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Vertex and Affinia form $1.6 Billion Collaboration to Develop Novel AAV Capsids for Genetic Therapies

April 28, 2020

Vertex Pharmaceuticals is ramping up its cell and gene therapy toolbox with a new research collaboration with Affinia Therapeutics to develop novel adeno-associated virus capsids to deliver transformative genetic therapies to people with serious diseases.

The collaboration will leverage Affinia’ 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).

Affinia’s proprietary AAVSmartLibrary and associated technology provides capsids that it says improve tissue tropism (the ability of cells and tissues of a host to support the viral delivery), manufacturability, and pre-existing immunity.

“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 Affinia CEO Rick Modi.

Under the terms of their agreement, Affinia will be eligible to receive more than $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 will apply its vector design and engineering technologies to develop novel capsids with improved properties. It will be responsible for the discovery of capsids that meet certain pre-determined criteria.

Vertex will have an exclusive license to intellectual property developed using Affinia’s technology in DMD and DM1 and 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. 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.

“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.

Photo: Bastiano Sanna, executive vice president and chief of Cell and Genetic Therapies at Vertex

Author: Rare Daily Staff

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