Biospace

• The FDA experienced a slight slowdown in regulatory productivity during the first half of 2024 due to significant leadership instability and ongoing workforce attrition. • Under acting commissioner Kyle Diamantas, the agency has shifted toward a more open stance, particularly regarding rare disease therapies and accelerated approval applications. • Key beneficiaries of this shift include REGENXBIO, which was told its clinical data for RGX-121 was sufficient, as well as Replimune and uniQure.

• HCA Healthcare has announced a new study published in the New England Journal of Medicine (NEJM) highlighting advancements in CRISPR-based therapies for children. • The research builds upon pioneering work at TriStar Centennial Medical Center and the Sarah Cannon Transplant and Cellular Therapy Network, which performs over 1,600 cellular therapies and transplants annually. • Dr. Frangoul, a key investigator, previously contributed to the first U.S. clinical trial for gene-editing in sickle cell disease, leading to the first FDA-approved CRISPR therapy.

• The U.S. Department of Health and Human Services (HHS) and the FDA have launched an "unprecedented" initiative to restore American leadership in clinical trials and counter competition from China. • A key component of the plan is an expedited investigational new drug pilot program designed to reduce early trial timelines by as much as 12 months. • Health Secretary Robert F. Califf stated the effort aims to reverse the trend of clinical research moving overseas by making the U.S. the premier location for medicine development.