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

Transforming the Treatment Landscape for Liver and Brain Disorders

Apterna develops precision-delivered RNA therapeutics for diseases where organ access determines what is possible, from severe hepatic dysfunction in metabolic disease to CNS-involved oncology and brain metastases. Our dual-platform approach enables targeted, non-viral delivery to the liver and the brain, supporting programmes designed to restore function, expand treatment eligibility, and create partner-ready opportunities across high-need indications.

Liver-Targeted Delivery

Precision RNA therapeutics for hepatic disorders

Brain-Targeted Delivery

Breaking through the blood-brain barrier

Focusing on Conditions of Highest Unmet Need

Many of the most serious diseases remain undertreated because therapies cannot reach the right organ at effective concentrations, or because patients in advanced stages cannot tolerate available regimens safely.

Validated RNA Chemistry

Proven molecular modalities paired with clinically validated, organ-selective delivery

Precision Targeting

Direct delivery to hepatocytes and CNS tissues to maximise on-target effect

Reduced Toxicity

Lower systemic exposure to support tolerability in fragile patient populations

Apterna’s platforms are built to address the delivery barrier that limits RNA medicine. Our initial focus is liver-related disease, where obesity, alcohol use, and chronic inflammation drive a growing global burden, and CNS-involved oncology, where effective treatment remains constrained by the blood-brain barrier.

Restoring Liver Health Where It Matters Most

Liver disease is one of the fastest-growing contributors to global morbidity and mortality. Because the liver governs metabolism, detoxification, and inflammatory signalling, dysfunction can rapidly destabilise multiple organ systems and restrict access to life-extending therapies.

Spectrum of Liver Disease Progression

Fatty Liver (Steatosis)

MASH (Steatohepatitis)

Fibrosis

Cirrhosis

Decompensated Cirrhosis and HCC Risk

Liver Failure

Liver failure occurs when the liver loses its ability to perform essential metabolic and synthetic functions. In patients with cirrhosis or severe hepatic injury, treatment options are extremely limited.

Unmet Need:

• 30-40% of patients with primary liver cancer present with severe dysfunction
• Current drugs contraindicated due to hepatic toxicity
• Only palliative care available for many patients

Apterna's Focus:

Restore liver function to make patients eligible for standard therapies, extending life expectancy and quality of life.

Hepatocellular Carcinoma

HCC is the most common form of primary liver cancer, frequently developing on a background of cirrhosis, NASH, or viral hepatitis. Global incidence is rising due to the obesity and diabetes epidemic.

Unmet Need:

• 40% of HCC patients have underlying liver failure
• Prevents use of chemotherapy, immunotherapy, or surgery
• Median survival as short as three months

Apterna's Focus:

Improve hepatic function to enable combination with existing cancer treatments, addressing a gap no current therapy fills.

Fatty Liver Disease

Hepatic steatosis arises when excessive fat accumulates in the liver, existing in alcohol-related (ARLD) and metabolic dysfunction-associated (MASLD) forms.

Unmet Need:

• Affects 1 in 4 adults globally
• No approved pharmacological treatment
• Limited efficacy of lifestyle modification

Apterna's Focus:

Modulate metabolic pathways to reduce liver fat and prevent progression to cirrhosis or cancer.

NASH / MASH

NASH (reclassified as MASH) is the inflammatory, fibrotic form of fatty liver disease, representing a major public health challenge linked to obesity and metabolic syndrome.

Unmet Need:

• Global therapeutics market remains underdeveloped
• Candidate therapies failed due to poor liver specificity
• Systemic toxicity limits treatment options

Apterna's Focus:

Direct liver action addressing metabolic and inflammatory processes driving NASH, demonstrating improvements in key biomarkers.

Extending Precision RNA Delivery Beyond the Liver

Many aggressive cancers have a high propensity to spread to the brain. Once metastasised, these cancers become extraordinarily difficult to treat because most drugs cannot cross the blood-brain barrier. Apterna’s liver-first strategy also supports broader metabolic care by improving hepatic resilience and enabling safer initiation, escalation, and durability of GLP-1R+ therapy in real-world patients.

Blood-Brain Barrier Penetration

Systemic RNA delivery to CNS tissue

Unmet Need

Apterna's Brain Platform

From Organ-Specific Delivery to Systemic Innovation

Apterna’s delivery technologies are modular and partner-ready. While our internal focus is on liver-first programmes in metabolic disease, including GLP-1R+ enablement, and oncology involving the brain, the same platforms can be adapted across a broad range of indications where organ-selective delivery determines clinical success.

Rare Liver Disorders

Genetic enzyme deficiencies and inherited metabolic conditions

Neurodegeneration

Alzheimer's, Parkinson's, and related CNS disorders

Musculoskeletal

Metabolic-inflammatory disorders affecting joints and muscles

Combination Therapies

RNA therapeutics with immunotherapy and gene repair tools

Platform Vision

By enabling precise control over where and how RNA drugs act, Apterna’s platform opens new frontiers for the entire field of nucleic acid medicine.

A Vision for Accessible RNA Medicine

Apterna’s ultimate goal is to make RNA therapy safe, targeted, and accessible. By focusing on delivery systems that are chemically defined, non-viral, and compatible with standard manufacturing, we aim to keep costs manageable and ensure broad patient access.

Global Access

Affordable RNA therapeutics for worldwide patient populations

Education

worldwide patient populations Education Advancing scientific understanding of precision RNA delivery

Collaboration

Partnerships to address real-world disease burden