A drop in endogenous Factor VIII expression was observed from treatment to 5-year follow-up despite continued demonstration of efficacy.
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.
The chief technology officer of Mustang Bio discussed the company’s approach and future plans in CAR T production.
Encouraging results from the phase 1 BrainChild-01 trial were recently published.
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 is designed to yield longer remission rates for pediatric and young adult patients with relapsed or refractory B-cell acute lymphoblastic leukemia.