Pioneering biotech extends collaboration with leading global drug researcher to boost search for rare disease therapies

SynaptixBio and Evotec are working to develop new candidate therapies for a specific leukodystrophy.

SynaptixBio, the only company licensed to commercialise a treatment for a rare, incurable and deadly disease, has today announced it is extending its collaboration agreement with Evotec, a leading life science company, to find further candidate drugs to treat H-ABC, the most severe form of TUBB4A leukodystrophy.

Drug research was originally carried out by the Children’s Hospital of Philadelphia (CHOP), the world’s leading centre for leukodystrophy research, under a sponsored research agreement.

CHOP identified a candidate antisense oligonucleotide (ASO) as a potential therapy for H-ABC.

SynaptixBio and Evotec started working together in April 2022, with the aim of turning CHOP’s candidate ASO into a therapeutic for H-ABC.

This extension to their collaboration agreement will see Evotec significantly broaden the pipeline with additional ASOs as candidate therapies.

Dan Williams, CEO at SynaptixBio, 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 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.”

SynaptixBio is a virtual company; there are only two full-time and three part-time employees. All drug research is contracted out, as is every business function.

ASOs work by stopping a gene, in this case the mutated TUBB4A gene, from forming toxic proteins, so it is a type of gene-silencing.

A mutated TUBB4A gene results ultimately in less myelin for insulating nerve fibres in the brain, leading to disruption of the signals between neurons.

The US Food and Drug Administration (FDA) recently awarded SynaptixBio a second Orphan Drug Designation (ODD), to allows research and development of a therapy for another form of TUBB4A leukodystrophy, Isolated Hypomyelination.

The company received its first ODD in early 2023 for the CHOP therapeutic that targets H-ABC.

In October 2023, SynaptixBio was awarded a £490,000 BioMedical Catalyst grant from Innovate UK to tackle less common variants of the disease.

Rare diseases predominantly have a genetic origin, affect the young, and are very often life-limiting, so the impacts on families are devastating.

ASO technology has been proven in the treatment of other dystrophies, including Duchenne muscular dystrophy, and is quick and cost-effective to develop.

According to a European Commission report from 2020, “1 in 17 people will be affected by a rare disease at some point in their lives. This amounts to 3.5 million people in the UK”.

There are over 8,000 known rare diseases, with more emerging all the time.

For more information about SynaptixBio, visit here: https://www.synaptixbio.com/