- A new U.K. company called AlveoGene launched Thursday with aspirations to develop gene therapies for rare respiratory diseases that are delivered via small machines known as nebulizers.
- The unusual approach could be a way to more reliably deliver gene therapies directly to the epithelial cells of the lungs. The company said preclinical research suggests its technology — dubbed InGenuiTy — could work with high efficiency and long-lasting effects.
- AlveoGene aims to bring its first candidate, a treatment for patients with alpha-1 antitrypsin deficiency or AATD, into clinical testing over the next two to three years. The company said it will also look to combine its platform with other technologies to target additional diseases such as lung surfactant deficiencies and idiopathic pulmonary fibrosis.
AlveoGene sprung out of the work of the U.K. Respiratory Gene Therapy Consortium, a group originally founded in 2001 to find a gene therapy for cystic fibrosis. In the process of developing a viral vector to deliver the cystic fibrosis gene to the lungs, it discovered its lentiviral technology could produce secreted proteins that are key to treating other diseases.
Six scientists from the group formed AlveoGene with funding from Oxford Science Enterprises, the Harrington Discovery Institute at University Hospitals and Old College Capital, the venture investment fund of the University of Edinburgh. The new biotech licensed the lentiviral delivery platform developed by the consortium for respiratory diseases with “high unmet need,” outside of cystic fibrosis.
AlveoGene’s first candidate, AVG-001, is designed to get the body to produce alpha-1 antitrypsin, a key protein that helps protect the lungs from inflammation and tissue damage. People with a deficiency of alpha-1 antitrypsin are more likely to suffer from diseases such as emphysema.
There’s no cure for AATD, which can affect either the liver or the lungs. The only treatment options for patients are symptom management or weekly injections of the alpha-1 antitrypsin protein. A number of companies are trying to change the treatment landscape, however; Wave Life Sciences, Arrowhead Pharmaceuticals and Vertex Pharmaceuticals are all working on new therapies.
AlveoGene estimates that at least 100,000 people in the U.S. suffer from AATD lung disease. Many are undiagnosed, though a new at-home test may help in identifying patients who would benefit from treatment, the company said.
David Hipkiss, a 25-year veteran of the industry, will lead the new company as executive chair. He formerly served as CEO of Prosonix and Enesi Pharma.