News

uniQure jumps on FDA breakthrough tag for Huntington’s disease drug
Qure (NASDAQ:QURE) shares added ~39% in the premarket on Thursday after the Dutch biotech announced that the FDA granted ...
Proof-of-Concept Data for Rare Disease Indications MVID and SBS-IF for Jaguar Health's Crofelemer to be Presented at April 2025 ELITE PED-GI Congress
Crofelemer, a novel plant-based anti-secretory prescription drug, has been granted Orphan Drug Designation by the FDA and the European Medicines ...
BioSpace1d
Glycomine Gets $115M Series C for Rare Disease, Aims for Phase II Launch Mid-Year
The raise will go toward trialing the company’s lead drug for phosphomannomutase-2 congenital disorder of glycosylation, a ...
CURE13d
FDA Grants Rare Pediatric Designation to Treatment With NEO100 in Brain Cancer
The FDA has granted Rare Pediatric Disease Designation for NEO100 in the treatment of pediatric-type diffuse high-grade ...
uniQure Announces FDA Breakthrough Therapy Designation Granted to AMT-130 for the Treatment of Huntington’s Disease
Breakthrough Therapy designation based on clinical evidence from Phase I/II trials showing meaningful slowing of disease progression ~~ ...
BioPharma Dive1d
Startup Glycomine raises $115M to push rare disease drug deeper into testing
The biotech is developing an experimental drug for the most common of a family of disorders all caused by errors in ...
BioPharma Dive3d
Translating FDA guidance into action: Regulatory considerations for orphan drug developers
This blog delves into the rise of rare disease drug approvals and the critical importance of regulatory guidance in shaping ...
Ultragenyx: Commercial Engine Fuels Rare Disease Pipeline
Ultragenyx's strong revenue growth, diverse streams, and late-stage pipeline make it a standout in rare disease and gene ...
Ophthalmology Times1d
FDA grants RMAT designation to Atsena Therapeutics' gene therapy for XLRS
ATSN-201 was given regenerative medicine advanced therapy designation to treat X-linked retinoschisis, for which there ...
Breakthrough stem cell therapy opens new treatment paths for autoimmune patients
“Our CB-SCs can target different compartments of the immune system, educating and correcting their dysfunctions at a fundamental level,” says Dr. Zhao. “This therapy has the potential to revolutionize ...