Home/Pipeline/Rugonersen (OHB-724)

Rugonersen (OHB-724)

Angelman Syndrome

Phase 3Active

Key Facts

Indication

Angelman Syndrome

Phase

Phase 3

Status

Active

Company

Oak Hill Bio

About Oak Hill Bio

Oak Hill Bio is a UK-based biotech with a unique 'relay' business model, acquiring and advancing late-stage rare disease programs abandoned by larger pharmaceutical companies. Its lead assets are rugonersen (OHB-724), an ASO for Angelman syndrome entering Phase 3, and OHB-607, a recombinant protein for bronchopulmonary dysplasia prevention in preemies, currently in Phase 2b with partner Chiesi. The company operates with a lean, asset-centric approach, aiming to de-risk development and deliver first- or best-in-class therapies to underserved patient populations.

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Therapeutic Areas

Rare Disease Neonatology Neurodevelopmental Disorders Ophthalmology

Other Angelman Syndrome Drugs

Drug	Company	Phase	Status
HLX-0553	Healx	Preclinical	Active
Undisclosed Pipeline Programs	AS2 Bio	Pre-clinical	Active
GTX-102	Ultragenyx Pharmaceutical	Phase 1/2	Active
NNZ-2591	Neuren Pharmaceuticals	Phase 2	Active