Alan Cohen
MD, Chief Medical Officer, Arcturus Therapeutics
Breakthroughs in mRNA medicines could be transformative for patients living with rare diseases such as cystic fibrosis and ornithine transcarbamylase deficiency.
Why mRNA medicines could be a rare disease breakthrough
During the Covid-19 pandemic, advances in messenger ribonucleic acid (mRNA) technology accelerated vaccine development. Now this same technology may offer new hope to patients with rare diseases, like those with cystic fibrosis (CF). CF is a chronic, progressive, life-shortening, currently incurable genetic disease that primarily damages the lungs and digestive system. Then there is ornithine transcarbamylase (OTC) deficiency – also currently incurable and estimated to affect more than 10,000 people worldwide a metabolic condition that causes a build-up of ammonia in the blood and is highly toxic to the brain and nervous system of newborns, children and adults.
Our bodies need millions of essential proteins to operate and survive. CF and OTC deficiency are a result of gene mutations which cause the body to produce a faulty protein (CF) or a non-functional protein (OTC). However, Arcturus Therapeutics, an mRNA medicines company, currently has therapies in development that could prove life-changing for patients. Rather than managing symptoms, mRNA medicines aim to address the underlying genetic cause of disease by providing a healthy version of missing or defective proteins.
Advances mean we now have the possibility of being able to better
identify rare conditions and bring something to the forefront that
could improve the quality and longevity of patients’ lives
Improving the quality and longevity of patients’ lives
Traditional medicines can often struggle to reach or replace complex proteins inside cells. The big advantage of mRNA medicines is that when they enter the cell, they provide ‘instructions’ to produce the proteins needed to prevent or treat a disease.
“We believe mRNA medicines are truly transformative for rare diseases,” explains Alan Cohen, MD, Chief Medical Officer at Arcturus Therapeutics. “Practically speaking, they help us make our own protein therapies.” Dr Cohen notes that the company has also developed its own specialised delivery system designed to send mRNA medicines to targeted organs and cell types.
In conditions like CF, different patients can have different genetic mutations. Yet mRNA medicines can potentially treat cystic fibrosis patients regardless of their specific mutation, so offering a unique ‘blueprint’ solution. They can do this safely, too, because unlike gene therapy, mRNA is temporary. Once its instructions (or ‘messages’) have been delivered, the body naturally breaks them down.
“It’s an exciting time,” says Dr Cohen. “Advances mean we now have the possibility of being able to better identify rare conditions and bring something to the forefront that could improve the quality and longevity of patients’ lives.”
[1] Arcturus Therapeutics. (2024). Arcturus Therapeutics Provides Updates for Ornithine Transcarbamylase (OTC) Deficiency and Cystic Fibrosis (CF) Programs.
