GenieUs and Perron Institute move into exciting next phase of RNA drug development

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Sydney, Australia, Perth, Western Australia, TBC November 2021: GenieUs Pty Ltd, a genomics discovery company that uses data analytics and machine learning to better understand the cause of neurodegenerative diseases, and the Perron Institute, Perth who develop RNA therapies called Antisense Oligonucleotides have developed two unique candidate therapeutics that could ultimately translate into treatment options for patients suffering from neurodegenerative diseases, including Amyotrophic Lateral Sclerosis (ALS), and Parkinson’s Disease.

Using a genomic medicine approach, the latest data from patients cells has shown that two microRNA therapies have strong target engagement and can potentially regulate genes known to affect ALS (the most common form of MND) and Parkinson’s. MicroRNA are small molecules that regulate genes by “RNA silencing” a process that neurons and other cells in the body use to regulate the amount of proteins required for biological processes like growth and repair. Micro RNA can regulate a number of genes at once and this is why GenieUs has focused on this RNA based cell process, to identify novel therapeutic candidates.

The GenieUs team uncovered a number of microRNA targets that were significantly dysregulated in motor neurons from MND/ALS patients using a comprehensive genomic medicine-based approach which included the spinal fluid from ALS patients. GenieUs and the Perron Institute have partnered to develop an antisense technology approach to directly target the dysregulated microRNA. In an exciting partnership with the MND research group at the Perron Institute, the two teams have been keenly working to evaluate these microRNA targets leveraging the Perron Institute MND research group’s extensive track record in gene targeting antisense therapeutics for ALS/MND and other neurological conditions. The Perron institute MND research 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.

Dr Loren Flynn (Perron institute postdoctoral research fellow), who is an inventor of several MND antisense therapeutics 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 of Matt Keon, GenieUs CEO, and Professor Anthony Akkari, head of the MND genetics and therapeutics group at the Perron Institute, will test RNA therapies in patient stem cells models over the next 12 months to show efficacy.

Founded in 2017, GenieUs’ analytics solution is at the forefront of bioanalytical analysis of datasets to map and sub-categorise neurodegenerative diseases for more personalised treatment options.  Neurodegenerative diseases are complex and vary between people, so it is important to understand  the unique genetic backgrounds and molecular events that cause the condition and create truly personalised care and treatment for people.The Perron Institute, established in 1982 is the longest established medical research institute in Western Australia and is researching a range of neurological disorders. It brings together a dedicated range of researchers and clinicians, many pre-eminent in their fields. The Perron Institute has contributed to a number of neuromuscular disease discoveries over the years, this includes the SOD1 gene the first gene discovered for MND back in 1992, and most recently the first antisense therapeutics for Duchenne muscular dystrophy.

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, CEO and co-founder of GenieUs, said: “As with everything we do, we seek to provide therapies safely and quickly to patients. These are encouraging early findings and we hope that we will be able to collect enough evidence to be able to move into clinical trials in the very near future.”

Professor Anthony Akkari, Head of Motor Neurone Disease Genetics and Therapeutics Research at the Perron Institute said: “It is our goal to provide new treatment options for people living with these devastating conditions as quickly as possible.”

GenieUs has been awarded a patent for the biomarker of one the microRNA’s they are developing.   

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About GenieUs

GenieUs is a science and technology company using  a proprietary model of data analytics and machine learning to solve the mystery of neurodegenerative diseases, one of the most misunderstood condition areas on the planet. Founded in 2017, its analytics solution uses in-house algorithms and the deepest integration of datasets to map and sub-categorise neurodegenerative diseases for more personalised treatment.

About Perron Institute

For over 39 years, the Perron Institute has continued to build its reputation as an innovative medical research institute which has made significant breakthroughs across a broad spectrum of neurological conditions. Perron Institute’s multidisciplinary approach enables the translation of research outcomes into treatments aimed at providing a better quality of life for millions of people around the world who suffer with devastating neurological conditions.

About Neurodegenerative Disease

Neurodegenerative diseases like motor neuron diseases and dementia are one of our fastest-growing health risks. There are 10 million new cases of dementia globally every year, yet research and diagnosis are years behind diseases like cancer.

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