CGTLive®’s Weekly Rewind – November 8, 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. RiboX's Circular RNA Therapy RXRG001 Cleared for Phase 1/2a Trial in Radiation-Induced Xerostomia and Hyposalivation

RiboX stated that this is the first IND clearance the FDA has granted for any circular RNA therapy.

2. Caroline Diorio, MD, FRCPC, FAAP, on Noninflammatory Neurotoxicity Events in Patients Receiving CAR-T

The attending physician at the Cancer Center at Children's Hospital of Philadelphia discussed a study she coauthored that was recently published in Blood.

3. Lexeo Therapeutics' Gene Therapy LX1001 Increases APOE2 Expression in Patients With Alzheimer Disease

LX1001's safety profile was characterized as well-tolerated with no amyloid-related imaging abnormalities reported.

4. Nanoscope Therapeutics’ Retinitis Pigmentosa Gene Therapy MCO-010 Improves Visual Acuity in Randomized Controlled Clinical Trial

The randomized, controlled clinical trial treated patients with either a high dose of MCO-010, a low dose of MCO-010, or a sham–control.

5. FDA Removes Clinical Holds Previously Placed on 3 of CARsgen's Oncology Cell Therapies

The clinical holds on the 3 products were originally placed by the FDA in December 2023 based on findings from an inspection of CARsgen’s manufacturing facility.