‘Virtual’ biotech targets R&D funding on rare disease therapy

Medical researcher

UK biotech firm SynaptixBio has secured £13.2 million to fund its development of the world’s first treatment for TUBB4A-related leukodystrophy, a rare, incurable and deadly disease.

The company successfully raised £11.05m in its latest funding round to add to the £2.125m of seed funding from the last two years. It hopes this will fund the rest of its research into a treatment and take it to human clinical trials next year.

First identified in 2015, TUBB4A-related leukodystrophy, which is believed to mainly affect babies and young children, is caused by a mutation in the TUBB4A gene. It disrupts the signals between nerve cells in the brain and can lead to significant impairment of motor skills such as walking, sitting up and swallowing.

Patients can also develop seizures, muscle contractions, hearing and speech difficulties, and uncontrollable limb movements, while others who have developed motor skills in early childhood can regress.

SynaptixBio co-founder and CEO Dr Dan Williams said: “This latest funding round is the final piece of the investment jigsaw. Depending on external economic factors, it should enable us to continue our work all the way to clinic, which will be a fantastic achievement. Our mission to develop a treatment for this life-limiting condition has taken a huge step closer.”

Reduced drug development costs

By adopting a virtual business model and outsourcing, SynaptixBio has been able to keep costs low.

Having secured a rare paediatrics disease (RPD) designation from the Food and Drug Administration (FDA) in the US earlier this year, the company founders hope its virtual framework will inspire others to develop treatments for rare diseases.

“Typically, biotechs have multiple molecules in their pipeline,” Dr Williams said. “SynaptixBio currently has a single therapeutic under development. As a virtual company with very low overheads and employees, all of the investment can go into research and development.”

The company has entered into a sponsored research agreement with the world-leading leukodystrophy centre, the Children’s Hospital of Philadelphia (CHOP) in the US, to develop a TUBB4A-related leukodystrophy treatment from antisense oligonucleotides (ASOs).

It is hoped ASOs, which have previously been used to treat conditions such as Duchenne muscular dystrophy and spinal muscular atrophy, will dramatically improve the quality of, and extend, the lives of TUBB4A patients.

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