Rare disease-focused SynaptixBio has identified SB H-19642, a therapeutic candidate it will take forward into clinical trials for the treatment of H-ABC.
The antisense oligonucleotide works by stopping mutated genese from producing toxic proteins, effectively silencing it.
Hypomyelination with Atrophy of the Basal Ganglia and Cerebellum (H-ABC) is a rare genetic disorder that causes progressive neurological symptoms, including movement impairments and intellectual disability.
“We are confident our candidate drug will deliver the same positive impacts that ASOs have made on other degenerative diseases such as Duchenne muscular dystrophy," noted Dan Williams, CEO at SynaptixBio.
"The dangerous proteins produced by the single-gene mutation that causes H-ABC, the most severe form of TUBB4A-related leukodystrophy, have been shown in animal testing to be far less prevalent following treatment."
By silencing this gene, Williams claims that disease progression "could be completed halted," with some evidence suggesting that symptoms could be reversed in time.
SB H-19642 was created off the back of SynaptixBio's partnership with Evotec, which was initiated to identify, develop and qualify the ASO candidate.
So far, research shows that the drug is safe and stable, with initial testing finding the ASO to be effective at reducing the number of toxic proteins arising from the mutated gene.
"ASOs target the molecular causes of disease, rather than just treating the symptoms. This makes them potentially game changing,” added Williams.
SynaptixBio was recently awarded a £2 million BioMedical Catalyst grant from Innovate UK to support first-in-human clinical trials of its therapeutic targeting H-ABC.