CGTLive®’s Weekly Rewind – October 25, 2024

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. MeiraGTx’s Parkinson Disease Gene Therapy AAV-GAD Improves Motor Scores and Patient-Reported Quality of Life in Sham-Controlled Study

For the patients treated at the high dose, an average improvement of 18 points from baseline was observed in UPDRS Part 3 “off” medication score at 26 weeks posttreatment.

2. Sharon Hesterlee, PhD, on MDA 2025 as a Mixer for Strategies and Collaboration

The chief research officer of the Muscular Dystrophy Association (MDA) discussed the upcoming MDA Clinical and Scientific Conference, which will be held March 16-19, in Dallas, Texas.

3. REGENXBIO’s ABBV-RGX-314 Reduces Need for AntiVEGF Injections After Application in Fellow Eyes of Patients With Wet AMD

The data comes from a phase 2 substudy (NCT03999801) that treated the fellow eyes of patients who had previously been treated with ABBV-RGX-314.

4. Payer Challenges for Patient Access to Autologous Cell and Gene Therapies

Autologous cell and gene therapies show promise but face barriers such as high costs, limited access, and payer concerns over long-term effectiveness.

5. Ocugen Gets Green Light to Move Onto Phase 2 Portion of Trial Evaluating Stargardt Gene Therapy OCU410ST

The DSMB’s decision was made with reference to safety data from the GARDian trial’s phase 1 portion.