Oxford’s SynaptixBio signs licensing deal with US children’s hospital
SynaptixBio, an Oxford-based biotech company aiming to develop the first treatment for TUBB4a leukodystrophy, has signed a licensing agreement with the Children’s Hospital of Philadelphia (CHOP).
The license enables the Oxford-based firm to further research started at the CHOP through clinical trials, developing CHOP’s innovative research into the rare, incurable and deadly disease.
SynaptixBio co-founder and CEO, Dr Dan Williams said: “This landmark agreement will enable SynaptixBio to develop and commercialise CHOP’s patents and research related to the treatment of TUBB4A leukodystrophy.
“We are naturally delighted to be working with CHOP on this extremely important project, which aims to accelerate the research and development of the world’s first treatment for the disease.”
The disease was first identified by the program director of CHOP’s Leukodystrophy Centre, Dr Adeline Vanderver, who is a pre-eminent figure in the research. TUBB4a leukodystrophy makes up 9% of a group of about 30 rare neurodegenerative disorders known as leukodystrophies.
Data from the University of Utah suggests that leukodystrophies affect 1 in 7,663 births. This would mean that, of the approximately 140 million global births in 2021, 18,000 could have leukodystrophy, of which almost 1,650 could have TUBB4a.
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Caused by a mutation in the TUBB4A gene, the disease disrupts myelin surrounding nerves, leading to interruption of the signals between nerve cells in the brain.
At its most severe, the condition can lead to significant impairment of motor skills such as walking, sitting up and even swallowing.
Patients can also develop seizures, muscle contractions, hearing and speech difficulties, and uncontrollable limb movements, while others who have developed motor skills in early childhood can regress.
The condition often results in an early death for babies and children who develop the mutation.
CHOP has identified Antisense Oligonucleotides (ASOs) as a potential treatment for the disease.
It is hoped ASOs, which have previously been used to treat conditions such as Duchenne muscular dystrophy and spinal muscular atrophy, will dramatically improve the quality of, and extend, the lives of leukodystrophy patients.
Dr Vanderver said: “ASOs provide the potential to stabilise, improve quality of life, and extend life expectancy of children suffering from the condition. Successful prevention of leukodystrophy progression would be revolutionary, life-saving, and life-enriching.”
SynaptixBio is aiming to begin trials in 2024.
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