UK company AlveoGene launches with first inhaled delivery treatment for rare respiratory disorders

AlveoGene was created and 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 has been created 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.

David Hipkiss, leader and 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.

First target - AATD

The first target indication is alpha-1 antitrypsin deficiency disease (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.

Hipkiss added: “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.”

The company has secured an exclusive licence to the 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 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 for more than a decade, supported by approximately £72 million ($89 million) in grant funding, including from the Wellcome Trust, Department of Health and Social Care, Medical Research Council and the Cystic Fibrosis Trust.

Potential for inhaled gene therapies pipeline

AlveoGene will also evaluate the potential of InGenuiTy 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 remains substantially underdiagnosed and it can take several years until a patient with the condition 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.

Eric Alton, Imperial College London and coordinator of the GTC, said: “My colleagues and I at the GTC have a long-standing commitment to improving the lives of patients with respiratory disorders. We are very excited to take the next step on our journey through the creation of AlveoGene and look forward to working with our new investors to apply these innovations to deliver effective inhaled gene th