Our Mission - Valentina's Village

The Plan

There is no treatment for Hyaline Fibromatosis Syndrome anywhere in the world.
So we are funding the creation of one, made specifically for Valentina.  

The science exists. The team exists.
What we need is the funding to get her there.

What We’re Building

Valentina’s condition is caused by a fault in a single gene, ANTXR2. We are funding a personalised genetic medicine built for that exact fault.

The treatment is called an ASO, short for antisense oligonucleotide.

Genes work like instructions. Before a cell acts on one, it makes a working copy of it. An ASO is a short, made-to-measure strand of genetic material that attaches to that working copy and changes how Valentina’s body reads it.

It does not permanently alter her DNA. That is a large part of why the team chose it.
It is lower risk, and it can be given again as she grows.

It is built for Valentina’s exact mutation. It would not work for anyone else in the world.

Why the Strategy Changed

Our early research pointed towards a gene therapy using a vector approach. As the team looked harder at Valentina’s condition, a different approach turned out to be the safer and more suitable one.

There is no single place to treat.

HFS affects cells lining blood vessels throughout the body. Gene therapies work best when there is one organ to aim at. Here there isn’t one.

The effect would fade.

Those cells replace themselves every few years, and the benefit would go with them.

You only get one attempt.

A vector gene therapy therapy cannot be given a second time. An ASO can be given again, and again, for as long as Valentina needs it.

There is no single place to treat.

HFS affects cells lining blood vessels throughout the body. Gene therapies work best when there is one organ to aim at. Here there isn’t one.

The effect would fade.

Those cells replace themselves every few years, and the benefit would go with them.

You only get one attempt.

A vector gene therapy therapy cannot be given a second time. An ASO can be given again, and again, for as long as Valentina needs it.

An ASO also works without permanently changing her DNA, which makes it the lower-risk option.

An ASO also works without permanently changing her DNA,
which makes it the lower-risk option.

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Who is Doing the Work

Our partner is Gene2Cure, a non-profit that develops genetic medicines for children with rare and ultra-rare conditions. They run the programme end to end, hold the contracts, and answer to us.

Prof. Leszek Lisowski

Leads the science for Gene2Cure. He is one of the world’s leading scientists in genetic medicine delivery.

Prof. Gina Ravenscroft’s team

At the Harry Perkins Institute of Medical Research in Perth identified the exact fault in Valentina’s gene, and continues to research the condition.

The treatment itself is being developed by a specialist laboratory working under contract to Gene2Cure.

How Your Money is Controlled

This is a service agreement, not a donation.

Gene2Cure contracts the laboratory to do the work. They set the timelines, they own the data, and if the work slows down they can take the programme elsewhere.

Funding is released
against milestones.

Each stage ends with a decision about whether the science is working. If it isn’t, we find out early rather than after years of spending.

Nothing is wasted
between stages.

Whatever is left at the end of a phase moves to the next.

We hear from the team every quarter,

and more often when there’s something real to report.

This is a service agreement, not a donation.

Gene2Cure contracts the laboratory to do the work. They set the timelines, they own the data, and if the work slows down they can take the programme elsewhere.

Funding is released
against milestones.

Each stage ends with a decision about whether the science is working. If it isn’t, we find out early rather than after years of spending.

Nothing is wasted
between stages.

Whatever is left at the end of a phase moves to the next.

We hear from the team every quarter,

and more often when there’s something real to report.

We believe in complete transparency,
so you can see exactly how funding is used.

We believe in complete transparency, so you can see exactly how funding is used.

Prof. Leszek Lisowski Leading the science for Gene2Cure

Prof. Leszek Lisowski
Leading the science for Gene2Cure

Where Your Money Goes

We’re working with Gene2Cure on a full costing for Valentina’s treatment, broken down stage by stage.

When it’s ready we’ll publish it here in full, so you can see exactly what your donation pays for. We would rather show you real numbers than approximate ones.

Donate

Being Honest About the Road Ahead

Pioneering research comes without guarantees, and we want to be clear about that. At every stage, the evidence will determine what happens next.

If the science supports moving forward, the work will continue. If it doesn’t, we will communicate openly and explore every responsible alternative.

Your support helps fund a rigorous scientific process, pursued with care, accountability and the determination to do everything possible for Valentina.

Fund the Cure

Changing Things Beyond Valentina

Valentina is the only person in Australia reported to have her condition. That means almost every system built to help children falls over when it reaches her.

So alongside funding her treatment, we are talking to government about two things.

Funding for ultra-rare conditions,

so families like ours are not left raising millions on their own.

Access here at home,

so that when a treatment like Valentina’s exists,
it can be given to her in Australia rather than overseas.

We are at the beginning of that conversation.
But we would rather start it now than leave it for the next family.

The Science Exists.

The Team is in Place.

With the funding behind them, the next step becomes possible.

HELP FUND THE NEXT STEP

Valentina’s Story Our Mission Gala Dinner

Life in the village

Valentina's Village Ltd is a registered charity with the Australian Charities and Not-for-profits Commission (ACNC).
ABN 95 700 589 064

Tax status: Valentina’s Village Ltd has applied for Deductible Gift Recipient (DGR) status. Donations are not currently tax deductible. If DGR is granted we will contact everyone who has given about receipts.