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Crispr Licensed for Eye Disease Gene Therapies

Retinal gene therapy

(National Human Genome Research Institute, NIH)

13 Oct. 2021. A developer of inherited eye disease therapies is acquiring rights to the gene editing technology Crispr for treatments that work inside the eyes. The deal with SparingVision in Paris could bring biotechnology company Intellia Therapeutics Inc. in Cambridge, Massachusetts as much as $600 million as well as an equity stake in SparingVision.

SparingVision develops treatments for retinitis pigmentosa, a progressive genetic disorder that destroys photoreceptor cells in the retina, leading to total blindness in some cases, which affects some 1 in 4,000 people worldwide. The company has three candidate therapies for the disease, all delivered with adeno-associated viruses, or AAVs. These viruses are benign, naturally occurring microbes that infect cells, but do not integrate with the cell’s genome or cause disease, and generate at most a mild immune response. One of SparingVision’s treatment candidates code-named SPVN06 is in preclinical development, while the other two therapies are in discovery stage.

Crispr, short for clustered, regularly interspaced short palindromic repeats, is adapted from a natural process used by bacteria to protect against attack by viruses, where an enzyme that deactivates or replaces genes binds to targeted RNA molecules generated by the genome. The RNA molecules then guide an editing enzyme, known as Crispr-associated protein 9 or Cas9, to specific genes needing changes.

Intellia Therapeutics is developing treatments with Crispr-Cas9 that remove disease-causing genes or mutations, repair genes with mutations found in small regions of DNA, and insert corrected or functioning genes when mutations are found in larger DNA regions. The company says its treatments are being designed to be either administered directly to work in vivo, i.e.  inside patients’ bodies, or delivered to cells taken from the patient, then cultured outside the body and transplanted back. In June 2021, Science & Enterprise reported on early clinical trial data from Intellia demonstrating the feasibility of in vivo Crispr delivery, with few adverse effects.

Jointly study and develop new Crispr-Cas9 delivery methods

In their agreement, SparingVision gains an exclusive license to Intellia’s Crispr-Cas9 technology for up to three disease targets affecting the eyes. SparingVision will lead preclinical work and clinical trials of therapy candidates developed by the two companies. Plus, the two companies will jointly study and develop more delivery methods for Crispr-Cas9 therapies to the retina: AAVs that self-deactivate and lipid nanoparticles.

Intellia is eligible for developmental and commercial milestone payments of some $200 million for each of the three products developed under the partnership, and royalties on sales. And Intellia is receiving a 10 percent equity stake in SparingVision. Intellia also has an option to obtain commercialization rights for up to two of the new products developed by the companies, with Intellia paying SparingVision opt-in fees, reimbursing or sharing development costs, and paying royalties on sales in the U.S.

“Intellia is the first company in history ” says Stéphane Boissel, president of SparingVision, in a statement, “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 believe,” adds Intellia CEO John Leonard, “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.”

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