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Neurxtem Neural Organoid Human Platform Development for Substance and Opioid Use Disorders

Award Information
Agency: Department of Health and Human Services
Branch: National Institutes of Health
Contract: 4R44DA051281-02
Agency Tracking Number: R44DA051281
Amount: $1,516,917.00
Phase: Phase II
Program: SBIR
Solicitation Topic Code: NIDA
Solicitation Number: DA19-019
Solicitation Year: 2019
Award Year: 2021
Award Start Date (Proposal Award Date): 2021-02-15
Award End Date (Contract End Date): 2023-01-31
Small Business Information
Columbus, OH 43212-1180
United States
DUNS: 079883378
HUBZone Owned: No
Woman Owned: No
Socially and Economically Disadvantaged: No
Principal Investigator
 (614) 292-1380
Business Contact
Phone: (985) 788-0994
Research Institution

This is a SBIR proposal from Neurxstem Inc., a company selected by NCET2 as a “Best University
Startups 2016” is in response to the NIH HEAL initiative. Neurxstem’s goals are to develop and
optimize a proprietary induced pluripotent stem cell derived human Neural Organoid Platform
(NNOP) to develop 1) an early diagnostic NNOP for substance (SUD) and opioid (OUD) use
disorder susceptibility and 2) to identify early therapeutic targets for future screens for effective
SUD/OUD disease-related drug discovery, efficacy and safety screening. The current state of the
art technology for such goals ranges from human primary and immortalized cell lines to rodent
models. Because these models are poor surrogates for the complex network structure of the
human brain, the proposed platform is expected to fill a crucial gap in translational neuroscience
goals to understand and treat human brain disorders. Neurxstem has developed the proprietary
methodology to make nearly complete human neural organoids and has shown that a superior
NNOP compared to other ‘organoids’ that has all the major parts of the brain – retina, cortex,
midbrain, hindbrain, and spinal cord - in a single, networked and scalable NNOP that also
expresses all the major cell types including the microglia critically involved in SUD and exhibits
responses to a drug of addiction that alters a multitude of biomarkers linked to multiple drugs of
abuse. Neurxstem, a small business founded by Dr. Rene Anand, one of the technology
inventors, proposes to further develop this clinically corroborated model system to test its
reliability, reproducibility, robustness and utility for commercial translational neuroscience
applications for SUD/OUD and initiate market entry after FDA approval. Phase I Aims: The
Phase I goals are to identify early therapeutic targets by: 1) mathematical modeling of gene
expression data responses to drugs of addiction to deduce early biomarkers for SUD/OUD. Phase
II Aims: One goal is identify all possible early therapeutic targets by mathematical modeling from
multiple data points and drug exposure samples for future SUD/OUD countermeasure
development with Pharma. A second goal is to scale and validate the platform with multiple SUD
patient iPSC lines to demonstrate: a) reliability, b) reproducibility and c) robustness metrics
needed to demonstrate the full utility for early diagnostic and drug discovery, efficacy and safety
testing commercial goals for SUD/OUD. Additionally, Neuxstem’s commercialization plan
includes 1) market feedback from prospective clients for product validation; 2) initial market entry
with beta customers/ business partners; and 3) continuing intellectual property development and
prosecution. Fundamentally, the further development and validation of Neurxstem’s NNOP
should provide a lower cost, more precise and accurate system for personalized, predictive
diagnostic medicine and development of countermeasure for SUD/OUD.

* Information listed above is at the time of submission. *

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