CGTLive®’s Weekly Rewind – December 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. End-of-Study Results From Hemgenix’s HOPE-B Trial

Steven W. Pipe, MD, a professor of pediatric hematology/oncology at the University of Michigan Health, discussed the final results of the phase 3 study that evaluated the hemophilia B gene therapy.

2. What the Field of Cell and Gene Therapy Needs Most in the Current Moment

Deborah Phippard, PhD, shared her thoughts with Renier Brentjens, MD, PhD, on the importance of big wins for those in research.

3. Rabi Hanna, MD, on Practical Considerations for Integrating Gene Therapy

The chairman of the Division of Pediatric Hematology & Oncology and BMT at Cleveland Clinic Children's discussed learnings from the institutions integration of gene therapy for sickle cell disease.

4. Emily Brown, MGC, CGC, on Models for Expanding Access to Cardiac Genetic Counseling

The genetic counselor at Johns Hopkins Hospital spoke on how cardiac genetics clinics are adopting alternative care models to broaden access to genetic testing and counseling.

5. Sangamo Starts Putting BLA for Fabry Disease Gene Therapy Isa-Vec in Front of FDA

The rolling BLA is supported by data from the phase 1/2 STAAR clinical trial (NCT04046224).