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).

Denali Therapeutics DNLI reported primary analysis data from an early to mid-stage study of its pipeline candidate, tividenofusp alfa (DNL310), in 47 patients with Hunter syndrome (MPS II ...

Denali remains on track to submit a BLA for tividenofusp alfa or DNL310 for the treatment of MPS II (Hunter syndrome), under the accelerated approval pathway in early 2025, and is preparing for a ...

The UK regulatory authorities have approved the first ever trial of a revolutionary gene therapy for young children diagnosed with Hunter syndrome ... also known as mucopolysaccharidosis type II (MPS ...

Feb. 06, 2025 (GLOBE NEWSWIRE) -- Denali Therapeutics Inc. (NASDAQ: DNLI), today announced the primary analysis of the Phase 1/2 study in 47 participants with Hunter syndrome (MPS II) in the 24-week ...

Shire’s attempts to develop a drug for cognitive impairment from the rare inherited disease Hunter Syndrome has failed ... and families living with MPS II.” “We are grateful to the children ...

MPS disorders are inherited in an autosomal recessive manner, except for MPS II (Hunter syndrome), which is X-linked. Symptoms vary depending on the specific type of MPS but commonly include ...

Regenxbio (RGNX) announced the closing of its previously announced strategic partnership with Nippon Shinyaku. Under the terms of the ...

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 ...

Fourth Quarter 2024 and Recent Program Updates CLINICAL PROGRAMS Tividenofusp alfa (DNL310, ETV:IDS) for Hunter syndrome (MPS II) In February 2025, Denali presented results of the primary analysis of ...