Raymond Wong - Centre for Eye Research Australia

Elaine Wziontek 0:01

With information on the latest developments in assistive technology and initiatives from the studios of 2RPH in Sydney and around the country on the Radio Reading Network of Australia, we bring you Ablequest. Hello. I'm Elaine Wziontek. 

Many millions of people worldwide suffer from vision loss through damage to the retina. To date, there is no preventative or repair treatment available for conditions such as retinitis pigmentosa or age related macular degeneration, where faulty or damaged photoreceptors lead to blindness or impaired vision, but hope is on the horizon - as we hear today from our guest, leading stem cell biologist, Associate Professor Raymond Wong, who is head of cellular reprogramming research at the Centre for Eye Research Australia or CERA in Melbourne. 

Raymond and his team are working on a breakthrough approach to treatment which harnesses the regenerative power of the retina's own stem cells to prevent and reverse damage to the light sensing photoreceptors in the back of the eye to accelerate the pathway from the pre clinical stage to The market. Raymond has co founded with CERA, a gene therapy startup called Mirugen. Through Mirugen as a vehicle, the team at CERA aimed to switch on site for the millions of people living with incurable blindness. Barbara Sullivan spoke to Associate Professor Wong about this research effort at CERA.

Barbara Sullivan 1:43

Welcome to Ablequest, Ray, if I may call you that - we are so interested in having you on today on Ablequest, because many of our listeners live with vision loss. It's a daily challenge to lead an independent life with incurable blindness. So new research into alleviating, or in fact, reversing eye disease and damage is of great interest to us. So thank you for your time.

Raymond Wong 2:05

Thanks so much for having me.

Barbara Sullivan 2:07

As I understand it, to provide a vehicle for your groundbreaking research, which we will talk about. You've co-founded a startup, Mirogen, at CERA - the Centre for Eye Research Australia. But before we talk about Mirugen and your progress with that, what are the particular eye conditions you're addressing?

Raymond Wong 2:24

Certainly. So the problem we're addressing is blindness associated with the degeneration of the photoreceptors. So these are the light sensing cell at the back of our eye, in the retina, and that specifically is a common hallmark for many different form of blindness, including retinitis pigmentosa, age-related macular degeneration, thyroid guide disease and many more forms of blindness.

Barbara Sullivan 2:50

What exactly causes the blindness in these cases?

Raymond Wong 2:54

So the primary indication of our research is retinitis pigmentosa, and in this case it is this is caused by genetic mutations that resulted in the loss of photoreceptors and that ultimately lead to blindness. And unfortunately, once the photoreceptor cells are lost, there's really no cure for blindness at the moment.

Barbara Sullivan 3:18

And in the case of age related macular degeneration, which is in this category, I suppose that's more about damage, isn't it? Rather than genetic loss.

Raymond Wong 3:29

Genetics, it's also a contributing factor. But in age-related macular degeneration, the reason is more complex, but ultimately there is a degeneration of the photoreceptor that cause patient impairment.

Barbara Sullivan 3:43

And approximately how many people suffer from these conditions?

Raymond Wong 3:47

In the case of retinitis pigmentosa, we know there are more than one million people worldwide that is affected by retinitis pigmentosa - but overall, in general, when we talk about photoreceptor degeneration, affecting the many different form blindness that I mentioned, collectively, that's more than 190 million people worldwide, so a substantial number of patients with vision impairment.

Barbara Sullivan 4:13

So you touched on your research - can you explain the main focus of it?

Raymond Wong 4:17

Yes, I'll be very happy to do that. Our research is developing a gene therapy to stimulate the retina to regenerate new photoreceptors to treat blindness, and this will be in the form of an injection. We're using viruses to carry a set of reprogramming genes where we can inject them into the eye, targeting a specific stem cells within the retina called the Muller glial cells, and using our... using these genes to reprogram them, we can turn them into forming new photoreceptors to effectively restore vision in the patient. 

Now the Muller glial cells are really interesting. And in the retina, it turns out that in lower organisms such as fish and chicken, when the when the retina is injured, the Muller glial cells can regenerate the whole retina in effectively repair vision. So it's a remarkable regenerative ability, but this regenerative ability is lost in higher mammals, and we think that our gene therapy is a novel approach to unlock this regenerative ability in the retina to treat blindness.

Barbara Sullivan 5:31

You mentioned injecting the eye... at the moment with certain conditions people need to such as macular degeneration, people need to have injections every couple of months. You're talking here about a kind of single injection.

Raymond Wong 5:46

That's right, yeah, we're looking at a single injection that can reprogram the stem cells into forming photoreceptors.

Barbara Sullivan 5:54

This is very encompassing, this research. Do you break that down into particular projects that you work on sequentially or in parallel? Can you work in parallel in order to speed it up?

Raymond Wong 6:06

Oh, yes. So we have some projects that are working in sequential order. Some of them are in parallel to accelerate the progress. So for example, we're working on a number of projects. We're working on developing a human gene Atlas. So this is really a map that allows us to understand all the genes that are and the genetic signals that's happening in human retina cell types. And the reason to do that is that gives us a good idea of what genes we need to switch on in order to turn the stem cells into photoreceptors. 

We're also working on optimising the cell reprogramming technology that allow us to stimulate retina regeneration. And in parallel, we're also working on improving the gene delivery method to the retina using this kind of viruses called [?Adenosoci] viruses, right?

Barbara Sullivan 7:00

And you talk about, we, you've got a team working for you.

