CGTLive®’s Weekly Rewind – December 20, 2024

Welcome to CGTLive®’s Weekly Rewind! We’ve compiled 5 highlights from this week’s coverage of advances in gene and cell therapies, including FDA actions, notable research, and interviews with experts across the field.

1. FDA Approves Mesoblast’s Remestemcel-L for Steroid-Refractory GvHD

The product will be marketed under the name Ryoncil, and is indicated for patients aged 2 months and older.

2. Françoise Bernaudin, MD, on alloSCT Superiority Over SOC for Sickle Cell Anemia

The head of the Referral Center for Sickle Cell Disease and Clinical Research Department at Hôpital Intercommunal de Créteil of the Université Paris Cité discussed the Drepagreffe-1 and 2 studies and improvements seen over 10 years of follow-up.

3. uniQure and FDA Reach Accord on Approval Pathway for Huntington Disease Gene Therapy AMT-130

The agency agreed that data from the ongoing phase 1/2 studies compared to external control natural history data will be sufficient to support a BLA.

4. CGTLive® Presents: ImmunoLogic, A Video Podcast With Joseph Fraietta, PhD

Hosted by Joseph Fraietta, PhD, ImmunoLogic will be tailored for an audience fluent in the language of medicine and biotechnology, offering data-driven insights while maintaining accessibility.

5. The Power and Potential of Natural Killer Cell Therapies: A New Awakening

Natural killer cells could redefine medicine and offer new hope for cancer, autoimmune diseases, and neurological disorders through advanced CAR-NK therapies.