The company's lead candidate, SB H-19642, is an antisense oligonucleotide (ASO) designed to silence the mutated TUBB4A gene responsible for producing toxic proteins in Hypomyelination with Atrophy of the Basal ganglia and Cerebellum (H-ABC), the most severe form of the disease. 

SynaptixBio operates a virtual business model, outsourcing research and development activities to global partners. The company has collaborated with Evotec for the identification and development of its ASO candidate and has a sponsored research agreement with the Children's Hospital of Philadelphia (CHOP), granting exclusive rights to commercialize CHOP's intellectual property related to TUBB4A leukodystrophy treatments. 

SynaptixBio has received multiple designations from the U.S. Food and Drug Administration, including Orphan Drug Designation and Rare Pediatric Disease Designation for its H-ABC program. These designations may facilitate expedited development and review processes. 

The company has secured funding to advance its programs, including a £2 million BioMedical Catalyst grant from Innovate UK to support first-in-human clinical trials of SB H-19642. This follows earlier funding rounds totaling £13.2 million from private investors.