Affinia Therapeutics has signed a research collaboration deal worth more than $1.6bn with Vertex Pharmaceuticals to discover and develop adeno-associated virus capsids (AAV capsids) to be used in the latter's genetic therapies aimed at treating people with serious diseases.
The company’s AAVSmartLibrary and associated technology are said to be used for developing AAV capsids for enhanced tissue tropism, manufacturability, and pre-existing immunity.
The collaboration will make use of Affinia Therapeutics’ capsid engineering expertise and the scientific, clinical, and regulatory capabilities of Vertex Pharmaceuticals to fast track the development of genetic therapies for people suffering from Duchenne muscular dystrophy (DMD), myotonic dystrophy type 1 (DM1), and also cystic fibrosis (CF).
As per the terms of the deal, Affinia Therapeutics by applying its vector design and engineering technologies will develop novel AAV capsids with enhanced properties.
Affinia Therapeutics CEO Rick Modi said: “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.”
The agreement gives an exclusive license to Vertex Pharmaceuticals under Affinia Therapeutics’ technology and intellectual property (IP) in DMD and DM1 with an exclusive option to gain license rights for CF and another undisclosed disease. According to the partners, the agreement’s scope covers all genetic therapy modalities in these diseases.
Affinia Therapeutics will be eligible for payments totaling more than $1.6bn in upfront and development, regulatory, and commercial milestones. These will include $80m in upfront payments and research milestones that will be paid by Vertex Pharmaceuticals during the research term.
The company will also be paid tiered royalties by Vertex Pharmaceuticals on future net global sales on any products that emerge from the collaboration.
Affinia Therapeutics will handle the discovery of AAV capsids that meet some pre-determined criteria. On the other hand, Vertex Pharmaceuticals will take care of funding the design and manufacturing of genetic therapies that will incorporate the selected capsids along with their preclinical and clinical development activities, and marketing of any approved products in the licensed diseases.
Vertex Pharmaceuticals executive vice president and cell and genetic therapies chief Bastiano Sanna said: “This collaboration with Affinia Therapeutics will enhance our existing capabilities in discovering and developing transformative therapies for people with serious diseases.
“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.”