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

a fast-growing, innovative biotech company in Monrovia, California, has
been awarded a 3.7 million dollar Small Business Innovation Research
(SBIR) Fast-Track grant (#R44NS105156) by the National
Institute of Neurological Disorders and Stroke (NINDS) 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. Read
full press release.

About AcuraStem

AcuraStem is a biotechnology company formed in 2016 to create an
innovative precision medicine platform, iNeuroRX™, which leverages
patient stem cells and advanced machine learning technology to discover
drugs for neurodegenerative diseases. The platform has revealed a novel
ALS target from Dr. Justin Ichida’s lab at USC. AcuraStem is led by an
exceptional team of PhDs, professors, tech entrepreneurs, and successful
drug company veterans employing the latest scientific breakthroughs to
first address one of the most challenging, but tractable diseases,
amyotrophic lateral sclerosis (ALS).

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