The biotech out of King’s College London has raised $80 million (£56 million) to fund clinical trials of its “revolutionary” gene therapies with the potential to cure dementia.
AviadoBio aims to be a one-shot treatment that prevents cells in the brain and spinal cord from shrinking (neurodegeneration), which can lead to the conditions frontotemporal dementia (FTD) and motor neuron disease (MND).
There is currently no cure for any devastating disease, while current treatments are life-limiting and cumbersome.
Once the technology is proven, co-founder Professor Christopher Shaw believes that tackling more complex diseases 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 in Shaw’s lab over 25 years and uses a viral vector to deliver DNA into nerve cells, which have lost their ability to make proteins necessary to stay healthy.
Injecting a working copy of the gene helps the cell’s internal machinery to start making proteins again. The process can also work in reverse to knock out defective versions of the protein.
AviadoBio said the treatment completely eradicated symptoms in tests on rats and sheep.
The £56 million Series A round of funding will allow the team to expand in preparation for starting human trials, which are planned for late next year.
The treatments are seen as an important step towards both the prevention and treatment of many non-treatable degenerative conditions.
AviadoBio’s flagship project targets FTD, an aggressive and common form of youth-onset dementia characterized by changes in personality, behavior, and language.
The target protein is progranulin, which plays an important role in cell growth, survival and repair.
Current treatments include monthly lumbar puncture to inject supplements of this missing protein into the spinal fluid.
Since nerve cells persist for a lifetime, AviadoBio believes that a successful single dose of a working gene therapy has the potential to deliver lifelong results.
Shaw said: “Gene therapies are incredibly powerful, if we can deliver them safely we can really make a difference.
“These are powerful tools and we have a real chance to cure patients for whom there is no effective treatment.
“We’re starting with concrete causal factors that we think we can correct, then we’ll start working on a more complex disease like Alzheimer’s.
“We are far from being there but at least now we have the weapons to make it happen.”
Lisa Deschamps was appointed CEO of Aviedobio earlier this year, joining Novartis to create a drug that uses related technology to eliminate spinal muscle atrophy in infants with a single injection .
She said: “The goal is one-time therapy for lifelong benefits – it’s an overwhelming one, but it’s the goal.
“We believe we have the greatest potential for success. It won’t be easy but there is promise.”
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 was. LifeArch.
NEA’s Matt McVinney said: “Traditionally, there are very few options for dealing with the devastating course of neurodegenerative disorders. Aviedobio’s platform offers a revolutionary approach.”