Gene therapy biotech AlveoGene launches with lentiviral platform for respiratory diseases

New company AlveoGene, backed by experts at the UK Respiratory Gene Therapy Consortium (GTC), has made its debut with a lead program for rare respiratory disease alpha-1 antitrypsin deficiency (AATD).

OSE created the company together with Harrington Discovery Institute, Old College Capital and six GTC scientists. The UK biotech has an exclusive license to the GTC’s next-generation lentiviral delivery platform, InGenuiTy, except with regard to the CFTR gene, which is mutated in cystic fibrosis. Boehringer Ingelheim has taken the reins on that gene and is working on a CF gene therapy candidate using the tech.

Eric Alton

“One of the big advantages of [using a] lentivirus platform [as opposed to an AAV platform] is that it is the only viral vector we have ever tested that can produce an exciting, big therapeutic effect repeatedly,” GTC coordinator Eric Alton told Endpoints News in an interview. InGenuiTy can also design drugs for administration through a nebulizer, enabling more direct delivery to the lungs.

AlveoGene’s lead program, AVG-001, is in preclinical development for AATD, a rare disorder that sees patients produce lower levels of the protective plasma protein alpha-1 antitrypsin, leading to lung and liver damage. Grifols recently launched an at-home diagnostic test for the condition, AlphaID, which could help identify the more than 90% of AATD patients believed to be undiagnosed.

“What some of our competitors are doing is trying to make the liver more efficient at producing alpha-1 antitrypsin,” Alton said, whereas AVG-001 is designed to promote localized production of the protein in the lungs. AlveoGene will work to advance AVG-001 into the clinic over the next two to three years and has planned and reserved non-human primate studies, executive chair David Hipkiss told Endpoints.

Hipkiss stressed that the company is far from a one-trick pony — it has plans for a potential second program, AVG-002, to treat surfactant protein B deficiency. The rapidly fatal condition causes respiratory distress in newborn infants and has no viable long-term treatments save for challenging lung transplant.

AlveoGene also hopes to expand into bigger indications, such as idiopathic pulmonary fibrosis, and potentially explore novel non-viral approaches to gene therapy through its relationship with the GTC, Hipkiss said.

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