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
Iduronate 2-sulfatase MPS II (Hunter Syndrome)
Mid Late Clinical DNL310 (ETV:IDS)
LRRK2 Parkinson's
Mid Late Clinical BIIB122/DNL151
Progranulin Frontotemporal Dementia-Granulin
Mid Early Clinical TAK-594/DNL593 (PTV:PGRN)
Sulfamidase MPS IIIA (Sanfilippo Syndrome)
Mid IND-Enabling DNL126 (ETV:SGSH)
Alpha-L-iduronidase MPS I
Mid IND-Enabling DNL622 (ETV:IDUA)
Drug Discovery IND-Enabling Early Clinical Mid/Late Clinical Approved
Glial Biology
TARGET INDICATION DRUG CANDIDATE
RIPK1 (CNS) Neurodegeneration (ALS)
Mid Late Clinical SAR443820/DNL788
TREM2 Alzheimer's
Mid Early Clinical TAK-920/DNL919 (ATV:TREM2)
Drug Discovery IND-Enabling Early Clinical Mid/Late Clinical Approved
Cellular Homeostasis
TARGET INDICATION DRUG CANDIDATE
eIF2B ALS FTD
Mid Early Clinical DNL343
Multiple Multiple
Mid IND-Enabling OTV:Multiple
Drug Discovery IND-Enabling Early Clinical Mid/Late Clinical Approved
Other
TARGET INDICATION DRUG CANDIDATE
RIPK1 (Peripheral) Systemic Inflammation (CLE, UC)
Mid Late Clinical SAR443122/DNL758
Drug Discovery IND-Enabling Early Clinical Mid/Late Clinical Approved