CGTLive®’s Weekly Rewind – September 5, 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.

The rAAVrh8 gene therapy delivers HEXA and HEXB genes and has posted positive phase 1/2 results in infantile GM2 gangliosidosis, with enhancements in enzymatic activity and neurological outcomes.

The chief scientific officer and senior director of preclinical pharmacology Arbor Biotechnologies discussed ABO-101, the company’s gene therapy for primary hyperoxaluria type 1.

VG801 is intended to provide a functional copy of ABCA4 with the use of the company’s proprietary vgRNA REVeRT platform.

Annaiz Grimm, BS, a research scientist at Seattle Children's Research Institute, discussed the potential use of EngTregs to treat autoimmune disease.

Notably, the European Medicines Agency also granted MCO-010 orphan drug designation for 5 retinal dystrophy categories.