CGTLive®’s Weekly Rewind – November 1, 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. BridgeBio Pharma’s Gene Therapy BBP-812 Effects Progressive Improvement in Motor Function in Patients With Canavan Disease

The majority of patients treated with the gene therapy also showed improved myelination as measured by T-2-weighted MRI.

2. Overcoming AAV Barriers and Limitations to Treat Duchenne Muscular Dystrophy

Melissa Spencer, PhD, spoke about the emerging role of adeno-associated virus as a delivery system for gene therapies targeting muscular dystrophies, and the challenges associated with them.

3. Abeona Therapeutics Resubmits BLA to FDA for Epidermolysis Bullosa Gene Therapy Pz-Cel

The company made the resubmission after reaching an accord with the FDA in a Type A meeting with the agency that took place in August 2024.

4. R. Nolan Townsend, Sandi See Tai, MD, and Kim G. Johnson, MD, on Gene Therapy for APOE4 Homozygous Alzheimer Disease

The 3 experts discussed Lexeo Therapeutics’ LX1001 gene therapy trial that demonstrated promising safety and biomarker effects in patients with early-stage Alzheimer disease.

5. Intellia’s CRISPR Gene Editing Therapy NTLA-2002 Appears to Eliminate Hereditary Angioedema Attacks in Some Patients Following One-Time Treatment

Participants in the trial received either a single dose of 25 mg of NTLA-2002, a single dose of 50 mg of NTLA-2002, or a placebo.