SynaptixBio expands collaboration with Evotec to advance treatments for H-ABC

New Delhi: SynaptixBio has announced an extension of its collaboration with global life science company Evotec. This partnership aims to accelerate the development of therapies for H-ABC, a severe form of TUBB4A leukodystrophy, a rare and debilitating condition.

The collaboration builds on initial research conducted by the Children’s Hospital of Philadelphia (CHOP), a leading institution in leukodystrophy research. CHOP identified a candidate antisense oligonucleotide (ASO) as a potential therapy for H-ABC. Since April 2022, SynaptixBio and Evotec have been working together to advance this candidate ASO into a viable therapeutic option.

The agreement will significantly expand their pipeline with additional ASO candidates. ASOs work by targeting and silencing the mutated TUBB4A gene, which is responsible for producing toxic proteins. This gene-silencing approach is critical because a mutated TUBB4A gene leads to a deficiency in myelin, the protective sheath around nerve fibers in the brain. This deficiency disrupts neural signal transmission, causing severe neurological symptoms.

Dan Williams, CEO of SynaptixBio, emphasized, “The contract with Evotec is absolutely vital for us to ensure we have a range of viable candidates to take through further testing. Not only do they have the capabilities to identify and develop candidate drugs, but they can also help us take them right through clinical trials and beyond. They are instrumental in supporting us as we prepare for those clinical trials.”

In recent developments, SynaptixBio received its second Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA) for a therapy targeting Isolated Hypomyelination, another form of TUBB4A leukodystrophy. The first ODD was granted earlier in 2023 for the H-ABC therapeutic developed with CHOP.

Additionally, in October 2023, SynaptixBio was awarded a £490,000 BioMedical Catalyst grant by Innovate UK to address less common variants of the disease. This funding supports the company’s efforts to explore ASO technology, which has proven effective in treating other dystrophies like Duchenne muscular dystrophy.

With over 8,000 known rare diseases affecting a significant portion of the population, SynaptixBio’s work addresses a critical need. According to a 2020 European Commission report, approximately 1 in 17 people will be affected by a rare disease at some point in their lives amounting to 3.5 million people in the UK. It underscores the importance of continued research and innovation in this field.