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. MavriX Bio’s Angelman Syndrome Gene Therapy MVX-220 Garners FDA Fast Track Designation

MVX-220 is intended to provide a functional copy of UBE3A, the disease-targeted gene, to the neurons.

2. World Heart Day 2025: Looking Back at Recent Progress in Cardiology Cell and Gene Therapy

In observance of World Heart Day, held annually on September 29, we took a look back at news in cell/gene therapy for cardiovascular disease from the past few months.

3. FDA Publishes 3 New Draft Guidance for Industry Documents Aimed at Cell and Gene Therapy Development

The documents cover topics including efficient trial design, expedited review programs, and postapproval data collection.

4. CDKL5 and Gene Therapy

CGTLive® interviewed Jainu Jogani, the cofounder of Child’s Cure Genetic Research, whose daughter Reyna has CDKL5 deficiency disorder (CDKL5).

5. Umoja Biopharma’s In Situ CAR-T Therapy UB-VV111 Fast Tracked for Several R/R B-Cell Malignancy Indications

UB-VV111 is currently being evaluated in a phase 1 clinical trial for adult patients with r/r CD19+ B-cell malignancies.