Regeneron Pharmaceuticals and Avalanche Biotechnologies have entered a collaboration to discover, develop and commercialize gene therapy products for ophthalmologic diseases. The collaboration covers gene therapy vectors and molecules discovered jointly by Avalanche and Regeneron, developed using the Avalanche Ocular BioFactory, an adeno-associated virus (AAV)-based next-generation platform.
 
Avalanche will receive an upfront payment and is eligible to receive as much as $640 million in development and regulatory milestones, as well as royalties on sales. The collaboration includes as many as eight therapeutic targets. Regeneron will have exclusive worldwide rights for each product that advances to clinical development and Avalanche has the option to select two targets to share product development costs and profits. 
 
Regeneron has first negotiation for certain rights to AVA-101, Avalanche’s gene therapy product targeting vascular endothelial growth factor (VEGF) currently in Phase II development for wet age-related macular degeneration (AMD).
 
“We look forward to the opportunity to collaborate with Avalanche, a leader in the field of next-generation gene therapy technologies,” said George D. Yancopoulos, M.D., Ph.D., chief scientific officer of Regeneron and president of Regeneron Labs. “This collaboration highlights the commitment by Regeneron to invest in potentially breakthrough therapies that could benefit patients with sight-threatening diseases.”
 
“We are excited to work with Regeneron to discover and develop novel gene therapy medicines for serious eye diseases,” said Thomas W. Chalberg, Ph.D., co-founder and chief executive officer of Avalanche Biotechnologies. “The collaboration will bring together Avalanche’s novel platform technology with Regeneron’s proprietary molecules and research capabilities, with the goal of creating a new class of next-generation biologics in ophthalmology.”