New biotech firm launched to develop ‘revolutionary’ treatment for one of world’s rarest neurodegenerative diseases

A new biotech business SynaptixBio has been launched to tackle one of the world’s rarest neurodegenerative diseases.

SynaptixBio is working to develop the world’s first disease-modifying treatment for TUBB4a leukodystrophy, including H-ABC - a debilitating and potentially life-limiting condition.

Founded by a worldwide team of leading medical and pharmaceutical experts, the Oxford-based research company has entered into a sponsored research agreement with Children’s Hospital of Philadelphia (CHOP) in the US related to a new method for treating TUBB4a leukodystrophy.

SynaptixBio also has an option to exclusively license CHOP research related to this project. Antisense Oligonucleotides (ASOs) therapy, which has previously been used to treat conditions such as Duchenne muscular dystrophy and spinal muscular atrophy, is also hoped to dramatically improve the quality of - and extend – the lives of leukodystrophy patients.

Dr Dan Willams, CEO and co-founder of SynaptixBio, said the treatment had the potential to “modify the disease, increase survival and significantly improve motor skills development.

“The new approach provides the potential to stabilise, improve quality of life and extend life expectancy in children suffering from the condition.“Successful prevention of leukodystrophy progression would be a revolutionary life-saving and life-enriching treatment.”

TUBB4a leukodystrophy causes hypomyelination in the brain, which can lead to developmental delays in motor skills such as walking, sitting, speaking and swallowing; learning difficulties, seizures, paralysis and an early death.

Research has already begun, with the firm aiming to launch clinical trials in 2024.“This project will change people’s lives,” Williams said. “The research and development of a clinically-proven treatment for TUBB4a would be a real game-changer for patients and their families.

“There is a real chance to improve the lives of leukodystrophy sufferers. We want to ensure that dream becomes a reality.”