CGTLive®’s Weekly Rewind – April 19, 2024

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Review top news and interview highlights from the week ending April 19, 2024.

CGTLive®’s Weekly Rewind – April 19, 2024

CGTLive®’s Weekly Rewind – April 19, 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. A Slow Embrace: Hemophilia's Gradual Adoption of Gene Therapy

For World Hemophilia Day, CGTLive took a look at how the hemophilia field has adapted to the introduction of gene therapy to the treatment landscape.

2. John DiPersio, MD, PhD, on Ongoing Advancements in Stem Cell Transplantation and Cell Therapy

The director of the Center for Gene and Cellular Immunotherapy at Washington University School of Medicine discussed novel innovations that lie on the horizon.

3. 4DMT Aligns on Path Forward for Cystic Fibrosis Gene Therapy

The company plans to initiate a pivotal trial of 4D-710 in the second half of 2025.

4. PJ Brooks, PhD, on Developing New Strategies for Novel Clinical Trials

The deputy director, Division of Rare Diseases Research Innovation, NCATS, NIH, discussed the NIH’s genome editing program and more.

5. MCO-010 Phase 2b Data Supports BLA Submission for Retinitis Pigmentosa

Nanoscope Therapeutics received feedback from the FDA endorsing significant change in BCVA as a primary endpoint supporting approval.

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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
Sarah Larson, MD, the medical director of the Immune Effector Cell Therapy Program in the Division of Hematology/Oncology at David Geffen School of Medicine at University of California, Los Angeles (UCLA)
Manali Kamdar, MD, the associate professor of medicine–hematology and clinical director of lymphoma services at the University of Colorado
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