Value proposition

Developing therapies for severe leukodystrophies


Overview
SynaptixBio is a rare disease biotech company that is pioneering ground-breaking treatments for severe leukodystrophies and other childhood neurodegenerative diseases.

SynaptixBio’s mission is to deliver gene therapies for fatal leukodystrophies in children caused by loss of function or mutation in a single gene. Using a breadth of platforms including antisense oligonucleotide (ASO)-based technology, we can silence the expression of the toxic gene to reverse disease progression. This approach will create life saving treatments for severe genetic diseases that affect the central nervous system.


Pharmaceutical, Biotech, Leukodystrophy, Rare Disease, H-ABC, Hypomyelination, Basal Ganglia, Cerebrllum, Neurology, Brain, Preclincal, Drug Discovery, Life Sciences, Development, and Paediatric Disorders