Gene Therapy Collaboration

Genzyme Corporation and Applied Genetic Technologies (AGTC), a private development-stage biotechnology company, announced today that they 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.

Genzyme brings to the collaboration more than a decade of experience with gene therapy, involving multiple clinical trials in cardiovascular disease, oncology, and cystic fibrosis. Financial terms were not disclosed.

"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."

"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. AGTC's first product candidate is a treatment for Alpha One Antitrypsin Deficiency, an inherited form of emphysema; Phase I trials are underway with a potential product launch in early 2009. AGTC's investors include Interwest Partners (Menlo Park, California), Intersouth Partners (Durham, North Carolina), MedImmune Ventures (Gaithersburg, Maryland) and Skyline Ventures (Palo Alto, California). The company is located in Gainesville, Florida in the University of Florida's business development park.

Genzyme Corporation is a global biotechnology company dedicated to making a major positive impact on the lives of people with serious diseases. The company's broad product portfolio is focused on rare genetic disorders, renal disease, osteoarthritis and immune-mediated diseases, and includes an industry-leading array of diagnostic products and services. Genzyme's commitment to innovation continues today with research into novel approaches to cancer, heart disease, and other areas of unmet medical need. More than 7,000 Genzyme employees in offices around the globe serve patients in over 80 countries.

© 2002-2004 Genzyme Corporation.

Genzyme Corporation: http://www.genzyme.com/
Applied Genetic Technologies, Corp.: http://www.agtcfl.com/

Message posted by: Frank S. Zollmann