A team of medical and pharmaceutical experts has founded SynaptixBio, an Oxford-based research company working to develop the world’s first disease-modifying treatment for TUBB4a leukodystrophy, including H-ABC – a debilitating and potentially life-limiting condition. This is a sponsored research agreement with Children’s Hospital of Philadelphia (CHOP) in the US.
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 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. He said: “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.