Denali Therapeutics Inc.
Denali Therapeutics is dedicated to defeating neurodegenerative and lysosomal storage diseases through rigorous therapeutic discovery and development. They focus on addressing the genetic causes and biological processes underlying neurodegenerative diseases, leveraging scientific expertise to discover effective molecular therapeutics. Their mission is to develop treatments that can cross the blood-brain barrier and improve the lives of patients suffering from conditions such as Alzheimer’s, Parkinson’s, ALS, and other neurodegenerative diseases.
Industries
Nr. of Employees
large (251-1000)
Denali Therapeutics Inc.
Patents
Products
Recombinant iduronate-2-sulfatase engineered for CNS delivery
A recombinant iduronate-2-sulfatase protein engineered to cross the blood-brain barrier to replace deficient enzyme activity in neuropathic and systemic forms of MPS II.
LRRK2 small-molecule brain-penetrant inhibitor
A potent, selective, orally administered small-molecule inhibitor of LRRK2 designed to penetrate the brain and modulate lysosomal function implicated in Parkinson's disease.
Recombinant progranulin engineered for CNS delivery
A recombinant progranulin protein modified for brain delivery to restore progranulin function and lysosomal homeostasis in progranulin-deficient frontotemporal dementia.
Recombinant sulfamidase engineered for CNS delivery
A recombinant N-sulfoglucosamine sulfohydrolase protein engineered for blood-brain barrier penetration to treat Sanfilippo syndrome A (MPS IIIA).
Recombinant alpha-L-iduronidase engineered for CNS delivery
A recombinant alpha-L-iduronidase modified to cross the blood-brain barrier to treat neuropathic and systemic forms of MPS I.
Oligonucleotide CNS programs (multiple targets)
Development of oligonucleotide therapeutic candidates using CNS delivery technology to target multiple disease-relevant transcripts.
Recombinant iduronate-2-sulfatase engineered for CNS delivery
A recombinant iduronate-2-sulfatase protein engineered to cross the blood-brain barrier to replace deficient enzyme activity in neuropathic and systemic forms of MPS II.
LRRK2 small-molecule brain-penetrant inhibitor
A potent, selective, orally administered small-molecule inhibitor of LRRK2 designed to penetrate the brain and modulate lysosomal function implicated in Parkinson's disease.
Recombinant progranulin engineered for CNS delivery
A recombinant progranulin protein modified for brain delivery to restore progranulin function and lysosomal homeostasis in progranulin-deficient frontotemporal dementia.
Recombinant sulfamidase engineered for CNS delivery
A recombinant N-sulfoglucosamine sulfohydrolase protein engineered for blood-brain barrier penetration to treat Sanfilippo syndrome A (MPS IIIA).
Recombinant alpha-L-iduronidase engineered for CNS delivery
A recombinant alpha-L-iduronidase modified to cross the blood-brain barrier to treat neuropathic and systemic forms of MPS I.
Oligonucleotide CNS programs (multiple targets)
Development of oligonucleotide therapeutic candidates using CNS delivery technology to target multiple disease-relevant transcripts.
Services
Structuring and executing collaborative research and development agreements with academic, industry and patient-advocacy partners to co-develop therapeutic programs.
End-to-end clinical development capabilities including protocol design, biomarker-driven study design, trial operations and data-driven decision making from pre-IND through Phase 3.
Development and scale-up of manufacturing processes for biologics and other modalities, quality systems, and management of clinical supply chains.
Design, development and validation of analytical and biomarker assays to support translational studies and clinical endpoints.
Structured engagement programs with patients and advocacy organizations to collect input for trial design, communication and community outreach.
Structuring and executing collaborative research and development agreements with academic, industry and patient-advocacy partners to co-develop therapeutic programs.
End-to-end clinical development capabilities including protocol design, biomarker-driven study design, trial operations and data-driven decision making from pre-IND through Phase 3.
Development and scale-up of manufacturing processes for biologics and other modalities, quality systems, and management of clinical supply chains.
Design, development and validation of analytical and biomarker assays to support translational studies and clinical endpoints.
Structured engagement programs with patients and advocacy organizations to collect input for trial design, communication and community outreach.
Expertise Areas
- Neurodegenerative disease therapeutics
- Blood-brain barrier delivery
- Lysosomal enzyme replacement
- Oligonucleotide therapeutics for CNS
Key Technologies
- Blood-brain barrier transport platform
- Receptor-mediated transcytosis (transferrin receptor)
- Recombinant enzyme engineering
- Oligonucleotide (ASO) delivery