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Grant Awarded to Company for Kennedy’s Disease Research

Neuron illustration (NIH)

(National Institute on Aging, NIH)

AndroScience Corp., a drug discovery and development company in San Diego, California, said it received a $3.8 million translational research grant to help develop a treatment for spinal and bulbar muscular atrophy, also called Kennedy’s Disease. The National Institute of Neurological Disorders and Stroke (NINDS) of the National Institutes of Health awarded the grant.

The company says it will conduct the research as part of a joint effort with the Neurogenetics Branch of the NINDS to develop an oral drug treatment for Kennedy’s Disease. The disease is a rare hereditary neurodegenerative condition that currently has no approved drug available to patients. It results in progressive motor neuropathy and androgen insensitivity syndrome — inability of genetic males to respond to androgen male sex hormones — caused by a mutation in the androgen receptor (AR) gene.

AndroScience anticipates using its platform of therapeutic small molecule drugs that enhance degradation of the AR protein, termed AR degradation (ARD) enhancers enhancers. The project is expected to first validate an orally administered ARD enhancer drug in a transgenic animal model. The next stage will complete preclinical toxicology, safety pharmacology, and absorption-distribution-metabolism-elimination (ADME) tests needed to file an investigational new drug application for human clinical studies.

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