Gene therapy pioneer uniQure launched the first-in-human adeno-associated virus-based gene therapy clinical trial for Huntington’s disease.
Locanabio combines gene therapy with RNA modification for a novel, highly flexible platform that can modify, replace or eliminate dysfunctional RNA.
Cambridge, Massachusetts-based Carmine Therapeutics inked a research collaboration deal with Japan’s Takeda Pharmaceutical to develop and commercialize non-viral gene therapies for two rare diseases.
CSL Behring bolstered the company’s growing gene therapy portfolio with the acquisition of global rights to uniQure’s gene therapy program for hemophilia B that has a price tag of up to $2 billion.
Cambridge, Massachusetts-based Dyno Therapeutics came out of stealth mode with an official launch of the company’s proprietary CapsidMap platform.
Amicus Therapeutics announced positive interim results from the company’s CLN6 Batten disease gene therapy program.
Rockville, Maryland-based RegenxBio entered a license deal with Pfizer for a gene therapy for Friedreich’s ataxia, the most common hereditary ataxia.
Pfizer’s IV Revatio, when added to inhaled nitric oxide, failed to meet the primary efficacy endpoint in treating newborns with persistent pulmonary hypertension in a Phase III study.
Cranbury, N.J.-based Amicus Therapeutics and the Perelman School of Medicine at the University of Pennsylvania are expanding their gene therapy collaboration.
Asklepios BioPharmaceutical (AskBio) secured a $235 million investment from Vida Ventures and TPG Capital.
