CGTLive’s Weekly Rewind – April 28, 2023

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Review top news and interview highlights from the week ending April 28, 2023.

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. GenSight Biologics’ Lumevoq Demonstrates Clinically Meaningful Improvements in Early Access Programs

The new data builds upon positive long-term follow-up data from clinical trials.

2. Bart P. Leroy, MD, PhD, on Continuing to Verify Luxturna’s Efficacy

The head of the Department of Ophthalmology at the Center for Medical Genetics, Ghent University Hospital, discussed the results of a post-marketing study presented at ARVO’s 2023 conference.

3. bluebird bio Submits BLA for Sickle Cell Gene-Edited Cell Therapy, Lovo-cel

The BLA submission comes after a series of delays which have pushed lovo-cel behind Vertex’s exa-cel in the race to approval.

4. LCA1 Gene Therapy Improves Sensitivity, BCVA at Highest Dose

No serious TEAEs have been related to ATSN-101 with data up to 6 months after treatment.

5. Rachel Lynn, PhD, on Reprogramming Cell Therapies to Combat Exhaustion

The associate director of research at Lyell Immunopharma discussed the company’s programs and newest technologies aimed at solid tumors.

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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
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