CGTLive®’s Weekly Rewind – January 30, 2026

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. Ocugen’s Gene Therapy OCU410ST Continues to Show Efficacy in Phase 1 Stargardt Disease Trial

The therapy provided a +6-letter gain in BCVA-evaluable patients.

2. Integrating Gene Therapy into Clinical Care for Hemophilia B

Steven W. Pipe, MD, a professor of pediatric hematology/oncology at the University of Michigan Health, also discussed open questions that remain for the future of the hemophilia B gene therapy.

3. Pivotal Trial for Taysha's Rett Syndrome Gene Therapy TSHA-102 Doses First Patient

Taysha also disclosed that it has received written alignment from the FDA on elements of a potential BLA strategy for TSHA-102.

4. TIL expansion and more in vivo CAR data in 2026?

ImmunoLogic cohosts Janna Minehart, MD, and Joseph Fraietta, PhD, discussed their thoughts on the potential of 2026 to bring major new data on in vivo CARs and the expansion of tumor infiltrating lymphocyte (TIL) therapy outside melanoma.

5. FDA Lifts Clinical Hold on Intellia’s Trial for Gene Editing Therapy Nex-Z in ATTRv-PN, Places Holds on REGENXBIO MPS Trials

Intellia’s trial for nex-z in ATTR-CM remains under clinical hold.