Moderna Selected for FDA’s Rare Disease Therapeutics Pilot Program

On June 6th, Moderna announced that its investigational therapy for methylmalonic acidemia (MMA), mRNA-3705, has been chosen for the FDA’s Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot program. First launched in September 2023, the...

Ultragenyx to seek accelerated approval for rare disease gene therapy

Ultragenyx moved one step closer to establishing a new surrogate biomarker for accelerated approval in rare disease gene therapies. The biotech said Wednesday it will seek accelerated approval for its Sanfilippo syndrome type A gene therapy after the FDA appeared...