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 |
---|---|---|
Iduronate 2-sulfatase | MPS II (Hunter Syndrome) | |
LRRK2 | Parkinson's | |
Progranulin | Frontotemporal Dementia-Granulin | |
Sulfamidase | MPS IIIA (Sanfilippo Syndrome) | |
Alpha-L-iduronidase | MPS I |
Drug Discovery IND-Enabling Early Clinical Mid/Late Clinical Approved
Glial Biology
TARGET | INDICATION | DRUG CANDIDATE |
---|---|---|
RIPK1 (CNS) | Neurodegeneration (ALS) | |
TREM2 | Alzheimer's |
Drug Discovery IND-Enabling Early Clinical Mid/Late Clinical Approved
Cellular Homeostasis
TARGET | INDICATION | DRUG CANDIDATE |
---|---|---|
eIF2B | ALS FTD | |
Multiple | Multiple |
Drug Discovery IND-Enabling Early Clinical Mid/Late Clinical Approved
Other
TARGET | INDICATION | DRUG CANDIDATE |
---|---|---|
RIPK1 (Peripheral) | Systemic Inflammation (CLE, UC) |
Drug Discovery IND-Enabling Early Clinical Mid/Late Clinical Approved