The grant will support clinical trials for rare disease treatments for leukodystrophy.
Oxford-based SynaptixBio has been awarded a £2 million BioMedical Catalyst grant from Innovate UK to support first-in-human clinical trials of a treatment targeting H-ABC, the most severe form of TUBB4A leukodystrophy.
In November 2023, SynaptixBio received a grant from Innovate UK to expand its search for rare disease treatments.
In February of this year, the company received its second orphan drug designation from the U.S. FDA for its treatment targeting isolated hypomyelination, a mild form of TUBB4A leukodystrophy.
In the UK, a rare disease is defined as one that affects fewer than 1 in 2,000 of the population, and SynaptixBio is the only company licensed to commercialise a treatment for this rare, deadly and currently incurable disease.
There is currently no treatment for TUBB4A-related leukodystrophies, a group of rare neurodegenerative disorders caused by mutations in the TUBB4A gene. The diseases cause disruptions to signals between nerve cells in the brain and primarily affect young children.
SynaptixBio is using antisense oligonucleotide (ASO) technology to address TUBB4A-related leukodystrophies. ASOs can alter gene expression, in this case, a specific ASO molecule targets the mutated TUBB4A gene to block the formation of a toxic protein, thus promoting the proper function of cells that form the myelin sheath that surrounds nerve fibers in the brain. With the toxic protein inhibited, other proteins step in to help form normal myelin.
The technology was rapidly developed, cost-effective, and has proven efficacy in treating Duchenne muscular dystrophy and other dystrophies. Research to date has been supported by Children’s Hospital of Philadelphia (CHOP), the world’s leading leukodystrophy research center, under a Sponsored Research Agreement.
