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Speaker 1: We've got a key. We as a first person in

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Speaker 1: the world to trial the groundbreaking gene therapy. So it's

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Speaker 1: a gene silencing drug targets a form of muscular dystrophy

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Speaker 1: known as FSHD. It if it's one in eight thousand trials.

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Speaker 1: Principal Advisor describes the drug as being as significant as

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Speaker 1: having antibiotics versus not having antibiotics. Associate Professor Richard Rocksbury

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Speaker 1: as well, it's Richard, morning to you.

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Speaker 2: Oh, good morning.

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Speaker 1: What's the technology that's allowed us to get here? Has

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Speaker 1: something happened? Have we discovered something changed something?

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Speaker 2: Yeah, In the last ten years or so, various drug

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Speaker 2: companies have been working very hard on finding ways to

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Speaker 2: interrupt the messenger RNA at that level and do that safely.

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Speaker 2: So it's been a big and also finding ways to

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Speaker 2: direct that technology to the muscles where the problem is.

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Speaker 1: Right, So that was ten years ago. They've been working

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Speaker 1: on that since they got that breakthrough. Has that been

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Speaker 1: a complicated journey?

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Speaker 2: Yes, As you know, there's been a series of experiments

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Speaker 2: in the lad doing that first with cells and then

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Speaker 2: with animals of course, then and now we're ready for

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Speaker 2: human trials. Yeah, And what is.

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Speaker 1: It about FSHD that's different from any other form of

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Speaker 1: muscular dystrophy, and that this particular drug may well work

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Speaker 1: for it.

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Speaker 2: Well, it's unusual because the whole our bodies actually do

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Speaker 2: everything we can to suppress the expression of a gene

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Speaker 2: called Ducks four. So we're basically by doing something where

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Speaker 2: we silence this gene, we're actually doing the job that

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Speaker 2: the body is trying to do for itself. But with

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Speaker 2: people who have these conditions have a genetic abnormality, so

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Speaker 2: that the Ducks four escapes from the surveillance and suppression.

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Speaker 1: Is gene silencing the future and all sorts of things.

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Speaker 1: If we can isolate one, isolate out the gene for

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Speaker 1: whatever it may be, and then go about silencing it,

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Speaker 1: you can solve pretty much anything, not.

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Speaker 2: Just about everything, because some things are the problem is

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Speaker 2: a lack of you know, the genetic abnormality causes a

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Speaker 2: lack of something. But there are some diseases, such as

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Speaker 2: f SHD, where there's an over expression of something toxic.

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Speaker 2: Another example would be my atonic dystrophy. That's that's that's

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Speaker 2: probably the commonest muscular dystrophy in New Zealand, and that

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Speaker 2: we've already got trials, which is showing amazing benefits for

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Speaker 2: the patients.

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Speaker 1: And when you talk about gene silencing, so you're not

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Speaker 1: I mean, you're not solving the problem. It's like a headache.

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Speaker 1: You've still got the head out. You just can't feel.

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Speaker 1: Is it that simple or not?

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Speaker 2: Yeah, it's well, it's no, you don't. Don't you don't

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Speaker 2: have it's gene silencing in that we're not taking away

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Speaker 2: the gene. So you're right, it's not getting we're not

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Speaker 2: doing crisper at this stage. Maybe that's for the future,

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Speaker 2: but we're not changing the gene itself. But we're changing

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Speaker 2: the very first message from the DNA. Your body makes

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Speaker 2: messenger RNA, which goes out to that the cells to

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Speaker 2: make proteins. And it's at that very first step where

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Speaker 2: the DNA makes messenger RNA. We're interrupting at that very

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Speaker 2: fundamental level. So really getting to the root of things

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Speaker 2: that would.

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Speaker 1: Not about side effects at this point and whether or

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Speaker 1: not it works with everybody, Oh.

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Speaker 2: Good question. I mean, the whole of this this thing

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Speaker 2: where the New Zealanders are having the first doses and

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Speaker 2: last week and this week, the whole purpose of this

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Speaker 2: study is to make sure it's safe.

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Speaker 1: All right, will we when will we know? And when's

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Speaker 1: the path to commercialization?

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Speaker 2: Huh, Well, it's early days, but I was if it

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Speaker 2: works as well as this other study that we've just had,

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Speaker 2: I would think over the next three years, it may

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Speaker 2: may maybe in clinical practice and within about three years time.

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Speaker 1: It's amazing, all right, Richard go well, appreciate it very much.

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Speaker 1: Richard Rocksborough, the trials principal investigate. It's amazing what science

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Speaker 1: can do, or will do, or is about to do.

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