Raymond Wong 7:04

That's right, yeah. So I am very fortunate to have great collaborators and mentor and my team members at the Centre for Eye Research Australia. So you know, we have tremendous support from the Centre for Eye Research Australia in particular. Our Managing Director, Professor Keith Martin, is very supportive of the project. He's also the co-founder of the spinoff company Mirogen, that is focusing on commercialising the retina gene therapy. My lab has some really incredibly talented researchers - in particular, Dr Daniel Urrutia, my postdoc fellows is driving a lot of the research that is happening in the lab right now. 

And we're also leveraging the expertise in Centre for Eye Research Australia, both in terms of basic science and also the clinicians. We have a fantastic collaboration with many of them, and here we also have a dedicated of ophthalmic clinical trial centres, at [?Sherulia], and a strong partnership with the Royal Victorian Eye Hospital. So, you know, we're very fortunate to have this really, really good research environment that enable our work to happen.

Barbara Sullivan 8:15

That's really the big guns, isn't it? I mean, it's... fantastic. And how does the startup that you talked about, Mirugen, fit into the picture. What is its particular purpose?

Raymond Wong 8:16

So Mirugen is a startup biotech company that's being incubated at the Centre for Eye Research Australia. The focus of this is to develop this gene therapy to stimulate photoreceptor regeneration. In my case, I would like to translate my research to help patients with vision impairment. That's my primary goal. And the way I see it is commercialisation is the essential pathway to bring our research funding, research findings in the lab to the clinic. And with Mirugen, we're looking to accelerate our work so this can happen faster with this commercialisation pathway. So we're really excited to come on this journey with Mirogen.

Barbara Sullivan 9:07

It's an interesting name. Does it have a particular meaning,Mirugen?

Raymond Wong 9:11

I'm glad you asked. (THEY LAUGH) We've put some thoughts into the name. So Miru, it's the Japanese word for vision or view, and Gen is gene therapy. So this is really paying homage to the Japanese discovery of the cell reprogramming technology, and in particular by Professor Shinya Yamanaka, who is really a pioneer in this field. And...he won the Nobel Prize for discovering cell reprogramming technology to generate stem cells. So he is also my scientific hero. This is in many ways related to this early reprogramming technology.

Barbara Sullivan 9:49

Now you've recently had a funding coup for Mirugen, something of a Christmas present. I believe it happened in December. Tell us about that.

Raymond Wong 9:57

Oh yeah, that's the best Christmas present we've got. Okay, I'm really happy to say that Mirugen was awarded... funding from the curator grant, and that's, that's a $1.92 million of funding. So this is a federal funding under the Medical Research Future Fund that is supporting biotech companies to accelerate translational research. So we're really excited to be awarded this prestigious funding - and that enable us to really advance our research.

Barbara Sullivan 10:30

So when you get funding like that, almost $2 million which is substantial, I'm sure there are conditions placed on that, other milestones that you have to meet - for example, with your projects that you outlined before?

Raymond Wong 10:43

Yes, absolutely. So with Mirugen, we've set up a development plan where we have to hit certain milestone in order to continue to get the funding. The Curator Plus program itself is actually a $5 million program, so there's an opportunity to get more if we hit the milestone. But this is all very milestone driven and looking to generate a very favorable outcome, to develop and advance our research to become a drug treatment. Yes,

Barbara Sullivan 11:12

Well, when you marry what you're doing with the numbers of people that are going to have a positive impact, if you've got a positive outcome, I'm sure that there will be a continued flow of funding. Do you feel confident it'll be sustained? 

Raymond Wong 11:26

Well, so far, we're we are fortunate to be supported by, you know, various funding, and you're absolutely right. You know, sustain the funding is critical for our research to happen. And you know, in the past, we've been supported by National Health and Medical Research Council, the current Medical Research Future Fund, and also retina, Australia, national stem cell Foundation of Australia, Australia, vision research and many other forms of philanthropic funding. We're all very grateful to this to get us to this point, and we are really excited about the potential of our research. 

So in many ways, we must continue so there will be ongoing fundraising and appropriate time point. But you know, I think there is tremendous potential with our research that I am personally very excited to advance this progress.

Barbara Sullivan 12:17

Well, it's not something you'll solve in a in a short time frame, even though you're accelerating your pre clinical work to get it to commercialisation. So what's your overall timeframe? Do you think and what are the various stages ahead of you? 

Raymond Wong 12:30

So our research is still at the early stages. We're at the pre-clinical stages, and that means we are working through optimisation of manufacturing efficacy and also safety, and those are really important before we bring it to clinical trial for, first in human testing in the patient, and we see that there will be a few years ahead of us before we head to clinical trial - but we're just really, really excited about the progress - we have certainly seen the work being accelerated in the past few years, and there's also a lot of new technologies coming in, into the current area, so we have to work hard.

Barbara Sullivan 13:13

Yes, now, if anyone listening wants to find out more, presumably, the CERA newsletter that seems to come out every month often highlights this, and people can go to the website and keep track of where you are.

Raymond Wong 13:25

Yeah, so the CERA website would be possibly the best way to keep track of it. There will be regular news release. We do have a regular newsletter that we send out to the general public, and there's a way to sign up for the newsletter online, through our CERA website. I believe we're also looking to publish our research, so that's also a good indication of where things are.

Barbara Sullivan 13:48

Well, it's extremely interesting, and I wish you the very best. I'd love to check in with you again before too long, Raymond to see how you're going.

Raymond Wong 13:55

Thank you so much for having me.

Barbara Sullivan 13:57

I have been speaking with Associate Professor Raymond Wong of the Centre for Eye Research Australia at the University of Melbourne - for more information, CERA's website is C, E, R, A, dot org, dot A,U.

Elaine Wziontek 14:15

You've just been listening to Ablequest - a program that looks at developments in assistive technology and initiatives. From Barbara Sullivan and Elaine Wziontek, thank you for listening and goodbye till next program.