With an FDA approval submission for RegenXBio’s Hunter syndrome gene therapy already underway, the biopharma has now found a commercialization partner for both the U.S. and Asian markets.

DNLI announced that the FDA has granted Breakthrough Therapy Designation to its pipeline candidate, tividenofusp alfa (DNL310), for the treatment of individuals with Hunter syndrome (MPS II).

If you have carpal tunnel syndrome, your symptoms appear gradually and are mild at first. As a result, it’s easy to ignore the tingling, pain, and clumsiness caused by a pinched nerve. But recognizing ...

Fifteen-year-old Dominic Henriquez from Prosper, Texas, is bringing hope to boys like him who live with Hunter syndrome, a rare genetic disorder that primarily affects males. Dominic’s journey ...

Denali Therapeutics Inc. (NASDAQ: DNLI), today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for tividenofusp alfa (DNL310) for the treatment ...

An approval would make RGX-121 the first gene therapy for Hunter syndrome, which is progressive and causes a range of ...

Regenxbio will receive $110m upfront from Nippon Shinyaku and is eligible to get up to $700m in development, regulatory, and ...

Tividenofusp alfa (or DNL310) is composed of iduronate 2-sulfatase (IDS) fused to Denali’s proprietary Enzyme TransportVehicle™ (ETV), which is engineered for active transport into the brain and broad ...