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>> Haemophilia B is a rare bleeding disorder.
It affects around one in 30,000 males.
There's a problem with blood clotting.
In patients with haemophilia,
they lack a protein called Factor IX.
Factor IX is a protein that circulates in blood
and when we cut ourselves it starts the coagulation of blood
to protect us from bleeding excessively.
Now, in patients with haemophilia,
the gene that's responsible for making factor IX is damaged.
>> There is currently a therapy.
Patients can be given recombinant protein,
but it has to be given very frequently.
It's extremely expensive
and therefore not readily available throughout the world
and it's a very invasive process.
So, it is a disease that's in need of better treatment.
>> One way of treating this condition is
to replace the damaged gene with a normal copy
of fully functional gene.
And so the way we've decided to deliver this gene to the liver
which is where factor IX is usually made is to use viruses.
We've taken out the properties of the virus which is important
for causing disease and replaced it with the factor IX gene
and so what we're doing essentially is using the virus
as a vehicle to deliver our gene to the liver.
In the incubators are cells that we use to make the virus.
These are our production line for making the vector.
When we treat patients, we infuse it directly
into the patient's vein and that's essentially it;
that's the treatment done.
>> It is impressing
that literally somebody is injecting cells
that making factor IX into a vector that is a virus.
When I cut myself shaving for example, the bleeding just stops
which wasn't happening before and I feel better
in general I don't have small bleeds anymore,
bruises and stuff like that.
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>> The collaboration between St. Jude
and University College London has really been critical
to the success of this project.
>> We've been able to develop this project together
and accelerate its passage to clinic.
Before it can used by the wider haemophilia community,
it needs to go through several other steps to show
that the safety aspects of this treatment are robust
and long lasting.
And for this treatment to become available,
it may take another five to ten years.
>> We're also interested in opening this project to children
who may in fact benefit more than adults from this approach.
>> I think the real take on this research is
that it has a potential to revolutionise the lifestyle
of these patients, to revolutionise haemophilia care.
>> This kind of a solution is something permanent
that can make a difference for a lot of people.
>> If we can make it work for haemophilia,
then there is every hope that we can make it work for a variety
of other disorders including diabetes, including cancer.
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