Gene and Cell Therapy Data Roundup: Updates in DLBCL, SMA, XLRP, and More
• REGENXBIO's RGX-121 shows sustained efficacy in MPSII, with significant reduction in heparan sulfate levels and reduced need for enzyme replacement therapy.
• Biogen's nusinersen demonstrates superior efficacy in SMA at a higher dose compared to the FDA-approved dose, showing statistically significant improvement in CHOP-INTEND scores.
• Allogeneic CAR-T Azer-Cel achieves complete responses in some DLBCL patients, with ongoing responses observed in cohort B of a phase 1/1b trial.
• Beacon Therapeutics' AGTC-501 for XLRP maintains a favorable safety profile at 36 months, with most adverse events resolving within the first three months post-treatment.
Orgenesis' ORG-101 CAR-T Therapy Shows Promise in Real-World ALL Study in China
• Orgenesis' ORG-101, a CD19-directed CAR-T therapy, demonstrated an 82% complete response rate in adults and a 93% complete response rate in children with B-cell acute lymphoblastic leukemia (ALL).
• The real-world data, gathered from 233 patients in China, indicated a low incidence of severe cytokine release syndrome (CRS), with 2% in adults and 6% in children.
• ORG-101 utilizes a decentralized manufacturing approach, potentially improving access and reducing costs, and Orgenesis plans to initiate a multicenter phase 1/2 clinical trial.
• Recent approvals of CAR-T therapies in China, including cilta-cel for multiple myeloma and relma-cel for mantle cell lymphoma, highlight the growing CAR-T landscape.
© Copyright 2025. All Rights Reserved by MedPath