Our Pipeline


We develop a broad portfolio of drug candidates that have the potential to meaningfully improve the lives of patients by directly addressing the causes and risk factors of neurodegenerative diseases.

Each of our programs strictly follows the principles of our R&D strategy: Selecting targets that are genetically linked to the disease, engineering our molecules to cross the blood-brain barrier, and guiding clinical development through the discovery and use of biomarkers.

Our Pipeline

Lysosomal Function
Target Indication Drug Candidate
LRRK2 Parkinson's Early Clinical Early Clinical DNL151
Iduronate 2-sulfatase MPS II (Hunter Syndrome) Early Clinical Early Clinical DNL310 (ETV:IDS)
Progranulin Frontotemporal Dementia IND-Enabling IND-Enabling DNL593 (PTV:PGRN)
Alpha-Synuclein Parkinson's, DLB, MSA Drug Discovery Drug Discovery ATV:aSyn
Sulfamidase MPS IIIA (Sanfilippo Syndrome) Drug Discovery Drug Discovery ETV:SGSH
Undisclosed Lysosomal storage disease with Neurodegeneration Drug Discovery Drug Discovery ETV:LF1
Undisclosed Parkinson's Drug Discovery Drug Discovery AAV:LF2
Drug Discovery IND-Enabling Early Clinical Late Clinical Approved
Glial Biology
Target Indication Drug Candidate
RIPK1 ALS, Alzheimer's, MS Early Clinical Early Clinical DNL747
TREM2 Alzheimer's IND-Enabling IND-Enabling DNL919 (ATV:TREM2)
Undisclosed ALS Drug Discovery Drug Discovery GB1
Drug Discovery IND-Enabling Early Clinical Late Clinical Approved
Cellular Homeostasis
Target Indication Drug Candidate
EIF2B ALS, FTD Early Clinical Early Clinical DNL343
Tau Alzheimer's Drug Discovery Drug Discovery ATV:Tau
Abeta Alzheimer's Drug Discovery Drug Discovery ATV:Abeta
Undisclosed ALS, Parkinson's Drug Discovery Drug Discovery CH1
Drug Discovery IND-Enabling Early Clinical Late Clinical Approved
Other
Target Indication Drug Candidate
RIPK1 Peripheral Inflammatory Disease, RA Early Clinical Early Clinical DNL758
LRRK2 Crohn's Disease Drug Discovery Drug Discovery DNL975 (LRRK2 Peripheral)
Undisclosed Undisclosed Drug Discovery Drug Discovery OT1
Drug Discovery IND-Enabling Early Clinical Late Clinical Approved
Patients