Healx has announced a collaboration with Boston Children’s Hospital and Barth Syndrome Foundation (BSF) to advance promising therapeutic compounds to treat Barth syndrome.
The announcement comes on World Rare Disease Day, an annual global awareness initiative designed to bring attention of rare diseases to the general public and policymakers.
Barth syndrome, a life-threatening, genetic mitochondrial disease, can cause skeletal and cardiac muscle weakness and potentially fatal arrhythmias.
Healx, along with many other companies, is capitalising on advances in artificial intelligence (AI) and merging technology with new findings in science to overcome the financial and time challenges to develop therapies in rare diseases.
“We have to consider alternatives to the traditional drug discovery model,” said Dr Tim Guilliams, chief executive and co-founder, Healx. “Rare disease patients simply cannot rely on the traditional drug development process which doesn’t work in a timely fashion for such complex, commonly fatal diseases in niche patient populations.”
He continued, “The R&D cost to develop effective treatments for a condition that affects less than 0.00000004% of the world population, such as Barth syndrome, is not something that appeals to the traditional economics of the drug development industry.”
The company’s new approach aims to reduce the time to get new drugs into the clinic to two years, from a decade or more.