RARE Daily

AlveoGene Launches to Develop Unique Inhaled Gene Therapies for Rare Respiratory Disorders

September 14, 2023

Rare Daily Staff

AlveoGene has launched in conjunction with seed funding to transform rare respiratory disease outcomes using inhaled gene therapy.

Oxford Science Enterprises and Harrington Discovery Institute at University Hospitals led the financing with participation from Old College Capital, the University of Edinburgh’s venture investment fund.

Oxford Science Enterprises, Harrington Discovery Institute, and Old College Capital created Alveogene in partnership with six leading scientists from the world-renowned UK Respiratory Gene Therapy Consortium (GTC). The GTC was founded in 2001 to catalyze the application of pioneering research to gene therapy development and manufacturing related to cystic fibrosis and other respiratory diseases and originated at Imperial College London and the Universities of Oxford and Edinburgh.

AlveoGene has secured an exclusive license to a proprietary and validated next-generation lentiviral delivery platform developed by the GTC for the treatment of respiratory diseases with high unmet need (excluding the use of the CFTR gene which is mutated in cystic fibrosis). Gene therapies developed using the “InGenuiTy” platform can be delivered through a nebuliser, transducing lung epithelial cells with high efficiency and producing a long duration of action and can achieve these effects following repeated administration. The platform has been developed over more than a decade, supported by approximately $90 million (£72 million) in grant funding, including from the Wellcome Trust, Department of Health and Social Care, Medical Research Council, and the Cystic Fibrosis Trust.

The GTC has demonstrated key characteristics of the platform, including a scalable manufacturing process, which will allow its rapid translation through to first-in-human trials. This foundation will now enable AlveoGene to fast-track the development of its first candidate AVG-001, a novel, inhaled gene therapy designed to promote localized production of alpha-1 antitrypsin to treat patients with alpha-1 antitrypsin deficiency (AATD). The company is aiming to progress this candidate towards clinical development over the next two to three years.

This [funding] will enable AlveoGene to rapidly advance our first candidate—AVG-001 a unique, inhaled gene therapy for AATD—towards clinical development,” said David Hipkiss, executive chair of AlveoGene. “In addition, we intend to explore other opportunities to leverage our powerful IngenuiTy platform alongside other complementary technologies to create first-in-class inhaled gene therapies with the potential to transform outcomes for patients with rare respiratory diseases.”

AlveoGene will also evaluate the potential of the InGenuiTy platform alongside other technologies to create a pipeline of novel inhaled gene therapies targeting other rare respiratory disease opportunities, such as lung surfactant deficiencies and idiopathic pulmonary fibrosis.

AATD is a rare inherited disorder in which patients produce reduced levels of alpha-1 antitrypsin, a protective plasma protein that safeguards the lungs from inflammation and tissue damage caused by infection and inhaled irritants. AATD affects at least 100,000 people in the United States and a similar number in Europe and is a major genetic risk factor for progression to emphysema.

The condition remains substantially underdiagnosed and it can take several years until a patient with AATD is identified. There is no available cure for patients with AATD and the current standard of care is either symptomatic treatment, or in some countries weekly IV infusions of human plasma-derived functional alpha-1 antitrypsin. Recently, the U.S. Food and Drug Administration approved a new at-home diagnostic test and is now commercially available. It is anticipated that this product will improve the rates of early diagnosis substantially and help identify the more than 90 percent of people with AATD believed to be undiagnosed, and who might benefit from treatment.

Photo: David Hipkiss, executive chair of AlveoGene

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