First Patient in ASPIRE-FTD Trial Receives AviadoBio's AVB-101 for FTD-GRN

A novel gene therapy, AVB-101, has been developed by AviadoBio, a leading company in the field of neurodegenerative disorder treatments. This therapy is a groundbreaking approach that aims to directly deliver a functional GRN gene copy to the brain, with the goal of replenishing progranulin levels and potentially halting the progression of frontotemporal dementia (FTD) for patients with progranulin (GRN) gene mutations, known as FTD-GRN.

The ASPIRE-FTD trial, which is currently enrolling patients, marks a significant advancement as it is the first study of its kind to administer gene therapy directly into the thalamus for adult neurodegenerative diseases. The trial is set to take place at various locations across Europe and the United States, with existing sites in Poland, Spain, and the Netherlands, and further expansion planned.

Lisa Deschamps, AviadoBio's CEO, emphasized the company's dedication to offering innovative treatments for FTD-GRN, highlighting the importance of this milestone for both the FTD community and the company. She expressed gratitude to the clinical investigators and families participating in the trials, acknowledging the potential of AVB-101 to transform the lives of those affected by FTD-GRN.

FTD is a severe form of dementia that typically results in death within a short timeframe following diagnosis. It is characterized by a range of symptoms including personality changes, behavioral issues, language loss, and reduced mobility. Being a leading cause of dementia among individuals under 65, FTD is often misdiagnosed. AVB-101 is designed as a one-time therapy that could potentially halt the disease by restoring the necessary levels of progranulin protein in the brain.

The Chief Medical Officer, David Cooper, M.D., underlined the urgent need for new treatments for FTD-GRN, given the absence of effective disease-modifying therapies. The treatment's initiation in the ASPIRE-FTD trial signifies a crucial step in assessing the potential of AVB-101, which has already shown positive results in preclinical studies.

Delivered via a minimally invasive neurosurgical procedure, AVB-101 targets the thalamus, a brain region with extensive connections to areas critical for FTD. This method is intended to safely bypass the blood-brain barrier, ensuring the therapy reaches the frontal and temporal cortex where it is most needed, while minimizing dosage and potential systemic exposure.

The Interventional Neurotherapy Center at Mazowiecki Szpital Bródnowski Hospital in Europe is pioneering the use of MRI-guided infusions for gene therapies. Prof. Mirosław Ząbek, chair of neurosurgery at the center, expressed excitement about participating in this research and performing the first intrathalamic administration of AVB-101.

Dr. Gabriela Klodowska, a neurologist and founder of the Neuro-Care Clinic, shared the significance of bringing such a study to Poland, offering hope to patients with a GRN mutation through a potential one-time treatment.

In November 2023, the FDA granted Fast Track designation to AVB-101, expediting the development and review of this new drug for a serious condition with an unmet medical need. The therapy has also received orphan drug status from both the FDA and the European Commission.

The ASPIRE-FTD study is an open-label, multi-center, Phase 1/2 dose-escalation trial aimed at evaluating the safety and preliminary efficacy of AVB-101 in FTD-GRN patients. The study involves a single administration of the therapy delivered to the thalamus via a stereotactic neurosurgical procedure at specialized neurosurgical centers.

FTD is a leading cause of dementia in individuals under 65, with a significant impact on various aspects of life due to its early onset. Genetic FTD cases, including those with GRN mutations, account for about one-third of all cases. The prevalence of FTD-GRN is substantial, with thousands of people affected in the U.S. and EU, and the numbers are expected to grow as awareness and understanding of the disease improve.

AviadoBio is driven by a mission to translate scientific breakthroughs into life-changing medicines for FTD and ALS patients. With a deep understanding of the brain and advanced gene therapy platforms, the company is uniquely positioned to develop transformative treatments for neurodegenerative diseases.

The company's investors include a consortium of renowned venture capital and health-focused funds, reflecting the confidence in AviadoBio's approach and potential impact on the field of neurodegenerative medicine.