Our client, a global leader in the development of pioneering therapies for rare genetic diseases, is looking for transformational therapeutic approaches for sickle cell disease (SCD) and beta-thalassemia. Specifically, our client is seeking disease modifying approaches using antibody, small molecule, oligo or in vivo gene therapy treatment approaches.
Approaches should target the underlying mechanisms of disease to transform outcomes. Approaches that only address the symptoms of sickle cell disease are not of interest, unless they also address the root ...