Application

Small‑molecule c‑Abl inhibitors designed to treat neurodegenerative diseases such as Parkinson’s and Alzheimer’s.

Key Benefits

• Targets c‑Abl, a key regulator linked to neurodegeneration, oxidative stress, and loss of dopaminergic neurons.
• Provides new small‑molecule scaffolds with potential for improved safety profiles over existing c‑Abl inhibitors.
• Supports oral pharmaceutical formulations suitable for chronic administration.

Market Summary

Neurodegenerative diseases—including Parkinson’s disease and Alzheimer’s disease—represent a major and rapidly expanding global health burden due to aging populations. Parkinson’s alone affects a significant percentage of adults over 65 and leads to progressive motor decline, cognitive impairment, and reduced quality of life. Current treatments primarily offer symptomatic relief and do not slow or reverse the underlying disease processes. Growing evidence links oxidative stress, mitochondrial dysfunction, and dysregulated c Abl signaling to the progression of these disorders. Existing c Abl inhibitors show potential but face limitations such as cardiotoxicity or poor long term tolerability. There is a critical need for safer, more targeted therapeutics capable of addressing the molecular mechanisms driving neurodegeneration.

Technical Summary

Emory researchers developed a series of small molecule compounds that inhibit the Abelson non tyrosine kinase (c Abl) for the treatment of neurological disorders. The patent discloses multiple chemical entities and their pharmaceutically acceptable salts, along with formulations suitable for oral delivery, including tablets, capsules, pills, gels, and granules. These compounds are intended for managing neurodegenerative diseases such as Parkinson’s and Alzheimer’s by targeting aberrant c Abl signaling associated with oxidative stress, dopaminergic neuron loss, and impaired mitochondrial quality control. Methods of treatment include administering the disclosed compounds alone or as part of pharmaceutical compositions to subjects diagnosed with c Abl related neurodegenerative conditions.

Development Stage

The technology is in the preclinical stage with active lead optimization of the c Abl–inhibiting small molecule compounds.

Publication ACS Med Chem Lett. 2020 Mar 12;11(4):491-496. doi: 10.1021/acsmedchemlett.9b00612.