- Intrathalamic delivery of AVB-101 shows promising efficacy in a pre-clinical disease model
- Gene therapy candidate also demonstrated broad cortical expression in a large animal biodistribution study
London, UK, May 4th, 2022 – AviadoBio, a pioneering, pre-clinical stage gene therapy company focused on developing and delivering transformative medicines for people with neurodegenerative disorders, announces that it will be presenting preclinical data at the upcoming ASGCT 25th Annual Meeting (American Society of Gene & Cell Therapy) on 16-19 May, 2022. The data are from studies it has conducted with its investigational, one-time, adeno-associated virus (AAV) gene therapy, AVB-101 (AVB-PGRN), for the treatment of frontotemporal dementia with GRN mutations (FTD-GRN).
The oral presentation, entitled “Intrathalamic Delivery of AVB.PGRN Rescues Pathology in Grn Null Mice and Achieves Widespread Cortical Expression in a Large Animal Model without Expression in the Liver”, will be presented by AviadoBio Co-Founder and Chief Scientific and Clinical Advisor, Professor Chris Shaw, on Tuesday 17 May at 3:45pm (EST). The presentation will form part of the session “Applications of Improved Gene Therapy Methods in Neurologic Disorders” and will be followed by a Q&A discussion.
The abstract number 468 can be viewed here.
In addition, AviadoBio CEO Lisa Deschamps will be presenting at the conference, on Monday 16 May at 12:00pm (EST), in a session entitled: “Startup Showcase: AviadoBio – A revolution in gene therapy for neurodegenerative disorders”.
At AviadoBio, our mission is to transform the lives of people living with neurodegenerative disorders by developing and delivering transformative gene therapies for diseases including frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). The Company’s technology is based on pioneering research from King’s College London and the UK Dementia Research Institute. AviadoBio’s unique platform combines next-generation gene therapy design with deep neuroscience expertise and a novel neuroanatomy-led approach to drug delivery. AviadoBio’s investors include New Enterprise Associates (NEA), Monograph Capital, Advent Life Sciences, EQT Lifesciences, Dementia Discovery Fund (DDF), F-Prime Capital, Johnson & Johnson Innovation – JJDC, Inc. (JJDC), and LifeArc.
The company is developing AVB-101 for patients with FTD-GRN. AVB-101 is an investigational AAV gene therapy designed to slow or stop disease progression by delivering a functional copy of the GRN gene throughout the central nervous system to restore progranulin levels.
For more information, please visit www.aviadobio.com and follow us at Twitter @AviadoBio and LinkedIn AviadoBio.
About Frontotemporal Dementia and AVB-101
Frontotemporal dementia (FTD) is of the second most common form of dementia in people under the age of 65 after Alzheimer’s disease. It affects 50,000 to 60,000 patients in the U.S. and over 100,000 in the E.U. Approximately one third of FTD cases are familial and linked to autosomal dominant mutations in three genes including the granulin gene (GRN) and FTD-GRN represents 5-10% of all patients with FTD. Progressive degeneration of the frontal and temporal lobes of the brain is characteristic of FTD, and is associated with progressive decline of behaviour, decision-making, language and emotion, typically leading to death within 7-10 years of diagnosis. There are currently no approved treatments to stop or slow the progression of FTD or FTD-GRN.
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