Oak Hill Bio is a clinical-stage biotechnology company focused on acquiring and developing late-stage rare disease drug candidates deprioritized by larger pharmaceutical companies. The company advances these therapeutic assets through clinical development to achieve regulatory approval and market access. This strategy aims to bring necessary treatments for rare conditions, such as Angelman syndrome and complications of extreme prematurity, to patients who otherwise face significant delays.
Funding
Funding not disclosed
Founders
Product
Problem
Many rare disease drugs are deprioritized during development due to corporate strategy shifts, leaving patients with unmet needs and promising therapies abandoned mid-development. Extremely preterm infants face severe complications due to underdeveloped organs, while individuals with Angelman syndrome lack disease-modifying therapies to address the underlying genetic cause.
Solution
Oak Hill Bio acquires and develops late-stage rare disease drugs that have been deprioritized by big pharma, bringing them to market for patients in need. Their pipeline includes rugonersen (OHB-724), an antisense oligonucleotide (ASO) in Phase 3 development for Angelman syndrome, designed to restore activity of the intact paternal UBE3A gene. They are also developing mecasermin rinfabate (OHB-607), a recombinant human IGF-1/IGFBP-3 currently in Phase 2b trials, for complications of extremely premature birth, addressing low levels of IGF-1 in these infants to promote organ development. Additionally, OHB-401, an oral small molecule plasma kallikrein inhibitor, is in preclinical development for diabetic macular edema.
Target Audience
The primary target audience includes patients with Angelman syndrome, extremely preterm infants at risk of complications, and individuals with diabetic macular edema, as well as the healthcare providers who treat these conditions.
Features
- Rugonersen (OHB-724): Antisense oligonucleotide (ASO) that binds to the UBE3A-ATS transcript, unsilencing the paternal UBE3A allele in Angelman syndrome patients.
- OHB-607: Recombinant human IGF-1/IGFBP-3 replacement therapy designed to address IGF-1 deficiency in extremely premature infants.
- OHB-401: Oral small molecule plasma kallikrein inhibitor for diabetic macular edema, targeting inflammation and edema in retinal blood vessels.
- ASO designed to trigger degradation of the UBE3A-ATS transcript in the CNS.
- IGF-1 replacement therapy to promote continued development and maturation of vital organs and vasculature.
- pKAL inhibition with an oral small molecule.