SynaptixBio receives incurable disease research boost

The Oxford-based biotech company has secured £13.2m to progress world’s vital treatment development

Oxford-based SynaptixBio – a company focused on treating the genetic central nervous system disease TUBB4A-related leukodystrophy – has gained £11.05m in its latest financing project. It adds to the £2.125m of seed funding during the last two years. Each of the pivotal rounds have arrived exclusively from private investment.

The research firm anticipates that the investment will cover the rest of its extensive research into a treatment, while also progressing it to human clinical trials next year.

Originally identified in 2015, TUBB4A-related leukodystrophy – which mainly impacts babies and young children – is triggered by a mutation in the TUBB4A gene and currently has no cure.

Consequently, it disrupts the signals between nerve cells in the brain and also cause impairment of motor skills such as walking, sitting up and swallowing. Meanwhile, effected individuals can also suffer from seizures, muscle contractions, hearing and speech difficulties.

To progress its research, SynaptixBio has entered into a sponsored research agreement with the Children’s Hospital of Philadelphia (CHOP) in the US – a world-leading leukodystrophy centre – to develop a treatment from antisense oligonucleotides, which can prevent or alter the production of proteins.

The partnership, which also incorporates exclusive patent rights – allows SynaptixBio to translate CHOP’s research to human clinical trials.

SynaptixBio co-founder and chief executive officer, Dr Dan Williams, believes the funding is a significant step: “This latest funding round is the final piece of the investment jigsaw. Depending on external economic factors, it should enable us to continue our work all the way to clinic, which will be a fantastic achievement.”

He added: “Our mission to develop a treatment for this life-limiting condition has taken a huge step closer.”