Latest news with #AviadoBio
Yahoo
13-06-2025
- Health
- Yahoo
‘Transformative': the UK lab working on a way to halt genetic type of dementia
Behind the gleaming glass facade of an office block in east London's Docklands, Dr Martina Esposito Soccoio is pipetting ribonucleic acid into test tubes. Here, not far from Canary Wharf's multinational banks, a British university spinout is working on a breakthrough treatment for a form of dementia that affects millions of people worldwide. There is no cure for dementia at present, but scientists at AviadoBio hope their clinical studies can stop the progression of a particular genetic type of frontotemporal dementia (FTD). 'It may be one of the first dementias to have a definitive treatment, a cure if you like, a really transformative treatment that allows people to live much longer and much more normal lives,' says Prof James Rowe, a consultant neurologist at Cambridge's Addenbrooke's hospital who is involved in the UK trial. FTD mainly affects the front and sides of the brain and, unlike Alzheimer's disease, does not begin with memory loss, which tends to occur later. It is characterised by progressive loss of language and changes in personality and behaviour. Most cases are diagnosed in people aged 45 to 65, but it can affect those in their 20s and 30s. There are an estimated 20,000 to 40,000 people living with FTD in the UK, and between 1 million and 2 million in the world. Rowe says: 'It's a double-edged sword: the young onset, the high genetic burden and rapid illness are also features that perhaps make it more tractable to treat.' The Die Hard and Pulp Fiction actor Bruce Willis, who recently celebrated his 70th birthday, was diagnosed two years ago with FTD, which his family described as a 'cruel disease'. They have not said whether he has a genetic form of FTD. The gene therapy developed by AviadoBio, which was spun out of Prof Christopher Shaw's research lab at King's College London in 2021, targets a type of FTD known as FTD-GRN. This is caused by mutations of a gene that lead to a deficiency of progranulin (GRN), a protein that is essential for maintaining healthy brain cells. AviadoBio, which employs 60 people, signed an exclusive licence agreement with the Japanese pharmaceutical firm Astellas last October to develop the therapy. It is now recruiting patients for its clinical trial in the UK, as well as the US, Poland, Spain, Sweden and the Netherlands. The infusion was administered in Warsaw in March 2024 in one of six patients who have had the treatment so far, in Poland and the US. All patients will be followed for up to five years as part of the trial. AviadoBio expects to publish the first data next year. Three years ago, Jessica Crawford, from Beverley in East Yorkshire, lost her mother, Julia, to FTD, caused by mutations of another gene, C9orf72. In 2014, when Julia was 58, her behaviour changed; the family suspected depression. Previously very sociable, she stopped going out and started playing games such as Candy Crush or watching TV shows 'over and over', her daughter recounts. Her mother initially did not want to see a doctor, and was only diagnosed with FTD in February 2019. By this time she was so confused she once put raw chicken in a sandwich. 'Getting the diagnosis wasn't easy because FTD wasn't well known; my mum was acing in the memory tests,' Jessica says. Julia became increasingly confused and lost the ability to speak, and then to communicate at all. Jessica became her full-time carer in 2020, but her mother deteriorated so much that she had to enter a care home in late 2021, and died the following year. Jessica, 33, found out she also carries the gene mutation, and with her husband decided to conceive through IVF with a pre-implantation genetic diagnosis. When their five embryos were screened, four had the gene mutation, and the fifth resulted in the birth of their son. The couple donated their other four embryos to science and she takes part in GENFI – a long-running UK-led global study of families with FTD across 40 sites. AviadoBio itself was born out of the research done at King's by Shaw, a neurologist who has focused on FTD and amyotrophic lateral sclerosis (ALS) for more than three decades, and Dr Youn Bok Lee and Dr Do Young Lee from the UK Dementia Research Institute's centre based at King's. ALS, the most common form of motor neurone disease, has also been linked to mutations of the GRN gene and leads to muscle weakness, paralysis and eventually death. Shaw acts as the company's chief scientific and clinical adviser, and while the Lees are no longer involved in the firm's research, all three remain shareholders. David Cooper, AviadoBio's chief medical officer, says: 'It was something that hit me when I first joined the company, [when] you look at the MRI scans of patients with a GRN mutation, the frontal and temporal parts of their brain are just melting away … So we really need earlier treatment, an earlier diagnosis and a more organised healthcare approach to deal with it.' AviadoBio's lead product, AVB-101, is infused directly into the brain by a neurosurgeon using a cannula as thin as a strand of angel hair pasta, during a 90-minute procedure guided by MRI. It delivers a functional copy of the progranulin gene to restore appropriate levels of the protein to affected areas of the brain. It is a one-off treatment, and no immunosuppressant drugs are needed afterwards. 'The patients who have FTD are born with almost half of the progranulin levels that you and I might have,' says the chief executive, Lisa Deschamps. 'Our goal in the study is to supplement the GRN gene and restore as much progranulin in these individuals as possible to normal levels to reduce the neurodegeneration effect.' Other medications in development around the world include two gene therapies, from Philadelphia-based Passage Bio and Eli Lilly-owned Prevail Therapeutics, but they do not target the thalamus, the 'relay station' in the brain. Passage Bio's therapies are delivered directly to the cerebrospinal fluid in a single treatment. Denmark's Vesper Bio has developed an oral capsule, designed to act on the GRN gene, that is being trialled at University College London hospital. AviadoBio, whose investors include Johnson & Johnson's innovation arm and the UK not-for-profit LifeArc Ventures, is part of a growing life science cluster in Canary Wharf. At its labs, scientists – assisted by robotics – research how to target a particular gene. 'The UK has real strengths in this area,' says Rowe, pointing to the international GENFI study, run since 2011 by Prof Jonathan Rohrer, a neurologist from UCL Queen Square Institute of Neurology who also sits on AviadoBio's scientific advisory board. 'It's a real win for the UK.'
