Business Wire

Vertex and CRISPR Therapeutics Announce Licensing Agreement to Accelerate Development of Vertex’s Hypoimmune Cell Therapies for the Treatment of Type 1 Diabetes

Share

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) today announced that they have entered into a new non-exclusive licensing agreement for the use of CRISPR Therapeutics’ gene editing technology, known as CRISPR/Cas9, to accelerate the development of Vertex’s hypoimmune cell therapies for type 1 diabetes (T1D).

This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20230324005339/en/

“We have multiple programs in our T1D portfolio including VX-880 and VX-264, which are in the clinic, as well as our hypoimmune program, in preclinical development,” said Bastiano Sanna, Ph.D., Executive Vice President and Chief of Cell and Genetic Therapies at Vertex. “Having successfully demonstrated clinical proof of concept in T1D in our VX-880 program, we are excited to deepen our relationship with CRISPR Therapeutics with this agreement, which will allow us to further accelerate our goal of generating fully differentiated, insulin-producing hypoimmune islet cells for T1D.”

“We are pleased to expand our long and successful relationship with Vertex with this collaboration which fully leverages our gene editing platform to develop hypoimmune cell therapies for T1D,” said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. “In parallel, we continue to expand our capabilities in regenerative medicine and advance our existing allogeneic gene-edited cell therapy programs.”

Under this agreement, Vertex will pay CRISPR Therapeutics $100 million up-front for non-exclusive rights to CRISPR Therapeutics’ technology for the development of hypoimmune gene-edited cell therapies for T1D. CRISPR Therapeutics will be eligible for up to an additional $230 million in research and development milestones and receive royalties on any future products resulting from this agreement.

CRISPR and ViaCyte, Inc., which was acquired by Vertex in 2022, will continue to collaborate on their existing gene-edited allogeneic stem cell therapies, using ViaCyte cells, for the treatment of diabetes under the terms of their collaboration. A Phase 1/2 study of VCTX211, an allogeneic, gene-edited, stem cell-derived product candidate for T1D, which originated under the CRISPR Therapeutics and ViaCyte collaboration, has been initiated and is ongoing. CRISPR Therapeutics will not obtain any interest in Vertex’s pre-existing pipeline of T1D products, including VX-880 and VX-264.

About Vertex

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 clinical pipeline of investigational small molecule, cell and genetic therapies in other serious diseases where it has deep insight into causal human biology, including sickle cell disease, beta thalassemia, APOL1-mediated kidney disease, pain, type 1 diabetes, and alpha-1 antitrypsin deficiency.

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. 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 13 consecutive years on Science magazine's Top Employers list and one of Fortune’s Best Workplaces in Biotechnology and Pharmaceuticals and Best Workplaces for Women. For company updates and to learn more about Vertex's history of innovation, visit www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

(VRTX-GEN)

Vertex Special Note Regarding Forward-Looking Statements

This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements by Bastiano Sanna, Ph.D., and Samarth Kulkarni, Ph.D., in this press release, statements about the terms of and expectations for Vertex’s collaboration with CRISPR, potential benefits and results that may be achieved through the collaboration, including acceleration of the development of Vertex’s hypoimmune cell therapies for T1D, statements regarding the future activities of the parties pursuant to the collaboration, statements regarding upfront and milestone payments, and potential royalties on future products, and statements regarding the collaboration between CRISPR and ViaCyte and that CRISPR will not obtain any interest in Vertex’s pre-existing pipeline of T1D products. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company’s beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that the anticipated benefits and potential of Vertex’s collaboration with CRISPR may not be achieved on the anticipated timeline, or at all, that data may not support further development of the therapies subject to the collaboration due to safety, efficacy, or other reasons, and other risks listed under the heading “Risk Factors” in Vertex's annual report filed with the Securities and Exchange Commission (SEC) and available through Vertex’s website at www.vrtx.com and on the SEC’s website at www.sec.gov. You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

About CRISPR Therapeutics

CRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations in Boston, Massachusetts and San Francisco, California, and business offices in London, United Kingdom. For more information, please visit www.crisprtx.com.

CRISPR THERAPEUTICS® word mark and design logo and VCTX211™ are trademarks and registered trademarks of CRISPR Therapeutics AG. All other trademarks and registered trademarks are the property of their respective owners.

(CRSP-GEN)

CRISPR Therapeutics Forward-Looking Statement

This press release may contain a number of “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including without limitation, statements made by Samarth Kulkarni, Ph.D. and Bastiano Sanna, Ph.D. in this press release, as well as statements regarding CRISPR Therapeutics’ expectations about any or all of the following: (i) the future activities of the parties pursuant to the ViaCyte collaboration and the expected benefits of such collaboration, including expectations regarding VCTX211; (ii) the safety, efficacy and progress of its clinical programs; (iii) upfront and milestone payments, and potential royalties on future products, under the non-exclusive license; and (vii) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies. Without limiting the foregoing, the words “believes,” “anticipates,” “plans,” “expects” and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: that it may not realize the potential benefits of its collaborations on the anticipated timeline, or at all; the potential that clinical trial results may not be favorable; that one or more of product candidate programs will not proceed as planned for technical, scientific or commercial reasons; and those risks and uncertainties described under the heading "Risk Factors" in CRISPR Therapeutics’ most recent annual report on Form 10-K, quarterly report on Form 10-Q and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC's website at www.sec.gov. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

To view this piece of content from cts.businesswire.com, please give your consent at the top of this page.

