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NSW Biosciences Fund

2023 BioSF Successful Recipients

RNAfold.AI

Funding: $1,025,238

To tackle challenges in drug discovery and design, RNAfold.AI have developed a suite of AI algorithms capable of optimising new RNA targeting drugs all the way to clinical trials, even for
previously ‘undruggable’ protein targets.

Initially, RNAfold.AI will focus on oncology, a leading cause of mortality. However, the software
package has the potential to identify drivers in other adverse and complex health disorders, too.
RNAfold.AI utilises AI to predict the secondary structure of RNA molecules, integrating the
thermodynamic model of RNA folding with pharmacological compounds. The AI platform is trained
on comprehensive data to experimentally identify a lead for RNA therapeutics with significantly
higher accuracy compared to current experiential trial-and-error approach, thereby enhancing
development cost and time efficiency.

RNAfold.AI will use the funding to create a distinctive database by integrating information from
RNA biology literature, molecular data and therapeutically informative experiments. This unique
database will be further enriched with their sequencing and experimental data to train the AI
assistant, streamlining discovery processes, enhancing success rates and facilitating the design
of new chemistries.

By minimising the number of compounds required for screening and licensing their in-house
developed drugs at preclinical stages, RNAfold.AI will become a valuable partner in advancing NSW
as an emerging biotechnology hub.

Skin2Neuron

Funding:  $1,996,762

Alzheimer’s disease stands out as one of humanity’s most pressing health challenges, with no
clinically meaningful treatment available to counter its relentless decline.

Skin2Neuron (S2N) is a NSW-based biotech company focused on developing all-new regenerative
medicine technology for Alzheimer’s disease and beyond.

The company’s core technology (Primaverah©) is a process for manufacturing Hair Follicle-derived
Neuroprecursors (HFNs) – a novel cell type discovered by our team with the potential to revolutionise the treatment of brain disorders. This pioneering approach starts with adult hair follicles, and is uniquely capable of the isolation, enrichment and expansion of these rare cells.

The final product is a clinically relevant number of HFN cells of exceptional purity. The company’s
preclinical research has shown the cells can replace lost neurons and synapses in ageing and
disease and reverse memory deficits. With the potential to bring back cognition and enhance quality
of life, S2N hopes to open up a whole new class of treatment: the anti-dementia biologic.

This innovative technology is only the first of several therapies in the pipeline. The funding will
expedite S2N’s critical development, scaling up and automating manufacture, helping to bring
this new technology closer to clinical trial and then the global market. S2N’s long-term vision is
to develop next-generation cell therapies for brain disorders such as Alzheimer’s, Parkinson’s and
Huntington’s disease.

ThromBio

Funding: $1,478,000

Stroke represents one of the most pressing health challenges globally, with over 12 million new
cases annually. There are very few safe and effective treatments, with less than 10 per cent of
patients benefiting from current standard of care. For over 40 years the pharmaceutical industry has
tried to develop safe and effective anticlotting agents that can be used in the hyperacute treatment
of stroke, however all agents tested to date cause life-threatening bleeding on the brain, which has a
high mortality rate.

ThromBio is leveraging research led by Professors Shaun Jackson and Richard Payne, renowned
experts in thrombotic disorders, to address this challenge. Their portfolio of first-in-class compounds
is characterised by a unique mechanism of action, and potentially represents a significant leap
forward in the treatment of ischemic strokes.

The company's small molecule New Chemical Entity, TBO-309 has now treated 10 patients, with promising early results (STARS - Safety and Tolerability of Adjunctive TBO-309 in Reperfusion for Stroke). The study commenced in late 2023 in leading stroke units in NSW, Victoria and South Australia.

The funding will support a Phase II clinical study, Co-STARS, to determine the optimal clinical dose of TBO-309 and further commercial discussions with potential partners for drug combination, monotherapy and stent (device) collaborations.

With no new approved pharmacological therapy for the hyperacute treatment for stroke since
1996, ThromBio’s potentially landmark antiplatelet agent, distinguished by its unprecedented
safety profile, offers a unique opportunity for Australia. Success in the Phase II clinical trial could
potentially revolutionise the global standard of care for stroke, leading to significant life-saving
impacts and economic benefits.