In October, U.K.-based gene therapy company AviadoBio, Ltd., and pharmaceutical company Astellas announced an exclusive option and license agreement for an investigational therapeutic, AVB-101, which is currently in a Phase 1/2 trial for people with FTD-GRN.

AFTD recently spoke with AviadoBio Chief Executive Officer Lisa Deschamps about her colleagues’ work and vision for a collaborative approach to improving the lives of all who are living with or facing FTD. The conversation has been edited and condensed for clarity.

You can learn more about the partnership between AviadoBio and Astellas in this press release.

What approach is your company using to treat FTD?

AviadoBio’s mission is to develop and deliver transformative gene therapy for neurological conditions, starting with FTD and ALS. Our ASPIRE-FTD trial is focused on treating a monogenic form of FTD where people have a variant in the GRN gene, which can impact the body’s ability to produce sufficient, functional levels of progranulin. Suppressed levels of the protein can lead to abnormal accumulation of TDP-43, implicating overall brain health.

Using adeno-associated virus (AAV) technology, coupled with intraparenchymal injection – a novel approach to delivery – we will be able to administer very small doses of our therapeutic, AVB-101, directly into the brain and be very targeted to the affected areas. This approach, which is a one-time dose delivered in an MRI-guided surgical procedure, enables our therapeutic to bypass the blood-brain barrier and pial membrane, thereby avoiding any unwanted systemic exposure that comes with administration into the cerebrospinal fluid.

Ultimately, the goal of our trial is to restore brain progranulin levels, thereby potentially slowing or stopping the progression of FTD-GRN. While it is still early days for our Phase 1/2 trial, where we’ve so far dosed three people, and we hope to enroll a total of 6-9 patients in the US, Spain, Netherlands, and Poland. Our main goals for our ASPIRE-FTD trial are to demonstrate the safety of AVB-101 as a one-time treatment for FTD-GRN, determine the dose required for potential clinical benefit, and evaluate our therapeutic’s effectiveness on disease progression over a five-year period.

What do you think is the most urgent need to be addressed for people facing FTD and their families?

I think there are a few very important categories I’d highlight. For one, I think there needs to be better education, awareness, and tools to identify and diagnose FTD. We know that for a large portion of the community, FTD is not familial; but even for those for whom it is familial, we’re often seeing the first generation of people getting an accurate diagnosis and then learning it is genetic through predictive testing. That’s a huge opportunity for us to understand how we can better reach people before diagnosis – how we should drive that urgency – and what resources are needed for the individual and the whole family afterward. Also, what can we change in our approach or policies to improve our monitoring for abnormalities and to be more proactive in giving people the information and help they need?

We also need therapeutic interventions. While nothing is approved for use today, many of us are working to bring a therapeutic to market. Our therapy will not cover the broader population at first – many of us are focused on the genetic forms of the disease – but we know there is a need to provide options for sporadic forms of the disease. We need a lot more education and awareness to help clinicians know what to look for since FTD presents so differently from other forms of dementia. Unfortunately, there are probably a lot of people with FTD who are sitting under the umbrella of Alzheimer’s or other disease and not getting the support they really need.

Collectively, as an industry and working with organizations like AFTD, we can better understand how we can make an impact in these areas. I don’t think we can do it alone.

What role do you hope you and your colleagues play in empowering this community?

Our goal is to be a part of introducing a therapy to patients, and we take this very seriously. We hope that in 10-15 years, maybe even sooner, the FTD disease area will look very different. Just like in other disease areas where there was no treatment previously, like multiple sclerosis or rheumatoid arthritis, the landscape today is very different compared to where it was. In all of these cases, there was a lot of work to raise awareness of the disease through education to ultimately better understand the pathogenesis of the disease and bring forward a treatment.

There’s still a lot about FTD we don’t know, especially for sporadic forms of the disease, so there’s a lot of research to be done. This is where industry, advocacy organizations, and policymakers can all work together to advance understanding of the disease and bring meaningful treatments or potential cures for people. We can do a lot together to educate, raise awareness, develop tools for diagnosis, encourage genetic testing, and get therapies into the hands of those who are in need.

What is most encouraging about today’s research landscape?

