MicroRNAs (miRNAs) for the Treatment of Neurological Disorders


Oligonucleotides that target Kv4.2 miRNAs for the upregulation of Kv4.2 protein in neurological disorders.

Key Benefits

  • Increases the abundance of the neuro-protective protein, Kv4.2.
  • Offers a potential treatment for neurological diseases, including Fragile X syndrome, autism spectrum disorders, epilepsy, and Alzheimer's disease.

Market Summary

Millions of people are living with neurological disorders in the US. These disorders include Fragile X Syndrome, autism spectrum disorders, epilepsy, and Alzheimer’s disease. Currently, the vast majority of available pharmacological treatments for these disorders rely on symptom management rather targeted disease therapies. There are currently no miRNA-based drugs on the market; however there are, or have been, many miRNA clinical trials progressing for ocular and retinal disorders, cancer, kidney disease, and antiviral therapies; unfortunately none of the potential therapeutics are in trials for neurological disorders.

Technical Summary

Neuronal excitability is tightly regulated, and defects in mechanisms involved in this regulation can lead to neurological disorders. A key player in the control of neuronal excitability in the brain is the A-type potassium channel Kv4.2. This potassium channel controls excitatory currents in the hippocampus and is thus critical to maintain a healthy excitatory balance in the brain. Emerging data suggests that Kv4.2 protein levels are dysregulated in a variety of disease states. Emory researchers have identified a microRNA that reduces Kv4.2 protein levels. An antagonist oligonucleotide, or antagomir, against this microRNA increases Kv4.2 protein levels in cultured neurons and induces an increase in cell survival after excitotoxicity-inducing treatments.

Developmental Stage

  • Antagomir can successfully rescue Kv4.2 protein levels and increase cell survival after excitotoxicity-inducing treatments in vitro.
  • Proof of principal experiments in mouse models of neurological disease have been initiated.

Patent Information

App Type Country Serial No. Patent No. File Date Issued Date Patent Status
Utility (parent) United States 14/474,965 9,458,458 9/2/2014 10/4/2016 Issued
Continuation United States 15/243,604 9,932,585 8/22/2016 4/3/2018 Issued
Tech ID: 12098
Published: 1/3/2014