Biogen, C4 Therapeutics Working to Treat Parkinson’s by ‘Naturally’ Degrading and Clearing Proteins

Biogen and C4 Therapeutics (C4T) have joined efforts to develop therapies using a cell’s natural protein degradation system as a way of treating neurological diseases such as Alzheimer’s and Parkinson’s.

This agreement allows the two to combine Biogen’s expertise in neuroscience and therapy development with C4T’s knowledge and research work into targeted protein degradation, or the breaking down of faulty or damaged proteins.

“We are excited to work with our Biogen colleagues to take on the challenge of researching new potential therapies for Alzheimer’s disease, Parkinson’s disease and other devastating neurological diseases,” Andrew Phillips, PhD, president and CEO of C4T, said in a press release.

“Our approach of using the cell’s innate mechanisms to eliminate specific, disease-causing proteins is a promising new way to tackle the challenges of central nervous system diseases,” he added.

C4T uses its proprietary Degronimid platform technology to develop small molecules that promote the targeted degrading and rapid clearance of disease-causing proteins. The Degronimid technology was initially developed by researchers at Dana-Farber Cancer Institute and was licensed to C4T in January 2016.

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This new strategy involves chemically attaching a small molecule ligand that binds the selected proteins to another molecule, which then hijacks an enzyme whose function is to direct proteins to the cell’s natural protein degradation machinery — the proteasome.

Degronimid holds promise as a way of removing  proteins that do not respond to treatments against them, as well as proteins known to develop resistance to therapies that work to inhibit or block them. Overall, taking advantage of the cell’s natural cleaning system to degrade targeted proteins represents a new approach to treatment.

“C4T’s platform enables the discovery of novel molecules that take advantage of endogenous protein degradation mechanisms to target disease-causing proteins,” said Michael Ehlers, MD, PhD, executive vice president, research and development at Biogen.

“This new collaboration with C4T complements our broader research strategy to develop potential therapies for neurological conditions across multiple modalities,” he said. “We look forward to discovering new potential therapeutic options for diseases that currently have limited-to-no treatment options available.”

Biogen and C4T will work together in researching targets, with Biogen responsible for advancing likely candidates along in development, the release stated. C4Twill earn up to $415 million in upfront and potential future royalties under the collaboration, it added.