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Regenerative Therapy for Corneal Endothelial Dystrophies and Injuries

Award Information
Agency: Department of Health and Human Services
Branch: National Institutes of Health
Contract: 1R42EY031196-01
Agency Tracking Number: R42EY031196
Amount: $300,000.00
Phase: Phase I
Program: STTR
Solicitation Topic Code: N
Solicitation Number: PA18-575
Solicitation Year: 2018
Award Year: 2020
Award Start Date (Proposal Award Date): 2020-09-30
Award End Date (Contract End Date): 2021-09-29
Small Business Information
Columbus, OH 43214-2120
United States
DUNS: 078687051
HUBZone Owned: No
Woman Owned: No
Socially and Economically Disadvantaged: No
Principal Investigator
 (806) 414-9623
Business Contact
Phone: (614) 579-9520
Research Institution
3601 4TH STREET - MS 6271
LUBBOCK, TX 79430-6271
United States

 Nonprofit College or University

Worldwide, only 1 out of 70 patients with corneal endothelial dystrophies and injuries receive a corneal
transplant due to the limited corneal donor pool, the need for advanced surgical facilities and talent, and
expense. RashmiVu is developing a stem cell based regenerative technology that addresses this large unmet
global need through a paradigm shift, wherein treatment could be as simple as an intracameral injection of
stem cells as an outpatient procedure. RashmiVu has repeatedly demonstrated successful isolation and
expansion of human corneal endothelial stem cells (hCESCs) into millions, confirmed that these cells are
hCESCs by analysis of phenotypic markers, and demonstrated their regenerative potential in preliminary
animal studies in both mice and rabbits. This Fast-Track STTR seeks to complete development and
demonstrate potential products including (a) a single cell suspension for intracameral injection, (b) a single cell
formulation that can be “painted” onto the corneal endothelium, and (c andamp; d) sheets of hCESCs and/or their
differentiated mature corneal endothelial phenotypes for use in DSEK and DMEK surgeries.
Phase I Specific Aims: 1. Cell Yield: Determine tissue procurement inclusion/exclusion criteria that reliably
yield 5M+ hCESCs/donor eye, and optimize differentiation to maximize yield of mature CEC phenotypes; 2.
Definitive healing: Rapid innate healing in rabbits obfuscates effects of hCESCs (see Prelim Data). The team
will complete development of a slow-healing cryoinjury rabbit model and demonstrate definitive hCESC
efficacy; 3. Mechanisms: Determine mechanisms potentiated by or convertible to a pharmaceutical pathway.
Phase II Specific Aims: 4. Cell suspension formulations: Optimize cell suspensions for injection and targeted
delivery including ROCKi pre-treatment, and optimize media and methods for high cell survival after freezing,
storage, thawing, and revival; 5. Cell Sheets: Develop monolayer sheets of hCESCs on transplantable
substrates, develop a method for consistent differentiation of the hCESC monolayer into a mature CEC
monolayer on these sheets, and demonstrate successful partial thickness transplantation (DSEK) in rabbits; 6.
Dosage, Safety andamp; Efficacy: Initiate FDA-recommended pre-clinical trials to demonstrate safety, efficacy,
effectiveness of targeted delivery approaches including dose response, cell survival, engraftment, distribution,
migration, proliferation, tumorigenicity, host immune response, and cellular toxicities.
A successful regenerative treatment for corneal endothelial dystrophies and damages could dramatically
reduce health care costs, eliminate blindness, and return people to fully functioning members of society, all
with an attractive market opportunity.PUBLIC HEALTH RELEVANCE
Diseases and trauma of the corneal endothelium lead to about 45k corneal transplants per year in the US.
Worldwide, only 1 out of 70 patients receive needed transplants due to the limited corneal donor pool and the
need for advanced surgical facilities and talent. RashmiVu, LLC, and its collaborators are developing a plentiful
source of human corneal endothelial precursor stem cells that could lead to a lower cost, globally available
treatment for corneal endothelial dystrophies and damages, and potentially eliminate most corneal transplants
for corneal endothelial dystrophies and damaged endothelium.

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

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