H-ABC Program
Hypomyelination with Atrophy of the Basal Ganglia and Cerebellum (TUBB4A-related)
Pre-clinicalActive
Key Facts
Indication
Hypomyelination with Atrophy of the Basal Ganglia and Cerebellum (TUBB4A-related)
Phase
Pre-clinical
Status
Active
Company
About SynaptixBio
SynaptixBio is a private, pre-clinical stage biotech developing antisense oligonucleotide (ASO) therapies for rare, fatal leukodystrophies caused by TUBB4A gene mutations. The company holds key regulatory designations from the FDA, including Rare Paediatric Drug and Orphan Drug Designations for its lead programs targeting H-ABC and Isolated Hypomyelination. With a seasoned leadership team, SynaptixBio is advancing candidate selection for clinical trials and positioning itself as the sole potential therapeutic provider for a significant unmet medical need affecting thousands of children globally.
View full company profile