CGTLive®’s Weekly Rewind – April 4, 2025
Review top news and interview highlights from the week ending April 4, 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. FDA Approves Sanofi’s siRNA Therapy Fitusiran for the Treatment of Hemophilia A and B
The product is marketed under the name Qfitlia.
2. David-Alexandre Gros, MD, on the Work to Bring Porcine Kidney Transplant to the Clinic
The chief executive officer of Eledon Pharmaceuticals also discussed other future plans for the company.
3. European Medicines Agency Requests Pause on Trials for Sarepta’s DMD Gene Therapy Elevidys
The move was made in relation to the previously-announced death of a US teenager who received the therapy.
4. Urgency of Funding and Equity in Neuromuscular Research and Care
Robert Califf, MD, MACC, a cardiologist and former FDA commissioner, discussed the necessity of policy and funding efforts for neuromuscular disorders, the stress on the clinical care system, and the need to acknowledge healthcare inequities.
5. Nanoscope Therapeutics’ Gene Therapy MCO-010 Restores Vision in Patients With Retinitis Pigmentosa
At 12 weeks posttreatment, all 4 of the patients showed improvement in the form of a lower logMAR.
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Jeffrey Chamberlain, PhD, on the Import of Early Intervention in DMD
July 10th 2025The McCaw Endowed Chair of Muscular Dystrophy at University of Washington, discussed how comprehensive care for DMD patients involves early diagnosis, steroid treatment, consideration of mutation-specific therapies, and more.
