| Version 2.1.1.0 |
|
|
Abstract
Grant Number: 5R44DK055933-03 PI Name: GREEN, ANTHONY P. PI Email: tony@puresyn.com PI Title: Project Title: Improved Adeno-Associated Virus Purification Abstract: DESCRIPTION (provided by applicant): Puresyn, Inc. has developed purification processes for recombinant adeno-associated virus gene therapy vectors utilizing its proprietary chromatographic resin, PolyFlo. In Phase I, two tandem chromatography processes were developed: heparin affinity and PolyFlo and tandem PolyFlo in two different modes. Each process resulted in product of high purity and recovery that exceeds gradient centrifugation processes. We demonstrated each process removes significant amounts of host and viral contaminants. We are seeking Phase II funding to concentrate on AAV production to include large-scale purification up to 5 x 10e14 particles per run without compromising purity, yield or biological activity. We will refine and expand our current processes to assure they are applicable to AAV serotypes 1, 2 and 5. We will also address the significant problem of AAV aggregation. Finally, we will conduct an in vivo mouse study to determine the correlation between purity, biological activity and safety through the evaluation of immune and histopathological responses to the vector. Results of this research will have immediate impact for those engaged in the use of recombinant AAV vectors for gene delivery because it will allow the large-scale development of AAV vectors, It will have positive commercialization implications for PoIyFlo and Puresyn, Inc. PROPOSED COMMERCIAL APPLICATION: NOT AVAILABLE
Thesaurus Terms:
adeno associated virus group, chromatography, ion exchange chromatography, protein purification, recombinant virus, resin, technology /technique development, transfection /expression vector
gene therapy, microorganism population study, serotyping
high performance liquid chromatography, histopathology, laboratory mouse, western blotting
Institution: PURESYN, INC. 87 GREAT VALLEY PARKWAY MALVERN, PA 19355 Fiscal Year: 2003 Department: Project Start: 01-JUN-1999 Project End: 29-FEB-2004 ICD: NATIONAL INSTITUTE OF DIABETES AND DIGESTIVE AND KIDNEY DISEASES IRG: ZRG1