CGTLive®’s Weekly Rewind – October 18, 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. Benitec Biopharma’s Oculopharyngeal Muscular Dystrophy Gene Therapy Produces Clinical Improvements in Swallowing in Phase 1b/2a Trial

Benitec reported that the first patient showed a 35% reduction, constituting clinically meaningful improvement, in total score on the Sydney Swallow Questionnaire.

2. The Role of Established Pharmaceutical Companies in Bringing Cell Therapy to Autoimmune Disease

Daniela van Eickels, MD, PhD, MPH, the vice president and head of medical affairs for Bristol Myers Squibb’s Cell Therapy Organization, also discussed the company’s planned presentation at ACR Convergence 2024.

3. Beacon Therapeutics’ XLRP Gene Therapy AGTC-501 Continues to Show Promise in Long-Term Data From SKYLINE Trial

Among 7 patients’ eyes that were treated with the study’s high dose, the response rate was 57% (4/7 eyes).

4. Wave Life Sciences Achieves RNA Editing in Trial for WVE-006 in Alpha-1 Antitrypsin Deficiency

According to the company, this is the first time therapeutic RNA editing has been clinically demonstrated in humans.

5. Adicet's T-Cell Therapy ADI-100 Snags Clearance for Evaluation in Idiopathic Inflammatory Myopathy and Stiff Person Syndrome

Originally, the phase 1 clinical trial was only cleared for the recruitment of patients with lupus nephritis, but IND amendments have added more eligible indications.