Hunter Syndrome (MPS II)
Hunter syndrome, or mucopolysaccharidosis type II (MPS II), is a rare progressively debilitating genetic disorder in which people cannot break down heparan sulfate, one type of large sugar molecules called glycosaminoglycans (also known as GAGs, or mucopolysaccharides), resulting in build up in brain and body tissues. Hunter syndrome is caused by a deficiency of the lysosomal enzyme iduronate-2-sulfatase (I2S), which is responsible for the breakdown of glycosaminoglycans.
“Ultimately the hope for all of our families is that we can find a therapeutic that's going to help their child and eventually a cure. Every day, they rely so much on the hope that industry can bring to them.”
Terri Klein - President, National MPS Society
Denali’s scientific approach to Hunter syndrome
Denali is evaluating DNL310, also known as tividenofusp alfa, an investigational weekly IV treatment, in clinical studies. This treatment is proposed to work by using Enzyme Transport Vehicle (ETV) technology to carry the missing enzyme, I2S, across the blood-brain barrier brain (BBB) and to other parts of the body. DNL310 is being studied to determine if this treatment addresses the behavioral, cognitive, and physical symptoms of Hunter syndrome.
DNL310 is being studied in two clinical trials. A Phase 1/2 study to access the safety of DNL310 is no longer recruiting. A separate Phase 2/3 study called COMPASS is currently enrolling participants. The objective of the COMPASS study is to determine the efficacy and safety of DNL310 vs idursulfase, an enzyme replacement therapy (ERT).
To learn more about this study, including details about who may be eligible, visit
ClinicalTrials.gov (COMPASS Phase 2/3)
DNL310 is an investigational drug, its clinical profile has not been established, and it has not been approved by any global health authority.
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Advocacy Organizations
These organizations are committed to supporting the needs of people and families affected by Hunter syndrome. Their websites may offer you more information, resources, and guidance.
By clicking these links, you will leave the Denali Therapeutics website and be redirected to a third-party site. Denali is not responsible for, nor does it endorse, the content, terms and conditions of use or privacy practices of external websites.
Project Alive was founded with the purpose of finding a cure for Hunter syndrome.
The Hunter Syndrome Foundation funds research, supports families, and raises awareness of this rare disease.
IMPSN is a global community of informed and empowered organizations, who promote access to medicines, new research, and programs that lead to better quality of lives for patients and those affected by MPS and related diseases.
MPS Society UK transforms lives through support, research and awareness. We provide professional support to individuals and families affected by MPS, Fabry and related lysosomal conditions in the UK.
The National MPS Society’s mission is to serve those impacted by MPS and mucolipidosis (ML). It supports research, provides families with invaluable resources, and works to increase public and professional awareness. Visit this site to see a list of country-specific organizations outside the US.
Courageous Parents Network is a non-profit organization that orients and empowers parents and others caring for children with serious medical conditions, by providing resources and tools that reflect the experience and perspective of other families and clinicians.
Denali values all MPS II community members and organizations. Additional resources may be available.
For questions, please contact the Denali Patient Advocacy team at patients@dnli.com.