IntraBio is at the forefront of developing innovative treatments for rare neurodegenerative diseases, focusing on their lead investigational drug, IB1001, which is an orally administered modified amino acid known as N-Acetyl-L-Leucine. This compound is designed to address significant unmet medical needs in conditions such as Niemann-Pick Disease Type C (NPC) and GM2 Gangliosidosis. IntraBio has conducted extensive clinical trials, including positive Phase IIb and Phase III studies, demonstrating that IB1001 can significantly improve motor and cognitive functions, quality of life, and overall cognition in both pediatric and adult patients suffering from these debilitating diseases.

The company’s commitment to advancing treatment options is evident through its rigorous clinical trial programs, which have yielded promising results. For instance, the Phase III trial (IB1001-301) for NPC showed substantial improvements in symptoms, while other trials for GM2 Gangliosidosis and Ataxia-Telangiectasia have also reported statistically significant benefits. IntraBio's research findings are published in leading medical journals, underscoring the scientific validity of their work and the potential impact of IB1001 on the treatment landscape for rare neurological disorders.

United Kingdom

IntraBio Ltd., which is a subsidiary of IntraBio Inc, is a private pharmaceutical company located in Oxford, United Kingdom, focusing on the testing and development of novel and repurposed drugs to treat rare neurodegenerative diseases including lysosomal storage disorders.

IntraBio

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