Inspire Biotherapeutics launches to develop lung-focused gene therapy

Company plans to launch a first-in-human clinical trial to test novel platform

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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A new company, Inspire Biotherapeutics, has launched with the goal of developing a new gene therapy platform that could be used in the treatment of cystic fibrosis (CF) and other disorders that affect the lungs.

Inspire is planning to launch a first-in-human clinical trial to test the novel gene therapy platform. The trial is planned to include patients with “a lethal neonatal [newborn] lung disease,” according to a press release from the company, which did not provide further details.

“Our lead indication is currently fatal in neonates — we are compelled to get this across the finish line!” said Sandra Donaldson, Inspire’s CEO.

Donaldson added that positive results from this trial would provide validation for the use of Inspire’s gene therapy platform, called AAVenger, in people.

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Inspire’s AAVenger platform uses a modified form of adeno-associated virus (AAV) to deliver genetic cargo to cells in the lungs. AAV has been widely used in the development of experimental gene therapy approaches because the virus is engineered to not cause serious illness in people and it’s relatively easy to manipulate in a lab.

The AAVenger platform has specifically been engineered to have better tropism for lung tissue. This means that the vector is designed to deliver its genetic cargo to cells in the lungs, while cells elsewhere in the body are unaffected. In theory, this greater specificity could allow better targeting of lung cells without increasing the risk of side effects affecting other parts of the body.

“Identifying vectors that can effectively deliver and express therapeutic transgenes [human-made genetic cargo] in the lung has been a major challenge in realizing a curative gene therapy for genetic lung diseases. The AAVenger platform has demonstrated quick and sustained expression and is amenable to repeated dosing in lung tissue,” said Bernard Thébaud, MD, PhD, chief medical officer of Inspire.

Experiments done in “disease-relevant animal models” have shown that the platform works as intended, delivering genes to lung cells without affecting other cell types, according to Inspire.

“Inspire’s AAVenger technology is tackling critical unmet need in diseases of the lung,” Donaldson said.

The launch of Inspire was supported by three business founders: C3i Center, Octane Medical Group of Companies, and the Ontario Institute for Regenerative Medicine.

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