At Intellia, we are building a full-spectrum, product-driven biotechnology company focused on developing and commercializing curative genome editing treatments that positively transform the lives of people living with severe and life-threatening diseases. Our approach to advancing the broad potential of genome editing includes our plans to:
- Focus on indications that enable us to fully develop the potential of the CRISPR/Cas9 system
- Aggressively pursue in vivo liver indications, both independently and in collaboration with Regeneron, to rapidly develop therapeutics with customized modular delivery technology
- Continue to develop and expand our engineered cell therapies on immuno-oncology and autoimmune/inflammatory diseases, as well as our collaboration with Novartis that focuses on hematopoietic stem cell-based treatments and enhanced CAR T oncological therapies
- Continue to leverage strategic partnerships to accelerate clinical development
- Grow our leadership position in the genome editing field
A multi-pronged diversified strategy
To maximize our opportunity to develop clinically successful products, we have applied a multi-pronged approach to selecting our initial disease targets, or “sentinel indications.” We chose those indications that have significant unmet medical needs, using four primary selection criteria:
- The type of edit involved
- Delivery approach – in vivo or ex vivo
- Presence of established therapeutic endpoints
- Potential benefits compared to existing therapies
These selection criteria allow us to build a pipeline that is not reliant on any single delivery technology or editing approach for success. In addition, we can apply the learnings from these sentinel indications to inform our selection of subsequent indications and targets of interest. Our approach serves to increase our probability of success and broadens the opportunity for potential strategic alliances to accelerate clinical development.
In short, we envision a better way to develop cures for patients.