CGTLive’s Weekly Rewind – February 9, 2024

CGTLive’s Weekly Rewind – February 9, 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. Sickle Cell Disease Gene Therapies to Be Part of Initial Focus for Biden-Harris Administration’s CGT Access Model

The new Cell and Gene Therapy Access Model will be led by the Centers for Medicare & Medicaid Services’ Innovation Center.

2. Michael Kelly, PhD, on Potential of Nonviral Approaches to DMD Gene Therapy

The chief scientific officer of CureDuchenne discussed how nonviral approaches may address issues including gene size and redosability.

3. Akouos Seeks to Tackle Otoferlin Gene-Mediated Hearing Loss With Phase 1/2 Clinical Trial and Associated Natural History Study

AK-OTOF delivers transgenes encoding OTOF, the disease-targeted gene, to the inner hair cells of the cochlea via a single unilateral intracochlear administration.

4. M. Peter Marinkovich, MD, on Increased Accessibility With Topical B-VEC DEB Gene Therapy

The associate professor of dermatology at Stanford University discussed the potential impact of Vyjuvek’s approval in the derm field.

5. Arsa-Cel Continues to Show Durable Benefit in MLD Outcomes Over Natural History

Orchard Therapeutics has also randomized the first patient with Hurler Syndrome in the phase 3, registrational HURCULES trial of OTL-203 gene therapy.