SynaptixBio, Evotec Extend Research and Development Collaboration

Image Credit: SynaptixBio

SynaptixBio, a company that commercializes rare disease treatments, is extending its deal with Evotec, a life science company, which involves finding candidate drugs to treat H-ABC, the most severe form of TUBB4A leukodystrophy.¹ Their partnership dates back to April 2022.

Per a sponsored research agreement, drug research was conducted by the Children’s Hospital of Philadelphia (CHOP). CHOP determined that antisense oligonucleotide (ASO) could serve as a potential therapy for H-ABC.

What does the extension of the partnership signify exactly? According to Evotec, it expands the company’s pipeline by adding more ASOs as candidate therapies. In this particular case, ASOs would have “gene-silencing” benefits, as they prevent a mutated TUBB4A gene from forming toxic proteins. A mutated TUBB4A gene can mean less myelin for insulating nerve fibers in the brain, resulting in neuron signal disruption.

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

The FDA recently granted SynaptixBio a second Orphan Drug Designation (ODD)—with the first coming last year for the CHOP therapeutic that targets H-ABC—granting the company permission to conduct research and development of a therapy for another form of TUBB4A leukodystrophy known as Isolated Hypomyelination.

In October 2023, Innovate UK, a national innovation agency, gave the company $630,000 BioMedical Catalyst grant that could be utilized toward treating other less common disease variants.

ASO technology has been effective when it comes to combatting other dystrophies—such as Duchenne muscular dystrophy—and is reportedly quick and economical to develop.

Reference

1. Pioneering biotech extends collaboration with leading global drug researcher to boost search for rare disease therapies. SynaptixBio. July 22, 2024. Accessed July 25, 2024. Press release delivered via email.