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 Prof Chris Shaw, and Dr Youn Bok Lee and Dr Do Young Lee from the UK DRI’s centre based at the School of Neuroscience at the King’s College London 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.
Prof Shaw said:
“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, the UK Dementia Research Institute, and John and Lucille van Geest Foundation which got us started.”
Roisin NicAmhlaoibh, UK DRI Chief Innovation & Business Officer, said:
“Congratulations to AviadoBio on this exciting milestone. The success of this work exemplifies the power of innovative partnerships in accelerating the development of urgently needed therapies for conditions like frontotemporal dementia. This collaboration with Astellas represents a pivotal moment in the advancement of gene therapy for neurodegenerative diseases.”
Source: King’s College London
Article published: 8 October 2024
Banner image: AviadoBio