Intellia and SparingVision See Eye to Eye in New Partnership

Intellia_Courtesy of company

Photo courtesy of Intellia

Genome-editing company Intellia Therapeutics and France-based SparingVision have partnered to develop new therapies for ocular diseases using CRISPR/Cas9 gene-editing technology. The deal could be worth up to $600 million for Intellia.

Under terms of the deal, Intellia, which is developing CRISPR-based therapies for leukemia and hereditary transthyretin (ATTR) amyloidosis, will grant SparingVision rights to its in vivo CRISPR/Cas9 technology for the development of ocular therapies directed at three different diseases of the eye that are considered hard to treat. The disease targets were not disclosed in the announcement.

SparingVision will take the lead for preclinical and clinical development of the programs. The French company will also be responsible for funding the development of the assets. In addition to the three targets, Intellia and SparingVision will work together to research and develop novel self-inactivating AAV vectors and LNP-based approaches to address the delivery of CRISPR/Cas9 genome-editing reagents to the retina.

SparingVision’s own research focuses on rod-cone dystrophies. Its two lead assets, SPVN06 and SPVN20, are aimed at mid and late stages of retinitis pigmentosa (RP) respectively.

Intellia President and Chief Executive Officer John Leonard said they opted to partner with SparingVision based on that company’s “unparalleled understanding of retinal diseases and track-record for developing novel therapies for patients with ocular diseases.” Providing the company with Intellia’s CRISPR/Cas-9 technology is another way to leverage the promise of genome editing to address diseases that, so far, have been unable to be equitably treated by existing therapies.

“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,” Leonard said in a statement.

Stéphane Boissel, president and chief executive officer of SparingVision, said the collaboration with Intellia is a pivotal moment in the company’s aim to disrupt the ophthalmology field through the use of cutting-edge technologies like CRISPR. The aim of the collaboration with Intellia is complementary to SparingVision’s ongoing work to develop gene therapies for ocular disease.

“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,” Boissel said in a statement.

For Intellia, the collaboration means the Cambridge, Mass. company will receive a 10% equity stake in SparingVision. The company will also be eligible for milestone payments associated with all three disease targets covered under the deal. The value of each target for Intellia is estimated at approximately $200 million. The deal provides Intellia with an option to obtain commercialization rights in the United States for two of the three assets.

The deal with SparingVision comes about a month after Intellia announced it was entering its wholly-owned ex vivo CRISPR genome-editing candidate NTLA-5001 into the clinic as a potential treatment for acute myeloid leukemia. In an exclusive interview with BioSpace, Leonard explained that NTLA-5001 is engineered to target the Wilms’ Tumor (WT1) antigen. That antigen is overexpressed in approximately 90% of AML patients regardless of genetic subtype, he explained. Additionally, WT1 is highly expressed across multiple hematological and solid tumor types. This means there is potential to target additional cancers.

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