Oak hill bio

What Oak hill bio does

Oak Hill Bio (legal entity OHB Pediatrics Ltd.) is a clinical-stage biotechnology company founded in 2024 and headquartered in Cambridge, Massachusetts with UK operations, focused on acquiring and developing rare disease therapies that have been deprioritized by large pharmaceutical companies. The company's stated strategy is to take late-stage assets across the finish line where big pharma corporate strategy shifts have interrupted promising programs. The lead asset is rugonersen (OHB-724), an antisense oligonucleotide acquired from Roche in February 2025 that targets the UBE3A-ATS transcript to restore paternal UBE3A expression in Angelman syndrome, a rare neurodevelopmental disorder affecting approximately 500,000 individuals globally with no approved disease-modifying treatments. Phase 1 TANGELO trial results published in Nature Medicine in July 2025 demonstrated dose-dependent normalization of EEG biomarkers and developmental gains above natural history; a pivotal Phase 3 study is expected to initiate in mid-2026.

The pipeline includes three programs. Rugonersen is the lead, targeting Angelman syndrome via intrathecal antisense oligonucleotide delivery. OHB-607 is a recombinant IGF-1/IGFBP-3 replacement therapy in a Phase 2b trial (Footprints study) partnered with Chiesi to prevent complications of extreme prematurity. OHB-401 is an oral small molecule plasma kallikrein inhibitor in preclinical development for diabetic macular edema, with Phase 1 planned for mid-2026.

The business model is asset acquisition and clinical development, with future revenue intended from commercialization of rare disease therapies through specialty pharmaceutical distribution channels and patient advocacy group partnerships upon regulatory approval. The company is pre-revenue, having raised $32.5 million in a June 2026 Series A financing co-led by Balyasny Asset Management, venBio, and Janus Henderson Investors. Customers are patient populations: primarily individuals with Angelman syndrome (~30,000 diagnosed in US and EU5), with secondary targets in extreme preterm infants and diabetic macular edema patients. Distribution will involve clinical trials, expanded access programs for eligible patients, and post-approval specialty rare disease channels with engagement from organizations such as the Angelman Syndrome Foundation and FAST.

Name

Oak hill bio

Legal name

OHB Pediatrics Ltd.

Website

https://oakhillbio.com

Company type

Private

Founded year

2024

Operating status

Operating

Headcount range

11–50 employees

Short description

Oak Hill Bio is a clinical-stage biotechnology company that acquires and develops late-stage rare disease drugs deprioritized by big pharma. Its lead asset rugonersen, an antisense oligonucleotide licensed from Roche, is advancing to Phase 3 for Angelman syndrome.

Ownership category

akta.pro rank

Product category

Rare Disease Therapeutics

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714)

SIC

Services-Commercial Physical & Biological Research (8731)

akta.pro primary industry

Genetic & Genomic Rare Disease Therapeutics (HLAIAIAA)

akta.pro secondary industries

Rare Endocrine & Growth Disorder Therapies (HLAIAIAJ), Rare Ophthalmology Disorder Therapies (HLAIAIAK), RNA Therapeutics Clinical Development (trial design, endpoints, patient stratification) (HLAAADAH), RNA Chemistry & Oligonucleotide Engineering (backbones, modifications, stereochemistry) (HLAAADAE)

Keywords

• Rare disease therapeutics
• Antisense oligonucleotide therapy
• Clinical-stage biotechnology
• Orphan drug development
• Neurodevelopmental disorder treatments

Headquarters

HQ city

Altrincham

HQ country

United Kingdom

HQ region

Europe

Offices2 records

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Personnel, Technology or R&D, Operations, Marketing or Sales, Infrastructure

Revenue model

1. Pharmaceutical product sales: Pre-revenue clinical-stage biotech company. Revenue will be generated through commercialization of rare disease therapies upon regulatory approval. The company acquired global rights to rugonersen from Roche in February 2025 and plans to advance it through Phase 3 and regulatory approval.

Go-to-market motion1 record

Distribution channels2 records

Marketing channels5 records

Core offering

Oak Hill Bio is a clinical-stage biotechnology company that acquires and develops late-stage rare disease drug programs that have been deprioritized by large pharmaceutical companies. Its pipeline centers on rugonersen (OHB-724), a Phase 3-ready antisense oligonucleotide licensed from Roche for Angelman syndrome, supported by OHB-607, a recombinant IGF-1/IGFBP-3 therapy for complications of extreme prematurity being co-developed with Chiesi, and OHB-401, an oral plasma kallikrein inhibitor in preclinical development for diabetic macular edema.

Product overview

Oak Hill Bio is a clinical-stage biotechnology company that acquires and develops rare disease drugs deprioritized by large pharmaceutical companies. The company's pipeline consists of three programs: Rugonersen (OHB-724), an antisense oligonucleotide for Angelman syndrome in Phase 3 preparation; OHB-607, a recombinant IGF-1/IGFBP-3 therapy for complications of prematurity in Phase 2b with partner Chiesi; and OHB-401, an oral small molecule for diabetic macular edema in preclinical development.

Differentiator

Problem solved

Functional benefit

Products and services

• Rugonersen (OHB-724) Investigational antisense oligonucleotide designed to restore UBE3A protein expression in neurons by suppressing the UBE3A-ATS transcript and unsilencing the paternal UBE3A allele. Phase 3-ready for the treatment of Angelman syndrome, a rare genetic neurodevelopmental disorder with no approved disease-modifying therapy. Selected as the most potent compound in its class after screening approximately 2,500 LNA-modified ASOs.
• OHB-607 Proprietary recombinant protein complex combining insulin-like growth factor 1 (IGF-1) with its binding protein IGFBP-3. Designed as a replacement therapy to compensate for IGF-1 deficiency in extremely preterm infants (less than 28 weeks gestational age) and prevent complications including bronchopulmonary dysplasia. Co-developed with Chiesi in the Footprints Phase 2b study.
• OHB-401 Oral small molecule plasma kallikrein (pKAL) inhibitor in preclinical development for diabetic macular edema, leveraging established research in plasma kallikrein inhibition.

Quantifiable outcome

• Dose-dependent partial normalization of pathological brain activity on EEG delta power biomarker in Angelman syndrome patients
• +3 more outcomes

Segments2 records

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature3 records

Partnerships

Two partnerships are on record, tiered core and major.

• RochecoreOEM/ Whitelabel/ Licensing PartnerExclusive license agreement in February 2025 for global rights to rugonersen (RO7248824), a Phase 3-ready antisense oligonucleotide for Angelman syndrome. Roche originally developed rugonersen and conducted Phase 1 TANGELO trial.
• ChiesimajorStrategic or Co-development PartnerPartner for OHB-607 development; Chiesi is involved in the Phase 2b trial (Footprints study) for OHB-607 to prevent complications of prematurity.

Scale indicators3 records

Recent moves7 records

Expansion highlights6 records

Market position

Competitive moat3 records

Key risks6 records

Key highlights6 records

Customer concentration

• LinkedInlinkedin.com/company/oak-hill-bio

Revenue estimate

Valuation estimate

Number of profiles

Profiles9 records

Funding overview

Funding rounds1 record

Investors4 records

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Oak hill bio