Intellia is a pioneer in the development of CRISPR/Cas9 genome editing and is focused on rapidly moving transformative therapies towards the clinic. Our company’s unique strengths include the expertise of our team in discovering and developing genome editing products, our proprietary versatile lipid nanoparticle delivery system, and our focus on both in vivo and ex vivo product development.
We are proud to count biopharmaceutical companies and leading academic institutions as partners – organizations that share our determination to revolutionize medical science.
We seek strategic collaborators to help them unlock the full potential of CRISPR/Cas9 genome editing. We scout for cutting-edge technologies that advance the application of CRISPR/Cas9 for human therapeutics and our genome editing platforms, including:
- Genome modifying technologies
- Recombination and DNA repair technologies
- RNA therapeutic technologies
- Therapeutic delivery of nucleic acids or ribonucleoproteins – viral and chemical
- Engineered immune cell technologies
- Novel target identification
If you are interested in exploring genome editing science and its translation into human therapies, or if your technology could advance the science, please contact us.
- In December 2014, Intellia entered into a strategic collaboration and license agreement with Novartis, focusing on accelerating the development of new ex vivo CRISPR/Cas9-based therapies using chimeric antigen receptor T cells and hematopoietic stem cells (HSCs). View Press Release
- In December 2018, Intellia and Novartis expanded their cell therapy collaboration to research ex vivo CRISPR/Cas9-based cell therapies using certain ocular stem cells. View Press Release
- The collaboration portion of the agreement ended in December 2019, with each party continuing to advance selected programs after the collaboration term. Unselected rights reverted to Intellia.
- In March 2020, the U.S. Food and Drug Administration (FDA) accepted an Investigational New Drug (IND) application submitted by Novartis for a sickle cell disease treatment. This therapeutic candidate is based on genome editing of HSCs, using CRISPR/Cas9 RNA guides identified through Intellia’s cell therapy research collaboration with Novartis. Intellia is eligible to receive additional downstream success-based milestones and royalties. View Press Release
- In parallel with its ex vivo collaboration with Novartis, Intellia has been advancing its wholly owned ex vivo pipeline of immuno-oncology and autoimmune cell therapies.
- Novartis provides innovative healthcare solutions that address the evolving needs of patients and societies. Headquartered in Basel, Switzerland, Novartis offers a diversified portfolio to best meet these needs: innovative medicines, cost-saving generic and biosimilar pharmaceuticals and eye care. Novartis has leading positions globally in each of these areas. In 2019, the Group achieved net sales of USD 47.4 billion. Novartis Group companies employ approximately 119,000 full-time-equivalent associates. Novartis products are sold in approximately 155 countries around the world. For more information, please visit http://www.novartis.com.
- In April 2016, Intellia entered into a license and collaboration agreement with Regeneron. Under the agreement, the companies are researching, developing and commercializing CRISPR/Cas-based therapeutic products primarily focused on genome editing in the liver. Intellia and Regeneron are also researching and developing novel technologies and improvements to CRISPR/Cas technology to enhance our therapeutic genome editing platform. View Press Release
- In June 2020, Regeneron and Intellia expanded and extended their collaboration by two years, from 2022 to April 2024, with Regeneron having an option to extend by two more years after that. In connection with this collaboration expansion, Regeneron paid an additional $100 million through a $70 million upfront cash payment and $30 million equity investment. View Press Release
- As part of the updated 2020 agreement, Intellia and Regeneron are co-developing potential hemophilia A and B CRISPR/Cas9-based treatments using their jointly owned targeted transgene insertion capabilities. Regeneron is leading development for both hemophilia A and hemophilia B development programs.
- Under the terms of the original 2016 agreement, Regeneron had the exclusive right to discover and develop CRISPR-based therapeutic products against up to 10 target genes, focused primarily on editing genes in the liver. In June 2020, Regeneron gained rights to develop five additional in vivo liver targets, increasing their target cap to 15 (excluding the hemophilia A and B programs being co-developed by Regeneron and Intellia). Intellia may receive milestones and royalties for in vivo targets developed by Regeneron alone, and share proportionally on any profits generated by co-developed in vivo products.
- Intellia’s first in vivo product candidate, NTLA-2001, seeks to treat transthyretin amyloidosis (ATTR), and is being co-developed with Regeneron under a co-development and co-commercialization (Co/Co) agreement. As the lead party, Intellia plans to submit an IND or IND-equivalent in mid-2020 for NTLA-2001.
- Intellia’s hereditary angioedema (HAE) program is subject to an option by Regeneron to enter into a Co/Co agreement, under which Intellia would remain the lead party. Intellia expects to submit an IND or IND-equivalent for NTLA-2002 for the treatment of HAE in the second half of 2021.
- As of June 2020, Regeneron also has a royalty-bearing, non-exclusive license to certain Intellia intellectual property (IP) to independently develop and commercialize up to 10 ex vivo CRISPR/Cas9 products limited to defined cell types. Intellia’s license to Regeneron does not include access to IP directed to NTLA-5001, Intellia’s wholly owned development candidate for the treatment of acute myeloid leukemia (AML), or other IP directed to Intellia’s ex vivo targets, programs or proprietary cell engineering processes.
- Regeneron (NASDAQ: REGN) is a leading biotechnology company that invents life-transforming medicines for people with serious diseases. Founded and led for nearly 30 years by physician-scientists, Regeneron’s unique ability to repeatedly and consistently translate science into medicine has led to six FDA-approved treatments and over a dozen product candidates in development. Regeneron’s medicines and pipeline are designed to help patients with eye disease, heart disease, allergic and inflammatory diseases, pain, cancer and infectious and rare diseases. Regeneron is accelerating and improving the traditional drug development process through its unique VelociSuite® technologies and ambitious initiatives such as The Regeneron Genetics Center, one of the largest genetics sequencing efforts in the world. For additional information about the company, please visit www.regeneron.com or follow @Regeneron on Twitter.
- In June 2017, Intellia entered into a three-year research collaboration, option and license agreement with Ospedale San Raffaele (OSR) to engineer optimized T cell-based cancer therapies. View Press Release
- The collaboration has resulted in Intellia’s first ex vivo development candidate, which is a T cell therapy for acute myeloid leukemia targeting the Wilms’ Tumor protein (WT1). In December 2019, Intellia exercised its option to obtain an exclusive license to OSR’s intellectual property developed under the collaboration, including patent applications directed to WT1-specific T cell receptors.
- OSR is a leading European research-university hospital near Milan, Italy. Scientific research at OSR covers virtually all fields of biomedical research. OSR seeks to integrate basic, translational and clinical research to provide the best assistance to patients. For further information, visit hsr.it.