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SynaptixBio
Oxford
Founded 2021
Antisense oligonucleotide gene therapies for rare childhood leukodystrophies
SynaptixBio is a rare disease biotech company developing gene therapies for severe leukodystrophies and childhood neurodegenerative diseases, using antisense oligonucleotide technology to silence disease-causing genes.
Its lead programme targets a rare, fatal degenerative disorder caused by mutations in the TUBB4A gene, for which it holds licensed rights and has received FDA Orphan Drug Designation.
Having selected a clinical development candidate, SynaptixBio is advancing towards first-in-human trials, backed by Innovate UK grant funding and private investment.