A Modified AAV Gene Therapy for Cystic Fibrosis

ABSTRACT Talee Bio is developing a gene therapy for cystic fibrosis CF the first curative treatment for this disease CF is an autosomal recessive disease involving any of a number of mutations to the gene for cystic fibrosis transmembrane conductance regulator CFTR a membrane channel that influences the composition of mucous secretions in the gut and respiratory tract CF is primarily characterized by progressive airway infection and inflammation and in the majority of individuals death from respiratory failure The standard of care for CF includes therapies that address symptoms of the disease such as bronchodilators mucolytics and antibiotics and those that attempt to modulate the activity of defective mutated CFTR However none of these treatments are curative Talee has developed a novel transgene and a number of innovations in a well characterized delivery vector that collectively will overcome challenges that have previously limited the clinical viability of gene therapy for CF In this phase proposal Talee will demonstrate the feasibility of its approach by demonstrating functional transformation of human CF cells in vitro and the transduction efficiency and distribution of a marker transgene delivered with its proprietary vector If successful this work will pave the way to future development in animal models of CF and for safety and PKPD studies in Phase that will inform a pre IND meeting with the FDA Talee Bio is a collaboration among drug development professionals with more than years of combined experience and academic founders of the company from the University of Iowa who have been investigating CF pathogenesis and treatment including gene therapy strategies for more than years PROJECT NARRATIVE Talee Bio is developing a treatment for cystic fibrosis Cystic fibrosis is a genetic disease Talee Bioandapos s approach will deliver a fully functional gene to cystic fibrosis patients that will overcome the defect causing their disease