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BCX17725

Netherton Syndrome

Pre-Clinical/Phase 1Investigational

Key Facts

Indication
Netherton Syndrome
Phase
Pre-Clinical/Phase 1
Status
Investigational
Company

About BioCryst Pharmaceuticals

BioCryst Pharmaceuticals is a mission-driven, commercial-stage biotech focused on delivering first-in-class or best-in-class oral therapies for rare diseases with significant unmet need. Its strategic evolution from a discovery entity was cemented by the 2020 FDA approval of ORLADEYO® (berotralstat), a once-daily oral prophylactic for HAE, establishing a sustainable revenue base. The company leverages its proprietary structure-guided drug design platform to build a pipeline in HAE and complement-mediated diseases, aiming to deepen its market penetration and expand into new therapeutic areas like Netherton syndrome.

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SXR1096Sixera PharmaPhase 1
KB104Krystal BiotechPreclinical
QRX003Quoin PharmaceuticalsPhase 2/3