CGTLive®’s Weekly Rewind – January 17, 2025

CGTLive®’s Weekly Rewind

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 Issues CRL for Atara Biotherapeutics’ BLA for T-Cell Immunotherapy Tabelecleucel for EBV+ PTLD

The CRL was related inspection of a third-party manufacturer rather than any efficacy or safety concerns regarding the product itself.

2. Georg Schett, MD, on Future Goals for CAR-T in Autoimmune Disease

The vice president research and chair of internal medicine at the University of Erlangen – Nuremberg discussed potential upcoming innovations in the field.

3. ViGeneron Snags FDA Rare Pediatric Disease Designation for CNGA1-Associated Retinitis Pigmentosa Gene Therapy VG901

In addition to the new designation, ViGeneron also announced that the data safety monitoring board for the trial has given the green light for dose escalation.

4. Transplant Eligibility Versus CAR-T Eligibility

Manali Kamdar, MD, the associate professor of medicine–hematology and clinical director of lymphoma services at the University of Colorado, discussed the importance of referring patients with r/r LBCL who are transplant ineligible for CAR-T treatment.

5. Ocugen’s Retinitis Pigmentosa Gene Therapy OCU400 Sustains or Improves Visual Function in 2-Year Follow-Up Data

Among 9 patients treated in the study who were evaluable at 2 years posttreatment, all 9 showed improved or preserved visual function in comparison to their untreated fellow eyes.