- Gene editing company Intellia Therapeutics is broadening its research efforts by targeting diseases of the eye through a new deal announced Wednesday.
- The deal has Intellia granting exclusive rights to its CRISPR technology to SparingVision, a young French biotech focused on genomic medicine. SparingVision will fund and lead both the preclinical and clinical development of therapies directed at three ocular targets. In exchange, Intellia will take a 10% equity stake in its partner.
- To date, Intellia's work has centered around potential one-time fixes for blood disorders, uncommon diseases and cancer. The deal with SparingVision now adds ocular illnesses to that list, and puts Intellia in closer competition with Editas Medicine, a gene editing company which for years has been advancing new treatments for the eyes, among others.
In the field of genetic medicine, eyes have been a focal point.
The first gene therapy for an inherited disease approved in the U.S., Roche's Luxturna, treats vision loss caused by a mutation in a gene that makes important retinal proteins. And just last month, Editas unveiled long-awaited data from the first clinical trial of a CRISPR-based gene editing treatment that works inside the body and, specifically, attempts to fix a different form of inherited vision loss.
For developers, the eyes offer several advantages. They're easy to reach in surgical procedures, and generally more sequestered from the body's immune defenses. They also can be treated with small doses of therapy compared to other organs, which lowers some of the potential manufacturing challenges.
The past few years have seen major pharmaceutical companies like Novartis, Biogen, AbbVie and Johnson & Johnson invest in gene therapies for the eye, typically through deals with smaller, specialized biotechs.
Now, Intellia is doing the same. While it has extensive experience with CRISPR gene editing — enough so that Novartis and Regeneron partner with Intellia on programs targeting hemophilia, sickle cell disease and transthyretin amyloidosis — the company hasn't spent as much time investigating eye diseases. Intellia hopes SparingVision can help.
"We have been thoroughly impressed with the team at SparingVision, particularly regarding their unparalleled understanding of retinal diseases," said Intellia CEO John Leonard in a statement Wednesday.
Deal terms hold that, should they hit certain development and commercial milestones, Intellia could take home as much as $200 million from each therapy that stems from the collaboration. The company may also receive royalties on future sales of any resulting products.
What's more, Intellia has the option to claim U.S. commercialization rights to product candidates from two of the three collaboration targets. For any candidate Intellia options, it will pay SparingVision an opt-in fee, certain reimbursement costs, 50% of development expenses and, later, royalties on any U.S. sales.
Aside from the three targets, Intellia and SparingVision plan to research and develop new, so-called "self-inactivating" adeno-associated viral vectors, as well as lipid nanoparticle-based approaches to address challenges delivering CRISPR-based gene editing treatments to the retina.
Intellia will also be able to use technological advances made through this collaboration for any targets not included in the original deal.