Alveogene Ltd. has announced its novel inhaled gene therapy for lethal neonatal surfactant protein B (SP-B) deficiency, AVG-002, has been awarded orphan drug designation by the FDA.
After the initial approvals in monogenic inherited diseases, the scope of gene therapy is widening, with new delivery routes, novel vectors, cell-specific targeting and products aiming to treat chronic disorders, all making headway in 2023.
Arriving on the gene therapy scene with an undisclosed seed funding sum, Alveogene is tackling respiratory diseases with high unmet need via a next-generation lentiviral delivery platform to advance into the clinic a candidate for rare inherited disorder alpha-1 antitrypsin deficiency.
Though data won’t be available for a few years, the disclosure in mid-July that Grifols SA completed enrollment in the phase III study called Sparta caused some ears to perk up in the alpha-1-antitrypsin deficiency (AATD) space, where a number of contenders are busy.
Alveogene Ltd. has launched with a focus on inhaled gene therapies for respiratory disorders. The company has been created by Oxford Science Enterprises, Harrington Discovery Institute at University Hospitals, and Old College Capital in partnership with six scientists from the UK Respiratory Gene Therapy Consortium (GTC).
