approval pathway
Diamyd Medical’s Pivotal Phase 3 Type 1 Diabetes Trial Clears Final Safety Review Ahead of Early Readout in March 2026
Diamyd Medical; Type 1 Diabetes; Phase 3 trial; DIAGNODE-3; safety review; Data Safety Monitoring Board (DSMB); interim readout; March 2026; HLA DR3-DQ2 genotype; Orphan Drug Designation; Fast Track Designation; accelerated approval pathway; precision therapy
Researchers Publish Roadmap of FDA Interactions After Baby KJ’s Groundbreaking Gene Editing Therapy, as FDA Signals New Pathway for Patient-Specific Treatments
Baby KJ; CRISPR gene editing; CPS1 deficiency; FDA approval pathway; personalized medicine; rare disease; regulatory roadmap; platform trials; N-of-1 therapies; rare disease gene therapies
Sangamo Therapeutics Aligns with FDA on Accelerated Approval Pathway for ST-920 Gene Therapy in Fabry Disease
Sangamo Therapeutics, FDA, Accelerated Approval Pathway, ST-920, Fabry Disease, Gene Therapy, BLA Submission
Sangamo Therapeutics Fast-Tracks Fabry Gene Therapy with FDA Accelerated Approval Pathway
Sangamo Therapeutics, Fabry disease, gene therapy, isaralgagene civaparvovec, FDA, accelerated approval pathway, ST-920, STAAR study
Bristol Myers’ KRAS Confirmatory Data Avoid Amgen’s Flaws but Leave FDA Prospect Unclear
Bristol Myers Squibb, KRAS confirmatory data, Amgen, FDA, accelerated approval pathway, oncology therapies, clinical benefits, confirmatory trials