Oxford-based SynaptixBio, which is developing a therapy for a rare, deadly, and currently incurable rare disease (H-ABC), says it has identified the drug it will take forward into clinical trials. 

The drug, SB H-19642, is an antisense oligonucleotide (ASO), which is a short strand of synthetic DNA that stops mutated genes from producing toxic proteins. The mutated gene is ‘silenced’. 

Dan Williams, CEO at SynaptixBio, said: “This is a hugely significant milestone. 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. 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. 

“This means disease progression could be completely halted, and there is some evidence of symptoms being reversed.” 

ASOs offer a range of benefits compared to gene editing; they are highly targeted, generally produce much fewer side effects, and have broad applicability. Most significantly, they are non-permanent, so can be stopped if dangerous side-effects do appear. 

ASOs have already been approved for SOD1 dependent ALS (Motor Neuron Disease), and are currently in trials for Alzheimer’s disease and being investigated as a potential therapy for Parkinson’s disease, amongst others. 

SynaptixBio has been working with Evotec to identify, develop, and qualify the ASO candidate. Results so far indicate that the drug is safe and stable, and initial testing has shown it to be effective in reducing the volume of toxic proteins arising from the mutated gene. 

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.

DIANA SPENCER, Senior Digital Content Editor, DDW