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 Vertexs 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 were excited to partner with them to bring together their technology platform with our research and development expertise.
At Affinia Therapeutics, were 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.
About Affinia Therapeutics
At Affinia Therapeutics, our purpose is to develop gene therapies that can have a transformative impact on people affected by devastating genetic diseases. Our proprietary platform enables us to methodically engineer novel AAV vectors and gene therapies that have remarkable tissue targeting and other properties. We are building world-class capabilities to discover, develop, manufacture and commercialize gene therapy products with an initial focus on muscle and central nervous system (CNS) diseases with significant unmet need. http://www.affiniatx.com.
About Vertex Pharmaceuticals
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) a rare, life-threatening genetic disease and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.
Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London, UK. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 10 consecutive years on Science magazine's Top Employers list and top five on the 2019 Best Employers for Diversity list by Forbes. For company updates and to learn more about Vertex's history of innovation, visit http://www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.
Special Note Regarding Forward-Looking Statements
This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, Dr. Sannas statements in the second paragraph of the press release, Mr. Modis statements in the third paragraph of the press release, and statements regarding future activities of the parties pursuant to the collaboration. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent Vertex's beliefs only as of the date of this press release and there are a number of factors that could cause actual events or results to differ materially from those indicated by such forward-looking statements. Those risks and uncertainties include, among other things, Vertex may not realize the anticipated benefits of the collaboration, and the other risks listed under Risk Factors in Vertex's annual report and quarterly reports filed with the Securities and Exchange Commission and available through the company's website at http://www.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.
(VRTX-GEN)
- Main Show Only - Genetic Engineering - Coast to Coast AM - Video [Last Updated On: September 7th, 2012] [Originally Added On: September 7th, 2012]
- RTPB | Genetic Engineering [Last Updated On: September 7th, 2012] [Originally Added On: September 7th, 2012]
- Stop Monsanto From Poisoning Hawai'i: Genetic Engineering Chemical Warfare - Video [Last Updated On: September 7th, 2012] [Originally Added On: September 7th, 2012]
- Informatics 161 PSA: Human Genetic Engineering - Video [Last Updated On: September 7th, 2012] [Originally Added On: September 7th, 2012]
- Don't Trust Whole Foods or Other Health Food Stores For Non- GMO Foods | Genetic Engineering - Video [Last Updated On: September 7th, 2012] [Originally Added On: September 7th, 2012]
- Genetic Engineering - Video [Last Updated On: September 7th, 2012] [Originally Added On: September 7th, 2012]
- Easier Genetic Engineering! (Brainstorm Ep69) - Video [Last Updated On: September 7th, 2012] [Originally Added On: September 7th, 2012]
- TEDxUCIrvine - Francisco Ayala - Cloning, Genetic Engineering, [Last Updated On: September 7th, 2012] [Originally Added On: September 7th, 2012]
- Genetic Engineering in Agriculture - Video [Last Updated On: September 7th, 2012] [Originally Added On: September 7th, 2012]
- Infowars Special Report : Genetic Engineering - Video [Last Updated On: September 7th, 2012] [Originally Added On: September 7th, 2012]
- Glowing Rats and Extreme Genetic Engineering - Video [Last Updated On: September 7th, 2012] [Originally Added On: September 7th, 2012]
- Endangered Cat Born at Audubon - Video [Last Updated On: September 7th, 2012] [Originally Added On: September 7th, 2012]
- Danger of Genetic Engineering - Video [Last Updated On: September 7th, 2012] [Originally Added On: September 7th, 2012]
- AQA GCSE core science and B1 - Genetic Engineering - Video [Last Updated On: September 7th, 2012] [Originally Added On: September 7th, 2012]
- genetic engineering rDNA.wmv - Video [Last Updated On: September 7th, 2012] [Originally Added On: September 7th, 2012]
