The proposed research is intended to develop a large scale cell process and purification system for the production of recombinant AAV-CFTR vector. A suspension adapted packaging cell line will be constructed. This cell line will be used to develop a large scale batch suspension process utilizing a temperature-sensitive adenovirus mutant for bulk vector production. Vector recovery and purification methods will be optimized for large scale clinical manufacture. Formulation and drug delivery development will be initiated with purified vector. Clinical production of large quantities of AAV-CHR vector will be required for mid- and late-stage trials in patients with cystic fibrosis. Development of efficient and large scale methods to produce AAV vectors could also lead to new products for the treatment of other diseases in addition to cystic fibrosis.

Proposed Commercial Applications

Development of AAV-CFTR vectors for the treatment of cystic fibrosis could result in a gene delivery """"""""drug"""""""" that could slow or halt disease progression. This product would serve an existing market of 30,000 affected individuals. In addition, this AAV vector production technology would be of utility in other gene transfer applications, including treatment of genetic disease, cardiovascular disease, and cancer.

Agency
National Institute of Health (NIH)
Institute
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Type
Small Business Innovation Research Grants (SBIR) - Phase II (R44)
Project #
5R44DK049460-03
Application #
2518464
Study Section
Special Emphasis Panel (ZRG2-SSS-Y (09))
Program Officer
Mckeon, Catherine T
Project Start
1995-02-01
Project End
1998-08-31
Budget Start
1997-09-01
Budget End
1998-08-31
Support Year
3
Fiscal Year
1997
Total Cost
Indirect Cost
Name
Targeted Genetics Corporation
Department
Type
DUNS #
City
Seattle
State
WA
Country
United States
Zip Code
98101