CGTLive’s Weekly Rewind – January 12, 2024

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Review top news and interview highlights from the week ending January 12, 2024.

CGTLive’s Weekly Rewind – January 12, 2024

CGTLive’s Weekly Rewind – January 12, 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. FDA Activity Recap: December 2023 Features 2 Major Approvals, Meetings, and Clinical Holds

Catch up on any of the key FDA news stories you may have missed last month, with coverage highlights from the CGTLive™ team.

2. Mazyar Shadman, MD, on Investigating Treatment Choices With CAR-T and Auto-HSCT in LBCL

The associate professor at Fred Hutch Cancer Center discussed data from a retrospective analysis of patients treated with tisa-cel, axi-cel, or liso-cel.

3. Myasthenia Gravis mRNA CAR-T Demonstrates Clinically Meaningful Improvements in Phase 2a Trial

In terms of safety, Descartes-08 was characterized as well-tolerated during the study and long-term follow-up period.

4. Tami John, MD, on the FDA Approvals of Exa-cel and Lovo-cel

The clinical associate professor at Stanford Medicine also discussed ongoing trends in sickle cell disease research.

5. Vertex and CRISPR’s Ongoing Trial for Sickle Cell Disease Gene Therapy Seeks to Gather Patient Reported Outcomes

Exa-cel was recently approved for the treatment of SCD in the US, but data from the pivotal clinical trial continues to be collected.

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Ben Samelson-Jones, MD, PhD, assistant professor pediatric hematology, Perelman School of Medicine, University of Pennsylvania and Associate Director, Clinical In Vivo Gene Therapy, Children’s Hospital of Philadelphia
Manali Kamdar, MD, the associate professor of medicine–hematology and clinical director of lymphoma services at the University of Colorado
Steven W. Pipe, MD, a professor of pediatric hematology/oncology at CS Mott Children’s Hospital
Haydar Frangoul, MD, the medical director of pediatric hematology/oncology at Sarah Cannon Research Institute and Pediatric Transplant and Cellular Therapy Program at TriStar Centennial
Bhagirathbhai R. Dholaria, MD, an associate professor of medicine in malignant hematology & stem cell transplantation at Vanderbilt University Medical Center
Caroline Diorio, MD, FRCPC, FAAP, an attending physician at the Cancer Center at Children's Hospital of Philadelphia
R. Nolan Townsend; Sandi See Tai, MD; Kim G. Johnson, MD
Paul Melmeyer, MPP, the executive vice president of public policy & advocacy at MDA
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