Apterna · REGEN001

Restore function.Reopen possibility.

A three-minute introduction for investors.

The moment

A tumour the surgeon can remove.
A liver too weak to survive it.

The scan shows a small tumour. The surgeon can see exactly how to take it out. Then the blood tests come back, and the liver is too damaged to survive the operation.

The tumour is treatable. The patient is not.

They are offered palliative care instead.

The scale

This is a common situation

2.5 billion
people worldwide with a fatty liver
112 million
living with cirrhosis
~900,000
diagnosed with liver cancer each year
And at the moment that decides everything — whether the liver is strong enough — there is no approved medicine at all.
WHO, 2024 · Teng et al., 2023 · Luo et al., 2025 · Li et al., 2024
What we are building

One injection. One gene. One idea.

1

An injection under the skin

Given every few months, not every day.

2

It switches off a single gene

The one that holds a damaged liver back from recovering.

3

The liver does the rest

Take the brake off, and it repairs itself — as it has always known how to do.

REGEN001
Is it real?

In animals, it works

Doctors use two blood tests to decide whether someone is strong enough to be offered treatment at all. In published results, both were normalised.

✓

Albumin

normalised — the liver making what the body needs

✓

Bilirubin

normalised — the liver clearing what it should

—

No liver damage

an important result in its own right

This is early work. REGEN001 has not been given to a person yet. But it is real, it is published, and it points where we hoped it would.
Khorsandi et al., Molecular Therapy: Nucleic Acids, 2026
Who is behind it

They have done this before

Prof. Nagy Habib
Co-Founder, Chair & CEO

Professor of Liver Surgery, Imperial College London. Founded EMcision, sold to Boston Scientific. Co-founded MiNA Therapeutics — a $1.25bn agreement with Eli Lilly.

Dr Declan Doogan
Chief Medical Officer

Ran global drug development at Pfizer. Co-founded Biohaven, sold to Pfizer for $11bn.

Prof. John Rossi
Co-Founder · Science

Co-founded Dicerna, sold to Novo Nordisk for $3.3bn. Beckman Research Institute, City of Hope.

The idea is simple.The people have done it before.

If this is the kind of thing you back, we would like to hear from you.

Prof. Nagy Habib
Co-Founder, Chair & CEO
nagy@apterna.com
Mr Peter Bains
Co-Founder & Chief Operating Officer
peter@apterna.com
Apterna Ltd · Imperial Scale Space, 58 Wood Lane, London W12 7RZ

Apterna Ltd is a preclinical company. REGEN001 is investigational and is not an approved treatment. Nothing here is an offer of investment or a promise of future performance.