Sangamo will receive more than 120 issued, pending or in-licensed patents that include the AAV vector platform and manufacturing methods, therapeutic transgenes, and technology for direct administration of AAV to the brain. Sangamo will also have access to GMP master cell banks, materials and manufacturing know-how, as well as a database of preclinical efficacy and toxicology studies and other documentation supporting Ceregene's IND applications. Sangamo will also acquire all of Ceregene's preclinical and clinical therapeutic programs including its ongoing Phase II trial evaluating NGF-AAV (CERE-110) in Alzheimer's disease, and the proprietary needle device for brain delivery of AAV with supporting regulatory and clinical documentation.
"Ceregene is a leader in development and manufacturing of AAV-based therapies with significant clinical development experience," said Edward Lanphier, Sangamo's president and chief executive officer. "Since their inception in 2001, the company has safely treated over 115 subjects in four clinical trials. Sangamo has acquired all of Ceregene's AAV assets including CERE-110, AAV delivery of nerve growth factor (NGF) to the brain for the treatment of Alzheimer's disease. CERE-110 is being evaluated in a fully enrolled and fully funded Phase 2 clinical trial. In addition to the AAV platform, the assets also include one of the world's largest databases of AAV GMP manufacturing know-how, toxicology data, and safety data from their human clinical trials, which will be an invaluable resource as we advance our ZFP Therapeutics."
"Over 5 million people in the U.S. live with Alzheimer's, which is a devastating disease for both patients and their families, and more effective treatment options are needed," said Geoff Nichol, M.B. Ch.B., Sangamo's executive vice president of research and development. "Early data from the CERE-110 program are encouraging. Clinical data from a Phase 1 study demonstrate that CERE-110 can be safely injected into a specific area of the brain and preclinical studies suggest that treatment can produce levels of NGF that protect cholinergic neurons. The award granted by the NIH to fund the Phase 2 trial, and participation of the ADCS, a pre-eminent research consortium for testing new treatments for Alzheimer's, demonstrate strong support for the development of this novel therapeutic."