SB H-19642

SB H-19642 is an antisense oligonucleotide (ASO) therapeutic being developed by SynaptixBio for the treatment of Hypomyelination with Atrophy of the Basal Ganglia and Cerebellum (H-ABC), the most severe form of TUBB4A leukodystrophy. H-ABC is a rare, life-limiting neurodegenerative disease caused by mutations in the *TUBB4A* gene, which leads to the failure of myelin formation and maintenance in the brain. SB H-19642 is designed to specifically target and modulate the expression of the *TUBB4A* gene, aiming to reduce the toxic effects of the mutant protein and potentially halt disease progression. The program has received a £2 million BioMedical Catalyst grant from Innovate UK to support its transition into first-in-human clinical trials.

Other names

TUBB4A ASOTUBB-4A ASOTUBB 4A ASOTUBB4A antisense oligonucleotideTUBB-4A antisense oligonucleotideTUBB 4A antisense oligonucleotidebeta tubulin 4a gene silencing ASO