The InGenuiTy^® platform combines the lung-targeting ability of the Sendai virus with the durability and integration strength of lentiviral technology. The result is a gene therapy that can be delivered directly to the lungs, integrates efficiently, carries large genetic payloads, and – uniquely – can be safely redosed directly to the lung if needed.

AlveoGene is developing the InGenuiTy^®’ platform for a range of ultra-rare and rare indications including surfactant protein deficiencies, alpha-1 antitrypsin deficiency lung disease, and idiopathic pulmonary fibrosis.

ABSOLUTE UNMET NEED

For newborns with lethal Surfactant Protein B (SP-B) deficiency or certain forms of ABCA3 deficiency, that could mean a single administration delivered via an endotracheal tube, switching on production of the missing protein and allowing the child to breathe independently for life. In diseases where there is currently 100% mortality, the impact is hard to overstate: a baby who would otherwise die could go home.

Rare disease is often misunderstood as a niche endeavour. In reality, it’s a compelling commercial opportunity as well as a moral imperative. Ultra-rare conditions may affect only dozens of patients globally, but the health economic burden is immense and the unmet need absolute.

Rapidly evolving regulatory frameworks such as paediatric rare disease and orphan drug designation with associated priority review vouchers are designed to accelerate development and reward innovation. When therapies are transformative and precisely targeted, they can become standard of care rapidly – delivering meaningful patient outcomes alongside sustainable returns.

A QUARTER-CENTURY OF RESEARCH

AlveoGene’s journey reflects the strength of Oxford’s translational ecosystem. The company builds on more than 25 years of research from the UK Respiratory Gene Therapy Consortium (GTC), including Professors Deborah Gill and Steve Hyde and colleagues at The University of Oxford, Professors Eric Alton and Uta Griesenbach at Imperial College London, and Dr Christopher Boyd and Gerry McLachlan at the University of Edinburgh. Their work underpins AlveoGene’s inhaled gene therapy programmes across all indications.

Support from the Oxford-Harrington Rare Disease partnership has helped advance the science, while Oxford Science Enterprises (OSE) played a central role in company formation and early backing.

AlveoGene reflects what can happen when deep science, entrepreneurial experience and long-term capital come together around a shared ambition: to develop new therapeutic treatments for patients with extreme unmet medical need – and their parents – where today no options exist.