Pipeline
Our mission is to develop breakthrough rare disease therapies for the patients and families who need them.
Pipeline Overview
Our pipeline includes two late-stage therapeutic candidates: rugonersen (OHB-724), an antisense oligonucleotide (ASO) for Angelman syndrome, and mecasermin rinfabate (OHB-607) for complications of extremely premature birth. We expect to initiate a pivotal Phase 3 trial for OHB-724 in Q1 2026. OHB-607 is currently in a Phase 2b trial:
Candidate
Anticipated Milestones
Rugonersen
OHB-607
OHB-401
