CGTLive’s Weekly Rewind – June 30, 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. FDA Approves BioMarin’s Hemophilia A Gene Therapy Roctavian

The FDA’s decision to approve this first gene therapy in hemophilia A was based on data from the phase 3 GENEr8-1 clinical trial.

2. Frank Borriello, MD, PhD, on Next Steps for SUPLEXA-101 for Solid Tumors

The scientific cofounder and chief executive officer of Alloplex Biotherapeutics discussed progress of the phase 1 trial and possible future evaluations of the therapy.

3. FDA Approves Lantidra Cell Therapy for Type 1 Diabetes

With an original PDUFA date in August 2021, the decision is a long time coming.

4. James Gulley, MD, PhD, FACP, on Assessing a Novel TCR Bifunctional Antibody in CPI-Resistant Solid Tumors

The director of the Medical Oncology Service at the National Cancer Institute discussed the START-001 trial.

5. Gene Therapy Modifies Disease Progression in Patients With Giant Axonal Neuropathy

Taysha also provided updates on its gene therapy program for Rett syndrome.