ALN-CFB / Regeneron, Alnylam - LARVOL DELTA

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 (clinicaltrials.gov) - P1/2 | N=24 | Recruiting | Sponsor: Regeneron Pharmaceuticals | Trial completion date: Jan 2030 ➔ Jul 2031

First-in-human • Trial completion date • Anemia • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases