CGTLive®’s Weekly Rewind – September 26, 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. Phase 1/2 Trial for uniQure's Huntington Disease Gene Therapy AMT-130 Hits Primary End Point

The study also met a key secondary end point.

2. Jainu Jogani on the Potential to Address CDKL5 With Gene Therapy

The cofounder of Child’s Cure Genetic Research discussed his daughter’s rare genetic disease and the need for new treatment options.

3. Atsena Therapeutics’ Trial for X-Linked Retinoschisis Gene Therapy ATSN-201 Finishes Dosing Adult Patients in Part B

Part B of LIGHTHOUSE enrolled 9 patients and is expected to enroll 3 pediatric patients.

4. The Importance of Addressing Gene Therapy's Financial Viability in Ultra Rare Diseases

Roger Hajjar, MD, the director of the Mass General Brigham Gene and Cell Therapy Institute, discussed a trend of doctors and scientists working on models to make gene therapy products commercially viable.

5. Capricor Therapeutics Testing DMD Cell Therapy Deramiocel’s Mettle in Phase 3 HOPE-3 Clinical Trial

In honor of Duchenne Action Month and World Duchenne Day, held every year in September, CGTLive® is taking a closer look at this ongoing study.