CGTLive’s Weekly Rewind – January 5, 2024

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Review top news and interview highlights from the week ending January 5, 2024.

CGTLive’s Weekly Rewind – January 5, 2024

CGTLive’s Weekly Rewind – January 5, 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. After Exa-Cel: Exploring the Next Wave of CRISPR Gene Editing Strategies

The approval of exa-cel, the first gene editing therapy, may herald a new era in developing gene editing strategies.

2. Alexis Thompson, MD, MPH, on the Simultaneous Approvals of Exa-cel and Lovo-cel For SCD

The chief of hematology at Children’s Hospital of Philadelphia discussed dual approval of the 2 gene therapies that coincided with ASH’s 2023 conference.

3. Pfizer’s Hemophilia B Gene Therapy Approved as Beqvez in Canada

The FDA and EMA have accepted marketing authorization applications and decisions are pending.

4. Piers Blombery, MBBS, PhD, on Looking Deeper Into Effects of CAR T After CAR+ Lymphoma

The associate professor at Peter MacCallum Cancer Centre discussed further research and questions that need to be investigated with the cell therapy.

5. Oncternal Therapeutics Proposes Protocol Changes Following Death of Patient Treated With CAR-T Therapy ONCT-808 in Phase 1/2 Trial

ONCT-808 targets ROR1 and is currently being evaluated for the treatment of r/r aggressive B-cell lymphoma.

Recent Videos
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
Chun-Yu Chen, PhD, a research scientist at Seattle Children’s Research Institute
Michael Severino on In Vivo Gene Editing With RNA Gene Writers
Chris Wright, MD, PhD, on Annelloviruses, a Potential Alternative to AAV for Gene Therapy
Carol Miao, PhD, a principal investigator at Seattle Children’s Research Institute
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