CGTLive®’s Weekly Rewind – February 7, 2025

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."