CGTLive’s Weekly Rewind – July 21, 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. ICER Rates Exa-Cel and Lovo-Cel Cost-Effective up to $2 Million

The updated report maintains exa-cel's C++ grade and lovo-cel's B+ grade.

2. Richard Fessler, MD, on Working Toward Cell Therapy for Spinal Cord Injury

The professor of neurosurgery at Rush University Medical School discussed challenges with trials for SCI and the 1st Annual SCIIS.

3. Dose-Limiting Toxicity Responsible for Cure Rare Disease’s DMD Trial Death

Unforeseen complications may arise when treating older patients with gene therapy.

4. Paul Harmatz, MD, on Assessing D2S6 in Trials for MPS Type 2

The pediatric gastroenterologist and professor in residence at University of California at San Francisco discussed biomarker findings from the phase 1/2 CAMPSIITE trial.

5. Refractory Angina Gene Therapy XC001 Sustains Effect at 12 Months

XyloCor Therapeutics reported that at the 12-month time point, sustained and continued increases in total exercise duration over baseline measurements were observed.