Overview
Our lab’s research focuses on questions residing at the interface of red blood cell (RBC) physiology, transfusion medicine, and complement biology. We leverage the use of various tools including pre-clinical animal models, human samples and microfluidics to explore the cellular mechanisms and effects of complement dysregulation underlying hyperhemolysis, thrombotic microangiopathy and organ injury in various diseases. The overall goal is to translate the basic and translational research findings to improve the care of patients with blood disorders.
He is actively involved in research and advocacy through United States Thrombotic Microangiopathy consortium.
Research Projects
- Explore the mechanisms of how complement proteins interact and modulate hemolysis and multi-organ damage in sickle cell disease
- Investigate pathways driving transplant associated thrombotic microangiopathy (TA-TMA)
- Use of endothelialized microfluidic model to study the role of complement and other cellular proteins on endothelial dysfunction in thrombotic microangiopathy (TMA)
- Study of the rheology (deformations and flow) of RBCs in red blood disorders
- Testing of new drugs for complement mediated TMA using novel in vivo and in vitro methods
- Various clinical research projects on thrombotic microangiopathies including atypical hemolytic uremic syndrome, paroxysmal nocturnal hemoglobinuriaand thrombotic thrombocytopenic purpura
Clinical Trials
- PI. APL2‑PNH‑209. An Open-Label, Single-Arm, Phase 2 Study to Evaluate the Safety, Pharmacokinetics, and Biologic Activity of Pegcetacoplan in Pediatric Patients with Paroxysmal Nocturnal Hemoglobinuria. 09/05/17- open
- PI. BO42452 (Crosswalk-a). Roche/Genentech. A Phase IB Randomized, Placebo-Controlled Study Evaluating the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Crovalimab for the Management of Acute Vaso-occlusive Episodes (VOE) in Patients with Sickle Cell Disease. 03/13/23- open
- PI. AG348-C-022. A Phase 3, Multicenter, Randomized, Double-blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Subjects With Pyruvate Kinase Deficiency Who Are Regularly Transfused, Followed by a 5-Year Open-label Extension Period. 2023- open
- PI. AG348-C-023. A Phase 3, Multicenter, Randomized, Double-blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Subjects With Pyruvate Kinase Deficiency Who Are Not Regularly Transfused, Followed by a 5-Year Open-label Extension Period. 2023- open