Company profile
Synaptixbio Ltd
SynaptixBio | Leukodystrophies
SynaptixBio is a rare disease biotech pioneering ground-breaking development for treatment of severe leukodystrophies which affect the brain. SynaptixBio will tackle a specific acute subset of leukodystrophies by altering the expression of a mutated gene identified as causing the disease.
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SynaptixBio: A demonstration of antisense oligonucleotide therapeutics for the treatment of a rare paediatric TUBB4A-related leukodystrophy - A first-in-human clinical trial
1 Jul 2024 to 30 Jun 2027
SynaptixBio is a rare disease biotech pioneering ground-breaking development for treatment of severe leukodystrophies which affect the central nervous system (CNS) in both adults (milder) and children (the most common and severe). TUBB4A-related leukodystrophies are a group of newly described neurodegenerative diseases which are genetic, debilitating, and...
SynaptixBio: Development of antisense oligonucleotide therapeutics for the treatment of rare paediatric TUBB4A-related leukodystrophies
1 Nov 2023 to 30 Apr 2025
SynaptixBio Ltd is a UK-based rare disease biotech SME pioneering ground-breaking development of therapeutics for treatment of TUBB4A-related leukodystrophies - the diseases affect the central nervous system (CNS) in both adults (milder) and children (the most common and severe). Rare diseases affect less than 1:2,000 people and are often severely disabli...