GeneTherapyLive’s Weekly Rewind – January 7, 2022

Welcome to GeneTherapyLive’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. Gene Therapy Yields Complete Wound Healing in Dystrophic Epidermolysis Bullosa

Krystal Biotech’s investigational gene therapy beremagenegeperpavec (B-VEC, Vyjuvek) improved wound healing in patients with dystrophic epidermolysis bullosa, according to topline results from the placebo-controlled phase 3 GEM-3 trial (NCT04491604).

2. Gene Therapy for GM1 Gangliosidosis: Latest Updates

Ralph Laufer, PhD, chief scientific officer, Lysogene, discussed the gene therapy LYS-GM101 for GM1 gangliosidosis and a new program in Fragile X syndrome in which the company is partnering with SATT Conectus and the Institute of Genetics and Molecular and Cellular Biology.

3. Angelman Syndrome Gene Therapy Well Tolerated

The gene therapy GTX-102 has been well-tolerated in the first 4 patients with Angelman syndrome dosed in the phase 1/2 open-label, multiple-dose, dose-escalating KIK-AS study (NCT04259281) in Canada and the UK. Patients in the US are receiving lower doses after a recent clinical hold.

4. Using Cell Therapy to Reduce Immunosuppression in Living Kidney Transplant Participants

Scott Requadt, chief executive officer, Talaris Therapeutics, discussed the long-term follow-up and FREEDOM-1 trials of FCR-001 as well as the use of chimerism within study participants as a biomarker of efficacy.

5. Fabry Gene Therapy Program Discontinued Following Transient Engraftment Issues

AVROBIO is deprioritizing their Fabry disease gene therapy program and stopping recruitment in the phase 1/2 FAB-GT trial (NCT03454893) of AVR-RD-01 following disappointing engraftment data in the 5 most recently treated participants.