Partnership Provides Processing and Scale-up for AAV Vectors

Best-in-class production process technology will advance Avalanche's robust clinical program and be available to third parties through Lonza, a premier contract manufacturer of cell and viral-based therapeutics.

Companies announced a manufacturing collaboration focused on process development and scale-up efforts for the manufacturing of adeno-associated viral (AAV) vectors for gene therapy. AAV vectors are rapidly becoming one of the most promising gene delivery vehicles for the treatment of various diseases including inherited retinal disorders, age-related macular degeneration (AMD), hemophilia B, congestive heart failure, Parkinson's disease and others.

As part of the agreement, Avalanche and Lonza will make the technology available to third parties and share in the revenue. The Avalanche/Lonza manufacturing collaboration will focus on the development and high-yield production of AAV vectors based on a novel technology that uses stable baculovirus. This technology was licensed by Avalanche from Virovek, who will also play a key role in the collaboration.

"We are delighted to have such a skilled and reputable manufacturing partner in Lonza to help us advance our robust research and development programs using AAV to treat macular degeneration and other blinding diseases," said Thomas W. Chalberg, Ph.D., chief executive officer, Avalanche Biotechnologies, Inc. "By making our combined technology available to third parties, we hope to allow the entire AAV field to benefit from this best-in-class production process."

"Lonza's viral-based therapeutic business specializes in the development, GMP production, and fill and finish of multiple classes of viral vaccines and viral vector-delivered therapeutics," said David Enloe, Lonza's Head of Viral-based Therapeutics. "With our recent expansion of additional GMP suites that will increase working volumes up to 2,000L, we are poised for the growth we have seen in this sector. We look forward to partnering with Avalanche in this exciting collaboration and we are pleased to be able to add this novel, scalable method for AAV production to our capabilities."

AAV is a naturally-occurring virus that is not associated with any disease in humans. AAV-based vectors efficiently deliver genes or RNAi to numerous cell types at up to 100 percent efficiency. They are considered highly effective due to their high safety profile, lack of toxicity and ability to efficiently transduce and achieve long-term expression without integration in a wide range of tissues.