Lifeline for dementia and ALS sufferers as UK company develops groundbreaking treatment

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AviadoBio has raised $80million (£58.6million) to progress with their treatments for neurodegenerative disorders including frototemporal dementia and motor neurone disease – otherwise known as amyotrophic lateral sclerosis (ALS). AviadoBio co-founder Christopher Shaw is a Professor of neurology and neurogenetics at King’s College London. He has spent the last 25 years investigating ALS and frontotemporal dementia.

Both are extremely aggressive diseases, for which there are currently no effective treatments.

The severity of the illnesses will allow Professor Shaw and his team to determine whether their groundbreaking experimental therapies have an impact relatively early on.

AviadoBio’s unique platform combines next-generation gene therapy design with deep neuroscience expertise and a novel neuroanatomy-led approach to drug delivery.

Frontotemporal dementia is associated with behavioural change and language loss.

The first programme works by supplementing a gene called progranulin, which when mutated is deficient.

AviadoBio’s approach is to deliver the progranulin gene to the brain using an adeno associated virus as a vector.

Instead of administering the doses intravenously or through the spinal fluid, surgery will be used to apply small amounts of the virus to the brain which will then distribute it naturally via neural networks.

Professor Shaw told “Our approach is a surgical approach to get past the blood brain barrier, and also past the peel membrane to go into the brain – or into the spinal cord – to deliver the virus and we’re then using a sort of neural network to deliver the virus around the brain.

“And because we’re able to put it in the right place to do the right thing, we can use very small doses and we don’t see an inflammatory response, which obviously would be a worry when you’re when you’re putting these agents into the brain.

“We tap into this neural network, which then delivers to the cortex, which of course, is where the disease, frontotemporal dementia, has its greatest effect.

“So we have to be able to supplement the genes missing in those cells, and thereby prevent the degeneration.”

The team will start by treating symptomatic patients who are in the early phases of the disease.

But Professor Shaw hopes that if proven successful, the therapy can also be used to treat people who are “genetically at risk” from developing the disease thereby preventing them from becoming symptomatic later in life.

Another AviadoBio programme which will benefit from the huge funding works to “knock down” genes.

“Many of the genes which cause neurodegeneration are toxic, so you can’t just supplement them because that is not the problem, it is you body making a toxic protein,” Professor Shaw explained.

To combat this, the team intends to target the messenger RNA (mRNA) of the toxic genes and using a micro-RNA platform to knock it down.

This is the kind of therapy which AviadoBio hopes can transform the lives of those suffering from motor neurone disease.

Professor Shaw explained: “Motor neurone disease is a terrible paralysing illness usually kills people within three years of symptom onset and is the most common reason that people seek euthanasia.

“So, it’s a very, very severe disease that there is no effective treatment for.

“People are absolutely desperate and we think we’ve got a really, really powerful therapeutic approach, which is to knock down the genes that are causing the disease.

“And I think that’s a really fantastic opportunity to really have an impact.”

Working alongside Professor Shaw are his fellow co-founders: molecular neurobiologist Dr Youn Bok Lee and vector biologist Dr Do Young Lee from King’s College London and the UK Dementia Research Institute.

Lisa Deschamps, Chief Executive Officer, brings to the team 25 years of industry and extensive gene therapy experience.

Commenting on the $80million investment, she told “We’re extremely pleased with the raise and we feel very confident that there is tremendous interest.

“We feel very confident that the 80 million will help us to advance our lead programme in progranulin, as well as look to accelerate the other pipeline assets.”

Ms Deschamps said the money would help the team to collect more data and to hopefully get their therapies into clinics and dosing patients by the end of next year.

To get to that stage, they will need to get proof of safety and effective distribution in non-human primate studies.

After that data is collected, AviadoBio can apply for approval from the UK-based Medicines and Healthcare products Regulatory Agency (MHRA), and the American Food and Drug Administration (FDA) to allow them to run the human trial.

Professor Shaw added: “We’re so excited about this. Now we have got the funds to do the experiments properly and to actually offer this to patients who want to be part of the trial.

“It is the next generation of therapies that are coming through – we haven’t proven that they’re curative yet, but that is our goal.”

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