Oxford-based biotech company SynaptixBio has announced a major breakthrough in its quest to treat H-ABC, a rare, fatal, and currently incurable neurodegenerative disease. The company has identified its lead drug candidate, SB H-19642, which is now being prepared for clinical trials.

SB H-19642 is an antisense oligonucleotide (ASO)—a short strand of synthetic DNA designed to silence mutated genes that produce harmful proteins. This targeted therapy prevents the mutated gene from generating toxic proteins responsible for disease progression. ASOs are considered more precise and reversible than gene editing technologies, and typically result in fewer side effects.

“This is a hugely significant milestone,” said Dr Dan Williams, CEO of SynaptixBio. “We are confident our candidate drug will deliver the same positive impacts ASOs have shown in other degenerative conditions like Duchenne muscular dystrophy.”

H-ABC (Hypomyelination with Atrophy of the Basal Ganglia and Cerebellum) is the most severe form of TUBB4A-related leukodystrophy, caused by a single-gene mutation. Preclinical studies have shown that the toxic protein levels linked to the disease are significantly reduced following treatment with SB H-19642—suggesting the potential to halt or even reverse disease progression.

SynaptixBio has partnered with global drug discovery firm Evotec to develop and validate its ASO candidate. Early-stage research confirms that the drug is both safe and stable, with strong potential for clinical effectiveness.

The market for ASO therapies is expanding rapidly. According to Global Market Insights, it was valued at $4.4 billion in 2023 and is projected to reach $19.7 billion by 2032, driven by rising incidence of genetic disorders, technological advancements, and increasing regulatory approvals.

“ASOs don’t just manage symptoms—they target the root molecular causes of disease,” said Dr Williams. “That makes them truly game changing.”

SynaptixBio recently secured a £2 million BioMedical Catalyst grant from Innovate UK to support its upcoming first-in-human clinical trials, building on a previous grant awarded in November 2023 to expand its rare disease research.

Dr Williams also praised the H-ABC Foundation for raising awareness and advocating for early diagnosis and treatment.