The Guardian
13-06-2025
- Health
- The Guardian
‘Transformative': the UK lab working on a way to halt genetic type of dementia
Behind the gleaming glass facade of an office block in east London's docklands, Dr Martina Esposito Soccoio is pipetting ribonucleic acid into test tubes. Here, not far from Canary Wharf's multinational banks, a British university spinout is working on a breakthrough treatment for a form of dementia suffered by millions of people worldwide. There is no cure for dementia at present, but scientists at AviadoBio hope their clinical studies can stop the progression of a particular genetic type of frontotemporal dementia (FTD). 'It may be one of the first dementias to have a definitive treatment, a cure if you like, a really transformative treatment that allows people to live much longer and much more normal lives,' says Prof James Rowe, consultant neurologist at Cambridge's Addenbrooke's Hospital. FTD mainly affects the front and sides of the brain and unlike Alzheimer's disease, does not begin with memory loss, which tends to occur later. It is characterised by progressive loss of language and changes in personality and behaviour. Most cases are diagnosed in people aged 45 to 65, but it can affect people in their 20s and 30s. There are an estimated 20,000 to 40,000 people living with FTD in the UK, and between 1m and 2m in the world. Rowe says: 'It's a double-edged sword: the young onset, the high genetic burden and rapid illness are also features that perhaps make it more tractable to treat.' The Die Hard and Pulp Fiction actor Bruce Willis, who recently celebrated his 70th birthday, was diagnosed with FTD two years ago, with his family calling it a 'cruel disease'. They have not said whether he has a genetic form of FTD. The gene therapy developed by AviadoBio, which was spun out of Prof Christopher Shaw's research lab at King's College London in 2021, targets a type of FTD known as FTD-GRN. This is caused by mutations of a gene that lead to a deficiency of progranulin (GRN), a protein that is essential for maintaining healthy brain cells. AviadoBio, which employs 60 people, signed an exclusive licence agreement with the Japanese pharma firm Astellas last October to develop the therapy. It is now recruiting patients for its clinical trial in the UK, as well as the US, Poland, Spain, Sweden and the Netherlands. The first patient received the infusion in Warsaw in March 2024, out of six patients who have had the treatment so far, in Poland and the US. All patients will be followed for up to five years as part of the trial. AviadoBio expects to publish the first data next year. Three years ago, Jessica Crawford, from Beverley in Yorkshire, lost her mother to FTD, caused by mutations of another gene, C9orf72. In 2014, when her mother was 58, her behaviour changed; the family suspected depression. Previously very sociable, she stopped going out and started playing games like Candy Crush or watching TV shows 'over and over,' her daughter recounts. Her mother initially did not want to see a doctor, and was only diagnosed with FTD in February 2019. By this time she was so confused she once put raw chicken in a sandwich. 'Getting the diagnosis wasn't easy because FTD wasn't well known; my mum was aceing in the memory tests,' Crawford says. But her mother became increasingly confused and lost the ability to speak, and to communicate at all. Crawford became her full-time carer in 2020, until her mother deteriorated so much that she had to go to a care home in late 2021, and died the following year. Crawford, 33, found out that she herself carries the gene mutation, and with her husband decided to conceive through IVF with a pre-implantation genetic diagnosis. When their five embryos were screened, four had the gene mutation, and the fifth resulted in the birth of their son. The couple donated their other four embryos to science and she takes part in GENFI – a long-running UK-led global study of families with FTD across 40 sites. AviadoBio itself was born out of the research done at King's by Shaw, a neurologist who has focused on FTD and amyotrophic lateral sclerosis (ALS) for more than three decades, and Dr Youn Bok Lee and Dr Do Young Lee from the UK Dementia Research Institute's centre based at King's. ALS, the most common form of motor neurone disease, has also been linked to mutations of the GRN gene and leads to muscle weakness, paralysis and eventually death. Sign up to Business Today Get set for the working day – we'll point you to all the business news and analysis you need every morning after newsletter promotion Shaw acts as the company's chief scientific and clinical adviser, and while the Lees are no longer involved in the