1 capsid variant was validated in models of Duchenne muscular dystrophyDuchenne muscular dystrophy and limb-girdle muscular dystrophy. At lower doses, it effectively targeted muscle tissue without penetrating the liver, overcoming a major challenge in gene therapy delivery.
Frederic Revah, Genethon’s CEO, highlighted, “These results pave the way for next-generation gene therapies with fewer side effects. They showcase the potential of our AI-driven approach to AAV vector design, positioning Genethon as a leader in gene therapy innovation.”
Genethon’s methodology for designing AAV capsids using AI has opened up new possibilities for treating various target organs in different diseases. The research team successfully discovered several promising variants, demonstrating the potential of AI to accelerate gene therapy development.
Genethon’s pioneering work in gene therapy has led to the discovery of life-changing treatments for rare genetic diseases. The latest innovation with AI-designed capsids further enhances the efficacy and safety of gene therapies, bringing hope to patients suffering from neuromuscular diseases.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
