Areas of Innovation
Reimagining what science can do
We believe that science opens the door to unlimited possibilities. That’s why we’ve created a research engine that’s powered by cutting-edge technologies like gene therapy, AI-driven protein engineering, protein manufacturing, RNA end-joining, and synthetic rescue. Our global research labs are driven by one goal: to create the next generation of therapies that have the potential to change the lives of people facing serious diseases.
We’re exploring the following areas of innovative research
Gene Therapy with Targeted Delivery
We’re exploring intrathecally-delivered gene therapies for a range of serious diseases.
Protein Deimmunization
Our proprietary protein deimmunization platform uses AI to reengineer proteins to determine how we can lower their immunogenicity.
RNA End-Joining Technology
We’re pursuing a proprietary technology that could unlock new possibilities in gene therapy by allowing us to target large genes.
Synthetic Rescue
Our scalable platform couples cutting-edge molecular and cell biology techniques with sophisticated analysis of human genetics datasets to potentially deliver new ways of treating diseases.
Proprietary Manufacturing
Our proprietary protein manufacturing platform aims to cut the cost, time, and complexity of producing therapeutic proteins and viral vectors for gene therapy.
Our commitment to innovation elevates everything we do
Leading the way in what’s next energizes all of us—and drives us to push the boundaries of possibility across our organization. These innovative platforms and technologies are synergistic with one another and complementary to the rest of our business. Taken together, they have the potential to reframe how we think about treating serious diseases.
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