CGTLive®’s Weekly Rewind – April 18, 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. Fate Therapeutics’ iPSC-derived CAR-T FT819 Nabs FDA RMAT Designation for Lupus

FT819 is currently being evaluated in a multicenter phase 1 clinical trial.

2. Matthew Wicklund, MD, on OPMD and Advancements in Genetic Research

The professor of neurology at the University of Texas Health Science Center San Antonio discussed the genetic basis of oculopharyngeal muscular dystrophy, as well as the primary challenges in diagnosing and treating the condition.

3. Verve Therapeutics’ Base Editing Therapy VERVE-102 Reduces LDL-C in Patients With HeFH and CAD

Verve noted that a patient treated at the 0.6 mg/kg dose showed the maximum decrease in LDL-C levels: 69%.

4. Exploring the ARCUS Approach to Next-Gen Gene Editing for Muscular Dystrophy

Cassandra Gorsuch, PhD, chief scientific officer at Precision Biosciences, discussed the company’s ARCUS gene editing platform, which is being assessed for Duchenne muscular dystrophy.

5. Using Armored CAR T-Cells to Tackle Solid Tumors

Renier Brentjens, MD, PhD, the chair of the department of medicine at Roswell Park Comprehensive Cancer Center, discussed the innovations necessary to make CAR-T therapy effective in solid tumor indications.