Pharma Focus Asia

AcuraStem Receives Fast-Track SBIR Grant for the Development of a Novel Small Molecule Therapy to Treat ALS

Tuesday, June 19, 2018

AcuraStem a fast-growing, innovative biotech company in Monrovia, California, has been awarded a 3.7 million dollar Small Business Innovation Research Fast-Track grant  by the National Institute of Neurological Disorders and Stroke to continue research for the development of a small molecule therapeutic, “AS2015”, focused on treating patients with the genetic form of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) caused by expansion repeats in the gene C9ORF72.

The grant supports an integrated drug discovery approach by AcuraStem to leverage the induced motor neuron cellular models developed at AcuraStem President and Co-Founder, Dr. Justin Ichida's lab at USC. AcuraStem’s innovative precision platform, iNeuroRx™, utilizes nerve cells derived from patient stem cells, in conjunction with artificial intelligence, to predict drug efficacy, and is transforming patient outcomes. By applying this to their cutting edge iNeuroxRx™ platform, AcuraStem can partner with patients and their clinicians to evaluate disease progression and evaluate existing approved therapeutics to identify the best treatments to slow the progression of ALS.

The NINDS SBIR program seeks to help researchers commercialize early-stage scientific discoveries. Small-molecule drugs are easier to administer to patients, are often less expensive, and more easily penetrate the blood brain barrier—a beneficial feature for drugs that treat neurodegenerative diseases. The path for small molecule drug approval is marred with hundreds to thousands of failed programs, so it is believed important to leverage relevant human disease and toxicity models as early as possible during the preclinical drug discovery phases. Dr. Ichida’s human motor neuron assay helps to mitigate some of these obstacles that can hinder new therapeutic progression.

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