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Boosting product confidence of a world-leading RNA therapeutics start-up

April 4, 2022

CSIRO’s Transformational Bioinformatics group validates the importance of long non-coding RNAs (lncRNAs) as novel cancer therapeutics targets.

The Client 

Amaroq Therapeutics is a New Zealand-based biotechnology company targeting long non-coding RNAs for next generation cancer therapies. The company is spun out of research from the University of Otago in Dunedin, New Zealand, and is funded by the Medical Research Commercialisation Fund (MRCF), managed by life science venture capital firm Brandon Capital Partners. Amaroq secured $14m in seed funding from the MRCF, NZ Innovation Booster, and Cure Kids Ventures to create this world-leading RNA therapy. Amaroq also received support from Callaghan Innovation’s Tech Incubator program. 

The Challenge 

Only 1-2% of the human genome encodes for proteins. Most cancer-associated mutations are located outside of protein-coding genes, in the “non-coding” genome, which means protein-centric drug searches might miss the biggest opportunities. Amaroq’s research team discovered molecules that are highly-expressed in cancer cells originating from these “non-coding” regions, called long non-coding RNAs (lncRNAs). Once these are removed, cancer cells grow slower. Amaroq wants to use this discovery to combat cancer by developing robust and safe methods to remove these cancer-promoting lncRNAs. 

However, lncRNA are the biggest and most diverse class of RNA in the cell, so their appearance in cancer cells could be coincidental rather than causative.  

To increase the confidence that the lncRNA targets chosen are the most effective therapeutic candidates, Amaroq approached CSIRO to independently replicate their findings.   

The Genomics Insights team has been instrumental in validating our findings and helping us improve our processes. We were very impressed with their deep knowledge, professionalism, and attention to detail. 

~Sarah Diermeier, Chief Scientific Officer & Founder 

The Solution 

To solve the problem, CSIRO analysed data generated by an advanced biotech-screen, to find lncRNAs that are important for cancer cell growth. The Genomics Insights team took advantage of their knowledge of computational tools for gene expression analysis to find these targets and help validate Amaroq’s findings. Following this, they analysed gene expression in patient data from various cancer types to find clinically-important targets.   

We also leveraged the expertise of the Digital Genome Engineering team to identify potential off-target risks for the interesting targets. This strengthens Amaroq’s capabilities by providing valuable insights to their identified targets and helps them further prioritise lncRNAs candidates. 

The Outcomes  

In less than five months, CSIRO and Amaroq have collaboratively identified lncRNAs that affect cancer cell growth, and deliver novel targets for cancer-directed intervention, guiding future drug development. 

Besides the biological findings, CSIRO has helped upskill the Amaroq bioinformatics team, strengthening their capability for experimentation by providing robust bioinformatics pipelines, which: 

  • detect differentially expressed lncRNA 
  • evaluate lncRNA targeting strategy    

With the replication study complete, Amaroq now has the confidence and strengthened capability to invest their seed funding into developing the lncRNA and targeting strategy into therapeutics ready for phase 1 clinical trials over the coming years. 

Originally published on CSIRO Australian e-Health Research Centre.