Genzyme Corporation and Applied Genetic Technologies (AGTC), a private development-stage biotechnology company, have entered into a research collaboration to jointly develop novel therapeutics involving gene therapy.

Through the collaboration, Genzyme gains access to AGTC's extensive expertise using Adeno-Associated Virus (AAV) vectors to deliver genes to patients, including access to AGTC's novel high-yield manufacturing capabilities for AAV vectors. These vectors may have therapeutic advantages in several disease areas where Genzyme has active preclinical development programs, including lysosomal storage disorders, cardiovascular disease, central nervous system disorders, immune-mediated disorders, and others.

"We are delighted to collaborate with an established biotechnology leader like Genzyme, which has contributed heavily to many advances in our understanding of gene therapy," said Sue Washer, CEO of AGTC. "This agreement is an important validation of AGTC's technology, and will position us to build on our early work in a number of therapeutic areas and enable us to bring products to market more efficiently," added Sue.

"Our work with AGTC will bring Genzyme an impressive technology platform, which includes a scalable, highly productive manufacturing capability with the potential to support future clinical trials and product development," said Sam Wadsworth, vice president, Translational Research at Genzyme.

AGTC has licensed a significant portion of its intellectual property from the University of Florida where researchers originated this ground-breaking work in gene therapy. AGTC is developing novel therapeutics for patients with unmet medical needs utilizing the non-pathogenic adeno-associated virus.