Pioneering research which sees the use of a gene therapy platform to explore treatments for neurodegenerative diseases has been backed by new investment of £58.6million.
AviadoBio has raised the funds to use its patented platform to initially look into therapies for Frontotemporal dementia (FTD) and Amyotrophic Lateral Sclerosis (ALS, or motor neurone disease, MND).
The substantial investment to the venture – developed at the UK DRI at King’s – follows £12million in seed funding, and will allow King’s spinout AviadoBio to take their research into human clinical trials in FTD patients for the first time.
The funds will also help advance other pre-clinical work into ALS and ultimately other neurogenerative diseases.
Frontotemporal dementia is a progressive neurodegenerative disease affecting the frontal and temporal lobes of the brain and is the second most common form of young-onset dementia (over 65 years). FTD is characterised by changes in personality, behaviour and language, rather than short-term memory deficits seen in Alzheimer’s disease.
ALS/MND causes progressive muscle paralysis affecting limb movements speech and swallowing, leading to death from respiratory failure within three to five years.
Crucially, there are no treatments that can slow or halt progression of either disease.
Gene therapy is a promising approach for the treatment of neurodegenerative diseases, based on the principle of delivering DNA into cells to supplement or knock down mutated genes.
AviadoBio – founded by Professor Christopher Shaw, Dr Youn Bok Lee and Dr Do Young Lee – is using the Adeno-associated virus (AAV), which can be engineered to carry theses gene therapies to target specific cells such as neurons in the brain and spinal cord. This technology proved life changing for babies with the genetic motor neuron disease, spinal muscular atrophy (SMA), who rarely lived beyond two years without being ventilated.
A single injection in infancy protected their motor neurons such that they could walk, talk and did not need ventilation.
The therapy will target the gene encoding the protein progranulin, which is mutated in about 20 per cent of inherited and five per cent of non-inherited or sporadic cases.
Progranulin is important for biological processes such as inflammation and the growth of neurons, and mutations of the gene in FTD cause a significant reduction in the levels of the protein. Delivering unmutated copies of the gene will help restore this imbalance in progranulin and improve the symptoms experienced in the disease.
“AviadoBio’s unique platform combines next-generation gene therapy design with deep neuroscience expertise and a novel neuroanatomy-led approach to drug delivery,” says Professor Christopher Shaw, professor of neurology and neurogenetics at the Institute of Psychiatry, Psychology & Neuroscience (IoPPN) at King’s College London, and co-founder, chief scientific and clinical advisor of AviadoBio.
“Precision micro dosing achieves extensive gene expression throughout the nervous system, maximising the therapeutic potential for patients living with devastating neurological diseases.
“While many neurodegenerative conditions start in a specific region gene, the pathology eventually spreads throughout the nervous system.
“We have seen that modifying gene expression can be curative, but achieving widespread distribution is the greatest challenge. We have shown that precision micro dosing to neural networks will deliver broad central nervous system (CNS) expression, providing safe and effective treatments.”
The investment marks a huge milestone for the new company, as well as for Professor Shaw personally, whose decades of discovery research into FTD and ALS have set the groundwork for these promising new disease therapies.
Professor Shaw continues “After spending my entire career to date researching the causes of these diseases and designing therapies that bring hope of a cure for patients, I believe that AviadoBio has the potential to achieve this.”
Professor Shitij Kapur, president and principal of King’s College London, said: “Chris has worked with King’s for over 25 years.
“I’m honoured that he chose our university to develop this ground-breaking research, and that staff and students have benefited from his world leading science along the way. I am pleased King’s is helping to take that work forward that has the potential to change the world and make a huge impact on many people’s lives.”
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