RTX-001

Name: RTX-001 Name (English): RTX-001

Description: RTX-001 is an innovative autologous engineered macrophage cell therapy being evaluated for the treatment of decompensated liver cirrhosis. It is classified as an autologous macrophage therapy, utilizing the patient's own immune cells. The macrophages are engineered with IL-10-MMP9 mRNA to enhance their anti-inflammatory and anti-fibrotic effects. RTX-001 is administered as a dispersion for infusion.

Mechanism of Action: The treatment involves collecting the patient's blood cells, modifying them in a laboratory to become specialized macrophages (RTX-001), and then infusing these modified cells back into the patient. The goal is to improve liver function and reduce complications associated with decompensated liver cirrhosis by leveraging the regenerative properties of these engineered macrophages.

Clinical Trial: RTX-001 is currently in a Phase 1/2 open-label, multicentre study called EMERALD (NCT06823713) to evaluate its safety, tolerability, and efficacy in patients with decompensated liver cirrhosis. The study is being conducted in the UK and Spain and began recruiting in October 2024.

Eligibility Criteria (for the EMERALD trial):

• Age 18 to 75 years
• Diagnosis of liver cirrhosis
• Recent hospitalization for a major hepatic decompensation event
• MELD score between 12 and 20
• No recent alcohol misuse
• And other specific criteria

Exclusion Criteria (for the EMERALD trial):

• Liver cirrhosis due to specific viral hepatitides or autoimmune/cholestatic etiologies
• Acute liver disease without underlying cirrhosis
• Current organ failure requiring more than outpatient supportive care not related to the qualifying hepatic decompensation
• Known splenomegaly ≥16 cm
• And other specific criteria

Primary Outcome Measures (for the EMERALD trial):

• Safety and tolerability
• Major clinical events, including death