AlveoGene launches to develop inhaled gene therapies for rare respiratory disorders

• New gene therapy company created and funded by Oxford Science Enterprises, Harrington Discovery Institute and Old College Capital in partnership with six leading scientists from the world-renowned UK Respiratory Gene Therapy Consortium (GTC)
• AlveoGene initially aims to leverage proprietary lentiviral gene therapy platform designed and developed by GTC specifically for inhaled delivery
• First target indication is alpha-1 antitrypsin deficiency disease (AATD), a degenerative condition that can lead to early onset emphysema
• AATD represents a rapidly growing multi-billion dollar opportunity based on improved diagnosis and patient identification
• David Hipkiss, an experienced life sciences company builder and leader, appointed as Executive Chair

Oxford, UK – AlveoGene (or ‘the Company’, www.alveogene.com), an innovative company focused on transforming rare respiratory disease outcomes using inhaled gene therapy, today announces its launch in conjunction with seed funding raised from Oxford Science Enterprises (OSE), Harrington Discovery Institute at University Hospitals (Harrington), and with participation from Old College Capital (OCC), the University of Edinburgh’s venture investment fund.

AlveoGene has been created by OSE, Harrington and OCC in partnership with six leading scientists from the world-renowned UK Respiratory Gene Therapy Consortium (GTC). The GTC was founded in 2001 to catalyse 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 licence 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 £72 million in grant funding, including from the Wellcome Trust, the 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 localised 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 2-3 years.

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 US 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, a new at-home diagnostic test (AlphaID™ at Home; Grifols, Spain) has been approved by the US Food & Drug Administration 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% of people with AATD believed to be undiagnosed, and who might benefit from treatment.

AlveoGene will be led by David Hipkiss as Executive Chair. David is a biopharmaceutical executive with over 25 years experience of building and leading innovative companies, particularly in the respiratory disease area, and a proven track record of delivering high-value outcomes for stakeholders. He is the former CEO and co-founder of respiratory medicine company Prosonix (acquired by Circassia for £100 million in 2015) and former CEO and founder of Enesi Pharma (now aVaxzipen), a company developing a novel solid dose injectable vaccine delivery platform.

David Hipkiss, Executive Chair of AlveoGene, said: “I am truly excited to lead this new venture. 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. This will enable AlveoGene to rapidly advance our first candidate – AVG-001 a unique, inhaled gene therapy for AATD – towards clinical development. 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.”

More news and updates