Gene therapy pioneer developing cure for dementia raises $80m for clinical trials

·3-min read
   (Dan Kitwood/Getty)
(Dan Kitwood/Getty)

A biotech spun out of King’s College London has raised $80 million (£56 million) to fund clinical trials of “revolutionary” gene therapies with the potential to cure dementia.

AviadoBio’s goal is a one-shot treatment that stops cells in the brain and spinal cord from shrinking (neurodegeneration), which can cause the conditions frontotemporal dementia (FTD) and motor neuron disease (MND).

There is currently no cure for either of the devastating diseases, while existing treatments are life-limiting and cumbersome.

Once the technology has been proven, co-founder Professor Christopher Shaw believes tackling more complex illnesses such as Alzheimer’s - which is caused by multiple gene mutations - will be more closely within reach.

The firm’s patented platform has been developed over 25 years in Shaw’s lab and uses a viral vector to deliver DNA into nerve cells which have lost the ability to make proteins essential for staying healthy.

Injecting a working copy of the gene directly enables the cell’s internal machinery to begin making the protein again. The process can also work in reverse, to knock-out faulty versions of protein.

AviadoBio said the treatment has completely cleared pathology in tests on mice and sheep.

The £56 million Series A round funding will allow the team to expand in preparation for human trials to begin, planned for later next year.

The therapies are seen as a vital step towards both the prevention and cure of a host of untreatable degenerative conditions.

AviadoBio’s lead project targets FTD, an aggressive and common form of young-onset dementia caused, in a third of cases, by a single mutated gene and characterised by changes in personality, behaviour and language.

The target protein is progranulin, which plays a key role in the growth, survival and repair of cells.

Existing therapies involve a monthly lumbar puncture to inject doses of this missing protein into spinal fluid.

Because nerve cells last for life, AviadoBio believes there is potential for a successful single dose of the working gene therapy to deliver lifelong results.

Shaw said: “Gene therapies are incredibly powerful, if we can deliver them safely we can really make a change.

“These are powerful tools and we have a real chance to cure patients for whom there are no effective treatments.

“We are starting with concrete causal factors which we think we can correct, then we will start to work on the more complicated disease like Alzheimer’s.

“We are long way from being there but at least we’ve now got the weapons to do it.”

Lisa Deschamps was appointed CEO of AviadoBio earlier this year, joining from Novartis which has used related technique to create a drug which knocks out spinal muscular atrophy in babies with a single injection.

She said: “The goal is a one-time therapy for a lifelong benefit - it’s a hefty one, but that’s the goal.

“We believe we have the greatest chance of success. It won’t be easy but the promise is there.”

The financing was led by New Enterprise Associates (NEA) and co-led by Monograph Capital, with participation from LSP, as well as seed investors Advent Life Sciences, Dementia Discovery Fund (DDF), F-Prime Capital, JJDC and medical research charity LifeArc.

Matt McAviney of NEA said: “Traditionally, there have been very few options to combat the devastating course of neurodegenerative disorders. AviadoBio’s platform introduces a revolutionary approach.”

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