GeneTherapyLive’s Weekly Rewind – October 8, 2021

Welcome to GeneTherapyLive’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. Cellular, Tissue, and Gene Therapies AdComm Meeting Takeaways: Barry Byrne, MD, PhD

The director of the Powell Gene Therapy Center at the University of Florida discussed the recent meeting of the FDA Cellular, Tissue, and Gene Therapies Advisory Committee. He touched on adverse events typically observed with gene therapies and takeaways from the meeting.

2. Angelman Syndrome Gene Therapy Trial Resumes Following Clinical Hold

The FDA has lifted their clinical hold on Ultragenyx and GeneTx’s phase 1/2 study (NCT04259281) of GTX-102 after trial protocols were amended to lower dose levels after lower extremity weakness was observed in patients treated with high-doses.

3. Regulating Cell and Gene Therapy Activity

Paul Wotton, PhD, chief executive officer, Obsidian Therapeutics, discussed the company’s pipeline and technologies. He also touched on partnerships the company has formed to collaborate on their cell and gene therapies in development.

4. Gavo-Cel Efficacious in Refractory Mesothelin-Expressing Solid Tumors

TCR2 Therapeutics has announced positive interim data from the phase 1/2 study (NCT03907852) of gavocabtagene autoleucel (gavo-cel; TC-210) for the potential treatment of refractory mesothelin-expressing solid tumors.

5. Exposing the Advantages of iPSC-Natural Killer Cell Therapies

Allen Feng, PhD, chief scientific officer, HebeCell, discussed advantages of induced pluripotent over hematopoietic stem cell therapies, as well as recent funding the company received from Jacobio Pharmaceuticals, intended to support the development of next-generation iPSC-CAR-NK therapies for oncologic, viral, and autoimmune indications.