With an FDA approval submission for RegenXBio’s Hunter syndrome gene therapy already underway, the biopharma has now ...

The UK regulatory authorities have approved the first ever trial of a revolutionary gene therapy for young children diagnosed with Hunter syndrome, a devastating rare lysosomal storage disorder. Five ...

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.

Denali Therapeutics Inc. DNLI announced that the FDA has granted Breakthrough Therapy Designation to its pipeline candidate, ...

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).

The Hunter syndrome program was developed by Brian Bigger, a professor of cell and gene therapy at The University of Manchester. Professor Bigger has published preclinical data demonstrating that the ...

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 ...

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 ...