Scalable Purification of AAV2, AAV4 or AAV5 Using Ion-Exchange Chromatography
Description of Invention:
Adeno-associated viruses (AAVs) constitute, as a group, the vehicle of choice for gene therapy because of several attractive features. Among others, AAVs are less pathogenic than other viruses, and they can be used for the long-term expression of therapeutic genes.
This invention describes a simple ion-exchange (HPLC) methodology to purify different AAV serotypes. The protocol, which can be readily scaled up, details the efficient concentration of fully infective AAV particles, and is applicable to a number of promising serotypes for which efficient purification methodologies are currently lacking. Significantly, the method consistently produces higher infectivity per particle ratios than standard methods.
This invention, coupled with NIH invention E-325-2001, entitled "Highly Scalable Production of AAV in Insect Cells," would give a licensee a purification system that can be readily scaled-up to efficiently produce recombinant adeno-associated viruses for clinical trial development.
Inventors:
Nikola Kaludov and John Chiorini (NIDCR)
Related Technologies: DHHS Reference No. E-325-2001/1 --
U.S. Patent No. 6,723,551 issued 20 Apr 2004, titled "Production of Adeno-Associated Virus in Insect Cells"
DHHS Reference No. E-325-2001/2 --
U.S. Patent No. 7,271,002 issued 18 Sep 2007, titled "Production of Adeno-Associated Virus in Insect Cells"
Relevant Publication: N Kaludov et al. Scalable purification of adeno-associated virus type 2, 4, or 5 using ion-exchange chromatography. Hum Gene Ther. 2002 Jul 1;13(10):1235-1243. [PubMed abs]
Portfolios: Gene Based Therapies
Gene Based Therapies -Therapeutics-Gene Therapy-Other Gene Based Therapies -Therapeutics
For Additional Information Please Contact: Betty Tong Ph.D.
NIH Office of Technology Transfer
6011 Executive Blvd, Suite 325
Rockville, MD 20852-3804
Phone: 301/594-6565
Email: tongb@mail.nih.gov
Fax: 301/402-0220
