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DNLI's Hunter Syndrome Drug Gets Breakthrough Therapy Designation
Denali Therapeutics Inc. DNLI announced that the FDA has granted Breakthrough Therapy Designation to its pipeline candidate, tividenofusp alfa (DNL310), for the treatment of individuals with Hunter ...
pharmaphorum7y
Shire's Hunter Syndrome cognitive impairment drug fails
Shire’s attempts to develop a drug for cognitive impairment from the rare inherited disease Hunter Syndrome has failed to produce results. The company’s Elaprase (idursulfase) is already ...
North Texas teen offers hope to others with Hunter Syndrome
Hunter syndrome results from the absence of an enzyme needed to break down cellular waste. Without it, waste builds up in the body, causing progressive damage to various systems.
FierceBiotech11d
RegenXBio sells Hunter syndrome gene therapy's U.S., Asia commercial rights for $110M upfront
With an FDA approval submission for RegenXBio’s Hunter syndrome gene therapy already underway, the biopharma has now ...
University of Manchester4y
University announces partnership with AVROBIO for Hunter syndrome gene therapy
The University of Manchester has announced today a groundbreaking gene therapy partnership to ease the lifelong suffering of people with Hunter syndrome. The University has agreed to a worldwide ...
BioWorld9d
Regenxbio, Nippon Shinyaku in $810M deal for Hunter and Hurler gene therapies
In a deal potentially worth $810 million for Regenxbio Inc., Nippon Shinyaku Co. Ltd. is partnering on the U.S. and Asian development and commercialization of iduronate-2-sulfatase enzyme RGX-121 for ...