Restore function.Reopen possibility.
A three-minute introduction for investors.
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.
This is a common situation
One injection. One gene. One idea.
An injection under the skin
Given every few months, not every day.
It switches off a single gene
The one that holds a damaged liver back from recovering.
The liver does the rest
Take the brake off, and it repairs itself — as it has always known how to do.
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
They have done this before
Professor of Liver Surgery, Imperial College London. Founded EMcision, sold to Boston Scientific. Co-founded MiNA Therapeutics — a $1.25bn agreement with Eli Lilly.
Ran global drug development at Pfizer. Co-founded Biohaven, sold to Pfizer for $11bn.
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.
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.