SynaptixBio, an Oxford-based biotech company aiming to develop the first treatment for TUBB4a leukodystrophy, has signed a licensing agreement with the Children\u2019s Hospital of Philadelphia (CHOP).<\/p>\n\n\n\n
The license enables the Oxford-based firm to further research started at the CHOP through clinical trials, developing CHOP\u2019s innovative research into the rare, incurable and deadly disease.<\/p>\n\n\n\n
SynaptixBio co-founder and CEO, Dr Dan Williams said: \u201cThis landmark agreement will enable SynaptixBio to develop and commercialise CHOP\u2019s patents and research related to the treatment of TUBB4A leukodystrophy. <\/p>\n\n\n\n
\u201cWe are naturally delighted to be working with CHOP on this extremely important project, which aims to accelerate the research and development of the world\u2019s first treatment for the disease.\u201d<\/p>\n\n\n\n
The disease was first identified by the program director of CHOP\u2019s 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.<\/p>\n\n\n\n
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.<\/p>\n\n\n\n