Reports from the US indicate that more than 8 million people in this country are currently battling or have had cancer. Replicon Technologies, Incorporated (RTI) has developed a unique gene therapy strategy based on RNA vectors (replicons) with the potential to overcome limitations in the treatment of malignancies of the CNS, including primary cancers of the brain. In the Phase I project, we used a xenogeneic intracranial glioma mouse model to evaluate the feasibility of replicons for the treatment of malignant brain tumors. We have demonstrated that the treatment of tumor-bearing mice with replicons results in a significant enhancement of survival. In the Phase II project, RTI will optimize manufacturing protocols which will be suitable for large-scale production under GMP conditions. Methods for quality control testing wil also be developed. These parameters will be implemented in a pilot feasibility study with a viral vector manufacturing company for small-scale production of GMP-quality replicon preparations that will then be evaluated for safety and toxicity in two appropriate animal species. Data generated from these studies will be used to support an Investigational New Drug (IND) application filing with the FDA to allow for a Phase I clinical study of the replicons.

Proposed Commercial Applications

Not Available

Agency
National Institute of Health (NIH)
Institute
National Cancer Institute (NCI)
Type
Small Business Innovation Research Grants (SBIR) - Phase II (R44)
Project #
5R44CA079355-03
Application #
6513189
Study Section
Special Emphasis Panel (ZRG1-SSS-1 (10))
Program Officer
Wolpert, Mary K
Project Start
1998-09-21
Project End
2003-12-31
Budget Start
2002-04-18
Budget End
2002-12-31
Support Year
3
Fiscal Year
2002
Total Cost
$224,703
Indirect Cost
Name
Replicon Technologies, Inc.
Department
Type
DUNS #
City
Birmingham
State
AL
Country
United States
Zip Code
35223