Bringing a new medicines to people around the world requires significant resources and may involve the concerted efforts of multiple major companies and organizations. Throughout our history, global collaborations have played an important role in helping us to fully realize the productivity of our drug discovery capabilities and advance the development and commercialization of Vertex-discovered medicines. We seek to work with companies that share our sense of urgency to improve the treatment of major diseases and who share our commitment to scientific and clinical excellence.
In 1998, Vertex initiated its cystic fibrosis research program in connection with its collaborative relationship with the Cystic Fibrosis Foundation. KALYDECO® (ivacaftor) and ORKAMBI® (lumacaftor/ivacaftor) were discovered by Vertex as part of this collaboration.
Vertex and Parion Sciences began a collaboration in June 2015 to develop investigational epithelial sodium channel (ENaC) inhibitors for the potential treatment of cystic fibrosis and other pulmonary diseases. Under the agreement, Vertex maintains worldwide development and commercial rights to Parion's investigational ENaC inhibitors, including P-1037 and P-1055, for CF and other pulmonary diseases. The collaboration with Parion complements our ongoing work in CF and supports our two key goals in this disease - to increase the number of people eligible for new CF medicines and to enhance the benefit of treatment while expanding our pipeline.
Vertex and CRISPR Therapeutics began a research collaboration in October of 2015 focused on the use of CRISPR’s gene editing technology, known as CRISPR-Cas9, to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. The collaboration will evaluate the use of CRISPR-Cas9 across multiple diseases where targets have been validated through human genetics. Vertex and CRISPR will focus their initial gene editing research on discovering potential treatments to address the mutations and genes known to cause and contribute to cystic fibrosis and sickle cell disease. Vertex and CRISPR will also evaluate a specified number of other genetic targets as part of the collaboration. Vertex will have exclusive rights to license up to six new CRISPR-Cas9-based treatments that emerge from the collaboration.
Vertex and Moderna Therapeutics began a research collaboration in July 2016 aimed at the discovery and development of messenger Ribonucleic Acid (mRNA) Therapeutics for the treatment of cystic fibrosis (CF). The three-year collaboration will focus on research of the use of mRNA therapies to treat the underlying cause of CF by enabling cells in the lungs to produce functional copies of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, which is known to be defective in people with CF. Through the collaboration, the companies will explore the potential utilization of pulmonary mRNA delivery.