IntraBio’s platform technologies result from decades of research and collaboration with universities and institutions worldwide. Its clinical programs are based upon the expertise in lysosomal function and intracellular signaling of its scientific founders from the University of Oxford and the University of Munich.

The company's lead candidate is IB1001, an orally administered, modified amino acid (N-Acetyl-L-Leucine). In March 2024, IntraBio announced that the US FDA had accepted its New Drug Application (NDA) for IB1001 for the treatment of Niemann-Pick disease Type C (NPC). The NDA was based on the results of a Pivotal Phase III trial (IB1001-301) for adult and pediatric patients with NPC that met all primary and key secondary endpoints and showed IB1001 improved neurological signs and symptoms, functioning, and quality of life versus placebo.