CGTLive’s Weekly Rewind – March 31, 2023
Review top news and interview highlights from the week ending March 31, 2023.
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. Lovo-cel BLA Submission Likely Delayed for Sickle Cell
The gene-edited cell therapy had previously been under a partial clinical hold restricting pediatric enrollment to the clinical trial.
2. Matthew B. Harms, MD, on the Necessity of Tracking Gene Mutations in ALS
The associate professor of neurology at Columbia University spoke about the importance of identifying patients with ALS-associated gene mutations as early as possible.
3. Multi-Characteristic Opsin Optogenetic Therapy Meets Phase 2 Endpoint in Retinitis Pigmentosa
Most patients with RP treated with MCO-010 had clinically meaningful improvements in vision.
4. Rita Perlingeiro, PhD, on the Potential of iPS Cell-based Regenerative Medicine in Muscular Dystrophy
The Lillehei professor in stem cell and regenerative cardiovascular medicine at the University of Minnesota spoke about preclinical research with an iPS cell-derived product.
5. First Patient With HPV16+ Solid Tumors in SQZ-AAC-HPV Trial Achieves CR
The Study Safety Committee has recommended that the trial proceed to enroll patients in the highest-dose cohort.
Conservative Management Following Treatment With DMD Gene Therapy Elevidys
May 19th 2025Ben Samelson-Jones, MD, PhD, the associate director of clinical in vivo gene therapy at Children’s Hospital of Philadelphia discussed a case he presented at the American Society of Gene and Cell Therapy’s 2025 meeting.
Expanding Access to Whole Genome Sequencing in Parkinson Research
May 12th 2025James Beck, PhD, 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.
