CGTLive’s Weekly Rewind – March 18, 2022

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. Proposed FDA Gene Editing Guidance Emphasizes Long-Term Safety Evaluations

The FDA’s Center for Biologics Evaluation and Research has issued draft guidance for industry players developing gene therapy products that involve gene editing. The document is a reflection of the concern surrounding safety of gene-editing strategies.

2. Presymptomatic SMA Treatment With Zolgensma Associated With Normal Development

Pediatric patients with spinal muscular atrophy (SMA) treated presymptomatically with onasemnogeneabeparvovec (Zolgensma; Novartis) continue to demonstrate age-appropriate development, according to new data from the phase 3 SPR1NT study (NCT03505099).

3. FDA Recognizes Canavan Disease Gene Therapy With Multiple Designations

The FDA has granted fast track, rare pediatric disease, and orphan drug designations to Myrtelle’s gene therapy rAAV-Olig001-ASPA for the treatment of Canavan disease.

4. 2-year Data for Duchenne Muscular Dystrophy Gene Therapy Shows Durable Improvements

SGT-001, Solid Biosciences’ investigational gene therapy for treating Duchenne muscular dystrophy (DMD), improved outcomes in treated patients over 2 years, according to new data from the phase 1/2 IGNITE-DMD study (NCT03368742).

5. First Patient Dosed in T-Cell Malignancy CAR T-Cell Therapy Trial

The first patient has been dosed in the phase 1/2 trial (NCT04984356) of Wugen’s WU-CART-007, which is being explored for the potential treatment of relapsed/refractory (R/R) T-cell acute lymphoblastic leukemia (T-ALL) or lymphoblastic lymphoma (LBL).