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Oral drugs to halt neurodegeneration
Origami Therapeutics, Inc. is a biotech company developing novel protein degraders with potential to delay or halt disease progression by eliminating toxic proteins from the body. Our drug discovery platform leverages our proprietary human disease models developed to select the small molecules with the best chance of clinical success. Our first indication is Huntington’s disease, a debilitating, progressive and ultimately fatal disorder caused by a genetic mutation. We have already identified small molecules that reduce the toxic mutated protein, suppress mutant protein-induced toxicities in human disease neurons and get into the brain. We anticipate that this approach will be broadly applicable to traditionally “undruggable” targets in other neurological diseases.
Origami Therapeutics is developing disease-modifying protein degraders and correctors aimed at neurodegenerative diseases. The approach focuses on selectively degrading disease-causing proteins to halt progression while sparing normal protein forms, leveraging small molecules for broad distribution and manufacturability.
The company uses ORICISION™ Drug Discovery Platform to identify and develop therapies that degrade disease-related proteins and correct faulty proteins. Their strategy centers on small-molecule–based degraders designed to selectively remove pathogenic proteins implicated in neurodegenerative diseases, with an emphasis on delivering broad distribution in the body to reach affected tissues.
Who it’s for: Pharmaceutical/biotech companies, researchers, and clinical development teams focused on neurodegenerative diseases such as Alzheimer’s, Huntington’s, Parkinson’s, ALS, and FTD.
At Origami Therapeutics, Dr. Hoffman is creating a new approach to drug discovery for neurodegenerative diseases. Prior to Origami, she was a Research & Development executive at Vertex Pharmaceuticals, Amgen and Eli Lilly. She has made major contributions to >30 clinical programs in neurology, pain and psychiatry and to four marketed drugs for Cystic Fibrosis. Dr. Hoffman received her A.B. in Molecular Biology from Wellesley College and a Ph.D. in Cell Biology from the Johns Hopkins University.
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