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 ...
BioSpace2d
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 ...
STAT1d
I don’t know what I’m supposed to call my rare kidney disease
I care deeply because this chronic disease is mine. It lives inside me. It’s radically changed my life and the life of my ...
BioPharma Dive5d
Translating FDA guidance into action: Regulatory considerations for orphan drug developers
Rare diseases, often referred to as orphan diseases, affect small patient populations, yet collectively they impact more than ...
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 Dive2d
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 ...
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 ...
Healio2d
FDA grants regenerative medicine advanced therapy designation to XLRS gene therapy
The FDA granted regenerative medicine advanced therapy designation to ATSN-201, a gene therapy candidate for the treatment of ...
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 ...
Ophthalmology Times2d
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 ...
Pharm Exec11d
FDA Grants Orphan Drug Designation to Sanofi’s Rare Disease Drug Rilzabrutinib for wAIHA & IgG4-RD
while a Phase IIa study demonstrated reduced disease flares and steroid use over a 52-week treatment period in patients with IgG4-RD. 1 “Orphan drug designation for these two rare, immune-mediated ...
Odylia Therapeutics drives commitment to rare disease drug development through Rare Disease Day event
Industry leaders gather to discuss innovative solutions for accelerating the development of therapeuticsBOSTON, MA, April 10, ...