AVG-002 uses the same proprietary lentiviral vector backbone that is used for all AlveoGene products to deliver the relevant transgene to the lung. AlveoGene believes that AVG-002 could transform the current treatment paradigm and provide a lasting solution to this ultra rare respiratory disease that affects newborns/infants, and in addition provide an important catalyst for a range of other respiratory indications.
SP-B deficiency is caused by inherited mutations in the SP-B gene (SFTPB) on chromosome 2, which leads to a partial or complete absence of surfactant protein B. It is an autosomal recessive condition. Infants present shortly after birth with respiratory distress and failure, despite assisted ventilation and short term synthetic surfactant replacement therapy.
Post presentation, a diagnosis is made by genetic testing for the mutation in the child and both parents. SP-B deficiency carries a very poor prognosis and children with this disorder are very unlikely to survive beyond the first few months of life, there being no current therapeutic solution at this time
We believe that a single instilled administration of AVG-002 could potentially deliver ‘switch-on’ the life-long local production of surfactant in the neonatal lung providing a viable treatment option and a potential functional cure.
AVG-002 has been granted Paediatric Rare Disease Designation by the FDA and has been granted Orphan Drug Status. On marketing authorisation, AVG-002 qualifies for a tradable Priority Review Voucher, several of which have sold in 2025 for ~$150m.
With AVG-002 and AVG-003, we believe we will be creating two first and best-in-class unique products which may generate in excess of $250m sale per annum in aggregate.
We accelerating both candidates into clinical trials.
AVG-003 uses the same proprietary lentiviral vector backbone that is used for all AlveoGene products to deliver the relevant transgene to the lung. AlveoGene believes that AVG-003 could transform the current treatment paradigm and provide a lasting solution to this very rare respiratory disease that affects newborns/infants.
ABCA3 surfactant protein disorder is an autosomal recessive condition caused by inherited mutations in the ABCA3 gene (ABCA3) on chromosome 16. This results in the loss or defective function of the phospholipid transporter required to produce pulmonary surfactant.
Individuals with ABCA3 deficiency may present at birth, with severe respiratory distress similar to SP-B deficiency, or in older children where the disease may be milder but with chronic breathing difficulties and failure to thrive. The prognosis is variable, depending on the severity of the disease. In the most severe cases children require lung transplantation to survive, with only limited availability of suitable organs.
With AVG-003 we believe we will be creating a first and best-in-class, life-changing product together with a new high value market opportunity, providing a lifelong solution where presently there is none.
AlveoGene intend to apply for Paediatric Rare Disease Designation by the FDA and Orphan Drug Status in 2025.
AVG-001 is a novel, inhaled gene therapy leveraging the same proprietary lentiviral vector backbone that is used for all AlveoGene products to deliver the relevant transgene to the lung.
It is has been specifically designed to promote long term localised production of alpha-1 antitrypsin in the lung to treat patients with AATD lung disease. The Company is aiming to progress this candidate towards clinical development over the next 2-3 years.
AATD is a rare, significantly under diagnosedinherited 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. The most severe form of 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. 80% of the severe cases have AATD Lung Disease with no associated liver disease.
The condition remains substantially underdiagnosed and it can take several years until a patient with AATD 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. This treatment option is not widely reimbursed.
Current commercial estimates put the market for AATD at circa $1Bn rising to $3Bn with improved disease awareness and diagnosis. In addition, it is estimated that 10% of all COPD GOLD II class patients have undiagnosed AATD lung disease representing a major potential upside. Transforming the treatment paradigm would be of major beneficial impact to patients and healthcare outcomes alike.
AVG-004 uses the same proprietary lentiviral vector backbone that is used for all AlveoGene products to deliver the relevant transgene to the lung.uses the same proprietary lentiviral vector that is used for both AVG-001, AVG-002 and AVG-003 to deliver the relevant transgene to the lung.
AlveoGene believes that AVG-004 could transform the current treatment paradigm for IPF, by slowing the progression of IPF and improving lung function to extend patient lifespan and improve quality of life.
AVG-004 consists of two or more transgenes packaged in the same vector backbone and delivered simultaneously to the site of action in the lung.
IPF is a serious chronic disease that affects the tissue surrounding the air sacs, or alveoli, in the lungs. This condition develops when lung tissue becomes thick and stiff for unknown reasons. Over time, these changes can cause permanent scarring in the lungs, called fibrosis, making it progressively more difficult to breathe.
Family history, smoking and increasing age are risk factors for developing IPF. The most common symptoms of IPF are shortness of breath and cough. Some people may not have symptoms at first, but symptoms can develop and get worse as the disease progresses.
The way that IPF progresses varies from person to person, and scarring may happen slowly or quickly. In some people, the disease stays the same for years. In other people, the condition quickly gets worse and they can die within 3 years of diagnosis.
Many people with IPF also experience acute exacerbations, where symptoms suddenly become much more serious. Other complications of IPF include pulmonary hypertension and respiratory failure, which happen when the lungs cannot deliver enough oxygen into the bloodstream without support. This prevents the brain and other organs from getting the oxygen they need.
There is currently no cure for IPF and third-party analysis estimates an ever growing market size of approximately $10B by 2033.
Given the chronic nature of the disease it is likely that the treatment of IPF will require multiple and repeat dosing of a genetic intervention. Being the only vector system in the world that can be reliably and repeatably re-dosed to the lung with no loss of efficacy, the InGenuiTy platform represents the ideal platform to exploit for such chronic conditions, identical to the strategy being deployed by Boehringer Ingelheim in their cystic fibrosis trial.
Our preferred candidate is currently being triaged and evaluated in a range of in-vitro and ex-vivo models prior to candidate selection later in 2025.
We also welcome strategic interest from third parties whom may wish to explore their own transgene candidates with InGenuiTy under collaborative arrangements
