CGTLive®’s Weekly Rewind – February 7, 2025
Review top news and interview highlights from the week ending February 7, 2025.
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. United Therapeutics’ Gene-Edited Kidney Xenotranspant UKidney Cleared for US Trial
The IND clearance constitutes the first for a xenokidney product.
2. Mark Hamilton, MD, PhD, on Analyzing PostCAR Myeloid Neoplasms
The hematology-oncology and BMT cell therapy fellow at Stanford University discussed implications of his institution’s findings on treatment-related secondary malignancies.
3. Gradalis' Personalized Immunotherapy Gemogenovatucel-T Receives FDA RMAT Designation
The product, also known as Vigil, is being evaluated in the ongoing randomized, placebo-controlled phase 2b VITAL clinical trial.
4. MDA Care Centers and the Future of Gene Therapy in Neuromuscular Disease
Barry J Byrne, MD, PhD, the chief medical advisor of MDA, also shared his thoughts on the 75th anniversary of the organization.
5. Ultragenyx’s Gene Therapy UX111 Improves Clinical Function in MPSIII
With regard to safety, Ultragenyx characterized the gene therapy as “generally well-tolerated."
News and Expert Insights in Hemophilia Gene Therapy for Bleeding Disorders Awareness Month 2025
March 9th 2025In observance of Bleeding Disorders Awareness Month, held annually in March, catch up on some of the latest news and expert insights in gene therapy for hemophilia, which is among the most common of these disorders.
