Review top news and interview highlights from the week ending 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.
The FDA’s Center for Biologics Evaluation and Research has issued draft guidance for industry players developing gene therapy products that involve gene editing.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.
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).
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.
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).
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).
Bendamustine Is an Effective Alternative to Fludarabine-Based Lymphodepletion in LBCL
December 7th 2024In the wake of fludarabine shortages, lemphodepletion with bendamustine was found to be an effective alternative compared for patients with large B-cell lymphoma being treated with a CD19-directed CAR T-cell therapy.