Taysha Gene Therapies is focusing on AAV-based gene therapies for severe monogenic CNS diseases, with findings supporting the effectiveness of intrathecal administration for delivering these therapies.

Dr. Jean-Philippe Combal discussed the limitations of the current standard of care for CTX, highlighting the need for innovative treatments like VTX-806.

The European Society of Gene and Cell Therapy (ESGCT) 2024 Annual Congress is currently taking place in Rome, Italy, from October 22-25, showcasing groundbreaking research in gene therapy.

Key presentations will cover research on AAV vectors for Duchenne muscular dystrophy, liver-directed AAV gene transfer for glycogen storage disease, and long-term gene therapy efficacy in canine models.

Additionally, Genethon collaborators will present posters on topics such as AAV vector purification, glycosylation tests, and the therapeutic efficacy of dystrophin variants.

Despite a total funding of $10.9 billion for cell and gene therapies in the first half of 2023, investments have become more strategic, focusing on efficacy data from Phase II trials.

Affinia Therapeutics is presenting promising data on its MYBPC3 HCM program, which has shown the ability to reverse disease pathophysiology and restore cardiac function in over 110,000 affected patients.

The company also revealed that its AFTX-201 gene therapy for BAG3 dilated cardiomyopathy demonstrated full restoration of cardiac function in a relevant mouse model after a single intravenous dose.

At the event, four oral presentations and 18 posters will highlight advancements in gene therapies for various diseases.

Cellectis has made strides in understanding C-to-T conversion efficiencies, which enhances the potential therapeutic applications of TALEB technology.

Frederic Revah, CEO of Genethon, emphasized the importance of sharing research results that could transform patient care and highlighted the organization's leadership in gene therapy.

Panelists at the congress noted a shift in investor interest towards obtaining efficacy data, moving away from preclinical acquisitions.