CGTLive®’s Weekly Rewind – December 6, 2024
Review top news and interview highlights from the week ending December 6, 2024.
CGTLive®’s Weekly Rewind
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.
Transplant Eligibility Versus CAR-T Eligibility
January 16th 2025Manali Kamdar, MD, the associate professor of medicine–hematology and clinical director of lymphoma services at the University of Colorado, discussed the importance of referring patients with r/r LBCL who are transplant ineligible for CAR-T treatment.
Sequencing of Treatment in Third-Line R/R LBCL
January 15th 2025Manali Kamdar, MD, the associate professor of medicine–hematology and clinical director of lymphoma services at the University of Colorado, discussed the choice between treating patients with liso-cel or bispecific T-cell engagers.
