ANTI-STATHMIN BIFUNCTIONAL SMALL HAIRPIN RNA: PRECLINICAL DEVE

Award Information
Agency:
Department of Health and Human Services
Branch
n/a
Amount:
$199,045.00
Award Year:
2010
Program:
SBIR
Phase:
Phase I
Contract:
N43CO201000108
Award Id:
96599
Agency Tracking Number:
N43CO201000108
Solicitation Year:
n/a
Solicitation Topic Code:
NCI
Solicitation Number:
n/a
Small Business Information
1717 MAIN STREET, 60TH FLOOR, DALLAS, TX, 75201-
Hubzone Owned:
N
Minority Owned:
N
Woman Owned:
N
Duns:
610368925
Principal Investigator:
ALEX TONG
Business Contact:
DAVE SHANAHAN
(214) 220-4303
Research Institute:
n/a
Abstract

The three basic requirements for an effective RNA interference-based therapy are 1) identification of key genetic defect[s] that provide pivotal survival advantage(s) to the overall cancer cell population; 2) construction of a potent and durable target-specific therapeutic agent; and 3) deployment of a tumor selective, non-immunogenic, RES-stealthed delivery vehicle. Stathmin 1 (STMN1), a critical regulator of tubulin polymerization/depolymerization, is overexpressed in 86% of our patients. We have constructed a unique bi-functional shRNA (bi-sh) with enhanced siRNA and miRNA activities. Bi-shSTMN1 is packaged as a decorated, reversibly masked, bilamellar invaginated lipoplex shorn of impurities through the use of a novel SuperClean DNA process. Our technical objective is to integrate this proprietary SuperClean DNA technology to extend the systemic therapeutic window of bi-shSTMN1. We will manufacture GMP grade SuperClean bi-shSTMN1 plasmid DNA and establish the systemic maximum tolerated dose of this lipoplex. By incorporating treatment controls of similarly-prepared parental GMP plasmid DNA, we can also identify toxicity that may be attributed to bi-shSTMN1 expression. These findings, together with the completion of our dose escalation Phase I protocol, will culminate in a phase I/II clinical study to determine safety and efficacy of systemic targeted delivery of BIV-bi-shSTMN1 with docetaxel in advanced melanoma.

* information listed above is at the time of submission.

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