CGTLive®’s Weekly Rewind – December 6, 2024

News
Article

Review top news and interview highlights from the week ending December 6, 2024.

CGTLive®’s Weekly Rewind

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.



Newsletter

Stay at the forefront of cutting-edge science with CGT—your direct line to expert insights, breakthrough data, and real-time coverage of the latest advancements in cell and gene therapy.

Recent Videos
Jeffrey Chamberlain, PhD
Tami John, MD
Tami John, MD
Tami John, MD
Matthew Ku, MBBS, FRACP, RACP, FRCPA/RCPA, PhD, an associate professor and the lymphoma stream lead at St Vincent’s Hospital
Saurabh Dahiya, MD, FACP, an associate professor of medicine at Stanford University School of Medicine; as well as clinical director of Cancer Cell Therapy in the Division of Blood and Marrow Transplantation and Cell Therapy at Stanford Medicine
Shahzad Raza, MD, a hematologist/oncologist at the Cleveland Clinic
Manali Kamdar, MD, the associate professor of medicine–hematology and clinical director of lymphoma services at the University of Colorado
Shahzad Raza, MD, a hematologist/oncologist at the Cleveland Clinic
Laura Aguilar MD, PhD, the chief medical officer of Diakonos Oncology
© 2025 MJH Life Sciences

All rights reserved.