CGTLive®’s Weekly Rewind – September 19, 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. Sanofi’s Neovascular Age-Related Macular Degeneration Gene Therapy SAR402663 Garners FDA Fast Track Designation

SAR402663 comprises a one-time intravitreal treatment and is intended to inhibit VEGF by providing a gene for soluble FLT01.

2. ImmunoLogic, Episode 6: "Tailor-Made Immunity: The Promise of Personalized Neoantigen Vaccines” With Stephen Schoenberger, PhD

In Episode 6 of ImmunoLogic, Stephen Schoenberger, PhD, discussed his research on customized cancer vaccines.

3. Ultragenyx's Glycogen Storage Disease Gene Therapy DTX401 Continues to Enable Decreases in Reductions in Daily Cornstarch Intake at 96 Weeks

With regard to safety, DTX401’s safety profile was characterized as “acceptable and expected.”

4. Daniel Galbraith, PhD, on Using Targeted Locus Amplification to Analyze Genetically Modified Cells

The chief scientific officer of Solvias discussed potential uses for the company’s platform.

5. Phase 1/2 Trial for Immix Biopharma’s AL Amyloidosis CAR-T NXC-201 Hits Over 50% Enrollment

The trial, which is taking place in the United States, is expected to enroll 40 patients in total.