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AlveoGene Debuts with a Mission to Create Inhaled Gene Therapies for Rare Respiratory Disorders

Friday, September 15, 2023

AlveoGene, a novel company focused on advancing inhaled gene therapy for rare respiratory diseases, has officially announced its launch. This announcement coincides with the company securing seed funding from Oxford Science Enterprises (OSE), Harrington Discovery Institute at University Hospitals (Harrington), and Old College Capital (OCC), the University of Edinburgh's venture investment fund.

AlveoGene has been established through a collaborative effort involving OSE, Harrington, OCC, and six prominent scientists associated with the renowned UK Respiratory Gene Therapy Consortium (GTC). Founded in 2001, the GTC's mission is to apply cutting-edge research to the development of gene therapies for respiratory diseases, with a particular focus on cystic fibrosis. It originated from institutions such as Imperial College London, the Universities of Oxford and Edinburgh.

The company has obtained an exclusive license for the "InGenuiTy™" platform, a state-of-the-art lentiviral delivery system developed by the GTC. This platform is designed for the treatment of respiratory diseases characterized by significant unmet medical needs, excluding cystic fibrosis. InGenuiTy™-based gene therapies can be administered through a nebulizer, efficiently targeting lung epithelial cells and providing long-lasting effects even with repeated use. The platform, which has received approximately £72 million in grant funding from organizations like the Wellcome Trust, the Department of Health and Social Care, the Medical Research Council, and the Cystic Fibrosis Trust, has undergone over a decade of development.

The platform has demonstrated key attributes, including a scalable manufacturing process, which will enable a rapid transition to first-in-human trials. AlveoGene plans to leverage this foundation to expedite the development of its first candidate, AVG-001, an innovative inhaled gene therapy aimed at stimulating localized production of alpha-1 antitrypsin to treat patients with Alpha-1 Antitrypsin Deficiency (AATD). The company's goal is to advance this candidate to clinical development within the next 2-3 years.

In addition to AVG-001, AlveoGene will explore the potential of the InGenuiTy™ platform in combination with other technologies to establish a pipeline of novel inhaled gene therapies for various rare respiratory diseases, including lung surfactant deficiencies and idiopathic pulmonary fibrosis.

AATD is a rare hereditary disorder characterized by reduced levels of alpha-1 antitrypsin, a protective protein that safeguards the lungs against inflammation and tissue damage resulting from infections and inhaled irritants. AATD affects a substantial number of individuals in the US and Europe, increasing the risk of developing emphysema. This condition frequently remains undiagnosed for an extended period. Currently, there is no cure, and treatment options are limited to symptomatic management or, in some regions, weekly IV infusions of human plasma-derived alpha-1 antitrypsin. The recent approval of an at-home diagnostic test, AlphaID™ at Home, by the US Food & Drug Administration holds promise for improving early diagnosis rates and identifying the significant proportion of undiagnosed AATD patients who could benefit from treatment."

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