Valoctocogene Roxaparvovec’s Treatment Durability in Hemophilia A in Question

A drop in endogenous Factor VIII expression was observed from treatment to 5-year follow-up despite continued demonstration of efficacy.

Novel Therapies in Development for Dravet Syndrome

Joseph Sullivan, MD, and Elaine C. Wirrell, MD, discuss new developments for the treatment of Dravet syndrome beyond seizure control including gene editing and therapy.

Improving Efficiency in CAR T Cell Therapy Production

The chief technology officer of Mustang Bio discussed the company’s approach and future plans in CAR T production.

HER2-Specific CAR T Therapy Shows Safety and Efficacy in Pediatric Tumors

Encouraging results from the phase 1 BrainChild-01 trial were recently published.

ctDNA Changes May Predict CD19-Targeted CAR T-cell Therapy Response in LBCL

Heterogeneity in the cellular and molecular features of CAR T-cell products contributes to variation in efficacy and toxicity follow treatment with axicabtagene ciloleucel.

HuCART19 Efficacious and Durable in B-cell Acute Lymphoblastic Leukemia

huCART19 is designed to yield longer remission rates for pediatric and young adult patients with relapsed or refractory B-cell acute lymphoblastic leukemia.