Review top news and interview highlights from the week ending May 9, 2025.
CGTLive®’s Weekly Rewind
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 company’s CEO announced via LinkedIn that the company is shutting down its operations, citing a “difficult finding environment.”
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.
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).
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.
In a mid-cycle review meeting, the agency also noted that the review committee found no major deficiencies with the BLA package.
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Jeffrey Chamberlain, PhD, on the Import of Early Intervention in DMD
July 10th 2025The McCaw Endowed Chair of Muscular Dystrophy at University of Washington, discussed how comprehensive care for DMD patients involves early diagnosis, steroid treatment, consideration of mutation-specific therapies, and more.