Review top news and interview highlights from the week ending March 29, 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.
The company noted that it plans to submit the BLA in the second quarter of 2024.
2. John A. Charlson, MD, on Afami-Cel’s Upcoming Review for Synovial Sarcoma
The associate professor of medicine, Medical College of Wisconsin, discussed advantages of afami-cel and data from the pivotal SPEARHEAD-1 trial.
3. AOC Therapy Yields Strength Improvements in Myotonic Dystrophy
Avidity is currently enrolling in the phase 3 HARBOR trial, which it expects to initiate in the second quarter of 2024.
The associate professor of pediatrics at Emory University discussed the need to empower patients and families to make their own treatment decisions.
5. SRP-9001 Improves DMD Disease Trajectory, But Falls Short on Primary End Point
In the phase 3 EMBARK trial, treatment with SRP-9001 improved secondary outcomes of time to rise, microdystrophin expression, and 10-meter walk/run.
Will the CMS CGT Model Support Patient Access to Sickle Cell Disease CGTs?
July 25th 2024Cell and gene therapies can transform the paradigm of care for patients with chronic, complex conditions, but these therapies come at an up-front cost of several million dollars per treatment, complicating the pipeline of access to them.