Drug Development and Clinical Trials

Drug discovery and therapy development has been an ongoing focus of the Department of Genetics and Genomics Sciences and the Icahn Genomics Institute (IGI) for many years with great success.

We have developed novel therapeutics and repurposed methods for drug discovery to treat a variety of genetic diseases. Some examples include the development—from preclinical studies through human clinical trials—of enzyme replacement therapy for Fabry disease (FDA-approved Fabrazyme®) and Niemann-Pick type B disease, development of pharmacological chaperone therapy and performing the first preclinical adeno-associated virus mediated gene therapy studies for Fabry disease, as well as preclinical studies for an RNA interference therapy for Acute Intermittent Porphyria.

We also have a longstanding history of designing and conducting innovative clinical trials for patients with rare genetic disorders through our Clinical Trials Office. These include first in human studies to late-stage pivotal clinical trials, with various drug mechanisms from enzyme-based therapies to gene therapies.