CGTLive®’s Weekly Rewind – August 16, 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. Uptake of Nononcology Gene Therapy Remains Slow in Hematology, With bluebird’s Lovo-Cel Particularly Behind Vetex's Exa-Cel in SCD

Despite approval by the FDA on December 8, 2023, only 4 patients have begun treatment with lovo-cel in the commercial setting.

2. William Chou, MD, on Targeting Progranulin With Gene Therapy for Frontotemporal Dementia

The president and chief executive officer of Passage Bio discussed feedback from a recent Type C meeting with the FDA.

3. FDA Lifts Clinical Hold on Trial for 4D Molecular Therapeutics’ Fabry Disease Gene Therapy 4D-310

In light of the FDA’s decision, 4DMT plans to continue enrolling patients in INGLAXA before the end of 2024.

4. Alexandra Collin de l’Hortet, PhD, on Evaluating Epigenetic Gene Therapy for the Treatment of FSHD

The head of therapeutics at Epic Bio discussed EPI-321, an investigational treatment for facioscapulohumeral muscular dystrophy.

5. Genprex Makes Progress in 2 of 3 Lung Cancer Trials for Gene Therapy Reqorsa, but Drops the Other

The company reported promising early efficacy data from the Acclaim-1 and Acclaim-3 trials, but has discontinued Acclaim-2, citing difficulty enrolling patients.