CGTLive®’s Weekly Rewind – September 27, 2024

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. Canada Approves Vertex and CRISPR Therapeutics’ Gene Therapy Casgevy for Sickle Cell Disease and TDT

The approvals are indicated for patients 12 years and older.

2. Gene and Cell Therapy Approaches Hold Promise for Usher Syndrome

In honor of Usher Syndrome Awareness Day, CGTLive® interviewed Zheng-Yi Chen, DPhil, associate professor, Otolaryngology–Head and Neck Surgery, Harvard Medical School, about the current state of research in this rare disease.

3. Beacon Therapeutics’ XLRP Gene Therapy AGTC-501 Continues to Show Favorable Benefit-Risk Profile at 36 Months

here were no SUSARS and no endophthalmitis observed among 29 patients treated in the study.

4. Arun Upadhyay, PhD, on the Potential of Gene Therapy in Inherited Retinal Disease

The chief scientific officer and head of research, development, and Medical at Ocugen, discussed OCU400, the company’s gene-agnostic gene therapy for IRDs.

5. AAVantgarde Bio Doses First Patient in Trial for Usher Syndrome Gene Therapy AAVB-081

AAVB-081 is intended to address retinitis pigmentosa that results from Usher syndrome type 1B.