AviadoBio was founded in London in 2019 based on research carried out at King’s College London and the UK Dementia Research Institute by Chris Shaw, Youn Bok Lee and Do Young Lee.
The biotech focuses on developing gene therapies for neurological conditions with a genetic component with a focus on amyotrophic lateral sclerosis (ALS), also known as motor neuron disease, and frontotemporal dementia (FTD).
AviadoBio’s lead candidate, AVB-101, which is being licensed by Astellas, is an adeno-associated viral (AAV) vector gene therapy targeting FTD with mutations in the progranulin (GRN) gene. If effective, this treatment is designed to be a one-time therapy that will restore progranulin protein levels in the brain.
The deal with Astellas entitles AviadoBio to a US$30 million upfront payment and the big pharma are also investing US$20 million in equity in AviadoBio. Astellas will be able to receive a worldwide license for development and commercialization of AVB-101, if trials go well. If the therapy reaches the market, AviadoBio also stands to gain a total of $2.18 billion in combined license fees, milestone payments and royalties from Astellas.
“As we complete dosing of the first cohort of patients in our phase 1/2 ASPIRE-FTD trial of AVB-101, we are excited about the potential of this collaboration to help address the unmet need that exists today in FTD,” said Lisa Deschamps, CEO, AviadoBio, in a press statement.
“This strategic collaboration will combine our promising gene therapy candidate for FTD-GRN and delivery expertise with Astellas’ global capabilities in development and commercialization of gene therapies. Together, we can further accelerate delivering this investigational medicine to families around the world who so desperately need treatment options for FTD-GRN and other neurological diseases.”
Fulfilling an unmet need
FTD comprises several different types of dementia that impact the frontal and temporal lobes of the brain. Compared with other types of dementia, FTD is early onset, and symptoms typically begin between the age of 45 and 65 years with an estimated prevalence of 4.6 cases per 1,000 people.
Some cases of FTD are sporadic and do not appear to be inherited, but at least a third of cases are familial and a strong family history of the disease is often seen in these patients. Symptoms are often severe, and patients typically die between three and 13 years after being diagnosed.
Several genes are implicated in the pathology of FTD, but most familial cases are caused by autosomal dominant inherence of mutations in three genes, one of which is GRN, located on chromosome 17.
There is a strong unmet need for treatments for FTD patients as there are currently no approved disease-modifying therapies on the market. Although AAV gene therapies are not a new concept, targeting them to reach specific organs such as the brain can be problematic. AviadoBio has shown it can successfully inject AVB-101 into the brain of mice, sheep and monkeys in small doses in preclinical trials. It has also shown good effects of the gene therapy in animal trials with an increase in progranulin seen in the brain and a significant reduction in disease symptoms.
Although early-stage trials have been promising, whether AVB-101 can also be successful in clinical trials remains to be seen. Many neurodegenerative disease treatments that showed early promise have failed in later clinical trials in recent years, but with two newly approved Alzheimer’s treatments on the market things are beginning to improve and there is increased optimism in the space.
