SynaptixBio bags a £2 million BioMedical Catalyst grant from Innovate UK for clinical trials of rare deadly disease
Summary
Click here SynaptixBio bags a \xa32 million BioMedical Catalyst grant from Innovate UK for clinical trials of rare deadly disease Oxford-based SynaptixBio, the only company licensed to commercialise a treatment for a rare, deadly and currently incurable disease, has been awarded a \xa32 million BioMedical Catalyst grant from Innovate UK to support first-in-human clinical trials of its therapeutic targeting H-ABC, the most severe form of TUBB4A leukodystrophy.This follows an earlier grant, in November 2023, from Innovate UK to expand SynaptixBios search for rare disease therapies; in February this year the company secured a second Orphan Drug Designation from the FDA in the US for a therapy targeting Isolated Hypomyelination a less severe form of TUBB4A leukodystrophy.Dan Williams, CEO at SynaptixBio, said; Getting to clinical trials will be a massive moment for us, and this grant is a major boost. They are caused by mutations in the TUBB4A gene, resulting in disruption to the signals between nerve cells in the brain. Currently, there is no cure.SynaptixBio is using antisense oligonucleotide (ASO) technology to tackle TUBB4A-related leukodystrophies; ASOs can alter the expression of genes, in this case a specific ASO molecule targets the mutated TUBB4A gene to stop it forming toxic proteins, which in turn help the proper function of the cells that form myelin sheaths surrounding nerve fibres in the brain. With the toxic protein suppressed, other proteins step in to help form normal myelin.The technology has been proven in the treatment of other dystrophies, including Duchenne muscular dystrophy, and is quick and cost-effective to develop.In the UK, a rare disease is defined as a condition that affects fewer than 1 in 2,000 in the population. This amounts to 3.5 million people in the UK and 30 million people across Europe.Earlier SynaptixBio successfully led a second round of investment, taking the total up to \xa313.2 million.Research to date has been supported by the worlds leading centre for leukodystrophy studies, the Childrens Hospital of Philadelphia (CHOP), under a sponsored research agreement.SynaptixBio has signed a worldwide exclusive license to intellectual property from CHOP, enabling commercialisation of a treatment.This award means we can begin to see the ultimate end-goal of an effective therapeutic getting so much closer, added Williams.