AFTD recently spoke with AviadoBio Chief Executive Officer Lisa Deschamps about her colleagues’ work to develop a therapeutic and treatment protocol targeting FTD.

*Responses have been edited for clarity and length.

 

Can you share some background on AviadoBio’s approach to developing an FTD-focused therapeutic?

We are a drug and delivery company focused on addressing the key challenges in therapeutic areas with significant unmet needs. Our mission is to develop and deliver transformative gene therapy for neurological conditions, starting with FTD and ALS. We know that drug delivery is a barrier in treating central nervous system (CNS) diseases, so our approach is to take what we know and what we’ve learned – and are continuing to learn – and do it better. In this case, we know a lot about using adeno-associated virus (AAV) technology in CNS conditions and we are coupling it with a novel approach to delivery.

By using intraparenchymal injections, we will be able to deliver very small doses of our therapeutic directly into the brain and be very targeted to the affected areas, which we hope will translate into maximal therapeutic benefit while managing any negative safety indications. Striking the right balance is so critical and it’s been a key challenge to understand how best to cross the blood-brain barrier and deliver drugs systemically.

We know the disease starts out more focally, but as it progresses and people develop more symptoms, FTD becomes more global in nature. By going directly into the brain, specifically into the thalamus, which is a central relay hub to the brain, we hope to achieve a broad cortical distribution to all the areas of the brain that are, not only at the onset but also over time, affected by the disease.

Currently, our lead program focuses on the monogenic form of FTD where people have a variant in the GRN gene, which we believe – based on the current literature – is about 10% of all diagnosed individuals. Additionally, up to half of the people in this group have a family history of the disease while others wouldn’t have any known history – so it’s also important to figure out how we can implement genetic testing to identify affected individuals earlier and develop ways to proactively monitor people as they age for early signs of CNS-related diseases.

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 you or your family’s condition has a genetic cause and can help you think through the benefits and risks of genetic testing and ways to talk to family members about genetic testing, privacy concerns, and more. For individuals who are interested in genetic testing, the genetic counselor will discuss the risks and benefits of testing and identify the specific test that can ensure the most accurate results possible. For more information about genetic counseling, click here.

What inspires you and your colleagues to work in the FTD and neurodegenerative disease space?

AviadoBio has a team of nearly 60 people who are rallied around this common cause to treat people across CNS diseases, specifically starting with neurodegeneration. We have neurologists and neurosurgeons on board with deep scientific capability and interest. The driving force, however, is when you look across therapeutic areas and consider the ones that still have such significant unmet needs, CNS diseases – especially neurodegenerative diseases – just scream out for solutions.

For any biopharmaceutical company, you look for where there’s an unmet need and where you can contribute to transforming care – the neurodegenerative area is certainly one of the biggest ones. Coupling the capabilities and expertise of the AviadoBio team members with the huge unmet need that exists, we aim to take things we know and find a different way to do them so we can work quickly and expeditiously on behalf of people affected by diseases like FTD where there’s currently no treatment.

What do you hope people will learn and understand about FTD?

Working with groups like AFTD and the community at large, we learn something new every day. Something I’ve learned hearing from many families is how FTD’s symptoms – which so often go misdiagnosed – frequently begin with changes in behavior or in ways that you would not associate with dementia. We’re also learning that people can present these symptoms for a very long time before diagnosis, all while time is ticking, which we know is bad in neurodegeneration. I think these are some of the biggest things we need to address and educate people about. We all need to be more aware of the early warning signs and understand that there might be an underlying medical cause for what’s going on.

First, we need to educate people about what this disease really looks like, but then we also need to think about our medical practices. If you take your average 40- or 50-year-old individuals, there would be normal proactive or preventative care protocols where doctors are able to identify issues or diseases before there are symptoms – much like a mammogram or colonoscopy. In neurodegeneration, there are currently no tests until a person is symptomatic.

If we think about things like policy reform and everything necessary to truly transform care, it’s going to take a lot of disciplines and stakeholders – certainly, biopharmaceutical companies are a part of this equation – coming together to educate not only about the disease but how we can effectively make a difference and treat it. 

This year, AFTD is focused on making sure every FTD story counts. Why do you think it is important for people to share their stories?

The power of the voices of people living and dealing with these diseases is so profound – and we as stakeholders collectively need to provide that platform for people to connect, share, educate, and most importantly, support one another. At the end of the day, it’s really about the connections that people make that will really supercharge education and awareness. For the families we’ve spoken to, a lot of their passion comes from being able to tell other people about their experience and educate others on the symptoms, coping mechanisms, and ways they’ve learned to live life to the fullest until there are therapeutics available.

I think the power of connections between people is so critical. Collectively, we need to think about how we can support people impacted – we are learning every day – and it’s so important to hear what families need as it relates to this disease. There’s power in that for us as we are thinking about developing clinical trials and continuing our development.

What are your hopes or visions for the future of FTD and the neurodegenerative disease landscape?

My hope is that we ultimately find ways to bring treatments to [people who are diagnosed] that are transformative, not just addressing symptoms. Although, in some of these areas like FTD there aren’t even symptomatic treatments yet – so any opportunity to make people feel better is a good thing. I hope that we end up in a world where people don’t feel helpless and where we have medical practices established for neurodegenerative diseases that are able to look for signs and symptoms early on. I hope we will truly understand the diseases inside and out so we know where to look and can provide the right tools for diagnosis and that we can begin treating the disease before it even starts, or – the ultimate goal – we can prevent or eradicate these diseases.

Finally, I hope we live in a world where all of us together, as multidisciplinary stakeholders, come together to collaborate on solutions because none of us can do it on our own. We really need collaboration. How would therapeutic development look different if we all came together and tried to do things better on behalf of the people we are trying to serve?

 

AFTD is sincerely grateful for AviadoBio’s support of the 2023 Education Conference as a Gold-level sponsor. You can now view recorded sessions of this year’s event on AFTD’s YouTube channel.