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Ascepion Pharmaceuticals

Baltimore, Maryland, USAdebiopharm.com ↗

Ownership: Private
Development stage: Preclinical

Overview

Asklepion has achieved FDA approval for Cholbam® (cholic acid) capsules—the first treatment approved for pediatric and adult patients with bile acid synthesis disorders due to single enzyme defects. The lead investigational asset is intravenous L-citrulline targeting acute lung injury following pediatric cardiac surgery; this program has received orphan drug designation and reached Phase III clinical trials. Additional programs include therapies targeting sickle cell crises and gene therapies for muscular dystrophy.

Therapeutic areas: PulmonologyMetabolic DisordersRare DiseasesHematologyCardiovascular
Modalities: Small molecules (e.g., L-citrulline)Gene therapy (mini-dystrophin for Duchenne Muscular Dystrophy)Bile acid derivatives (cholic acid/Cholbam®)
Industry: Biotech

Drug pipeline

1 asset

ASP-08001

Preclinical

Partnerships

Retrophin/Nasdaq RTRX acquired rights to cholic acid asset from Asklepion.

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