CGTLive’s Weekly Rewind – May 12, 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. Dopamine Transporter Deficiency Syndrome Gene Therapy BGT-DTDS Gets FDA Rare Pediatric Disease Designation

Bloomsbury Genetic Therapies’ BGT-DTDS, a neuron-targeted and AAV2-mediated therapy, previously received orphan drug designation from the FDA and EC.

2. Jonathan Yen, PhD, on Showing Proof-of-Concept With Prime Editing in Sickle Cell Disease

The director of therapeutic genome engineering, St. Jude Children’s Research Hospital discussed recent research published in Nature Biomedical Engineering.

3. NRTX-1001 Cell Therapy Shows Promise for Drug-Resistant Mesial Temporal Lobe Epilepsy

The single-administration allogeneic cell therapy has shown positive safety—and signs of efficacy—in preliminary data from the first 2 patients dosed with the Neurona Therapeutics’ product.

4. Arshad Khanani, MD, on Future Plans for anti-VEGF Gene Therapy 4D-150

The director of clinical research at Sierra Eye Associates discussed 4D Molecular Therapeutics’ plans for 4D-150 in both wet AMD and diabetic macular edema.

5. MSLN-Targeted KIR-CAR Therapy Trial Initiates at UPenn

SynKIR-110 was also granted fast track designation by the FDA in April 2023.