-AviadoBio, a pioneering gene therapy company dedicated to developing and delivering potentially transformative medicines for neurodegenerative disorders, today announced that the first patient has been treated in the Phase 1/2 ASPIRE-FTD trial evaluating AviadoBio’s investigational gene therapy, AVB-101, in people with frontotemporal dementia (FTD) with progranulin (GRN) gene mutations (FTD-GRN). Clinical trial sites are currently open in Poland, Spain, and the Netherlands with additional sites expected to open in multiple countries, including the United States.

“AviadoBio is committed to bringing forward an innovative gene therapy treatment for FTD-GRN and this moment marks an important milestone for the FTD community and our company,” said Lisa Deschamps, CEO. “We are thankful for the dedication of the ASPIRE-FTD clinical investigators studying AVB-101 and immensely grateful for the families who participate in clinical trials for new treatment options that may change the future for generations of families living with FTD-GRN.”

FTD is a devastating form of early-onset dementia that typically leads to death within three to 10 years from diagnosis.^1,2 People with FTD commonly experience personality changes, behavioral disturbance, loss of language, apathy, and reduced mobility.³ It is a leading cause of dementia in people under the age of 65 and is often misdiagnosed.⁴ People with FTD who have disease-causing GRN mutations produce a reduced amount of progranulin protein. AVB-101 is a potential one-time therapy designed to stop disease progression by delivering a functional copy of the GRN gene to restore appropriate progranulin levels to affected areas of the brain.

“There is a critical need for new treatments for people living with FTD-GRN. The lack of effective disease-modifying treatments makes this diagnosis particularly difficult for patients and their families who don’t currently have any available treatment options for this devastating and progressive disease,” said David Cooper, M.D., Chief Medical Officer. “Treating the first patient in ASPIRE-FTD marks an important step forward in understanding AVB-101’s potential, which has already shown promise in preclinical studies.”

AVB-101 is delivered using a minimally invasive, stereotactic neurosurgical procedure directly to the part of the brain called the thalamus. The thalamus has extensive connections to other parts of the brain, including the frontal and temporal lobes, which play a critical role in FTD and the symptoms that impact patients and their families. This targeted delivery method aims to safely and effectively cross the blood-brain barrier, delivering targeted treatment directly to the brain to restore progranulin levels in the frontal and temporal cortex where it is needed most, while at the same time minimizing the dose required and thereby limiting any potential systemic exposure.

The Interventional Neurotherapy Center (INC) at Mazowiecki Szpital Bródnowski Hospital is the first and only center in Europe currently performing MRI-guided infusions of gene therapies. “Our team is excited to participate in this important scientific research and to perform the first intrathalamic administration of AVB-101 in the ASPIRE-FTD global trial,” said Prof. Mirosław Ząbek, M.D., Ph.D., the chairperson of the department of neurosurgery at INC.

“As a neurologist and founder of the Neuro-Care Clinic, I’ve seen the difficult struggles of my patients with dementia and their families,” said Gabriela Klodowska, M.D., Ph.D. “It means a lot to our patients with FTD to bring this type of study offering a potential one-time treatment for patients with a GRN mutation to Poland and to be the first to enroll patients in this global study.”

In November 2023, the U.S. Food and Drug Administration (FDA) granted Fast Track designation to AVB-101. Fast Track is a process designed to facilitate the development and expedite the review of new drugs to treat serious conditions and fill an unmet medical need. This designation is intended to bring new medicines to patients earlier. It has also received orphan drug designation by both the FDA and European Commission.