I think we’re learning a lot about the pathogenesis of disease, but there’s still a lot we don’t know. It’s encouraging to see people like Emma Heming Willis coming out and raising the flag and showing the impact that this disease, unfortunately, has on their family. Her work alongside others has opened people’s eyes – the stage is there now; it’s what we do with it that will be so important. We are learning how we can effectively talk about the disease, but we also need to create and implement an action plan. Our recent attendance at the International Society for FTD conference in Amsterdam gives me hope. There was a lot of energy in the space around the scientific progress being made – there was a palpable sense that we are on the edge of something monumental, something that is about to be discovered. Each diagnosis, research study, or clinical trial gives us more information so we can put real treatments in the hands of real people.

We’ve also learned a lot about biomarkers, but there is still an urgent need and much work to be done there. These tools are necessary to help us not only develop treatments and assess the outcomes of therapies but also be aware of what we need to look for and introduce therapies in a timely way.

AFTD Note: On Thursday, May 1, 2025, AFTD will feature a half-day symposium on the genetics of FTD at the AFTD Education Conference in Broomfield, Colorado to increase awareness about the known genetic causes of FTD, debunk myths about FTD genetics and genetic testing, and provide information about ongoing clinical trials and the urgent need for study volunteers to further drug development. The agenda will include an opportunity for companies currently sponsoring clinical trials to present on their work while fostering dialogue to further FTD-focused research and improve available resources and support options. Sign up to receive updates on event registration here.

What are the challenges you see and what are your hopes for how stakeholders can overcome these together?

One of the biggest challenges is that while we have learned a lot about the pathogenesis of the disease, there’s still a lot we don’t know. When I think about the families that I’ve had the privilege to meet, including at AFTD’s events, I think about what makes a difference between the mother with FTD, who unfortunately just passed away when she was 55, and the 85-year-old grandmother, who was a carrier but is still doing well and is asymptomatic. How can we predict the disease’s progression, and what other factors were involved in making such a difference in people’s outcomes? In both cases, there are low progranulin levels, but one person’s symptoms progressed dramatically when another showed no symptoms; what makes the differences there? Scientifically, that’s a challenge. We need to better understand the disease pathology to know when it’s best to begin treating someone – ideally before people start showing symptoms. There is an enormous amount of natural history data, but as we go, we’ll need to continue building on that base.

Another big challenge in the United States is advancing genetic testing and access to diagnostic tools while ensuring this information doesn’t impact a person’s ability to get health insurance or long-term care coverage. Nothing should stop a person from fully understanding their health, especially when there is a family history of a disease and when there’s a possibility of an intervention or clinical trial participation. All these things take time – we need action and mobilization, to dial up the urgency to know all we can about the disease and move toward a more proactive model. Right now, it’s a conundrum for people who face the disease – there’s a risk in learning their genetic status, but there’s also a great expense when someone doesn’t have the information they need. As a community, how do we support people and empower them to make these decisions? People need to choose for themselves what’s right – but if they’re not educated and don’t have tools and resources available to them, then shame on us. Ultimately, the decision point lies with the individual and their families. Our job collectively is to make those resources available to them.

At AviadoBio, we’re really excited about and committed to the work we’re doing. We are so grateful to those who are willing to be a part of our ongoing ASPIRE-FTD clinical trial, and we want to thank each person, the community, and AFTD. I know we will learn a lot from these trials – and we are heads-down and focused on our mission.

AFTD Note: Whether you have a personal or family history of FTD, or whether past misdiagnosis could potentially be masking one, AFTD strongly recommends that you consider genetic counseling as a first step to answering questions about your genetic status.

A genetic counselor can evaluate the likelihood that your condition, or your family’s condition, has a genetic cause and can help you think through the specifics of genetic testing and how to talk to family members about the testing process, privacy concerns, and more. For people who are interested in pursuing testing, the counselor will discuss its risks and benefits and identify the specific test that can ensure the most accurate results possible. More information about genetic counseling can be found here.

AFTD sincerely appreciates AviadoBio’s continued support of our mission and the people we serve.

To learn more about AviadoBio’s ASPIRE-FTD trial, which is currently underway, please visit www.aspire-ftd.com, or visit the Studies Seeking Participants page to learn more about trials that are actively recruiting. Signing up for the FTD Disorders Registry is another way that persons diagnosed, care partners, and family can participate in research.