Small Molecules Targeting Protein-RNA Interactions in ALS and Related Neurodegenerative Diseases

Case ID:

This invention is an in silico molecule capable of mitigating locomotor dysfunctions through TDP-43 contained RNA remodeling.


Amyotrophic Lateral Sclerosis (ALS) is a neurodegenerative disease that affects the function of nerves and muscles. In the United States over 6,000 people are diagnosed each year with an estimated 15 cases diagnosed per day. Current treatments require drugs like riluzole and radicava. Riluzole is a drug that promises to slow the progress of ALS, giving patients more time to function. Riluzole also has minimal effects regarding survival. Radicava is similar to riluzole in that it improves the speed in which the slowing progress begins. There still remains no novel treatment to challenge the long-term survival of patients treated with ALS. This invention seeks to contest current ALS drugs as it aims to develop in vivo compounds to aid therapeutic development of ALS.



  • ALS therapeutics
  • Toxicity studies



  • Restoration of sequestered RNA targets
  • Increases lifespan in Drosophila model
  • Exhibits neuroprotective potential in ALS files
  • Mitigates TDP-43 mediated toxicity
  • Molecules include 4-aminoquinoline analog
Patent Information:
Contact For More Information:
Mitch Graffeo
Sr. Licensing Manager - COM-T
The University of Arizona
Lead Inventor(s):
Daniela Zarnescu
May Khanna
Vijay Gokhale