AviadoBio, Astella Ink Deal for Dementia Gene Therapy

King’s College London

The agreement leverages King's College London spin-out company's expertise in developing targeted gene therapy for neurodegenerative diseases

Brain Web

AviadoBio Ltd., a spin-out company founded by King's College London researchers, announced today an exclusive option and license agreement with Astellas Pharma Inc. for AVB-101, a gene therapy in Phase 1/2 development for patients with frontotemporal dementia with progranulin mutations (FTD-GRN).

The agreement gives Astellas the option to receive a worldwide exclusive license for the development and commercialisation rights to AVB-101 in FTD-GRN and other potential indications.

Frontotemporal dementia (FTD) is a form of early-onset dementia affecting the frontal and temporal lobes of the brain. FTD is characterised by a rapid decline in executive function and changes in personality, behaviour, and language. It is an important cause of dementia in those under the age of 65 and is underrecognized and often misdiagnosed.

AVB-101 is an investigational, adeno-associated virus-based (AAV) gene supplementation therapy for FTD patients with disease-causing mutations in the GRN gene. It is designed as a one-time therapy to potentially halt disease progression by delivering a functional copy of the GRN gene to restore progranulin levels in the brain.

AviadoBio was co-founded in 2021 by Christopher Shaw, Youn Bok Lee and Do Young Lee from the School of Neuroscience at the Institute of Psychiatry, Psychology & Neuroscience. The company focuses on gene therapy as an approach to treat neurodegenerative diseases, based on the principle of delivering DNA into cells to supplement or knock down mutated genes. They use adeno-associated (AAV) to carry these gene therapies with precision, targeting specific cells while delivering broader effect through the nervous system.

The agreement between Astellas and AviadoBio is a huge endorsement of the gene therapies we are developing for neurodegenerative diseases. The extra funding will accelerate progress on additional therapies and expand our range of gene targets. We are finally delivering on a promise made to our patients that genetic discovery would translate into genetic therapy. We are immensely grateful for the early funding from My Name'5 Doddie Foundation, UK Dementia Research Institute, and John and Lucille van Geest Foundation which got us started."

Professor Christopher Shaw, co-founder of AviadoBio and Professor of Neurology and Neurogenetics at King's College London

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