GeneTherapyLive’s Weekly Rewind – October 29, 2021

Welcome to GeneTherapyLive’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. Gene Therapy Yields Improvements in AADC-Deficiency: Paul Wuh-Liang Hwu, MD, PhD

The professor from National Taiwan University Hospital discussed 3 studies of PTC-AADC for AADC deficiency. He provided an overview of some of the improvements seen in treated patients.

2. Eli-cel Gene Therapy for Early CALD to Be Withdrawn From EU, UK

bluebird bio is withdrawing its EU marketing authorization and UK filing for Skysona (elivaldogene autotemcel; eli-cel) for the treatment of cerebral adrenoleukodystrophy (CALD). They are also planning to withdraw their β-thalassemia therapy, beti-cel, from the EU and UK.

3. Developing Customizable CAR T-Cell Therapies

Rick Fair, president and chief executive officer, Bellicum Pharmaceuticals, discussed the company’s GoCAR, iMC, and caspaCIDe technologies and how these technologies were used to develop the company’s lead programs, BPX-601 and BPX-603.

4. Gene Therapy Promotes Wound Closure of Diabetic Foot Ulcers

The novel gene therapy VM202 (Engensis; Helixmith) has shown efficacy in treating diabetic foot ulcers, according to data from a phase 3 study (NCT02563522) presented at the 2021 annual meeting of the Diabetic Foot Conference, October 21-23.

5. Developing Personalized Immunotherapies for Solid Tumors

Jessica Baker Flechtner, PhD, chief scientific officer, Genocea, discussed the company’s focus on solid tumors and manufacturing cell therapies. She also discussed challenges in manufacturing their cell therapies as well as upcoming data on GEN-009, a neoantigen vaccine in development.