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AviadoBio Initiates ASPIRE-FTD Clinical Trial Assessing AVB-101 for Frontotemporal Dementia

Monday, April 15, 2024

AviadoBio, a pioneering gene therapy company dedicated to developing transformative medicines for neurodegenerative disorders, has recently initiated the Phase 1/2 ASPIRE-FTD trial. This trial is evaluating the effectiveness of AVB-101, an investigational gene therapy, in individuals diagnosed with frontotemporal dementia (FTD) with progranulin (GRN) gene mutations (FTD-GRN). The treatment of the first patient represents a significant milestone in addressing this debilitating condition.

Lisa Deschamps, CEO of AviadoBio, expressed gratitude to the clinical investigators and participating families, emphasizing the company's commitment to introducing innovative treatments for FTD-GRN. FTD is a challenging form of dementia due to the lack of effective disease-modifying therapies. AVB-101 aims to address this gap by delivering a functional copy of the GRN gene to restore proper progranulin levels in the brain, potentially halting disease progression.

Dr. David Cooper, Chief Medical Officer, highlighted the urgency for new treatments in FTD-GRN, emphasizing the importance of AVB-101's advancement. The therapy is delivered via a minimally invasive neurosurgical procedure directly to the thalamus, a brain region crucially implicated in FTD, with the aim of restoring progranulin levels while minimizing systemic exposure.

Prof. Mirosław Ząbek, M.D., Ph.D., expressed excitement about conducting the first intrathalamic administration of AVB-101, underscoring the significance of this research. Dr. Gabriela Klodowska emphasized the impact of offering potential one-time treatments to FTD patients in Poland, reflecting the global significance of the ASPIRE-FTD study.

The Fast Track designation granted by the FDA underscores the urgency and importance of AVB-101's development. Additionally, AVB-101 has received orphan drug designation from regulatory bodies, further supporting its advancement.

ASPIRE-FTD is a Phase 1/2 study designed to assess the safety and efficacy of AVB-101 in FTD-GRN patients. The study involves a single administration of AVB-101 via stereotactic neurosurgery at expert centers across Europe and the United States.

FTD is a devastating condition, particularly affecting individuals under 65 years old. Genetic mutations, including those in the GRN gene, contribute to FTD pathology. With an estimated 11,000 individuals in the U.S. and EU living with FTD-GRN, the need for effective treatments is pressing. The ASPIRE-FTD study represents a significant step toward addressing this unmet medical need.



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