FierceBiotech7d
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 ...
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.
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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, ...
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Re­genxbio sells Hunter syn­drome li­cense with FDA fil­ing un­der­way
Nippon Shinyaku pays $110M upfront to Regenxbio for US/Asia rights to RGX-121 and RGX-111 gene therapies for ...
BioPharma Dive7d
Regenxbio licenses gene therapies to Japan’s Nippon Shinyaku
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 Announces U.S. FDA Breakthrough Therapy Designation Granted to Tividenofusp Alfa for the Treatment of Hunter Syndrome (MPS II)
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 ...
Why Is Denali Therapeutics Stock Trading Higher On Wednesday?
Denali Therapeutics showcases its BBB platform at J.P. Morgan Conference, highlighting DNL310's Hunter syndrome progress and plans for commercialization.
Denali: Despite ALS Primary Endpoint Failure, Possible Path Forward Remains
Denali Therapeutics Inc.'s stock remains stable despite trial setback in ALS. BLA filing for MPS II treatment on track. Click ...
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DNLI's Hunter Syndrome Drug Gets Breakthrough Therapy Designation
Denali’s wholly owned program, DNL310 or tividenofusp alfa, is an Enzyme Transport Vehicle-enabled iduronate-2-sulfatase (IDS) replacement therapy in development for MPS II (Hunter syndrome).