GenieUs collaboration for MND and Parkinson’s

In an exciting partnership with Western Australia’s Perron Institute, Sydney-based biotech company GenieUs will apply sophisticated data analytics to test genetic therapies known as antisense oligonucleotides in patient cell models.

Two unique candidate therapeutics that could ultimately translate into treatment options for neurodegenerative diseases such as motor neurone disease and Parkinson’s Disease have been developed in Australia for testing.

Perron Institute neuroscientist Professor Anthony Akkari said that using a genomic medicine approach, the latest data from patient cells had identified two microRNA therapies that could potentially regulate genes known to affect Parkinson’s and the most common form of MND, amyotrophic lateral sclerosis (ALS).  

“MicroRNAs are small molecules that regulate genes by ‘RNA silencing’. This is a mechanism that regulates the proteins required for biological processes such as neuron growth and repair,” Professor Akkari said.  

“MicroRNAs can regulate a number of genes at once which is why GenieUs has focused on this process to identify novel therapeutic candidates. The approach has included analysis of motor neurons and spinal fluid from ALS patients, uncovering a number of microRNA targets that were significantly dysregulated.”  

GenieUs and the Perron Institute have now developed an antisense technology approach to directly target these dysregulated microRNAs. The evaluation is leveraging the Perron Institute MND research group’s extensive track record in gene-targeting antisense therapeutics for ALS/MND and other neurological conditions.  

The team specialises in the development and translation of antisense therapeutics for patients, and has a number of candidate medicines in various stages of preclinical testing in preparation for first time in human studies in collaboration with Black Swan Pharmaceuticals USA. BSP is an ALS antisense therapeutic company focused on developing phase 1 ready MND/ALS drugs.  

Perron Institute postdoctoral research fellow Dr Loren Flynn and Dr Sam Brennan (GenieUs) have reviewed the preliminary data showing the potential for regulating these novel MND related microRNAs and selected two primary candidate targets to move forward in the coming 12 months.  

The joint team led by GenieUs CEO Matt Keon and Professor Akkari, who heads the MND Genetics and Therapeutics group at the Perron Institute and Murdoch University, will test RNA therapies in patient neuron cell models over the next 12 months to show efficacy. 

“It is our goal to provide new treatment options for people living with these devastating conditions as quickly as possible,” Professor Anthony Akkari said. 

“This advance is a further step towards unravelling one of the greatest health challenges we face – enabling earlier intervention to facilitate improved clinical outcomes for patients.”  

Matt Keon, co-founder of GenieUs, said: “As with everything we do, we seek to provide therapies safely and quickly to patients.  

“Neurodegenerative diseases are complex and vary between people, so it is important to understand the unique genetic backgrounds and molecular events that cause the conditions so we can create personalised care and treatment for people. 

“These are encouraging early findings and we hope we will be able to collect enough evidence to be able to move into clinical trials in the very near future.” 

Anthony Akkari Perron Institute Lab 2021

Professor Anthony Akkari.