CGTLive®’s Weekly Rewind – May 9, 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. Clinical Stage Biotech Bloomsbury Genetic Therapies Shuttering its Doors

The company’s CEO announced via LinkedIn that the company is shutting down its operations, citing a “difficult finding environment.”

2. James Beck, PhD, on Expanding Access to Whole Genome Sequencing in Parkinson Research

The chief scientific officer of the Parkinson’s Foundation discussed the foundation’s efforts to integrate whole genome sequencing into PD GENEration and expand access across diverse global populations.

3. J&J's XLRP Gene Therapy Bota-Vec Falls Short of Primary End Points

Data from the LUMEOS phase 3 clinical trial demonstrated a rare disease gene therapy referred to as botaretigene sparoparvovec (bota-vec) has failed to improve the vision-guided mobility of patients with X-linked retinitis pigmentosa (XLRP).

4. Diana Bharucha-Goebel, MD, and Bakri Elsheikh, MBBS, FRCP, on How New Therapies Are Changing SMA Care Guidelines

The clinical neurophysiologist at Children’s National and the professor of neurology at The Ohio State University Wexner Medical Center discussed the evolving SMA treatment landscape.

5. FDA States Advisory Committee Meeting Will be Held for Capricor’s BLA for DMD Cardiomyopathy Cell Therapy Deramiocel

In a mid-cycle review meeting, the agency also noted that the review committee found no major deficiencies with the BLA package.