Inspire Biotherapeutics Launches with Pre-seed Investment to Target Monogenic and Acquired Lung Diseases with Proprietary AAVenger Gene Therapy Platform

Inspire Biotherapeutics, an emerging biotech company with a mission to create life-saving gene therapies for monogenic and acquired diseases of the lung, is poised to move its novel lung tropic AAV gene therapy into clinical validation.

The AAVenger platform has demonstrated transduction and long-term expression in lung tissue without impact on other tissue types in disease-relevant animal models. The platform can be applied to treat cystic fibrosis, interstitial lung disease, pulmonary fibrosis, and other monogenic lung diseases. Inspire’s scientific founders – Dr. Bernard Thébaud, chief medical officer, – a neonatologist and cell therapy pioneer in neonates – and Dr. Sarah Wootton, chief scientific officer, – a vectorologist with extensive AAV experience – say they are pursuing initiation of a first-in-human clinical trial for a lethal neonatal lung disease with short time to data readout and success of therapy.

“Identifying vectors that can effectively deliver and express therapeutic transgenes in the lung has been a major challenge in realizing a curative gene therapy for genetic lung diseases,” says Thébaud. “The AAVenger platform has demonstrated quick and sustained expression and is amenable to repeated dosing in lung tissue.”

“Inspire’s AAVenger technology is tackling critical unmet need in diseases of the lung,” says Sandra Donaldson, chief executive officer for Inspire. “Our lead indication is currently fatal in neonates – we are compelled to get this across the finish line! A near-term clinical trial in this fragile population will validate Inspire’s AAVenger platform.”

The company launch is supported by three business founders – C3i Center Inc., Octane Medical Group of Companies, and the Ontario Institute for Regenerative Medicine – with deep expertise in company creation, translational teams, and regenerative medicine commercialization.

Inspire will be attending the Alliance for Regenerative Medicine’s Cell & Gene Meeting on the Mediterranean April 12-14, 2023, in Barcelona to engage investors and partners in a first-in-human clinical trial in a lethal neonatal lung disease model with short time to data readout and success of therapy.