The liver sits at the centre of metabolic control, detoxification, and immune regulation, yet it is also one of the hardest organs to treat safely in real-world patients with advanced dysfunction. Apterna’s liver-targeted RNA platform uses clinically validated GalNAc conjugate chemistry to deliver therapeutic RNA precisely to hepatocytes, enabling selective modulation of liver biology while minimising systemic exposure.
Precision-Delivered RNA Therapeutics Built on Two Complementary Platforms
Apterna’s science is built on a simple principle: RNA medicines only fulfil their promise when they reach the right cells safely, selectively, and efficiently. Our delivery technologies are designed to overcome the key limitations of conventional approaches, enabling organ-targeted programmes in the liver and the brain, two systems that often determine outcomes in metabolic disease, liver failure, and CNS-involved oncology.
Making RNA Therapeutics Organ-Specific, Safe, and Scalable
RNA medicine has expanded what is possible in drug discovery, but real-world impact depends on one decisive factor: delivery. Without organ-selective delivery, even the most promising RNA molecules cannot reach the right cells, achieve durable target engagement, or deliver sustained therapeutic benefit.
Apterna was founded to solve this problem. Our team has developed two complementary delivery platforms designed for two of the body’s most clinically challenging targets: the liver, where metabolic and inflammatory dysfunction can limit treatment options and outcomes, and the brain, protected by the blood-brain barrier, where effective systemic delivery remains a major hurdle. Both platforms are non-viral and chemically defined, combining molecular precision with scalable, manufacturable chemistry.
Targeting the Epicentre of Metabolic Disease
Preclinical Results:
- Favourable safety profile, supported by low systemic exposure and minimal immune activation
- High hepatocyte specificity, enabling efficient uptake with limited off-target distribution
- Durable activity, with the potential for dosing intervals measured in months rather than weeks
Applications:
Restoration of liver function in metabolic and inflammatory liver disorders
Potential to support patients with obesity-related hepatic dysfunction and advanced hepatic impairment
Platform expansion to additional liver pathways through strategic collaborations and licensing
Crossing the Blood–Brain Barrier Without Invasion
The brain is the most protected organ in the body, and that protection has long been one of the greatest barriers to treating CNS disease. Apterna’s brain-targeted platform uses a receptor-guided transport system designed to enable RNA and other macromolecular payloads to cross the blood-brain barrier following simple systemic administration.
Animal Model Results:
- Effective penetration into deep brain tissue following non-invasive systemic administration
- Successful delivery of multiple RNA payload types, including silencing, activating, and coding RNAs
- Modular architecture with potential adaptation across CNS oncology, neurodegenerative, and rare neurological indications
Validated Principles, Modern Engineering
Apterna’s technology builds on proven scientific foundations, enhanced with engineering advances that improve organ selectivity, consistency, and scalable development.
Specificity
Directing the therapeutic only to its target organ
Safety
Minimising off-target effects and systemic exposure
Stability
Engineering RNA constructs for optimal in-vivo persistence
Scalability
Using fully synthetic, non-viral chemistry compatible with global manufacturing standards
Adaptability
Allowing integration with multiple RNA formats
Specificity
Directing the therapeutic only to its target organ
Safety Is the Foundation of Every Apterna Programme
Safety sits at the centre of Apterna’s development philosophy. Every programme is designed and assessed through rigorous non-clinical evaluation, focusing on organ-selective biodistribution, immune tolerability, and longer-term safety in the target tissue.
- Advanced biodistribution profiling supported by state-of-the-art molecular imaging and quantitative tissue analytics
- GLP toxicology studies delivered with certified partners and regulatory-grade documentation
- Regulatory alignment with international standards and expectations, including ICH, GCP, and GMP pathways
- Early safety and pharmacovigilance planning to support robust monitoring from first-in-human studies onward
Discovery
Preclinical Safety
GLP Validation
Regulatory Readiness
First-in-Human
From Discovery to Clinical Proof-of-Concept
Apterna’s development roadmap is designed to de-risk progress at every stage. Each platform advances through clear, evidence-led steps, from molecular design and preclinical validation to regulatory readiness and clinical execution.
1
Target Discovery
Selection based on strong mechanistic rationale and unmet clinical need
2
Molecular Design
Optimisation of RNA constructs for potency, stability, and manufacturability
3
In-Vivo Validation
Testing for organ-specific delivery, biodistribution, and safety
4
Regulatory Preparation
Assembly of preclinical and CMC packages under ICH-compliant standards
5
Clinical Translation
Initiation of early-phase studies in defined high-need populations
Empowering the Broader RNA Ecosystem
While Apterna’s internal focus is on liver and brain indications, our delivery technologies are modular and partner-ready. We welcome collaboration with pharmaceutical, academic, and biotechnology teams seeking organ-targeted delivery for new RNA or nucleic acid programmes.
Platform Licensing
Platform Licensing for CNS or liver and metabolic applications
Co-development Agreements
Co-development for novel RNA payloads, combinations, or lifecycle extensions
Joint Research Ventures
Joint Research Ventures with universities and translational centres
Precision Delivery Is the Future of RNA Medicine — and It Starts Here
At Apterna, we believe RNA therapeutics should be as precise as they are powerful. Our dual-platform approach is designed to enable organ-targeted treatments that act at the source of disease while maintaining a strong focus on safety, manufacturability, and real-world clinical relevance.
We are preclinical, but we are building with intent: rigorous science, validated delivery principles, and a partnership-minded development model designed to support long-term therapeutic and commercial value.