NEW YORK – AlveoGene on Thursday announced its launch to advance inhaled gene therapies for rare respiratory conditions, starting with alpha-1 antitrypsin deficiency (AATD).
Oxford Science Enterprises (OSE), Harrington Discovery Institute at University Hospitals, and Old College Capital (OCC) in partnership with scientists at the UK Respiratory Gene Therapy Consortium created the new company. OSE and Harrington provided an undisclosed amount of seed funding for the newly launched Birmingham, UK-headquartered firm, which also received contributions from OCC and the University of Edinburgh's venture investment fund.
AlveoGene has an exclusive license to use the UK Respiratory Gene Therapy Consortium's proprietary next-generation lentiviral delivery platform, dubbed InGenuiTy, to develop gene therapies for respiratory diseases. The consortium, which formed in 2001 out of Imperial College London and the Universities of Oxford and Edinburgh, has developed a gene transfer agent for cystic fibrosis and licensed it to Boehringer Ingelheim. As such, AlveoGene's license to the consortium's platform precludes its use with CFTR, the gene mutated in cystic fibrosis.
The InGenuiTy platform, which took £72 million ($89.58 million) in grant funds and a decade to develop, enables the delivery of gene therapies through a nebulizer, which transduces lung epithelial cells and is designed to produce a long duration of action. Using this platform, AlveoGene hopes to speed its first inhaled gene therapy candidate, AVG-001, into clinical trials as a potential treatment for AATD.
The rare inherited disorder is caused by a deficiency in alpha-1 antitrypsin, a protein that protects the lungs from damage due to infection and environmental irritants. AATD patients are at increased risk for emphysema. Although the condition is underdiagnosed, diagnostic rates may improve with the recent regulatory clearance in the US of Grifols' AlphaID at-home test for detecting genetic mutations associated with the deficiency. Current treatments for AATD only address the symptoms, but in some countries, patients can receive weekly IV infusions of human plasma-derived functional alpha-1 antitrypsin.
AVG-001 is designed to promote localized production of alpha-1 antitrypsin, and AlveoGene is hoping to take this treatment into clinical trials in the next two to three years. Beyond AVG-001, AlveoGene has ambitions to use InGenuiTy alongside other complementary technologies to build an entire portfolio of inhaled gene therapies for various rare respiratory diseases, such as lung surfactant deficiencies and idiopathic pulmonary fibrosis.
"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," AlveoGene Executive Chair David Hipkiss said in a statement. "This will enable AlveoGene to rapidly advance our first candidate — AVG-001, a unique, inhaled gene therapy for AATD — towards clinical development."