Early Stage Proof Of Concept & Pharmacology And Toxicology Studies

From iPS cell to large animal models, our pre-clinical establishment has successfully served as the basis for IND submissions.   Our iPS core has generated multiple ocular disease models, while our large animal colonies have been used in multiple proof of concept studies. CAROT will assist investigators in the design and implementation of preclinical safety and efficacy studies that can ultimately support an ophthalmic gene therapy clinical trial. While every disease target is unique, suggestions for paths forward using in vivo and/or in vitro testing will be provided. CAROT will also assist with generating timelines, budgets, developing protocols and planning data analyses. Guidelines will draw upon CAROT’s substantial experience in designing and carrying out preclinical studies for Phase 1-3 human ocular gene therapy clinical trials.