Scalable Purification of AAV2, AAV4 or AAV5 Using Ion-Exchange Chromatography
Posted Oct 31 2008 5:00pm
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 (NIDCR) John A Chiorini (NIDCR)
Related Technologies: US, Patent No. 6,723,551, Issued 20 Apr 2004, Reference No. E-325-2001/1 ("Production of Adeno-Associated Virus in Insect Cells") US, Patent No. 7,271,002, Issued 18 Sep 2007, Reference No. E-325-2001/2 ("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
For Additional Information Please Contact: Patrick McCue Ph.D. NIH Office of Technology Transfer 6011 Executive Blvd. Suite 325, Rockville, MD 20852 United States Email: McCuepat@mail.nih.gov Phone: 301-496-7057 Fax: 301-402-0220
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 (NIDCR)
John A Chiorini (NIDCR)
Patent Status:
HHS, Reference No. E-308-2001/0
US, , Patent No. 7,419,817, Issued 02 Sep 2008
US, Application No. 60/381,180 filed 17 May 2002
Related Technologies:
US, Patent No. 6,723,551, Issued 20 Apr 2004, Reference No. E-325-2001/1 ("Production of Adeno-Associated Virus in Insect Cells")
US, Patent No. 7,271,002, Issued 18 Sep 2007, Reference No. E-325-2001/2 ("Production of Adeno-Associated Virus in Insect Cells")
Relevant Publication:
Portfolios:
Gene Based Therapies
Gene Based Therapies - Therapeutics
For Additional Information Please Contact:
Patrick McCue Ph.D.
NIH Office of Technology Transfer
6011 Executive Blvd. Suite 325,
Rockville, MD 20852
United States
Email: McCuepat@mail.nih.gov
Phone: 301-496-7057
Fax: 301-402-0220
Ref No: 1839
Updated: 11/2008