Collaborations A Long-Term Focus On Innovation

Regeneron and Intellia Therapeutics, Inc. are working together to advance potentially groundbreaking applications of CRISPR/Cas9 technology to address genetic diseases. CRISPR/Cas9 therapy holds promise to potentially cure disease with a single dose by selectively inactivating disease-causing genes (“knock-out” approach) and/or restoring necessary genetic functions by targeted insertion of a functional DNA sequence into the genome of patients deficient in an essential gene (“knock-in” approach).

"Regeneron and Intellia are working hard to find safe and innovative solutions to rare genetic diseases. We believe the precise CRISPR/Cas9 in vivo gene editing therapeutics jointly developed with Intellia could yield great advances in many diseases where significant unmet needs currently exist," said David Gutstein, M.D., Vice President, Global Program Head at Regeneron.

In the early days of the pandemic, Roche and Regeneron came together to develop, manufacture and distribute Regeneron’s investigational monoclonal antibody cocktail to treat and prevent COVID-19 to as many people as possible in record time.

While successfully completing the clinical development program, Regeneron and Roche moved swiftly to start producing the medicine at manufacturing sites around the globe — a herculean effort to increase the supply and reach more patients at a crucial and devastating time during the pandemic. The Roche collaboration more than tripled manufacturing capacity, and ultimately millions of doses of the antibody cocktail were supplied around the world.

"This major collaboration with Roche provided important scale in record time, enabling our medicine to reach millions of patients in the United States and around the globe. Together, we delivered when the world needed it most and provided an important option for those suffering from, or at high-risk for, COVID-19," said Kerry Reinertsen, Senior Vice President, Strategic Alliances at Regeneron.

In an example of a commercial-stage agreement, Regeneron granted Kiniksa Pharmaceuticals Ltd. an exclusive license to develop and commercialize ARCALYST in many countries around the world. As Regeneron’s very first therapy to enter the market, ARCALYST is an interleukin-1 (IL-1) blocker first approved in 2008. Since entering into the licensing agreement in 2017, Kiniksa has led the evaluation of ARCALYST in diseases believed to be mediated by both IL-1α and IL-1β. ARCALYST is now being commercialized in multiple indications.

“As our first FDA-approved treatment, ARCALYST exemplifies Regeneron’s commitment to discover and develop important medicines for patients with serious diseases, whether they are highly prevalent or ultrarare,” said Scott Mellis, Vice President, Early Clinical Development and Experimental Sciences, Rare Disease, at Regeneron. “Our agreement with Kiniksa has enabled expansion of approved indications so that this important medication can help many of those affected by IL-1-driven disease.”