4DMT is building a robust pipeline of wholly-owned and partnered gene therapy products delivered by routine aerosolized delivery for patients with rare diseases involving the lung. Aerosolized delivery procedures are performed routinely in the US and the rest of the world to deliver treatments to the lung airways and alveoli.
4DMT has completed and on-going vector discovery programs for AAV vectors designed to target various cells within the lung following aerosolized delivery. These are designed for superiority to 1st and 2nd generation vectors. We start by defining the Target Product Profile for each lung disease indication, and on that basis we will select the optimal customized vector from our portfolio of vectors.
We currently have two lung products in the discovery and engineering phase:
Lung Product for Cystic Fibrosis:
4D-710 is for the treatment of patients with Cystic Fibrosis. Our CF program benefits greatly from our partnership with the Cystic Fibrosis Foundation Therapeutics arm; the CFFT provides scientific and medical expertise, networks of key opinion leaders, research and financial support (over $5 Million committed to date). This product will express a truncated version of the human CFTR gene which is mutated in these patients.
Lung Product for Large Market Disease:
4DMT is developing customized vectors for large market lung diseases. 4DMT has a partnership with AstraZeneca focused on a single target in this therapeutic area.