CGTLive®’s Weekly Rewind – May 10, 2024

Welcome to CGTLive®’s Weekly Rewind! We’ve compiled 5 highlights from this week’s coverage of advances in gene and cell therapies, with a focus on this week's onsite coverage of the 2024 American Society of Gene & Cell Therapy Meeting.

1. Elevidys Shows Slight Statistical Improvements on Time to Rise, 10-Meter Walk/Run Despite NSAA Fail

The gene therapy is up for priority review with a PDUFA date of June 21, 2024.

2. Lawrence R. Lustig, MD, on Evaluating Gene Therapy for the Treatment of Otoferlin-Related Hearing Loss

The chair of the Department of Otolaryngology—Head and Neck Surgery at Columbia University College of Physicians discussed the phase 1/2 CHORD clinical trial evaluating DB-OTO.

3. TDT/SCD Lentiviral Therapy Boasts Quick, Well-Tolerated Engraftment in Patients

KL003 favorably compares to platelet and neutrophil recovery rates of approved gene therapies.

4. Travis Drow, BS, on Evaluating Engineered Tregs for Potential in Treating Multiple Sclerosis

The research scientist at Seattle Children's Research Institute discussed mouse model research he presented at ASGCT’s 2024 Meeting.

5. Patient With DMD Dies in Pfizer’s Phase 2 Gene Therapy Trial

In response to the death, Pfizer is pausing dosing associated with the crossover portion of the phase 3 CIFFREO trial.