firm's research, all three remain shareholders. David Cooper, AviadoBio's chief medical officer, says: 'It was something that hit me when I first joined the company, you look at the MRI scans of patients with a GRN mutation, the frontal and temporal parts of their brain are just melting away … So we really need earlier treatment, an, earlier diagnosis and a more organised healthcare approach to deal with it.' AviadoBio's lead product, known as AVB-101, is infused directly into the brain by a neurosurgeon using a cannula as thin as a strand of angel hair pasta, during a 90-minute procedure guided by MRI. It delivers a functional copy of the progranulin gene to restore appropriate levels of the protein to affected areas of the brain. It is a once-only treatment, and no immunosuppressant drugs are needed subsequently. 'The patients who have FTD are born with almost half of the progranulin levels that you and I might have,' says chief executive Lisa Deschamps. 'Our goal in the study is to supplement the GRN gene and restore as much progranulin in these individuals as possible to normal levels to reduce the neurodegeneration effect. Other medications in development at AviadoBio include two gene therapies, from Philadelphia-based Passage Bio and Eli Lilly-owned Prevail Therapeutics, but they do not target the thalamus, the 'relay station' in the brain. Passage Bio's therapies are delivered directly to the cerebrospinal fluid in a single treatment. Denmark's Vesper Bio has developed an oral capsule, designed to act on the GRN gene, that is being trialled at University College London Hospital. AviadoBio, whose investors include Johnson & Johnson's innovation arm and the UK not-for-profit LifeArc Ventures, is part of a growing life science cluster in Canary Wharf. At its labs, scientists – assisted by robotics – research how to target a particular gene. 'The UK has real strengths in this area,' says Rowe, pointing to the international GENFI study, run since 2011 by Prof Jonathan Rohrer, a neurologist from UCL Queen Square Institute of Neurology who also sits on AviadoBio's scientific advisory board. 'It's a real win for the UK.'
Business Wire
15-05-2025
- Health
- Business Wire
AviadoBio Announces ASPIRE-FTD Phase 1/2 Clinical Trial in the UK at Cambridge University Hospitals NHS Foundation Trust, Cardiff and Vale University Health Board, and University College London
LONDON--(BUSINESS WIRE)--AviadoBio, a pioneering gene therapy company dedicated to developing and delivering potentially transformative medicines for neurodegenerative disorders, today announced that its Phase 1/2 ASPIRE-FTD clinical trial is now open in the UK. The trial is evaluating AVB-101, an investigational gene therapy, in people with frontotemporal dementia (FTD) with GRN gene mutations (FTD-GRN). Cambridge University Hospitals NHS Foundation Trust (CUH), which hosts an internationally renowned center of excellence in providing support and care for families affected by FTD, is now recruiting patients. University College London (UCL) is also expected to open for patient recruitment shortly. The Advanced Neurotherapies Centre at Cardiff and Vale University Health Board, one of the first surgical centers in Europe currently able to perform MRI-guided infusions of gene therapies, will provide the capability to dose clinical trial participants from the UK and beyond in the ASPIRE-FTD study. FTD is a devastating form of early-onset dementia that varies in each individual. Symptoms of FTD can include changes in personality or uncharacteristic behaviors, progressive loss of language, loss of executive function and cognitive abilities, apathy, and reduced mobility. 1-3 People with FTD who have disease-causing GRN mutations produce a reduced amount of progranulin protein. AVB-101 is an investigational one-time therapy designed to deliver a functional copy of the GRN gene directly to the brain, thereby potentially restoring progranulin levels and stopping disease progression in patients with FTD-GRN. 'Launching ASPIRE-FTD and treating our first patients with AVB-101 have been significant milestones in FTD-GRN research and gene therapy development,' said David Cooper, M.D., Chief Medical Officer of AviadoBio. 'AviadoBio was built on pioneering research from King's College London and the UK Dementia Research Institute. The opening of our UK clinical trial sites reflects this strong research heritage, and we're proud to bring this clinical trial to the UK to make it more accessible to people living with familial FTD in the region.' 