CGTLive®’s Weekly Rewind – July 18, 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. Ultragenyx’s MPSIIIA Gene Therapy UX111 Met With CRL

According to Ultragenyx, the CRL relates to a need for additional chemistry, manufacturing, and controls (CMC) information and improvements and observations from inspections of manufacturing facilities.

2. Nathan Yozwiak, PhD, on Fostering Collaboration in Cell and Gene Therapy Research

The head of research at Mass General Brigham’s Gene and Cell Therapy Institute discussed the Institute’s efforts to bring about clinical translation of preclinical work.

3. Nanoscope Therapeutics Puts Rolling BLA for Retinitis Pigmentosa Gene Therapy MCO-010 in Front of FDA

According to the company, the BLA is the first for a gene-agnostic gene therapy for retinal disease to have been submitted to the FDA.

4. FDA Supports Pivotal Trial Expansion of ATSN-201 Gene Therapy for XLRS

Atsena Therapeutics will advance ATSN-201 into a pivotal phase 1/2/3 trial following FDA agreement on study design and end points.

5. High-Capacity Adenovirus Vectors for Gene Therapy

Derek Jackson, BS, MA, and Kilian Guse, PhD, of Pacira Biosciences, discussed the company’s symposium at ASGCT 2025.