- Genetic Engineering for Human Enhancement.mp4 - Video [Last Updated On: September 7th, 2012] [Originally Added On: September 7th, 2012]
- Genetic Engineering - Seven Wonders of the Microbe World (6/7) - Video [Last Updated On: September 7th, 2012] [Originally Added On: September 7th, 2012]
- Promising new drug target for inflammatory lung diseases [Last Updated On: September 8th, 2012] [Originally Added On: September 8th, 2012]
- Mandatory GM Labeling Would Require Major Change [Last Updated On: September 10th, 2012] [Originally Added On: September 10th, 2012]
- GEN reports on ocular therapeutics targeting the retina [Last Updated On: September 11th, 2012] [Originally Added On: September 11th, 2012]
- Prop. 37: Another example of the perils of the initiative process [Last Updated On: September 15th, 2012] [Originally Added On: September 15th, 2012]
- Genetic test predicts risk for Autism [Last Updated On: September 19th, 2012] [Originally Added On: September 19th, 2012]
- Immediate withdrawal of unsafe GE corn vital - Greens [Last Updated On: September 20th, 2012] [Originally Added On: September 20th, 2012]
- Immediate withdrawal of unsafe GE corn vital [Last Updated On: September 20th, 2012] [Originally Added On: September 20th, 2012]
- NZ out of step on GE [Last Updated On: September 21st, 2012] [Originally Added On: September 21st, 2012]
- Ballot Watch: Labeling genetically engineered foods [Last Updated On: September 30th, 2012] [Originally Added On: September 30th, 2012]
- Just another GE mirage [Last Updated On: October 2nd, 2012] [Originally Added On: October 2nd, 2012]
- The GM Barnyard [Last Updated On: October 3rd, 2012] [Originally Added On: October 3rd, 2012]
- Free Engine [Last Updated On: October 3rd, 2012] [Originally Added On: October 3rd, 2012]
- Cellectis Publishes Results Paving the Way for New Therapeutic Approaches against Cancer and Genetic Diseases [Last Updated On: October 3rd, 2012] [Originally Added On: October 3rd, 2012]
- Does moral decision-making in video games mirror the real world? [Last Updated On: October 3rd, 2012] [Originally Added On: October 3rd, 2012]
- Animals engineered with pinpoint accuracy [Last Updated On: October 3rd, 2012] [Originally Added On: October 3rd, 2012]
- Valley farmers fear 'modified' wording in Prop. 37 [Last Updated On: October 4th, 2012] [Originally Added On: October 4th, 2012]
- No on Proposition 37 [Last Updated On: October 4th, 2012] [Originally Added On: October 4th, 2012]
- The Dangers of Genetic Engineering [Last Updated On: October 5th, 2012] [Originally Added On: October 5th, 2012]
- Are inhaled medications effective and safe in critically ill patients on mechanical ventilation? [Last Updated On: October 5th, 2012] [Originally Added On: October 5th, 2012]
- Calif. initiative will test appetite for GMO food [Last Updated On: October 7th, 2012] [Originally Added On: October 7th, 2012]
- Calif. initiative will test appetite for GMO food - Sat, 06 Oct 2012 PST [Last Updated On: October 7th, 2012] [Originally Added On: October 7th, 2012]
- California to vote on 'genetically modified' labels [Last Updated On: October 7th, 2012] [Originally Added On: October 7th, 2012]
- California initiative will test appetite for genetically modified foods [Last Updated On: October 7th, 2012] [Originally Added On: October 7th, 2012]
- Do we have an appetite for genetically modified food? [Last Updated On: October 7th, 2012] [Originally Added On: October 7th, 2012]
- Genetic labelling mooted in California [Last Updated On: October 7th, 2012] [Originally Added On: October 7th, 2012]
- California initiative to test appetite for 'genetically engineered' food [Last Updated On: October 7th, 2012] [Originally Added On: October 7th, 2012]
- New tool for making genetic engineering of microbial circuits reliably predictable [Last Updated On: October 10th, 2012] [Originally Added On: October 10th, 2012]
- Researchers develop new tool for making genetic engineering of microbial circuits reliably predictable [Last Updated On: October 10th, 2012] [Originally Added On: October 10th, 2012]
- A Welcome Predictability [Last Updated On: October 10th, 2012] [Originally Added On: October 10th, 2012]
- Engineered flies spill secret of seizures [Last Updated On: October 12th, 2012] [Originally Added On: October 12th, 2012]
- Genetic 'remix' key to evolution of bee behavior, researchers find [Last Updated On: October 15th, 2012] [Originally Added On: October 15th, 2012]
