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October, 13 2021 • Articles

Intellia Therapeutics and SparingVision Announce Strategic Collaboration to Develop Novel Ocular Therapies Using CRISPR/Cas9 Technology

Intellia Therapeutics and SparingVision Announce Strategic Collaboration to Develop Novel Ocular Therapies Using CRISPR/Cas9 Technology

Cambridge, MA, and Paris, France– October 13, 2021 – Intellia Therapeutics, Inc. (NASDAQ: NTLA), a leading clinical-stage genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, and SparingVision, a genomic medicine company developing vision saving treatments for ocular diseases, today announced a strategic collaboration to develop novel genomic medicines utilizing CRISPR/Cas9 technology for the treatment of ocular diseases.

As part of this collaboration, Intellia will grant SparingVision exclusive rights to Intellia’s proprietary in vivo CRISPR/Cas9-based genome editing technology for up to three ocular targets addressing diseases with significant unmet medical need. SparingVision will lead and fund the preclinical and clinical development for the genome editing product candidates pursued under the collaboration. In addition, the parties will research and develop novel self-inactivating AAV vectors and LNP-based approaches to address delivery of CRISPR/Cas9 genome editing reagents to the retina.

As part of the licensing agreement, Intellia will receive a 10% equity ownership stake in SparingVision. Intellia will also be eligible to receive certain development and commercial milestone payments (around $200 million per product) as well as royalties on potential future sales of products arising from the collaboration. In addition, Intellia may exercise an option to obtain the US commercialization rights for product candidates arising from two of three collaboration targets. For product candidates Intellia chooses to option, Intellia will pay an opt-in fee, reimburse certain costs, share in 50% of development costs and pay royalties to SparingVision on US sales. Intellia will also maintain the ability to leverage technology advances established under this collaboration for any targets outside the partnership.

Stéphane Boissel, President and Chief Executive Officer of SparingVision, said:SparingVision’s aim has always been to disrupt the ophthalmology field by using cutting-edge technologies to address areas of significant unmet need. This collaboration with Intellia marks a pivotal moment in this mission and is highly complementary to our already mature and growing pipeline of unique mutation-agnostic gene therapies. Intellia is the first company in history to present clinical data supporting precision editing of a disease-causing gene within the body following a single, systemic dose of CRISPR/Cas9 and we are honored to have been selected as a strategic partner. We look forward to working together with the shared goal of radically changing the treatment of blinding ocular diseases.”

Intellia has been a pioneer in utilizing CRISPR/Cas9 technology to develop potentially curative treatments for genetic diseases. Today’s announcement is another step forward in more fully leveraging the power of our genome editing technology to address diseases inadequately treated with existing medicines,” said Intellia President and Chief Executive Officer John Leonard, M.D. “We have been thoroughly impressed with the team at SparingVision, particularly regarding their unparalleled understanding of retinal diseases and trackrecord for developing novel therapies for patients with ocular diseases. We believe SparingVision will be an excellent partner to expand our genome editing capabilities into the field of ophthalmology and we look forward to our new partnership.

The transaction is expected to close in the fourth quarter and is subject to certain closing conditions.

 

About Intellia Therapeutics

Intellia Therapeutics, a leading clinical-stage genome editing company, is developing novel, potentially curative therapeutics using CRISPR/Cas9 technology. To fully realize the transformative potential of CRISPR/Cas9, Intellia is pursuing two primary approaches. The company’s in vivo programs use intravenously administered CRISPR as the therapy, in which proprietary delivery technology enables highly precise editing of diseasecausing genes directly within specific target tissues. Intellia’s ex vivo programs use CRISPR to create the therapy by using engineered human cells to treat cancer and autoimmune diseases. Intellia’s deep scientific, technical and clinical development experience, along with its robust intellectual property portfolio, have enabled the company to take a leadership role in harnessing the full potential of CRISPR/Cas9 to create new classes of genetic medicine. Learn more at intelliatx.com. Follow us on Twitter @intelliatweets.

 

About SparingVision:

SparingVision is a genomic medicines company, translating pioneering science into vision-saving treatments. Founded to advance over 20 years of world-leading ophthalmic research from its scientific founders at the Paris Vision Institute, SparingVision is leading a step shift in how ocular diseases are treated, moving beyond single gene correction therapies. At the heart of this is a pipeline of gene independent treatments for rod-cone dystrophies. Lead products, SPVN06 and SPVN20, address mid and late stages of retinitis pigmentosa (RP)respectively. RP is the most common inherited retinal disease affecting two million people worldwide. These novel medicines could form the basis of a suite of new sight-saving treatments with potential applications across many other retinal diseases, regardless of genetic cause.

The Company is supported by a strong, internationally renowned team who aim to harness the potential of genomic medicine to deliver new treatments to all ocular disease patients as quickly as possible. SparingVision has raised €60 million to date and its investors include 4BIO Capital, Advent France Biotechnology, Bpifrance, Foundation Fighting Blindness (US), Fondation Voir & Entendre, UPMC Enterprises, Jeito Capital and Ysios Capital. For more information, please visit www.sparingvision.com.

 

Intellia’s Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, express or implied statements regarding Intellia’s beliefs and expectations regarding: its strategy, business plans and focus; its ability to quickly and efficiently realize the scope and potential of its genome-editing technology; its ability to maintain, expand and maximize its intellectual property portfolio and pipeline as well as accelerate clinical validation for its platform; the therapeutic value and development potential of CRISPR/Cas9 gene editing technologies and therapies; its ability to combine its CRISPR genome editing platform with SparingVision’s expertise in ocular diseases to create successful therapeutic products; and the expected strategic benefits of any current or future collaborations.

Any forward-looking statements in this press release are based on management's current expectations and beliefs of future events, and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, risks related to Intellia’s ability to protect and maintain its intellectual property portfolio; risks related to Intellia’s relationship with third parties, including its licensors and licensees; risks related to the ability of Intellia’s licensors to protect and maintain their intellectual property position; uncertainties related to the authorization, initiation and conduct of studies and other development requirements for the new company’s product candidates; the risk that any one or more of the collaboration product candidates will not be successfully developed and commercialized; the risk that the results of preclinical studies or clinical studies will not be predictive of future results in connection with future studies; and the risk that Intellia’s collaboration with SparingVision or its other collaborations will not continue or will not be successful. These and other risks and uncertainties are described in greater detail in the section entitled “Risk Factors” in Intellia’s most recent annual report on Form 10-K and quarterly report on Form 10-Q filed with the SEC, as well as discussions of potential risks, uncertainties, and other important factors in Intellia’s other filings with the Securities and Exchange Commission. Any forward-looking statements contained in this press release represent Intellia’s views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. Intellia explicitly disclaims any obligation to update any forward-looking statements, except as required by law.