Marnie:

In some ways, I’ve been working on a new therapy for a rare neurodevelopmental disorder, Prader-Willi syndrome (PWS), for my entire career. That’s over 20 years, so I have some bias and baggage!

As a venture principal scientist with WEHI Ventures, Nick brings a commercial mindset that’s completely different to mine and he comes to the project with fresh eyes. Having his expertise in our team makes us do better science.

There’s no effective treatment for PWS and it’s very medically complex, with symptoms like intellectual disability, low muscle tone and an insatiable appetite, often leading to obesity.

Our focus is on a protein called SMCHD1. We’re designing a drug that will target and destroy SMCHD1’s function. If we take away its mechanism, we can switch on some genes that should treat the cause of PWS.

Discovering SMCHD1 was an incredible career milestone. I discovered this protein myself, so it’s my ‘science baby’. When I started my scientific journey, I thought I’d be working at the basic end of science. I never imagined I’d be working on a world-first therapy to improve lives for hundreds of thousands of patients worldwide, and based on something that I discovered.

Nick’s intellectual capital is fast- tracking us towards a treatment for PWS. He has a venture capital outlook, but also the scientific skills to converse on the most technical level.

It takes a village for a medical discovery to finally reach patients. Critical funding like the investment from 66ten – WEHI’s first strategic investment fund, managed by WEHI Ventures – buys us the capacity of people’s time and expertise, which pushes us forward faster.

Other key players in ‘our village’ are the experts at the National Drug Discovery Centre (NDDC), based at WEHI, and also Professor James Murphy. With their skills, we discovered the chemical candidates that will help develop the future SMCHD1 inhibitor drug.

They analysed over 300,000 chemicals to make this discovery – like finding a needle in a haystack. You can’t do this enormous body of work on a shoestring and you can’t do it except with sophisticated robotics, like at the NDDC.

I’m looking forward to the day when I’ll place a SMCHD1 inhibitor pill in a patient’s palm, and see it work in a clinical trial. That’s when I’ll pop the champagne cork!

Nicholas:

As a venture principal scientist, I bring a unique skill set to the project team, blending academic scientific expertise with industrial research experience. It’s a pretty cool title and one that’s relatively new to the science world. It reflects WEHI Ventures’ ethos of fostering innovation and turning breakthrough science into commercial successes, to benefit patients.

I started my career as a lawyer, but then pursued a PhD in structural biology at WEHI. Seeking a commercial industry path, I then worked in San Francisco with biotech companies. Over there, it’s the global capital of biotech – a hotbed of innovation – and the scale of industrial science is out of this world.

Bringing that experience back to Melbourne has been invaluable. My role bridges the gap between scientists and investors. For me, bringing a scientific discovery to the market – as an actual treatment that can help a patient overcome a disease – is how I believe we can really make a difference.

It’s a colossal team effort and a difficult journey to get ideas to become an actual pill that can make a sick person better. Attracting funding early on is hard – they call it ‘the valley of death’ because many promising projects ‘die’ at that early- product development stage.

Our 66ten fund identifies high potential new ventures at WEHI, like Marnie’s. We fund the first critical step that’s going to advance a concept on its journey to commercialisation.

WEHI has proven that blockbuster returns can come from breakthrough science, if you play your cards right during the commercialisation process. Firstly, it can deliver life- changing treatments. Secondly, the royalties from the successful sale of a world-first therapy catapults funds back to WEHI, so we can continue to make discoveries that change the lives of millions of people. It’s sustainable science.

Most days I feel like I’m standing on the shoulders of giants. Marnie and the team’s project draws strength from decades of brilliant scientific research, and that’s what makes it such a compelling venture. Taking this to the next level feels like we’re on the verge of something incredible.

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Header image: Professor Marnie Blewitt and Dr Nicholas Liau at the National Drug Discovery Centre.