intrabio.comCrawled Oct 18, 2026
IntraBio’s lead compound IB1001 (N-acetyl-L-leucine) is in clinical trials for Niemann-Pick disease type C and other neurological disorders.
Ataxia-Telangiectasia is a rare, inherited disorder caused by mutations in the ATM gene, affecting the cerebellum and central nervous system.
IB1001, an orally administered modified amino acid, is being developed for orphan indications like GM2 Gangliosidosis and Ataxia-Telangiectasia.
A Phase III trial showed levacetylleucine improved neurological signs, symptoms, and functioning in pediatric and adult patients with Ataxia-Telangiectasia, with favorable safety and tolerability.
AQNEURSA is the only FDA-approved therapy for Niemann-Pick disease type C, showing improvements in neurological symptoms and function in adult and pediatric patients.
The European Commission approved AQNEURSA (levacetylleucine) for treating Niemann-Pick Type C disease in the European Union.
Cass Fields leads communications, marketing, brand, strategic partnerships, and patient advocacy for IntraBio Inc. across North America and Europe.
Board of Directors Mallory Factor, ChairmanFull bio above Elizabeth Factor, Chief Admin OfficerFull bio above Christopher YegenInvestor and Philanthropist Dr. Ali KhanDean of Public Health School, University of Nebraska Medical School. Former Director of Office of Health Emergency Preparedness at CDC
Professor Mallory Factor is the founder, president, and CEO of IntraBio Inc., with a career in launching and investing in early-stage companies, including medical device startups.
Professor Michael Strupp is a scientific founder of IntraBio Inc. and a professor at the University of Munich’s Department of Neurology, specializing in vestibular, ocular motor, and cerebellar disorders.
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IntraBio develops therapies for neurodegenerative diseases with high unmet medical needs, leveraging expertise from the University of Oxford and University of Munich.
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IntraBio is a leader in discovering, developing, and commercializing novel therapies and treatments for rare and neurodegenerative diseases with high unmet medical needs.
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IntraBio focuses on developing therapies for rare and common neurodegenerative diseases, addressing unmet medical needs in patient communities.