The HAVE system uses adeno-associated viruses (AAV), which are naturally-occurring, two-gene virus not known to cause disease in humans, to deliver DNA which can correct the underlying cause of genetic diseases.
The AGTC manufacturing method uses a recombinant herpes simplex virus (HSV) complementation system to produce high-titre, high potency AAV vector stocks.
Under the agreement, financial terms of which were not disclosed, AGTC will help customers engineer AAV-based vectors and associated helper viruses, after which SAFC will use HAVE to manufacture the vectors, either to support early research or at cGMP level for clinical trials and eventual commercial production.
Clients will also have access through this partnership to biomanufacturing testing services from BioReliance, SAFC's biologics services business.
‘Cost-effective’
"AGTC has established an effective and productive relationship with SAFC to manufacture AAV-based vectors for our own portfolio of gene-based product candidates for rare eye diseases," said Sue Washer, President and CEO of AGTC.
"AAV vectors have broad potential in treating a variety of genetic diseases, but until recently, could not be manufactured at clinical and commercial scale in a cost-effective manner. Our proprietary HAVE system overcomes these difficulties.
“In partnering with SAFC, we can now make our AAV manufacturing platform available to academic and corporate organizations that are pursuing gene-based therapies.”
AGTC said it has tested AAV vectors produced with this system in clinical trials involving more than 100 patients in several disease indications.