Rare disease drugmaker · based in South San Francisco, United States · founded 2015 · ticker DNLI · denalitherapeutics.com
Denali Therapeutics works on 5 rare diseases we track, including Amyotrophic Lateral Sclerosis, Hunter Syndrome and 3 more. It has 4 active clinical trials, and 2 are looking for volunteers now. It also has 1 FDA-approved rare disease drug.
Denali Therapeutics develops therapies for neurodegenerative and lysosomal storage diseases using its Transport Vehicle (TV) platform to cross the blood-brain barrier. Avlayah (tividenofusp alfa) received FDA accelerated approval in March 2026 for neurologic manifestations of Hunter syndrome (MPS II). DNL952 (ETV:GAA) for Pompe disease is in Phase 1 development.
Denali Therapeutics pipeline
Each row lists the diseases Denali Therapeutics is studying at that stage, from the ones closest to approval down to the earliest work.
These are Denali Therapeutics trials for the rare diseases we track, from ClinicalTrials.gov. Statuses there can lag behind company news, so confirm with the study team before you plan around one.
2 trials looking for volunteers now
Looking for volunteersPhase 1/2Recently updatedNCT06181136
Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)
This is a multicenter, open-label, Phase 1/2 study to assess the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and clinical efficacy of DNL126 in participants with Sanfilippo syndrome Type A (MPS IIIA). In Part 1, the core study period is 25 weeks (approximately 6 months); followed by an open-label extension (OLE), which extends through Week 97 (approximately 18 months); and a long-term extension (LTE), which extends through Week 241 (Year 5). In Part 2, the core study period is 25 weeks in duration followed by an OLE, which extends through Week 49, and an LTE, which extends through Week 241 (Year 5). Participants with MPS IIIA will be enrolled in three planned cohorts, and additional participants with MPS IIIA may be enrolled in three optional cohorts.
The study will enroll up to approximately 30 participants in total: 26 participants in Part 1 (Cohorts A1, A2, A3, B1, and B2 - not enrolling) and 4 participants in Part 2 (Cohort C1 - actively enrolling).
This is a Phase 1, multicenter, open-label study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of DNL952 in adult participants with late-onset Pompe disease. The principal aim of this study is to obtain safety and tolerability data across varous dose levels of DNL952 in participants with late-onset Pompe disease (LOPD).
Underway, not taking new volunteersPhase 2/3Recently updatedNCT05371613
A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)
Teststividenofusp alfa, idursulfase
For Mucopolysaccharidosis II
This is a Phase 2/3, multiregional, two-arm, double-blind, randomized, active (standard-of-care)-controlled study of the efficacy and safety of tividenofusp alfa (DNL310), an investigational central nervous system (CNS)-penetrant enzyme-replacement therapy (ERT) for mucopolysaccharidosis type II (MPS II).
Participants may also qualify to enter an open-label treatment phase with DNL310 or idursulfase based on pre-specified criteria.
Underway, not taking new volunteersPhase 1/2Hasn't posted an update in over a yearNCT04251026
A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome
Teststividenofusp alfa
For Mucopolysaccharidosis II
This is a multicenter, multiregional, open-label study to assess the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of tividenofusp alfa (DNL310), an investigational central nervous system (CNS)-penetrant enzyme replacement therapy (ERT), designed to treat both the peripheral and CNS manifestations of Mucopolysaccharidosis type II (MPS II; Hunter syndrome).
Participants, whose physicians feel they are deriving benefit, will have the opportunity to be reconsented into a safety extension and then an open-label extension for continued evaluation.
Follow a condition Denali Therapeutics is studying
Pick a condition. One email when a new trial opens, a trial changes or the FDA decides, from any company, not only Denali Therapeutics. Unsubscribe anytime.
You'll get, for Amyotrophic Lateral Sclerosis:
Trial Alerts, when a new trial opens or a trial changes
Disease Updates, FDA decisions and condition news, usually 1 or 2 emails a month
Turn either off anytime from the link in every email.
For Hunter Syndrome. Approved Mar 24, 2026. Taken by intravenous route.
More about Denali Therapeutics
As a rare disease specialist, Denali Therapeutics has active clinical trial programs and drug development efforts across 5 rare diseases, including Amyotrophic Lateral Sclerosis, Hunter Syndrome, Mucopolysaccharidosis Type I, Sanfilippo Syndrome, Pompe Disease. These programs may span orphan drug designation, novel therapeutic mechanisms, and precision medicine approaches targeting the underlying causes of each disease.
The clinical trials section below shows active and recruiting rare disease studies sponsored by Denali Therapeutics, sourced live from ClinicalTrials.gov. Each trial includes its current recruitment status, study phase (Phase 1 through Phase 4), conditions under investigation, and the number of active trial sites.
The FDA-approved drugs section lists treatments that have received U.S. Food and Drug Administration approval, with brand names, generic names, approval dates, and matched rare disease indications from the openFDA database.
