CGTLive’s Weekly Rewind – October 20, 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. Intellia’s CRISPR Therapy for ATTR-CM First to Enter Late-Stage Trials

Intellia Therapeutics plans to initiate the phase 3 trial of NTLA-2001 by the end of 2023.

2. Milind Desai, MD, MBA, on Addressing Unmet Needs in MYBPC3-associated Hypertrophic Cardiomyopathy

The director of the Center for Hypertrophic Cardiomyopathy at the Cleveland Clinic discussed TN-201, an investigational gene therapy for MYBPC3-associated HCM.

3. Canavan Gene Therapy Improves Developmental Measures Compared to Natural History

MSEL 5-mean domain, receptive language, and expressive language scores were significantly improved from baseline in treated participants.

4. David Rawlings, MD, on Research That Supports the Development of Engineered B-cell Therapies

The director of the Center for Immunity and Immunotherapies at Seattle Children's Research Institute discussed findings from several preclinical studies that could help bring engineered B-cell therapies to clinical trials.

5. bluebird's HGB-210 Trial Hopes to Support First SCD Gene Therapy Approval

Lovo-cel is currently being evaluated in the phase 3 HGB-210 study and has a PDUFA date of December 20, 2023.