Approach and Progress
What Precision Therapeutics Means to Us
Human genetics is at the core of our therapeutic and development strategies.
To build our pipeline, we focused on patients first — particularly patients with a high genetic risk profile. We assessed the functional implications of their underlying mutations. We then matched each molecular abnormality with an appropriate therapeutic candidate. We are currently pursuing three distinct genetically defined patient populations.
A Truly Multimodal Process
We’re focused on finding the best therapeutic solutions for patients — and we’re not limited to a single approach. We have carefully selected a panel of technologies well-suited to each target that human genetics has defined for us. This strategy has resulted in a broad and diverse pipeline, including monoclonal antibodies, recombinant proteins and gene therapies.
Our multimodal approach allows us to pursue a variety of attractive therapeutic targets not compatible with traditional inhibitor-based approaches.
Human genetics emphasizes the importance of a class of complement regulatory proteins. Loss of function mutations in these proteins can lead to complement dysregulation and risk of pathology both in the eye and systemically. Some of our candidates are designed to simultaneously suppress excessive complement activation and restore complement’s homeostatic roles crucial to maintaining health and preventing disease.
Our approaches are distinct from other complement therapeutics, which block the complement pathway and both the beneficial and detrimental effects of complement activation.
Our broad pipeline spans three genetically-defined targets, seven research projects, one development program and three biologic modalities including monoclonal antibodies, recombinant proteins and gene therapies.