Contact information

Vertex Pharmaceuticals Incorporated

Investors:
investor_info@vrtx.com
Susie Lisa, +1 617-341-6108
or
Manisha Pai, +1 617-961-1899
or
Miroslava Minkova, +1 617-341-6135

Media:
mediainfo@vrtx.com
or
U.S.: +1 617-341-6992
or
Heather Nichols: +1 617-839-3607
or
International: +44 20 3204 5275

CRISPR Therapeutics

Investors:
Susan Kim, +1 617-307-7503
susan.kim@crisprtx.com

Media:
Rachel Eides, +1-617-315-4493
rachel.eides@crisprtx.com

About Business Wire

Business Wire
Business Wire
24 Martin Lane
EC4R 0DR London

+44 20 7626 1982http://www.businesswire.co.uk

(c) 2018 Business Wire, Inc., All rights reserved.

Business Wire, a Berkshire Hathaway company, is the global leader in multiplatform press release distribution.

Subscribe to releases from Business Wire

Subscribe to all the latest releases from Business Wire by registering your e-mail address below. You can unsubscribe at any time.

Latest releases from Business Wire

Philip Morris International Demonstrates Clear Progress Toward Its Purpose as It Releases 2023 Integrated Report28.3.2024 12:38:00 CET | Press release

Philip Morris International Inc. (PMI) (NYSE: PM) today releases its fifth annual Integrated Report, which outlines the company’s ongoing work to advance toward its 2025 Roadmap goals, with the primary focus on addressing the health impacts of the company’s products. The 2023 Integrated Report highlights PMI’s continued expansion of smoke-free alternatives in markets worldwide, as well as social and environmental programs deployed with and in parallel to these products—including the deployment of responsible marketing and sales practices and efforts to reduce post-consumer waste. Further, PMI reports its progress toward fostering an empowered and inclusive workplace, improving the quality of life of people in its supply chain, decarbonizing its operations and value chain, and preserving nature and biodiversity. “2023 was a year marked by unity, determination, and a continued commitment to our vision of a smoke-free future,” said Jacek Olczak, Chief Executive Officer of PMI. “As we enco

Wolters Kluwer named Market Leader in BARC Score Financial Performance Management 202428.3.2024 12:09:00 CET | Press release

Wolters Kluwer, a global leader in information, software, and services for professionals, today announces that its CCH® Tagetik expert solution has retained its leadership position for the seventh consecutive year in both the Global and DACH region issues of the prestigious BARC Score Financial Performance Management (FPM) Report. Developed by BARC, one of Europe’s leading analyst firms for business software, the reports highlight CCH Tagetik’s strength as a unified platform for financial corporate management (financial consolidation and close; budgeting, planning, and forecasting; ESG and regulatory reporting; corporate tax) that can be used by individual subsidiaries or at group level. Ralf Gärtner, Senior Vice President and General Manager of Corporate Performance & ESG, Wolters Kluwer, said: “The BARC Score FPM Reports have ranked CCH Tagetik as a market leader year after year, recognizing our consistent commitment to product excellence and innovation. We are focused on empowering

Cirium’s Airfare Data Shows Increases in Economy Airfares in 2023 in North America and Europe With a Decrease in Transatlantic Business Class Fares28.3.2024 11:37:00 CET | Press release

Cirium, the leading aviation analytics company, analysed airfare trends for 2023, using Cirium FM Traffic. Economy airfare has generally increased since 2023 in North America and Europe, but lower than the U.S. inflation rate. “While airfare has risen in nominal terms in U.S. and Europe, consumers will appreciate that the increases are less than other consumables,” said Jeremy Bowen, CEO of Cirium. “Cirium’s data teams will continue to monitor the impact on fares of increased capacity in the market, despite challenges related to the availability of aircraft, be it due to groundings, engine issues, or supply chain constraints.” Cirium analysed airfare for the U.S. domestic market (including 13 U.S. airlines), intra-European travel, and the Transatlantic market. The data is non-directionally averaged, in U.S. dollars, not including taxes and fees. Unless otherwise noted, the fares indicated are for economy class. U.S. Domestic Airfare The average U.S. domestic airfare by the 13 airlines

Ministry of Education and Science of Ukraine and Palantir Partner to Create Safe Conditions for In-Person Education in Schools28.3.2024 10:00:00 CET | Press release

The Ministry of Education and Science, along with Palantir (NYSE:PLTR), a leading provider of AI systems, have signed an agreement. The main goal is to facilitate qualitative transformations in the field of education and ensure the maximum number of children have access to safe in-person learning. The agreement, initially for 12 months, will support the Government’s official School Offline initiative which aims to provide 300,000 more Ukrainian pupils with safe access to a school. New software tooling will enable: Funding allocated for school bomb shelters to be targeted where it will deliver the greatest benefit for children and young people of a school age. Every school to receive a risk rating according to a methodology recently adopted in law as a Cabinet of Ministers decree, in order to help identify targeted measures that will improve the safety of a school. The rating will take into account factors such as historic shelling in the area, presence of a bomb shelter, safety of acce

Guidewire Jasper Release Boosts Commercial Lines Agility for P&C Insurers and Strengthens Functionality Across Entire Product Portfolio28.3.2024 08:05:00 CET | Press release

Guidewire (NYSE: GWRE) announces Jasper, its latest release, which boosts commercial lines agility for P&C insurers with support for layered coinsurance, schedule import, and a new U.S. Bureau Content Solution.* Additionally, expanded HazardHub data for Canada gives insurers deeper property risk insights for underwriting precision. Jasper release will be generally available on April 5, 2024. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20240328607054/en/ “Commercial lines insurers face both risk and opportunity. On the one hand, there are economic fluctuations, including inflation, geopolitical complexities, capital limitations, and environmental challenges. On the other hand, the protection gap creates growth opportunities for insurers that innovate to stay ahead of the evolving risk landscape,” said Eugene Lee, senior vice president and general manager, InsuranceSuite, Guidewire. For P&C insurers seeking to improve commerc

HiddenA line styled icon from Orion Icon Library.Eye