Pipeline

We develop breakthrough therapies for neonatology and rare diseases, bringing science-driven solutions to patients who need them most.

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:

Oak Hill Bio Pipeline
CANDIDATE
INDICATION
MODALITY
PRE-CLINICAL
PHASE 1
PHASE 2
PHASE 3
STATUS
Rugonersen
Angelman
Syndrome
Antisense Oligonucleotide
Phase 3 start in Q1 2026
OHB-607
Complications
of Prematurity
Recombinant Protein
With Chiesi
Phase 2b trial restarted
Q2 2024
OHB-401
Diabetic
Macular Edema
Small Molecule
Phase 1 start in mid-2026
Oak Hill Bio Pipeline
Candidate
Progress
Rugonersen
Phase 3 start in Q1 2026
OHB-607
With Chiesi
Phase 2b trial restarted
Q2 2024
OHB-401
Phase 1 start in mid-2026
PROGRAMS

Rugonersen

Best-in-class antisense oligonucleotide for Angelman syndrome.
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OHB-607

First-in-class therapy for neonatology with transformational potential.
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OHB-401

Best in-class plasma kallikrein inhibitor for diabetic macular edema
Learn More →