rare genetic diseases
rare genetic diseases
Revolutionizing Gene Therapy with Engineered CASTs!
Unlocking the potential of CRISPR-associated transposons (CASTs) for precise gene insertion! 🌱💡 Discover how this innovation could transform genetic therapies! 🔍✨
Game-Changing mRNA Partnership Boosts Drug Development!
🌟 Wacker Biotech and RNAV8 Bio join forces to enhance mRNA therapeutics, promising better outcomes and faster drug development! 🚀💡📈
CEO of Généthon Reveals Future of Gene Therapy
🔍 Frédéric Revah, CEO of Généthon, reflects on the past, present, and future of the organization in an interview. 🧬 Généthon was created in 1990 with a focus on gene therapy research for rare genetic diseases. 💡 Revah discusses the challenges of financing development for rare diseases and the need for equity in distribution. 🏭 Généthon is working on improving bioprocessing and reducing the cost of production for gene therapies. 🤝 The organization collaborates with partners in various fields to advance their research. ⚙️ Exciting developments include producing AAV in plants and using artificial intelligence to optimize bioreactors. ❌ The industry faces challenges such as improving the full-to-empty ratio of virus capsids. ❗️ Revah comments on the Pfizer incident and assures that Généthon is closely monitoring safety in their own clinical trial.
