Investors – Apterna | Precision Therapies for Liver Disease, Metabolic Syndrome & Obesity

Investing in the Next Generation of RNA Therapeutics

Apterna is advancing a new generation of organ-targeted RNA therapeutics built on clinically validated delivery platforms. Our mission is to demonstrate that RNA medicines can be both precise and practical — restoring liver function, reaching the brain, and transforming outcomes where conventional drugs cannot.

Precision. Partnership. Progress.

Brain-Targeted

Liver-Targeted

A De-Risked, Delivery-Led RNA Therapeutics Strategy

Apterna represents a unique investment opportunity in the fast-growing field of RNA therapeutics — distinguished not by its payloads, but by its delivery technology. Our two complementary platforms overcome one of medicine’s hardest challenges: ensuring that RNA molecules reach their target organs safely, effectively, and reproducibly.

Unlike traditional gene or RNA therapy companies that rely on viral vectors, Apterna’s systems are non-viral, fully synthetic, and clinically validated in concept. They are scalable, cost-efficient, and supported by regulatory precedent from previously approved RNA medicines.

Precision Delivery

Targeting specific organs (liver and brain) to maximise therapeutic effect and minimise side effects.

Translational Focus

Moving rapidly yet responsibly from preclinical validation to early clinical proof-of-concept.

Partnership Potential

Creating multiple entry points for investors and licensees through platform flexibility.

A De-Risked, Delivery-Led RNA Therapeutics Strategy

Apterna occupies a distinctive position within the RNA ecosystem — combining the scientific credibility of an academic spinout with the agility of a privately held biotech.

Clinically Anchored Science

Founded by pioneers in RNA delivery and hepatobiliary medicine from Imperial College London and City of Hope.

Validated Platforms

Both liver- and brain-targeted systems use delivery chemistries supported by FDA precedent.

High-Impact Indications

Targeting diseases with no effective treatment options — severe liver failure, NASH, and brain metastases.

De-Risked Development Model

Leveraging known delivery mechanisms reduces technical uncertainty and regulatory risk.

Scalable Manufacturing

Synthetic, non-viral production compatible with existing RNA manufacturing infrastructure.

Proven Leadership

Founders with multiple successful biotech exits, including EMcision (Boston Scientific) and Biohaven (Pfizer, $11B).

Partnering Pathways

Licensing-ready platform with broad potential across oncology, metabolic, and CNS indications.

Structured Growth Through Defined Milestones

Apterna’s development plan is staged to maximise value creation and maintain investor transparency. We are currently raising funds to complete preclinical validation and initiate first-in-human readiness for our liver-targeted programme.

Phase

Use of Proceeds

Timeline

Expected Outcome

Seed / Pre-Series A

Preclinical validation, IP expansion, CRO partnerships

2025–2026

Completion of IND/CTA-enabling studies for lead liver programme

Series A (Tranche 1)

Manufacturing scale-up, regulatory submission, initial clinical sites

2026–2027

First-in-human (Phase 1b) initiation in advanced liver disease

Series A (Tranche 2)

Extended clinical evaluation, platform data package expansion

2027–2028

Phase 1b topline data and platform partnership announcements

Series B (Prospective)

Additional clinical indications, CNS platform development

2028–2029

Expansion into fatty liver (MASH) and oncology programmes

A Dual-Platform Portfolio Designed for Sustainable Growth

Apterna’s two delivery platforms create a multi-asset pipeline within a single company structure. Each platform has standalone commercial potential and can generate value through internal development or external licensing.

Primary Value Drivers

Lead Clinical Candidate (Liver)

Targeted therapy for patients with advanced liver disease — early proof-of-concept opportunity.

Platform Expansion (Brain)

CNS and oncology delivery technology with broad licensing appeal.

Partnerships

Revenue-generating collaborations across pharma and biotech sectors.

Intellectual Property

Multi-jurisdictional protection through 2041 for composition and use.

Accelerating Discovery Through Partnership

Apterna welcomes partnerships that extend the reach and impact of its delivery technologies. Our platform architecture allows seamless integration with a variety of RNA, oligonucleotide, and biologic payloads.

Partnership Models

Platform Licensing

Access to liver or brain delivery systems for specific indications.

Co-Development Agreements

Joint discovery and preclinical studies on shared targets.

Strategic Alliances

Multi-year collaborations with pharma and research institutions.

Academic Collaborations

Translational partnerships for rare or early-stage indications.

Partner Benefits

A Proven Heritage in Translational Medicine and Biotech Entrepreneurship

Apterna’s leadership team has a consistent record of transforming scientific innovation into commercial and clinical success. Their combined experience spans multiple successful biotech ventures.

Habib 4X

Novel Bloodless Liver Resection Devices

MiNA Therapeutics

Small activating RNA therapeutics

Biohaven

Co-founded and chaired by Dr Declan Doogan

Apterna’s leadership team has a consistent record of transforming scientific innovation into commercial and clinical success. Their combined experience spans multiple successful biotech venturea

Apterna builds on this foundation of scientific credibility, executional discipline, and successful deal-making to deliver sustainable long-term value to investors and collaborators.

Creating Value Through Clinical Validation and Platform Partnerships

Apterna’s near-term objective is to generate first-in-human proof-of-concept in liver disease — a milestone that typically drives significant value inflection in RNA therapeutics. Beyond that, the company’s long-term vision is to establish its delivery platforms as enabling technologies across multiple therapeutic areas.

Potential Pathways for Value Realisation

Strategic acquisition by a large pharmaceutical company following Phase 1b/2a data.
Multi-indication licensing partnerships generating upfront and milestone revenue.
Joint ventures with pharma or biotech collaborators for specific disease programmes.
Progressive expansion into CNS and metabolic indications to diversify pipeline assets.

Strategic Advantages

Apterna’s development and governance structure is designed to be capital-efficient, partnership-oriented, and globally scalable — ensuring that scientific progress translates directly into tangible value creation.

Connect With Our Leadership Team

For investor relations, partnership discussions, or due diligence access, please contact us directly. We welcome confidential introductions and strategic conversations with qualified institutional investors, venture partners, and corporate development teams.

Investor Enquiries

investors@apterna.ltd

Partnership Enquiries

partnerships@apterna.ltd

Head Office

London, United Kingdom

Together, We Can Deliver the Next Generation of RNA Medicine

Apterna stands at the forefront of precision RNA delivery — advancing a clinically grounded, scientifically de-risked strategy toward transformative therapies for liver and brain disease. We invite investors and partners who share our vision of responsible innovation to join us in shaping the future of RNA therapeutics.