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.

Development Programs

Our Pipeline

Lysosomal Function
TARGET INDICATION DRUG CANDIDATE
LRRK2 Parkinson's
Early Clinical DNL151
Iduronate 2-sulfatase MPS II (Hunter Syndrome)
Early Clinical DNL310 (ETV:IDS)
Progranulin Frontotemporal Dementia
IND-Enabling DNL593 (PTV:PGRN)
Alpha-Synuclein Parkinson's DLB MSA
Drug Discovery ATV:aSyn
Sulfamidase MPS IIIA (Sanfilippo Syndrome)
IND-Enabling DNL126 (ETV:SGSH)
GCase Parkinson's, Gaucher disease
Drug Discovery ETV:GCase
Arylsulfatase A MLD
Drug Discovery ETV:ARSA
Alpha-N-acetylglucosaminidase MPS IIIB
Drug Discovery ETV:NAGLU
Alpha-L-iduronidase MPS I
Drug Discovery ETV:IDUA
Acid-α-glucosidase Pompe
Drug Discovery ETV:GAA
Undisclosed Parkinson's
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 DNL788
TREM2 Alzheimer's
IND-Enabling DNL919 (ATV:TREM2)
Undisclosed ALS
Drug Discovery GB1
Drug Discovery IND-Enabling Early Clinical Late Clinical Approved
Cellular Homeostasis
TARGET INDICATION DRUG CANDIDATE
eIF2B ALS FTD
Early Clinical DNL343
Tau Alzheimer's
Drug Discovery ATV:Tau
Abeta Alzheimer's
Drug Discovery ATV:Abeta
Undisclosed ALS Parkinson's
Drug Discovery CH1
Drug Discovery IND-Enabling Early Clinical Late Clinical Approved
Other
TARGET INDICATION DRUG CANDIDATE
RIPK1 Cutaneous Lupus Erythematosus (CLE) COVID-19
Early Clinical DNL758
LRRK2 Crohn's Disease
Drug Discovery DNL975 (LRRK2 Peripheral)
HER2 Oncology
Drug Discovery ATV:HER2
Drug Discovery IND-Enabling Early Clinical Late Clinical Approved