Intellia and SparingVision Collaborate to Develop Novel Ocular Therapies Using CRISPR/Cas9 Technology

Intellia Therapeutics and SparingVision, a genomic medicine company developing vision saving treatments for ocular diseases, entered a strategic collaboration to develop novel genomic medicines utilizing CRISPR/Cas9 technology for the treatment of ocular diseases.

Photo: John Leonard, president and CEO of Intellia

As part of this collaboration, Intellia will grant SparingVision exclusive rights to its 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 percent 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 U.S. 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 percent of development costs and pay royalties to SparingVision on U.S. sales. Intellia will also maintain the ability to leverage technology advances established under this collaboration for any targets outside the partnership.

“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,” said Stéphane Boissel, president and CEO of SparingVision. “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 look forward to working together with the shared goal of radically changing the treatment of blinding ocular diseases.”

SparingVision was founded to advance more than 20 years of ophthalmic research from its scientific founders at the Paris Vision Institute. It is leading taking 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 addresses mid-stages retinitis pigmentosa (RP) and SPVN20 addresses the late-stage form of the condition. 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.

“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 CEO John Leonard. “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.

Author: Rare Daily Staff