Transforming RNA Medicine Through Liver Rescue
Apterna is a UK-based, preclinical biotechnology company developing precision-delivered RNA therapeutics designed to restore liver function in advanced metabolic liver disease and liver-associated oncology. Our lead programme targets hepatic biology directly, helping remove the liver bottleneck that prevents many patients from safely starting, escalating, or remaining on GLP-1 receptor agonist therapy and other disease-modifying regimens.
Precision for the Liver
Our liver-directed platform uses GalNAc-conjugated siRNA to selectively deliver gene-silencing therapeutics to hepatocytes via the asialoglycoprotein receptor. This enables targeted modulation of disease-driving pathways in the liver while minimising systemic exposure, an approach designed for the realities of fragile patients with severe hepatic impairment.
Access to the Brain
Alongside our liver programme, Apterna has developed a receptor-guided delivery platform based on Transferrin Receptor (TfR) targeting to support systemic transport of RNA payloads across the blood-brain barrier. While outside the scope of our liver-focused development plan, this platform provides future partnering optionality in CNS oncology and brain metastases.
Science With Purpose
Our mission is to unlock new clinical options for patients who have been left behind by current treatments, including those with decompensated liver disease, advanced metabolic dysfunction, and liver cancer complicated by hepatic failure. We focus on programmes that can deliver meaningful outcomes in the clinic, such as enabling safer access to established therapies and improving tolerance of life-extending regimens.
Partnership and Progress
Apterna is built for collaboration. Our strategy is to advance the lead liver programme through IND and early human data, then partner with, or be acquired by, organisations with established franchises in GLP-1 therapies and liver oncology. By restoring liver function, we aim to broaden the addressable patient population for existing blockbusters while improving outcomes for patients who are currently ineligible or unable to tolerate standard of care.
Redefining RNA Therapeutics for the 21st Century
Apterna is a UK-based, preclinical biotechnology company developing a new generation of precision-delivered RNA therapeutics. Founded by scientists and clinicians from Imperial College London and the City of Hope, we are addressing one of the most important challenges in modern medicine: how to deliver RNA safely and effectively to the right organ, at the right dose, with the right durability.
Our work bridges two areas of profound unmet need. The first is metabolic and liver disease driven by obesity, where liver dysfunction can limit the real-world safety, escalation, and long-term durability of GLP-1 receptor agonist therapy. The second is oncology involving the brain, where effective delivery across the blood-brain barrier remains a central barrier to progress. Using validated, non-viral delivery technologies, Apterna’s goal is to demonstrate that RNA medicines can be both powerful and precise: acting exactly where they are needed, while minimising unwanted systemic exposure.
As a preclinical-stage company, we are preparing for IND and CTA-enabling studies for our lead liver-directed programme and continuing validation of our brain-targeted platform. Every experiment advances our vision of a future where complex diseases can be addressed at their molecular roots through safe, organ-selective RNA therapeutics.
Two Platforms. One Mission: Precision Delivery.
Apterna’s research is built around two complementary RNA delivery platforms, each designed to solve a different delivery challenge and unlock new clinical options.
Liver-Targeted RNA Platform
Our lead platform uses clinically validated GalNAc conjugate chemistry to deliver therapeutic RNA precisely to hepatocytes via the asialoglycoprotein receptor. By concentrating activity in the liver, we minimise systemic exposure and enable targeted modulation of liver inflammation, fibrosis, and metabolic dysfunction.
This approach is designed for high-need settings where liver impairment limits treatment choices, including obesity-driven metabolic liver disease and advanced hepatic dysfunction. A central objective is to restore hepatic resilience so more patients can safely initiate, escalate, and sustain therapies whose benefits are otherwise constrained in real-world care, including GLP-1 receptor agonists.
Brain-Targeted RNA Platform
Our second platform is a receptor-guided aptamer delivery system designed to enable systemic transport across the blood-brain barrier. It supports delivery of RNA and other biological payloads to the central nervous system and intracranial tumours, creating partnering and licensing optionality in indications where effective CNS delivery remains a core limitation.
Together, these platforms reflect a single mission: precision delivery that turns promising biology into practical, patient-relevant therapeutics.
Both platforms are non-viral, scalable, and modular, designed to accommodate diverse RNA modalities, including small-interfering, small-activating, and messenger RNA constructs.
Focused Where the Need Is Greatest.
Millions of patients worldwide suffer from diseases that remain out of reach for current therapies — either because drugs cannot access the target organ or because their toxicity outweighs their benefit.
Liver and Metabolic Disorders
- Targeting metabolic dysfunction and fatty liver disease associated with obesity
- Designed to restore liver function and prevent disease progression
- Aims to serve patients with limited or no access to approved therapies
Brain and CNS Disorders
- Targeting neurodegenerative and rare brain diseases with unmet need
- Designed to cross the BBB & restore function while reducing off-target effects
- Aims to reach patients who lack effective or durable treatments
Triple-Negative Breast Cancer
- Targeting aggressive TNBC with tumour-selective RNA therapeutics
- Designed to suppress drivers of growth and metastasis while sparing healthy tissue
- Aims to expand options for patients with limited or no targeted therapies
A Vision Turning Into Milestones.
Apterna’s lead RNA programme is advancing through the final stages of preclinical development, with first-in-human readiness targeted within the next two years. This programme is designed to restore liver function and improve survival in patients with severe hepatic impairment — a population underserved by today’s therapies.
Our second programme, built on the brain-targeted delivery platform, is focused on oncology indications involving the central nervous system. Early studies demonstrate the ability to transport RNA payloads across the blood–brain barrier, supporting a pipeline that could redefine treatment in neuro-oncology.
As we progress, our focus remains firmly on safety, reproducibility, and validation — ensuring that each step towards the clinic builds upon a foundation of robust preclinical evidence.
Building the Furure of RAN Medicine Together
Apterna’s model combines scientific excellence with strategic partnerships. We collaborate with leading academic institutions, biotech innovators, and manufacturing partners to advance our technologies efficiently and responsibly.
Our upcoming funding round aims to complete preclinical validation, prepare for regulatory submissions, and initiate our first human studies. We welcome engagement from investors and partners who share our belief that RNA therapeutics can change the trajectory of global health.
The Apterna leadership team has a proven record of biotech creation and successful exits — including companies that have achieved multi-billion-dollar acquisitions. With this heritage, Apterna is positioned to follow a similarly disciplined and value-driven path toward clinical translation and long-term success.
Latest Updates and Highlights
CONFERENCE
Apterna selected to present at the UK RNA Therapeutics Innovation Forum 2025
Join us as we showcase our latest developments in precision RNA delivery platforms.
RESEARCH UPDATE
Apterna reports BBB transcytosis data for brain-targeted RNA delivery in preclinical models
Early studies demonstrate efficient receptor-mediated transport and neuronal uptake, supporting our CNS technology pipeline.
PRESS RELEASE
Apterna to go ahead with GMP manufacturing and Toxicology
Apterna secures an early investment to go ahead with GMP manufacturing and Toxicology studies to scale iLNP platform for global first-in-human studies
Together, We Can Redefine What RNA Therapeutics Can Do.
Every breakthrough begins with collaboration. Whether you are an academic researcher, potential partner, or investor, we invite you to join us in advancing RNA medicine to new frontiers.