CGTLive®’s Weekly Rewind – April 25, 2025
Review top news and interview highlights from the week ending April 25, 2025.
CGTLive®’s Weekly Rewind
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. Cartesian Therapeutics’ mRNA CAR-T Descartes-08 Shows Efficacy and Safety in Myasthenia Gravis in Phase 2 Study
During the 12-month study, Descartes-08 was deemed well-tolerated.
2. Linda Marbán, PhD, on Evaluating Capricor’s Cardiomyopathy Cell Therapy Deramiocel for DMD
The CEO of Capricor Therapeutics discussed clinical data presented at MDA’s 2025 meeting.
3. UniQure's Huntington Disease Gene Therapy AMT-130 Snags FDA Breakthrough Therapy Designation
Notably, AMT-130 has previously been granted regenerative medicine advanced therapy, orphan drug, and fast track designations by the agency.
4. Questions of Access: Gene Therapy’s Next Chapter in β-Thalassemia
Tami John, MD and Shalini Shenoy, MD, MBBS, offer insight into the clinical experience of patients treated with Casgevy and Zynteglo thus far.
5. BrainChild Bio's Pediatric Brain Tumor CAR-T BCB-276 Reels in Breakthrough Therapy Designation
The FDA’s decision was supported by data indicating overall survival benefit for patients with brain tumors from the phase 1 BrainChild-03 clinical trial.
OPMD and Advancements in Genetic Research
April 22nd 2025Matthew Wicklund, MD, a professor of neurology at the University of Texas Health Science Center San Antonio, discussed the genetic basis of oculopharyngeal muscular dystrophy, as well as the primary challenges in diagnosing and treating the condition.
