RARE Daily

Aquinnah Enters Collaboration with Roche to Advance Therapies for ALS and Other Neurodegenerative Diseases

February 17, 2022

Aquinnah Pharmaceuticals said it has signed an agreement with Roche to advance the development of brain penetrant oral small molecules to treat amyotrophic lateral sclerosis and other neurodegenerative diseases.

Recent advances suggest that the chronic response of RNA metabolism to stress plays a key role in the pathophysiology of neurodegenerative diseases, particularly ALS, frontotemporal dementias, and Alzheimer’s disease. Aquinnah said it has leveraged these recent findings into a pipeline of novel drugs that hold great promise for treating groups of patients who have few treatment options.

Aquinnah’s small molecule compounds reduce TDP-43 pathology, which is the hallmark characteristic in more than 95 percent of patients with amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease.

Under the terms of the agreement Aquinnah and Roche will collaborate in research activities up to the selection of an advanced drug candidate, after which Roche will be responsible for further research, development, and global commercialization.

“This collaboration with Roche validates our scientific approach and highlights our potential in this area of biology,” said Glenn Larsen, president and CEO of Aquinnah.

Photo: Glenn Larson, president and CEO of Aquinnah 

Author: Rare Daily Staff

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