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pp-277

Solution for injection/infusion

Dossier type
US CTD
Dossier status
Under development
Country of origin
United States
GMP approvals
FDA
Comments
Indication: Hematological disease, Immune disorder, Gastrointestinal disease Clinical stage: Phase 2 Modality: RNA therapeutic Mechanism & Target: Genetic modulator, Pathway modulator, Gene target Route & form: SC injection / IV infusion Differentiation: A best-in-class potential complement factor B (CFB) - targeting siRNA therapeutic for Paroxysmal Nocturnal Hemoglobinuria (PNH) in Ph2/3 stage. Complement-mediated kidney diseases (IgAN, Lupus Nephritis, C3G, IC-MPGN) in Ph2 stage
Manufacturer #39686

A clinical-stage biopharmaceutical company focused on immunology / inflammatory diseases. Developing a RNA-based therapeutic targeting immunology / inflammatory diseases with global development focus. Pipeline positioned for regulatory advancement in key markets.

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