Intellia looks at eye disease in latest deal

Dive brief:

  • Gene-publishing company Intellia Therapeutics is expanding its research efforts by targeting eye diseases with a new agreement announced on Wednesday.
  • The agreement allowed Intellia to grant the exclusive rights to its CRISPR technology to SparingVision, a young French biotech focused on genomic medicine. SparingVision will fund and lead the preclinical and clinical development of therapies targeting three ocular targets. In exchange, Intellia will take a 10% stake in the capital of its partner.
  • To date, Intellia’s work has focused on potential point solutions for blood disorders, rare diseases and cancer. The deal with SparingVision now adds eye diseases to that list and puts Intellia in closer competition with Editas Medicine, a gene-editing company that has been offering new eye treatments and more for years.

Dive overview:

In the field of genetic medicine, the eyes have been a focal point.

The first gene therapy for an inherited disease approved in the United States, Luxturna by Roche, treats vision loss caused by a mutation in a gene that makes important retinal proteins. And 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, in particular, attempts to correct another form of loss of blood. hereditary vision.

For developers, the eyes offer several advantages. They are easy to reach during surgery and generally more isolated from the body’s immune system. They can also be treated with small doses of therapy compared to other organs, reducing some of the potential manufacturing challenges.

In recent years, large pharmaceutical companies like Novartis, Biogen, AbbVie, and Johnson & Johnson have invested in gene therapies for the eye, typically through contracts with smaller, specialist biotechnology companies.

Now Intellia is doing the same. Although it has extensive experience in editing CRISPR genes – enough for Novartis and Regeneron to partner with Intellia on programs targeting hemophilia, sickle cell anemia, and transthyretin amyloidosis – the company has not. spent so much time investigating eye diseases. Intellia hopes SparingVision can help you.

“We have been very impressed with the SparingVision team, especially with their unparalleled understanding of retinal disease,” Intellia CEO John Leonard said in a statement on Wednesday.

The terms of the deal state that if they reach certain development and commercial milestones, Intellia could make up to $ 200 million from each therapy resulting from the collaboration. The company may also receive royalties on future sales of any resulting product.

In addition, Intellia has the ability to claim US commercialization rights to product candidates for two of the three collaboration targets. For any candidate for Intellia options, he will pay SparingVision a registration fee, some reimbursement fee, 50% development fee, and later royalties on all sales in the United States.

In addition to the three targets, Intellia and SparingVision plan to research and develop novel adeno-associated viral vectors known as “self-inactivating”, as well as lipid nanoparticle-based approaches to address the challenges of delivering drug-editing treatments. CRISPR-based genes at the retina.

Intellia will also be able to use the technological advances made thanks to this collaboration for all targets not included in the initial agreement.


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