CGTLive’s Weekly Rewind – June 3, 2022

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Review top news and interview highlights from the week ending June 3, 2022.

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. ICER Finds Beti-Cel Superior to Standard-of-Care for β-Thalassemia

Beti-cel was rated a B+ for lingering questions about durability and unknown risks.

2. Val-Rox BLA for Hemophilia A Delayed Again

BioMarin now expects to file in September 2022 instead of June.

3. FDA Approves Tisa-Cel Expanded Indication in R/R Follicular Lymphoma

The approval was based on data from the phase 2 ELARA clinical trial, in which a complete response of more than 65% was observed.

4. Expediting the Development of Patient-Specific iSPC Cell Therapies

Stefan Braam, PhD, chief executive officer of Ncardia and Cellistic discussed the companies’ work in the cell therapy space.

5. Liso-Cel Yields High Response Rates and Positive PROs in LBCL

Treated patients had significant improvements in fatigue and lymphoma symptoms.

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
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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