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 ...

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

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.

One of the treatments, for a progressive disorder known as Hunter syndrome, could secure approval in the U.S. as soon as late ...

Denali Therapeutics DNLI has performed well in the past 12 months amid a volatile market. Shares of the company have gained ...

Nippon Shinyaku pays $110M upfront to Regenxbio for US/Asia rights to RGX-121 and RGX-111 gene therapies for ...

Denali Therapeutics showcases its BBB platform at J.P. Morgan Conference, highlighting DNL310's Hunter syndrome progress and plans for commercialization.

About a year ago, Tenvie President and Chief Scientific Officer Tony Estrada approached Denali CEO Ryan Watts and others in the Denali team with a proposal to spin out the small-molecule drugs with ...

Finally, a dancer cried out: "Stop the music!" A group of people rushed to the stage. Someone recognized that Hunter's heart had stopped. She was in cardiac arrest. One audience member started ...

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 ...

Jeffrey Hung, an analyst from Morgan Stanley, maintained the Buy rating on Denali Therapeutics (DNLI – Research Report). The associated price ...

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 ...