FDA Accepts BMS's NDA for Repotrectinib in ROS1-Positive NSCLC
The agency is reviewing data from the Phase II TRIDENT trial and expects to decide whether to approve the drug by Nov. 27.
FDA Delays Decision for Sarepta Therapeutics Duchenne Muscular Dystrophy Gene Therapy
According to Sarepta, the federal agency has indicated it may grant accelerated approval to SRP-9001 as a treatment for a subgroup of patients based on age.
FDA, Industry Taking Steps to Streamline Cell Therapy Regulation, Though Uncertainty Remains
At two recent meetings, representatives from regulatory agencies, industry, and academia discussed ways to foster efficient, more predictable cell therapy regulation.
Bespoke Gene Therapy Consortium to 'Pressure Test' IND Roadmap with Eight Rare Disease Candidates
The gene therapies selected by the public-private consortium will inform a playbook that walks developers through preclinical work to IND submission.
Sangamo Therapeutics Prepping for Phase III Fabry Disease Gene Therapy Trial
Having garnered fast-track designation from the FDA and spurred by promising Phase I/II data, the firm is hoping to begin a late-stage trial of ST-920 by year-end.