A three-way collaboration has recently been announced to push for a new treatment in cystic fibrosis. This collaboration between Boehringer Ingelheim (BI), the UK Cystic Fibrosis Gene Therapy Consortium (GTC) and Oxford BioMedica (OXB), was formed to bring a fusion of clinical, scientific, manufacturing and commercial expertise in the hope of bringing a novel therapy to market.
They are looking to develop a first-in-class gene therapy using a strain of lentivirus (same virus class as HIV) as a vector, through an inhaler. OXB are world leaders in the development and manufacturing of lentivirus vectors. These lentivirus vectors hold many advantages including: being able to carry large payloads of genetic information, the ability to to permanently modify both dividing and non-dividing cells and a lack of pre-existing immunity found with many earlier vector models.
The GTC are bringing 15 years of experience in cystic fibrosis gene therapy research to help enable a universal treatment option for all cystic fibrosis patients, something only gene therapy can offer.
And finally BI, they will be using their expertise within the respiratory market to help develop and optimise the formulation of the drug as well as assist financially and within the commercial phase of the treatments lifecycle.
This collaboration will be interesting to watch as the novel therapy moves forward in development, though it will be a while before any clinical data will be available.
The collaboration will focus on a novel approach using a replication-deficient lentiviral vector in an inhaled formulation, to introduce a healthy copy of the CFTR gene into the cells of the lung. This method has demonstrated high gene transfer efficiency and offers the possibility of repeated administration to maintain the therapeutic effect. Gene therapy is the only therapeutic approach to date that can address all CFTR gene mutations, thus potentially offering a universal treatment option.