CGTLive®’s Weekly Rewind – July 26, 2024

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. FDA Accepts Mesoblast’s BLA for Remestemcel-L for Steroid-Refractory GvHD

After 3 tries, Mesoblast finally has a PDUFA date for its MSC therapy.

2. Stephanie Tagliatela on Researching AAV for Lennox-Gastaut, Alzheimer Disease, SCN9a Pain

The cofounder and chief scientific officer at Encoded Therapeutics shared preclinical research with the company’s AAV and miRNA platforms.

3. Stanford Study Finds Low Risk of Secondary Blood Cancers After CAR-T

Of 724 patients treated at the center, the cumulative incidence of secondary hematologic malignancy at 3 years posttreatment was 6.5%.

4. Lucas Harrington, PhD, on Using an Ultracompact CRISPR System to Target Cells Outside the Liver

The cofounder and chief scientific officer of Mammoth Biosciences discussed the company’s mouse model research on treating hypertriglyceridemia.

5. BlueRock Therapeutics Assessing the Potential of Cell Therapy for Parkinson Disease With Phase 1 Trial for Bemdaneprocel

In honor of World Brain Day, observed annually on July 22 by patient and clinician communities, CGTLive is taking a closer look at this ongoing study.