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  • Mass General Brigham Ventures

Vertex Pharmaceuticals and Affinia Therapeutics Establish Multi-Year Collaboration

Affinia Therapeutics’ proprietary AAV vector technology to be used in Vertex’s genetic therapy efforts with focus on Duchenne muscular dystrophy, myotonic dystrophy type 1 and cystic fibrosis

BOSTON & WALTHAM, Mass.--(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq:VRTX) and Affinia Therapeutics announced today that the two companies have entered into a strategic research collaboration to engineer novel adeno-associated virus (AAV) capsids to deliver transformative genetic therapies to people with serious diseases. Affinia Therapeutics’ proprietary AAVSmartLibrary and associated technology provides capsids for improved tissue tropism, manufacturability and pre-existing immunity. The collaboration will leverage Affinia Therapeutics’ capsid engineering expertise and Vertex’s scientific, clinical and regulatory capabilities to accelerate the development of genetic therapies for people affected by Duchenne muscular dystrophy (DMD), myotonic dystrophy type 1 (DM1) and cystic fibrosis (CF). “This collaboration with Affinia Therapeutics will enhance our existing capabilities in discovering and developing transformative therapies for people with serious diseases,” said Bastiano Sanna, Executive Vice President and Chief of Cell and Genetic Therapies at Vertex. “Affinia Therapeutics’ innovative approach to the discovery and design of AAV capsids brings yet another tool to our Vertex Cell and Genetic Therapies toolkit, and we’re excited to partner with them to bring together their technology platform with our research and development expertise.” “At Affinia Therapeutics, we’re setting a new standard in genetic therapy by leveraging our platform to methodically engineer novel AAV vectors that have unique therapeutic properties,” said Rick Modi, Chief Executive Officer. “Vertex is an established leader in developing transformative medicines for genetic diseases and renowned for its scientific rigor. We are thankful for the scientific validation this partnership brings and look forward to working closely with them to advance life-changing, differentiated genetic therapies and make a meaningful difference to those affected by these diseases.”

About the Collaboration Under the terms of the agreement, Affinia Therapeutics will apply its vector design and engineering technologies to develop novel capsids with improved properties. The agreement provides Vertex an exclusive license under Affinia Therapeutics’ proprietary technology and intellectual property (IP) in DMD and DM1 with an exclusive option to license rights for CF and an additional undisclosed disease. The scope of the agreement covers all genetic therapy modalities in these diseases. Affinia Therapeutics will be eligible to receive over $1.6 billion in upfront and development, regulatory and commercial milestones, including $80 million in upfront payments and research milestones that will be paid during the research term, plus tiered royalties on future net global sales on any products that result from the collaboration. Affinia Therapeutics will be responsible for the discovery of capsids that meet certain pre-determined criteria. Vertex will be responsible for and will fund the design and manufacturing of genetic therapies incorporating the selected capsids, preclinical and clinical development efforts, and commercialization of any approved products in the licensed diseases.


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