CGTLive®’s Weekly Rewind – April 4, 2025

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.