CGTLive®’s Weekly Rewind – December 6, 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. Gene Therapy RP-A501 Shows Promising Phase 1 Results in Danon Disease

The Rocket Pharmaceuticals AAV9 treatment was assessed in a 3-cohort trial of 7 patients, showing good safety and encouraging efficacy signals.

2. REGENXBIO Doses First Patient in Phase 3 Trial for DMD Gene Therapy RGX-202

The company also announced updated efficacy data from the trial’s phase 1/2 portion.

3. Genethon and Hansa Biopharma Launch Trial for Gene Therapy and Antibody Cleaving Enzyme Therapy Combination Treatment in Crigler-Najjar Syndrome

The phase 2 trial will assess Hansa’s imlifidase as a pretreatment for Genethon’s GNT-0003.

4. SC291 CAR T-Cell Therapy Fast Tracked for Relapsed/Refractory SLE

Sana Biotechnology’s SC291 chimeric antigen receptor T-cell therapy is being developed to treat relapsed/refractory systemic lupus erythematosus, including extrarenal lupus and lupus nephritis.

5. Cartesian Therapeutics’ mRNA CAR-T Descartes-08 Produces Durable Responses in Myasthenia Gravis

Alongside the updated data, Cartesian announced that it plans to launch a phase 3 clinical trial, dubbed AURORA, in the first half of next year.