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University Start-Up Developing Inhaled Gene Therapies

Inspecting lung X-rays

(National Heart, Lung, and Blood Institute, NIH)

14 Sept. 2023. A new company spun-off from a university lab consortium is advancing gene therapies delivered through the lungs to treat rare inherited respiratory diseases. The work of AlveoGene in Oxford, U.K. is based on discoveries from the U.K. Respiratory Gene Therapy Consortium or GTC, a group of researchers studying inherited respiratory diseases at universities of Oxford and Edinburgh, and Imperial College London.

A technology developed by GTC researchers uses engineered lentiviruses to deliver gene therapies. Among the well-known lentiviruses is HIV, but other lentivirus types offer the ability to deliver genes to cells for long durations. Studies by GTC researchers have enabled the modifying of lentiviruses for safe and efficient gene delivery into cells of epithelium tissue lining the airways. The consortium researchers engineered lentiviruses further for better airway cell delivery, with key proteins from Sendai viruses that affect mice. And the labs are adapting gene therapies for inhaled delivery with hand-held nebulizer devices that create a fine aerosol mist.

The GTC’s first major disease target was cystic fibrosis, a progressive inherited respiratory disease from mutations in the cystic fibrosis transmembrane conductance regulator or CFTR gene. In Oct. 2021, the consortium licensed its technology to drug maker Boehringer Ingelheim, giving Boehringer exclusive worldwide rights to develop and commercialize GTC’s lentiviral delivery technology for gene therapies treating cystic fibrosis. Part of that deal involved manufacturing processes for lentiviral gene therapies from Oxford Biomedica, a company featured in a March 2019 Science & Enterprise report.

Symptoms similar to emphysema

AlveoGene, formed last year, is gaining an exclusive license for GTC’s advanced lentivirus-delivery technology for disease targets other than the CFTR gene associated with cystic fibrosis. The licensed technology includes the ability to deliver gene therapies with an aerosol nebulizer. AlveoGene’s lead product, code-named AVG-001, is an inhaled treatment for alpha-1 antitrypsin deficiency or AATD, a rare inherited lung disease. People with AATD have symptoms similar to emphysema, but can also lead to scar tissue or cirrhosis in the liver. Earlier this year, the Food and Drug Administration cleared an at-home test for mutations responsible for AATD, which is expected to increase the number of diagnosed cases. AlveoGene says AVG-001 is currently in preclinical development.

The company is raising seed funds from Oxford Science Enterprises or OSE that creates and finances start-up businesses based on research at its university, and Old College Capital or OCC, the venture investment arm of Edinburgh Innovations supporting new companies from that university’s labs. Joining the seed round is Harrington Discovery Institute, affiliated with University Hospitals in Cleveland, Ohio, but also a funder of life science start-ups in the U.K. Investment amounts were not disclosed.

“The combination of pioneering science, an extensively validated platform, access to world-leading expertise through our founding scientists and the backing of OSE, Harrington, and OCC, provides a fantastic foundation for the company,” says AlveoGene executive chair David Hipkiss in a company statement released through Globe NewsWire. “This will enable AlveoGene to rapidly advance our first candidate – AVG-001 a unique, inhaled gene therapy for AATD – towards clinical development.”

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