Cirrhosis & transplant avoidance
Enabled by proof of concept in the lead indication.
One focused beginning.A considered path ahead.
REGEN001 anchors our liver-first programme, with evidence-led progression from preclinical research towards clinical translation.
Explore REGEN001
OUR DEVELOPMENT PROGRAMME
Liver-first.
Evidence-led.
REGEN001 is Apterna’s lead programme, with an initial focus on restoring liver reserve in people with liver cancer who cannot currently access potentially curative surgery.
Expansion into other liver settings is intended to follow evidence from the lead programme.
LEAD PROGRAMME
REGEN001
Liver functional restoration
PreclinicalINITIAL SETTING
Liver cancer surgery
THERAPEUTIC APPROACH
Liver-directed RNA
NEXT MILESTONE
Lead selection & in vitro proof of concept
Discovery
PreclinicalCurrent programme stage
Phase 1
Phase 2
Programme overview
The programme is designed to support liver function in patients whose reserve limits access to curative care. The current source pipeline lists REGEN001 as preclinical, with lead selection and in vitro proof of concept as its next milestone.
Metabolic liver disease
Strategic expansion following validation in earlier liver settings.
PLANNED DEVELOPMENT
A considered path to translation.
PLANNED · 2026
Lead selection
Optimisation and in vitro proof of concept to test the intended biological effect.
Company development plan. Timelines are prospective and depend on funding, study outcomes, and regulatory review.
CONTINUE EXPLORING
Explore what comes next.
A CLEARER UNDERSTANDING
Questions about our work.
What is REGEN001?
REGEN001 is Apterna’s lead preclinical liver-directed RNA programme. It is being developed with the aim of restoring liver function in liver cancer patients whose poor liver reserve limits access to potentially curative surgery.
What stage of development is REGEN001 at?
REGEN001 is in preclinical development. The company’s plan includes lead optimisation, proof-of-concept work and disease-relevant validation before subsequent development decisions.
Is REGEN001 available as a treatment?
No. REGEN001 is investigational and is not an approved treatment. Development milestones are prospective and depend on funding, research outcomes and regulatory review.