CGTLive®’s Weekly Rewind – April 4, 2025

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Review top news and interview highlights from the week ending April 4, 2025.

CGTLive®’s Weekly Rewind

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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Robert Califf, MD, MACC, a cardiologist and former FDA commissioner
Natalie Goedeker, CPNP, on Handling Neuromuscular Gene Therapy at Real-World Sites
Alfred L. Garfall, MD, MS, associate professor of medicine (hematology-oncology) and director, Autologous Hematopoietic Cell Transplantation, Cell Therapy and Transplant Program, Hospital of the University of Pennsylvania; and section chief, Multiple Myeloma, Division of Hematology/Oncology, Department of Medicine, Perelman School of Medicine, University of Pennsylvania,
Reena Sharma, MD, an adult metabolic consultant at Salford Royal Hospital
Nirav Shah, MD, MSHP, associate professor of medicine, at the Medical College of Wisconsin
Bhagirathbhai R. Dholaria, MD, an associate professor of medicine in malignant hematology & stem cell transplantation at Vanderbilt University Medical Center
Reena Sharma, MD, an adult metabolic consultant at Salford Royal Hospital
Mark Hamilton, MD, PhD, a hematology-oncology and bone marrow transplant (BMT) cell therapy fellow at Stanford University
Barry J Byrne, MD, PhD, the chief medical advisor of MDA and a physician-scientist at the University of Florida
Barry J Byrne, MD, PhD, the chief medical advisor of MDA and a physician-scientist at the University of Florida
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