Discovery
Approach
Our discovery approach begins with careful consideration of what diseases to target and how best to target them.  We then design cell based assays that closely model the fundamental biological processes that underlie the disease state.  These assays are then fed into our integrated suite of proprietary discovery, validation and development technologies.  This approach has two major benefits: the ability to identify best-in-class therapeutics and the ability to accelerate traditional drug discovery and development timelines.  The key points in our approach are as follows:

Identify those diseases where protein therapeutics have: 
  • a convincing medical utility
  • a compelling scientific rationale
  • a clear commercial opportunity
  • a reasonable path to the clinic

Design a suite of cell-based assays that best approximates the clinical indication: 
  • use primary human cells whenever possible
  • use the disease to model the assay, not vice versa
  • use multiple assay readouts
  • have the assay readouts measure the key intervention points, not proxies
  • make the assays flexible enough to allow for unexpected discoveries

Screen all human secreted proteins to rapidly identify the best therapeutic candidate(s): 
  • our world-class clone collection and proprietary bioinformatic analyses enable identification and cloning of all secreted proteins and receptor extracellular domains (ECD's)
  • integrated protein expression and assay systems allow high-throughput production and screening of our protein library
  • proprietary expression systems using human cells result in proper glycosylation patterns and therapeutically relevant protein levels

Confirm the candidate(s) using an innovative and rapid in vivo validation technology: 
  • validate function in vivo in weeks vs. months
  • determine efficacy in multiple animal models
  • simultaneously assess toxicological and pharmacological parameters
  • mimic therapeutic administration
Feed candidates downstream into development: 
  • leverage our platform technologies to screen for high-producing cell lines and optimize protein expression and production
  • evaluate and in-license proven technologies for rapid scale-up of protein production
  • build internal process development capabilities to enable transfer of processes to cGMP facilities

We believe that this integrated approach to discovery, validation and development maximizes the opportunity of discovering the best protein therapies for a broad variety of diseases and reduces the timelines from moving therapeutics from the bench to the clinic.