SynaptixBio, a rare disease biotech, is partnering with Evotec, a life sciences company, to find further drug candidates which can be used to treat H-ABC, a severe form of TUBB4A leukodystrophy.
This follows on from the previous collaboration between the Children’s Hospital of Philadelphia (CHOP), where an antisense oligonucleotide with the potential to treat H-ABC (hypomyelination with atrophy of the basal ganglia and cerebellum) was developed.
Together, SynaptixBio and Evotec will expand their pipeline of drugs for H-ABC, with a specific focus on antisense oligonucleotides (ASOs).
ASOs: how and why?
ASOs work by silencing a gene, thus stopping its functionality in the body. In the case of H-ABC, TUBB4a is present in a mutant form, and is responsible for the life-altering symptoms experienced by patients.
Therefore, employing TUBB4a silencers for the treatment of H-ABC could hold promise as an efficacious method.
ASO technology has been proven in the treatment of other dystrophies, including Duchenne muscular dystrophy and is quick and cost-effective to develop.
CEO at SynaptixBio, Dan Williams said: “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 does Evotec have the capability 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 US Food and Drug Administration (FDA) recently awarded SynaptixBio a second Orphan Drug Designation (ODD) to facilitate the research and development of a therapy for another form of TUBB4A leukodystrophy, Isolated Hypomyelination.