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King's spinout to develop ground-breaking neurodegenerative gene therapy

AviadoBio is an exciting new spin-out company founded by King’s College London researchers Professor Christopher Shaw, Dr Youn Bok Lee and Dr Do Young Lee

Chris Shaw brain

AviadoBio, an exciting new spin-out company founded by King’s College London researchers Professor Christopher Shaw, Dr Younbok Lee and Dr Do Young Lee, has raised funds to use a patented gene therapy platform developed at the UK DRI at King’s to explore treatments initially for Frontotemporal dementia (FTD) and Amyotrophic Lateral Sclerosis (ALS, motor neuron disease, MND).

The substantial new investment of £58.6M ($80m) following £12M ($16.5m) seed funding will allow the company to take their pioneering 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 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% of inherited and 5% 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. Precision micro dosing achieves extensive gene expression throughout the nervous system, maximizing the therapeutic potential for patients living with devastating neurological diseases.”– Professor Chris Shaw, Professor of Neurology and Neurogenetics at the Institute of Psychiatry, Psychology & Neuroscience (IoPPN) at King’s College London, Co-founder and Chief Scientific and Clinical Advisor of AviadoBio

Professor Christopher Shaw, Professor of Neurology and Neurogenetics at the Institute of Psychiatry, Psychology & Neuroscience (IoPPN) at King’s College London, Co-founder and Chief Scientific and Clinical Advisor of AviadoBio explains, “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 Co-founder Professor Christopher 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.”

“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.”– Professor Shitij Kapur, President and Principal of King’s College London

Professor Ian Everall, Executive Dean of the Institute of Psychiatry, Psychology & Neuroscience where the work was completed said: “This is a hugely exciting development which will change the treatment landscape for neurodegenerative diseases.”

Kay Penicud, UK DRI Director of Business and Innovation, “We are very proud to see our work grow and flourish. Our core model at the UK Dementia Research Institute is to incubate ideas, unlock their potential and ultimately attract further investment to take them forwards so they can improve the lives of the millions of people affected by dementia.”

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

Click here for more information on the UK DRI.

For more information, please contact Patrick O’Brien (Senior Media Officer)

In this story

Christopher Shaw

Christopher Shaw

Professor of Neurology and Neurogenetics

Younbok Lee

Younbok Lee

Lecturer