'We are delighted to be able to surgically deliver AVB-101 in the ASPIRE-FTD trial in Cardiff, offering hope to patients living with FTD in the UK and beyond. The Advanced Neurotherapies Centre is funded by Health and Care Research Wales and is currently the only center in the UK, and one of two European centers, conducting these groundbreaking first in human clinical trials, delivering advanced gene and cell therapies directly to the brain for neurodegenerative diseases. This trial represents a major step forward in the search for a treatment in FTD, potentially bringing a new therapy to reality for patients,' commented Professor William Gray, Local PI and Neurosurgeon at Cardiff University and Cardiff and Vale University Health Board. 'The potential to halt FTD-GRN with a single procedure could be truly transformative for patients and their families,' said Professor James Rowe, Consultant Neurologist at Addenbrooke's Hospital and Professor of Cognitive Neurology at the Cambridge Centre for Frontotemporal Dementia. 'Combining excellence in clinical care with cutting-edge research is vital to driving progress in this extremely challenging field. Through studies like this we hope to one day be able to provide a potential cure for FTD.' 'UCL is a leader in the Genetic Frontotemporal dementia Initiative, a group of research centers across Europe and Canada with expertise in familial frontotemporal dementia. We are proud to be included in this groundbreaking trial where targeted delivery of a single low dose of AAV gene therapy to the thalamus could be potentially disease-modifying for FTD,' said Jonathan Rohrer, Professor of Neurology at the UCL Dementia Research Centre. AVB-101 is delivered as a one-time-only treatment using a minimally invasive stereotactic neurosurgical procedure directly to the part of the brain called the thalamus. The thalamus is a key hub for connectivity in the brain with widespread projections across the brain including the cortex – a key area affected in FTD-GRN. The neurosurgery procedure to deliver AVB-101 bypasses the blood-brain barrier, thereby limiting it to only the brain itself, where it is needed the most. At the same time, this potentially reduces the amount of dose required and systemic exposure in other parts of the body. More information about the ASPIRE-FTD study can be found at About ASPIRE-FTD ASPIRE-FTD is a Phase 1/2 open-label, multi-center study designed to evaluate the safety and preliminary efficacy of AVB-101 in patients with FTD-GRN. In the study, eligible patients receive a one-time administration of AVB-101 delivered as a set of MRI-guided infusions into the thalamus during a minimally invasive stereotactic neurosurgical procedure at an expert neurosurgical center in the US, UK, or EU. More information about the ASPIRE-FTD study can be found at or About AviadoBio At AviadoBio, we are relentlessly chasing cures by translating groundbreaking science and precision delivery into life-changing medicines for people living with neurological conditions. With our deep understanding of the brain and suite of proprietary gene therapy platforms and delivery technologies, AviadoBio is working to overcome the challenges of delivering the right drug to the right place. Its innovative, neuroanatomy-led approach is designed to maximize the therapeutic potential of gene therapy to halt or potentially reverse neurodegenerative diseases. AviadoBio was founded on pioneering research from King's College London and the UK Dementia Research Institute and has a leadership team with extensive gene therapy development, delivery, and commercialization experience which uniquely positions the company for success in bringing transformative medicines to patients. AviadoBio's investors include New Enterprise Associates (NEA), Monograph Capital, F-Prime Capital, Johnson & Johnson Innovation – JJDC, Inc., SV Health Investor's Dementia Discovery Fund (DDF), Advent Life Sciences, EQT Life Sciences (Dementia Fund), LifeArc Ventures, and Astellas Pharma. For more information, please visit and follow us on X @AviadoBio and LinkedIn at AviadoBio. References: Pressman PS and Miller BL. Biol Psychiatry 2014;75:574–81; Young JJ et al. Ther Adv Psychopharmacol 2018;8:33–48; Hogan DB et al. Can J Neurol Sci 2016;43 Suppl 1:S96–109. Notes to editors: The Advanced Neurotherapies Centre, formerly known as the BRAIN Unit, is funded by the Welsh Government through Health and Care Research Wales. About GENFI The Genetic Frontotemporal dementia Initiative (GENFI) is a group of research centers across Europe and Canada with expertise in familial frontotemporal dementia (FTD) and is coordinated by Professor Jonathan Rohrer at University College London. GENFI collaborates closely with other similar studies around the world through the FTD Prevention Initiative, which is helping to design clinical trials for genetic FTD.
