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Improving Obesity Outcomes Through Interactive Web-Based Clinical Skills…

Award Information

Agency:
Department of Health and Human Services
Branch:
N/A
Award ID:
Program Year/Program:
2013 / SBIR
Agency Tracking Number:
R43GM102972
Solicitation Year:
2013
Solicitation Topic Code:
NIGMS
Solicitation Number:
PA12-088
Small Business Information
NATURE TECHNOLOGY CORPORATION
4701 INNOVATION DRIVE LINCOLN, NE 68521-
View profile »
Woman-Owned: No
Minority-Owned: No
HUBZone-Owned: No
 
Phase 1
Fiscal Year: 2013
Title: Improving Obesity Outcomes Through Interactive Web-Based Clinical Skills Training
Agency: HHS
Contract: 1R43GM102972-01A1
Award Amount: $247,934.00
 

Abstract:

DESCRIPTION (provided by applicant): To commercialize non-viral gene medicines, it is critical that both vector potency (i.e. therapeutic transgene expression levels) and the duration of the therapeutic effect be improved. Potent dose-sparing extended duration gene therapies will have a cost and efficacy competitive advantage over alternative technologies. In this Phase I proof of concept study, we will create a novel antibiotic-free MiniPlasmid gene therapy platform for extended duration gene therapy. Thevectors combine transient expression enhancers that improve transgene expression levels with a novel 270 base pair replication origin-antibiotic free selection cassette that we hypothesize will promote long duration gene expression after vector delivery tothe body. The MiniPlasmid platform will be applied to create a wound healing gene therapy product to treat diabetic neuropathic foot ulcers. In Specific Aims 1 and 2 a high yielding MiniPlasmid fermentation manufacturing platform is created. In SpecificAim 3 the MiniPlasmid vector platform is validated in vivo for extended duration expression compared to conventional plasmids. A hypoxia- inducible factor 1 (HIF-1 ) based gene medicine for diabetic foot ulcer treatment is developed utilizing an extendedhalf-life oxygen resistant highly active HIF-1 mutant (CA5-HIF-1 ). Specific Aim 3 is performed in collaboration with wound healing gene therapy expert Dr. John Harmon at Johns Hopkins University. The MiniPlasmid vector platform is designed to improvetransgene expression levels and duration to enable gene medicine development for multiple applications requiring extended duration expression. MiniPlasmid vectors developed in Phase I will be marketed to investigators for a variety of gene therapy applications through publications, trade shows, and the Nature Technology Corporation (NTC) website. In Phase II the HIF-1 MiniPlasmid gene therapeutic will undergo preclinical safety and efficacy evaluations for treatment of diabetic foot ulcers prior to clinical development in Phase III. PUBLIC HEALTH RELEVANCE PUBLIC HEALTH RELEVANCE: The objective of this proposal is to develop a novel antibiotic-free non-viral MiniPlasmid gene therapy platform for extended duration gene therapy, and as such is responsive to NIGMS SBIR high-priority area of interest in development of improved vectors for gene transfer. The vectors combine transient expression enhancers that improve transgene expression levels with a novel replication origin-antibiotic free selectioncassette that affords long duration gene expression after gene delivery to the body. The platform will be applied to create a wound healing gene therapy product to treat diabetic neuropathic foot ulcers.

Principal Investigator:

James Williams
402-472-6530
jim@natx.com

Business Contact:

James A. Williams
402-323-6289
jim@natx.com
Small Business Information at Submission:

NATURE TECHNOLOGY CORPORATION
4701 INNOVATION DRIVE LINCOLN, NE 68521-

EIN/Tax ID: 134161210
DUNS: N/A
Number of Employees: N/A
Woman-Owned: No
Minority-Owned: No
HUBZone-Owned: No