SynaptixBio Ltd. is a biotechnology company focused on developing innovative therapies for severe leukodystrophies, a group of rare genetic disorders that primarily affect the brain and lead to significant developmental delays and motor function deterioration. These conditions are caused by mutations in specific genes, resulting in the abnormal development or destruction of the brain's white matter, which is essential for proper nerve function. The company aims to address a particular subset of leukodystrophies by targeting the expression of a mutated gene responsible for the disease, thereby offering a potential therapeutic approach that goes beyond symptomatic treatment.

To achieve its goals, SynaptixBio employs a broad platform that includes antisense oligonucleotide (ASO) technology. This approach is designed to silence the expression of the toxic mutated gene associated with conditions like TUBB4a leukodystrophy, which disrupts the production of myelin, the protective sheath around nerve fibers. By preventing the production of this harmful protein, SynaptixBio's therapies have the potential to preserve myelination and improve the quality of life for affected individuals. The company is committed to advancing its pipeline through various stages of development, from discovery to clinical trials, with the aim of providing effective treatments for these debilitating diseases.

United Kingdom

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.

SynaptixBio Ltd.

Company Details