Astellas Pharma and AviadoBio have announced an exclusive license agreement for AVB-101, an experimental gene therapy for FTD-NRG currently undergoing a phase 1/2 clinical trial in the United States and Europe.

FTD caused by an inherited NRG mutation génétique inhibits the production of the essential protein progranulin, which leads to the accumulation of proteins such as TDP-43 in the brain. AVB-101 works by introducing a functional copy of the NRG gène to the brain to restore progranulin production.

“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 frontotemporal dementia,” said AviadoBio CEO Lisa Deschamps. “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.”

Through the terms of the agreement, Astellas has the option to receive a worldwide exclusive license for the development and commercialization rights to AVB-101 in FTD-NRG, as well as other potential uses. Astellas will invest $20 million in AviadoBio in addition to up to $30 million in upfront payments, and up to $2.18 billion if Astellas exercises its option. The deal reflects the potential of AVB-101, and sets a precedent for further investment in similar interventions for FTD.

More Resources Behind Research and Potential Production of AVB-101

A crucial aspect of the agreement between Astellas and AviadoBio is the significant increase in resources behind the research and development of AVB-101.

As therapies progress through the clinical trial process, the costs of research and development increase considerably. Not only must companies contend with the greater complexity of later-stage trials, they must navigate regulatory processes for each country their trial is in, and commit considerable resources to recruit and support participants. Even if a trial is successful, there are further regulatory and logistical hurdles to be cleared before a company can begin production and distribution of a therapy.

Astellas, a multinational pharmaceutical company based in Tokyo, has the resources and infrastructure to support larger-scale trials and potentially bring AVB-101 to market. Additionally, Astellas has some clinical experience with gene therapies, along with the manufacturing capabilities to support production of AVB-101. By partnering with one another, AviadoBio gains considerable support in furthering the development of its gene therapy, while Astellas can leverage its experience and resources to ensure that the therapy reaches people affected by FTD.

Are you interested in learning more about the steps of the clinical trial process AVB-101 has cleared so far? The gene therapy was first approved for clinical trials in the U.S. in November 2023, with the first person being dosed with the therapy in Europe later in 2024. In July 2024, clinical trials began in the U.S. at The Ohio State University.