Clinical Trial Update: A First-in-Human Safety and Efficacy Study of ALN-CFB, a Small Interfering RNA (siRNA) Targeting Complement Factor B, in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria With Persistent Anemia on a C5 Inhibitor [Phase 1, Phase 2]

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Summary

Phase 1/2 first-in-human trial evaluating ALN-CFB, an siRNA against complement factor B, for adults with paroxysmal nocturnal hemoglobinuria and persistent anem…

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Diseases / indications

• Anemia
• Paroxysmal Nocturnal Hemoglobinuria

Drugs mentioned

• ALN-CFB (siRNA targeting complement factor B)

Snippet

Sponsor: Regeneron Pharmaceuticals | Conditions: Paroxysmal Nocturnal Hemoglobinuria (PNH), Persistent Anemia | Phase 1, Phase 2 | Status: RECRUITING