Oak Hill Bio Ltd.
Oak Hill Bio is a clinical-stage biotechnology company focused on acquiring and developing rare disease drugs that have been deprioritized by big pharma. Their mission is to take the baton where others left off by developing late-stage rare disease drugs and bringing them across the finish line. They aim to transform discoveries into therapies for rare diseases, with a pipeline that includes candidates like rugonersen for Angelman syndrome and OHB-607 for complications of extremely premature birth.
Industries
Nr. of Employees
small (1-50)
Products
Rugonersen (antisense oligonucleotide for Angelman syndrome)
An antisense oligonucleotide therapeutic designed to degrade an antisense transcript in neurons to unsilence the paternal UBE3A allele and restore UBE3A expression; advanced into clinical development with Phase 3 planned.
OHB-607 (recombinant IGF-1/IGFBP-3 for complications of prematurity)
A recombinant human IGF-1/IGFBP-3 replacement therapy developed to increase IGF-1 exposure in extremely premature infants to reduce complications such as bronchopulmonary dysplasia; evaluated in preclinical and Phase 2a studies and currently in Phase 2b.
OHB-401 (oral plasma kallikrein inhibitor for diabetic macular edema)
An oral small-molecule inhibitor of plasma kallikrein in preclinical development for diabetic macular edema, progressing toward Phase 1 evaluation.
Rugonersen (antisense oligonucleotide for Angelman syndrome)
An antisense oligonucleotide therapeutic designed to degrade an antisense transcript in neurons to unsilence the paternal UBE3A allele and restore UBE3A expression; advanced into clinical development with Phase 3 planned.
OHB-607 (recombinant IGF-1/IGFBP-3 for complications of prematurity)
A recombinant human IGF-1/IGFBP-3 replacement therapy developed to increase IGF-1 exposure in extremely premature infants to reduce complications such as bronchopulmonary dysplasia; evaluated in preclinical and Phase 2a studies and currently in Phase 2b.
OHB-401 (oral plasma kallikrein inhibitor for diabetic macular edema)
An oral small-molecule inhibitor of plasma kallikrein in preclinical development for diabetic macular edema, progressing toward Phase 1 evaluation.
Services
Asset acquisition and in-licensing
Acquiring deprioritized late‑stage therapeutic assets and in‑licensing programs for continued clinical development.
Clinical development and trial sponsorship
Sponsorship and operational management of clinical trials including neonatal global studies and multi‑phase development programs.
Partnerships for manufacturing and commercialization
Negotiation and management of development, manufacturing, and commercialization agreements with external partners.
Preclinical and translational research
Conduct of nonclinical pharmacology, biomarker, and efficacy studies to support clinical entry and target validation.
Asset acquisition and in-licensing
Acquiring deprioritized late‑stage therapeutic assets and in‑licensing programs for continued clinical development.
Clinical development and trial sponsorship
Sponsorship and operational management of clinical trials including neonatal global studies and multi‑phase development programs.
Partnerships for manufacturing and commercialization
Negotiation and management of development, manufacturing, and commercialization agreements with external partners.
Preclinical and translational research
Conduct of nonclinical pharmacology, biomarker, and efficacy studies to support clinical entry and target validation.
Expertise Areas
- Rare disease drug development
- Antisense oligonucleotide therapeutics for CNS disorders
- Neonatal therapeutics and clinical development
- Recombinant protein therapeutics (IGF-1/IGFBP-3)
Key Technologies
- Antisense oligonucleotides (ASO)
- Recombinant protein therapeutics (IGF-1/IGFBP-3)
- Oral small-molecule plasma kallikrein inhibitors
- EEG-based pharmacodynamic biomarker (delta power)