Moderna Inc. (NASDAQ:MRNA) has entered a strategic licensing and research collaboration agreement with Vertex Pharmaceuticals Inc. (NASDAQ:VRTX) for the research and development of mRNAs and lipid nanoparticles for delivery of gene-editing therapies in treating cystic fibrosis (CF).
Moderna in a collaborative research agreement
The collaboration agreement will run for three years and will initially focus on the discovery and optimization of proprietary mRNAs and LNPs that can deliver gene-editing therapies to lung cells, thus enabling the production of functional cystic fibrosis transmembrane conductance regulator protein (CFTR). Moderna’s CEO, Stephane Bancel, said they are delighted to sign a second collaboration with Vertex to develop and deliver CF patients’ proprietary treatment using gene editing. Bancel added that the first collaboration agreement with Vertex for delivery of mRNA coding for CF protein in lung cells is coming on well. The second agreement will leverage Moderna’s technologies in exploring the use of gene editing in lung cells.
Vertex’s executive VP Global Research and Chief Scientific Officer, David Altshuler, said that the company’s CFTR modulator therapies can potentially treat most CF patients and address the underlying cause of the disease. Nevertheless, around 10% of patients don’t produce CFTR protein and are unlikely to benefit from current medicines. In the last five years, David said that the company has made significant progress in researching genetic therapies aimed to address CF whose delivery has remained a significant scientific and technological challenge.
Vertex committed to the development of gene-editing therapies for CF patients
The combination of Vertex’s scientific, regulatory, and clinical capabilities in CF and Moderna’s expertise in discovering and manufacturing mRNA technologies and proprietary LNP delivery systems will expedite the development of gene therapies for CF patients.
As per the terms of the collaboration agreement, Moderna is expected to carry research in discovering and optimizing the LNPs for gene-editing therapies delivery to lung cells in treating CF. The company is expected to receive an upfront payment of $75 million and regulatory and commercial milestones of up to 380 million, including tiered royalties.