- Can vaccines be delivered via the lungs instead of by injection? [Last Updated On: October 15th, 2012] [Originally Added On: October 15th, 2012]
- Food labeling regulations [Last Updated On: October 16th, 2012] [Originally Added On: October 16th, 2012]
- Research and Markets: Therapeutic Antibody Engineering: Current and Future Advances Driving the Strongest Growth Area ... [Last Updated On: November 2nd, 2012] [Originally Added On: November 2nd, 2012]
- Is there a difference between GE & GMO? [Last Updated On: November 2nd, 2012] [Originally Added On: November 2nd, 2012]
- Port Townsend Food Coop: A Sad Story of GMO Crop Cross Pollination - Video [Last Updated On: November 2nd, 2012] [Originally Added On: November 2nd, 2012]
- PUT IT IN YOUR MOUTH! Yes to Prop 37 — it's your right to know what's in your food! - Video [Last Updated On: November 2nd, 2012] [Originally Added On: November 2nd, 2012]
- editta braun company: planet LUVOS (clip, 4:30) - Video [Last Updated On: November 2nd, 2012] [Originally Added On: November 2nd, 2012]
- Mimic: The Director's Cut (Blu-ray Trailer) - Video [Last Updated On: November 2nd, 2012] [Originally Added On: November 2nd, 2012]
- South Park Season 1 Episode 7 - An Elephant Makes Love to a Pig - Video [Last Updated On: November 2nd, 2012] [Originally Added On: November 2nd, 2012]
- Yes on Prop 37: Animated Video - Video [Last Updated On: November 2nd, 2012] [Originally Added On: November 2nd, 2012]
- The Day of the Triffids Part 1 Full Movie - Video [Last Updated On: November 2nd, 2012] [Originally Added On: November 2nd, 2012]
- YES on prop 37 "flashmob" march - Santa Cruz, CA Sept. 2012 - Video [Last Updated On: November 2nd, 2012] [Originally Added On: November 2nd, 2012]
- World's Weirdest Places Discussed - Video [Last Updated On: November 2nd, 2012] [Originally Added On: November 2nd, 2012]
- THE CLUB OF ROME - DEPOPULATION AGENDA 21 - THE UNHIVED MIND - Video [Last Updated On: November 2nd, 2012] [Originally Added On: November 2nd, 2012]
- 27-Medical BiotechnologySG Part II.Gene Therapy, Tissue Engineering and Nanotechnology.mov - Video [Last Updated On: November 2nd, 2012] [Originally Added On: November 2nd, 2012]
- 26-Medical BiotechnologySG Part Ic. Animal and Human Cloning and Genetic Engineering.mov - Video [Last Updated On: November 2nd, 2012] [Originally Added On: November 2nd, 2012]
- How to Clone Animals - Video [Last Updated On: November 2nd, 2012] [Originally Added On: November 2nd, 2012]
- Fringe Opening sequence (Season 3 - The Day We Died) - Video [Last Updated On: November 4th, 2012] [Originally Added On: November 4th, 2012]
- Fringe Opening sequence (Season 3 - Olivia) - Video [Last Updated On: November 4th, 2012] [Originally Added On: November 4th, 2012]
- Fringe Opening sequence (Season 3 - Entrada) - Video [Last Updated On: November 4th, 2012] [Originally Added On: November 4th, 2012]
- Fringe Opening sequence (Season 4) - Video [Last Updated On: November 4th, 2012] [Originally Added On: November 4th, 2012]
- Fringe Opening sequence (Season 3) - Video [Last Updated On: November 4th, 2012] [Originally Added On: November 4th, 2012]
- Fringe Opening sequence (Season 2) - Video [Last Updated On: November 4th, 2012] [Originally Added On: November 4th, 2012]
- Fringe Opening sequence (1985) - Video [Last Updated On: November 4th, 2012] [Originally Added On: November 4th, 2012]
- Dendritic Cells: Biology And Clinical Applications - Video [Last Updated On: November 4th, 2012] [Originally Added On: November 4th, 2012]
- Port Townsend Food Coop: "I want my food untainted by GMOs" - Video [Last Updated On: November 4th, 2012] [Originally Added On: November 4th, 2012]
- Catholic Priest vs. Biologist - Video [Last Updated On: November 4th, 2012] [Originally Added On: November 4th, 2012]
- Watch Real Life Superhero Muscles Like Marvel's The Avengers - Video [Last Updated On: November 4th, 2012] [Originally Added On: November 4th, 2012]
- Fringe Opening sequence (Season 1) - Video [Last Updated On: November 4th, 2012] [Originally Added On: November 4th, 2012]
- Fringe Opening sequence (The Final Season: "Fight For The Future") - Video [Last Updated On: November 4th, 2012] [Originally Added On: November 4th, 2012]
- Port Townsend Food Coop: I Want To Know If GMOs Are In My Food - Video [Last Updated On: November 4th, 2012] [Originally Added On: November 4th, 2012]
- ProCognia jumps on expanded biosimilar collaboration [Last Updated On: November 7th, 2012] [Originally Added On: November 7th, 2012]