Denali Therapeutics
Denali Therapeutics works on 6 rare diseases tracked on Trial Friend, including Amyotrophic Lateral Sclerosis, Glycogen Storage Disease Type II, Hunter Syndrome and 3 more, with 4 recruiting clinical trials and 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.
Focus areas at Denali Therapeutics
As a rare disease specialist, Denali Therapeutics has active clinical trial programs and drug development efforts across 6 rare diseases, including Amyotrophic Lateral Sclerosis, Glycogen Storage Disease Type II, Hunter Syndrome, Mucopolysaccharidosis Type I, Sanfilippo Syndrome, and 1 additional rare conditions. 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 all active and recruiting 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.
Denali Therapeutics Drug Pipeline
Denali Therapeutics has 4 active clinical trials across 3 development stages, with 4 currently recruiting participants. Clinical trials advance through phases: Phase 1 tests safety in a small group, Phase 2 evaluates effectiveness and side effects, Phase 3 confirms benefit in a larger population, and Phase 4 monitors long-term safety after FDA approval.
Note: This pipeline includes all of Denali Therapeutics's active interventional trials, not only those targeting rare diseases. We show the full pipeline because a company's broader research activity, therapeutic expertise, and development infrastructure directly shape its ability to advance rare disease programs. A strong overall pipeline often signals deeper clinical operations, faster enrollment capabilities, and greater commitment to bringing new treatments to patients.
Denali Therapeutics Clinical Trials (4)
Active and recruiting clinical trials sponsored by Denali Therapeutics, sourced live from ClinicalTrials.gov. Each trial card shows the study phase, current recruitment status, conditions under investigation, study locations, eligibility criteria, and a direct link to the full ClinicalTrials.gov record. You can also download a one-page PDF summary to share with your doctor.
Note: Recruitment statuses on ClinicalTrials.gov may not immediately reflect recent FDA decisions, sponsor announcements, or enrollment changes. Always confirm a trial's current status directly with the study coordinator.
Denali Therapeutics FDA-Approved Drugs (1)
Medications developed or marketed by Denali Therapeutics that have received U.S. Food and Drug Administration approval. Drug data is sourced from the openFDA database and includes brand names, generic names, approval dates, and matched rare disease indications. Where a drug treats a condition covered by Trial Friend, the disease name links directly to that disease page.
| Drug Name | Brand Name | Rare Diseases | Approval Date |
|---|---|---|---|
| TIVIDENOFUSP ALFA-EKNM Hydrolytic Lysosomal Glycosaminoglycan-specific Enzyme [EPC] | AVLAYAH intravenous | Mar 24, 2026 |
Denali Therapeutics Trial Locations
Denali Therapeutics clinical trials are running at 45 sites in 14 countries. Click any country to drill down by state, city, and individual research facility. Proximity to a trial site is one of the most important factors in deciding whether to participate.
Rare Disease Focus Areas (6)
Diseases targeted by Denali Therapeutics's clinical trial and drug development programs
Amyotrophic lateral sclerosis is a progressive neurodegenerative disease destroying motor neurons in the brain and spinal cord. This causes progressive weakness and paralysis of voluntary muscles whil...
Glycogen Storage Disease Type II (GSD II, also called Pompe Disease) is a lysosomal storage disorder caused by deficiency of the enzyme acid alpha-glucosidase (GAA), which breaks down glycogen. This l...
Hunter syndrome is an X-linked lysosomal storage disorder caused by deficiency of iduronate-2-sulfatase enzyme, leading to accumulation of glycosaminoglycans in cells and tissues. This multisystem dis...
Mucopolysaccharidosis Type I (MPS I) is a lysosomal storage disorder caused by deficiency of the enzyme alpha-L-iduronidase, which breaks down glycosaminoglycans (GAGs). This results in accumulation o...
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. It builds up mainly in...
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. This buildu...
Patient Resources
Organizations and resources related to Denali Therapeutics's rare disease focus areas
Frequently Asked Questions About Denali Therapeutics
Common questions about Denali Therapeutics's rare disease programs, clinical trials, and treatments.