Vertex and Obsidian Therapeutics Establish Collaboration to Discover New Therapies Governing Gene Editing

Many years of strategic research collaboration leverage Obsidian’s proprietary cytoDRiVE® platform to discover manageable genetic therapies to treat serious diseases

Vertex will pay Obsidian up to $ 75 million in equity, down payments and possible research milestones

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and Obsidian Therapeutics announced today that the two companies have entered into a strategic research collaborative and licensing agreement focused on the discovery of new therapies that regulate gene editing for treating serious diseases. The collaboration leverages Obsidian platform’s cytoDRiVE® platform to discover gene-editing drugs whose therapeutic activity can be precisely controlled by small molecules and Vertex’s established scientific and clinical capabilities in small molecules, cells and genetic therapies to more quickly bring these approaches to patients.

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“This collaboration with Obsidian is growing and expanding Vertex’s leadership in small molecules and genetic therapies, and we are excited to work with the team at Obsidian to explore the capabilities of their technology,” said David Altshuler of Vertex, executive vice president, Global. Research and Chief Scientific Officer. “The ability to set gender editing activity to a specific level is an important innovation that can treat several major diseases.”

“At Obsidian, we use our cytoDRiVE technology to pioneer a new generation of realized cell and gene therapies for patients with serious diseases,” said Paul Wotton, Chief Executive of Obsidian Therapy. “Collaborating with Vertex, an established leader at the forefront of gene therapies, will further advance our technology in the field of gene therapy and accelerate development of manageable gene therapy therapies for patients.”

About the Cooperation

Under the terms of the agreement, Obsidian will use its cytoDRiVE technology to develop new regulated gene-editing therapy candidates for many major diseases. Obsidian gives Vertex the exclusive opportunity to authorize global rights to candidates discovered and developed in accordance with the cooperation. Following the exercise of its Vertex options, Vertex will be responsible for further preclinical and clinical development and trading.

Vertex will pay Obsidian up to $ 75 million in down payments and research milestones that can be paid during the research period, including an equity investment in Obsidian. Obsidian is eligible to receive up to $ 1.3 billion in potential payments based on the successful achievement of specific research, development, regulatory and trade milestones through up to five potential programs. In addition, Vertex will pay tier rights for future net sales of certain products that may result from this partnership. Specific diseases that are the subject of this collaboration are not disclosed.

About Obsidian Therapy

Obsidian Therapeutics, Inc. is a biotechnology company pioneering realized cell and gene therapies to deliver transformative outcomes for patients with insoluble diseases. The Company expects to send IND in mid-2022 for its main citoTIL ™ program, a newly created tumor infiltrating lymphocyte therapy armored with regulated membrane-bound IL15 that does not require patients to receive toxic IL2. Obsidian’s own citoDRiVE® technology provides a way to control protein degradation by small molecules approved by the FDA, allowing precise control of the time and level of protein expression. Obsidian is headquartered in Cambridge, Massachusetts. For more information, please visit www.obsidiantx.com.

About Vertex Pharmaceuticals

Vertex is a global biotechnology company that invests in scientific innovation to create transformational drugs for people with serious diseases. The company has many approved drugs 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 researching small molecular drugs in other major diseases, where it has in-depth knowledge of causal human biology, including pain, alpha-1 antiprypsin deficiency, and APOL1-mediated kidney disease. In addition Vertex has a rapidly growing pipeline of genetic and cellular therapies for diseases such as serous cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

Founded in 1989 in Cambridge, Massachusetts, Vertex’s global headquarters are now located in Boston’s Innovation District and its international headquarters are in London. Additionally, the company has research and development websites and sales offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry’s best jobs, including 11 consecutive years on Science magazine’s Top Employers list and best place to work for LGBT equality through the Human Rights Campaign For Corporate Updates and Learning more about the history of Vertex innovation, visit www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

Special Note On Forecast Statements

This press release contains forward-looking statements defined in the Private Securities Reform Act of 1995, including, without limitation, statements by David Altshuler and Paul Wotton in this press release, statements of the potential benefits and results that may be achieved by Vertex’s. collaboration with Obsidian, including Obsidian’s CytoDRiVE® technology platform, statements on prepayment and mileage payments and potential royalties on future sales, and expectations about the ability to quickly bring therapies to patients. Although Vertex believes that 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. . differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, inter alia, that the anticipated benefits and potential of Vertex’s collaboration with Obsidian may not be achieved along the intended timeline, or that data may not support further development of the therapies under the cooperation for safety, efficacy or other reasons, and other risks listed under the heading “Risk Factors” in Vertex’s annual report registered 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. .sek.gov. You should not overly trust these statements. Vertex disclaims any obligation to update the information contained in this press release when new information becomes available.

(VRTX-GEN)

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Contacts

Summit Contacts:
Investors:
Michael Partridge, 617-341-6108
or
Brenda Eustace, 617-341-6187
or
Manisha Pai, 617-429-6891

Media:
Heather Nichols, 617-839-3607
[email protected]

Contacts on Obsidian Therapy
Maggie Beller
[email protected]
646-942-5631

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