AAV

Our innovative delivery system

Adeno-associated virus (AAV) is a small virus that infects humans but does not cause disease.  It is uniquely suited for gene therapy application – because it cannot replicate on its own, it is unlikely to spread within the body. Human clinical trials have consistently found AAV to be safe. The same clinical trials have found that vectors (gene delivery vehicles based on AAV) are capable of causing long-term therapeutic protein expression in humans, meaning long-lasting treatment for patients.  Therefore, AAV vectors have advantages compared to many other viral and non-viral based gene delivery platforms in both function and safety for correcting genetic-based diseases.

AGTC has developed an AAV vector manufacturing platform comprised of a proprietary, patented production method and proven downstream purification operations integrated into a GMP-compliant format capable of AAV vector generation at a commercial scale.  The manufacturing platform is flexible and efficient, accommodating multiple genetic targets and AAV variants, or serotypes, while producing vector in sufficient quantity for treatment of both confined-space and systemic disease candidates.  AGTC is currently partnering with SAFC Pharma to make its proprietary production system available to potential academic and corporate partners.

Related Publications

Efficient Clearance of HSV Using a GMP-compliant Method for Production of rAAV Vectors 

HSV clearance during purification of a rAAV serotype 1 vector. 

An efficient rHSV-based complementation system for the production of multiple rAAV vector serotypes. 

Scalable recombinant adeno-associated virus production using recombinant herpes simplex virus type 1 coinfection of suspension-adapted mammalian cells. 

Large-scale adeno-associated viral vector production using a herpesvirus-based system enables manufacturing for clinical studies. 

Bioreactor production of recombinant herpes simplex virus vectors. 

Clearance and characterization of residual HSV DNA in recombinant adeno-associated virus produced by an HSV complementation system. 

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Alachua – Headquarters
14193 NW 119th Terrace, Suite #10
Alachua, Florida 32615
386.462.2204
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Cambridge
One Kendall Square, 1400W, Suite B14305
Cambridge, MA 02139
617.843.5728
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