Denali Therapeutics is headquartered in South San Francisco, United States, founded in 2015, publicly traded under the ticker symbol DNLI. The company dedicates its research and development resources exclusively to rare and orphan diseases, working to bring innovative treatments to patients with conditions that have historically had limited or no treatment options.
Where Denali Therapeutics trials take place
The trials above run at 45 study sites in 14 countries. Tap a country to see its states, cities and hospitals.
United States
20▼
United Kingdom
5▼
Canada
4▼
Belgium
2▼
Germany
2▼
Netherlands
2▼
Spain
2▼
Turkey (Türkiye)
2▼
Argentina
1▼
Brazil
1▼
Czechia
1▼
France
1▼
Rare diseases Denali Therapeutics works on
Each disease page lists every open trial, not only Denali Therapeutics's, plus approved treatments.
Amyotrophic lateral sclerosis is a progressive neurodegenerative disease destroying motor neurons in the brain and spinal cord.
How common: About 5,000 new cases per year in the U.S.; CDC's National ALS Registry estimates about 33,000 Americans were living with ALS in 2022, a number projected to pass 36,000 by 2030
Hunter syndrome is an X-linkedlysosomal storage disorder caused by deficiency of iduronate-2-sulfatase enzyme, leading to accumulation of glycosaminoglycans in cells and tissues.
How common: 1 in 100,000 to 150,000 males; females with disease are rare
Mucopolysaccharidosis Type I (MPS I) is a lysosomal storage disorder caused by deficiency of the enzyme alpha-L-iduronidase, which breaks down glycosaminoglycans (GAGs).
How common: Approximately 1 per 100,000 live births; Hurler form is the most common phenotype
Sanfilippo syndrome, formally mucopolysaccharidosis type III (MPS III), is a lysosomal storage disorder in which the body cannot break down a sugar chain called heparan sulfate.
How common: Approximately 1 per 70,000 to 1 per 100,000 live births; highest prevalence in certain populations including Australia and Scandinavia
Pompe disease is a rare inherited lysosomal storage disorder caused by deficiency of the enzyme acid alpha-glucosidase, leading to excessive accumulation of glycogen in muscles and organs.
How common: 1 in 40,000 people (infantile form: 1 in 138,500; late-onset: 1 in 60,000)
Where to get support
Patient groups can explain a diagnosis, point you to specialists and tell you about trials before they open. Denali Therapeutics's own site is denalitherapeutics.com, and our help paying for treatment page lists drugmaker programs and charity funds.
1 shared disease, including Amyotrophic Lateral Sclerosis
Questions about Denali Therapeutics
Denali Therapeutics develops therapies for neurodegenerative and lysosomal storage diseases using its Transport Vehicle (TV) platform to cross the blood-brain barrier. Avlayah (tividenofusp alfa) received FDA accelerated approval in March 2026 for neurologic manifestations of Hunter syndrome (MPS II). DNL952 (ETV:GAA) for Pompe disease is in Phase 1 development.
Denali Therapeutics has active programs across 5 rare diseases: Amyotrophic Lateral Sclerosis, Hunter Syndrome, Mucopolysaccharidosis Type I, Sanfilippo Syndrome, Pompe Disease. Each disease page on Trial Friend shows the latest recruiting trials, FDA-approved treatments, and patient resources.
Yes. Denali Therapeutics is a rare disease specialist headquartered in South San Francisco, United States, founded in 2015. The company focuses primarily on developing treatments for rare and orphan diseases, with programs targeting Amyotrophic Lateral Sclerosis, Hunter Syndrome, Mucopolysaccharidosis Type I among others.
The clinical trials section on this page shows active and recruiting rare disease trials sponsored by Denali Therapeutics, pulled live from ClinicalTrials.gov. Each trial card includes the study phase, recruitment status, conditions being studied, number of trial sites, and a direct link to the full study record. You can also explore individual disease pages to see trials from all sponsors for a specific condition.
To explore enrollment, find a recruiting trial in the clinical trials list on this page and click "View full study" to see the full ClinicalTrials.gov record, which includes eligibility criteria, study locations, and contact information for each trial site. Your doctor can also help determine if a specific trial is appropriate for your condition. Not all trials are open at every location, so check the site list for one near you.
Denali Therapeutics has 1 FDA-approved drug with a rare disease use that we track, as of November 2026.
Source: the FDA's openFDA label database, matched to the rare diseases on Trial Friend; the list updates automatically and can include older products.
Yes, for 1 of its FDA-approved rare disease medicine that we track, each checked on Denali Therapeutics' own website.
Avlayah: Denali Patient Services, 1-844-365-4365. Offers copay help with private insurance and insurance help. Checked October 5, 2026.
Medicare and Medicaid: drugmaker copay cards are limited to private insurance, so people with government coverage usually look to a free medicine program or a charity fund, both on the help paying page.
Eligibility is decided by each program, so call the number listed before a prescription is filled.
Denali Therapeutics was founded in 2015 and is headquartered in South San Francisco, United States. The company has been active for 11 years. It trades publicly under the ticker symbol DNLI. As a rare disease specialist, the company dedicates its resources to developing therapies for patients with rare and orphan conditions.