Vertex, Moderna Collaborate on mRNA Therapeutics for CF Treatment

Vertex, Moderna Collaborate on mRNA Therapeutics for CF Treatment

Vertex Pharmaceuticals and Moderna Therapeutics recently announced an exclusive research collaboration and licensing agreement to discover and develop the messenger ribonucleic acid trademarked (mRNA) Therapeutics for the treatment of cystic fibrosis (CF).

Vertex Pharmaceuticals is a global biotech with clinical development programs focused on CF, and Moderna Therapeutics is a clinical-stage pioneer of mRNA Therapeutic – an entirely in vivo drug technology that produces human proteins, antibodies and novel protein constructs inside patient’s cells.

“Vertex’s deep scientific expertise and commitment to creating the best treatment regimens for cystic fibrosis patients make them an ideal partner to advance mRNA Therapeutics for this disease,” said Stéphane Bancel, Moderna’s chief executive officer, in a press release. “This collaboration is illustrative of our consistent strategy to partner with world class therapeutic experts in order to maximize the potential of leveraging mRNA as a novel therapeutic approach across a spectrum of serious diseases. We’re particularly excited to explore the potential of pulmonary delivery as a new modality to bring transformative mRNA medicines to patients and address unmet needs.”

Vertex is strategically increasing the number of people eligible for treatment with its two currently approved CF medicines, known as CF transmembrane conductance regulator (CFTR) modulators, which are designed to target the defective CFTR protein in people with specific mutations in the CF gene. The company is also working on additional programs. The most advanced approach is the development of an additional two-drug combination of CFTR modulators, currently under evaluation in a broad Phase 3 development program. The company plans to advance a three-drug combination of CFTR modulators into Phase 2 in CF patients by the second half of 2016.

The three-year collaboration will focus on the use of mRNA therapies to treat the underlying cause of CF by enabling cells in the lungs to produce functional copies of the defective CFTR protein in CF patients. The companies will explore the potential of pulmonary mRNA delivery.

Responsible for carrying genetic instructions transcribed from DNA, mRNA is translated in cells into proteins that, when defective or missing, can underlie certain diseases including CF.

The approach could be applicable to anyone with CF regardless of their specific CFTR mutations. There are currently two Phase 1 clinical studies underway to test mRNA vaccines for the prevention of infectious diseases.

“We are excited to begin this collaboration with Moderna to further broaden our pipeline and support our goal of treating the underlying cause of CF for all people with this disease,” said Dr. David Altshuler, executive vice President of Global Research and chief scientific officer at Vertex. “Moderna’s messenger RNA Therapeutics represent a promising new approach that could provide functional CFTR proteins to the lungs of people with CF, which would complement our existing approach of using CFTR modulators to treat CF.”

Moderna will receive $20 million as part of Vertex’s upfront commitment to the collaboration, along with another $20 million in the form of a note that converts to equity. Vertex will become part owner of Moderna and will pay for future developments and regulatory milestones of up to $275 million, including $220 million in approval and reimbursement milestones, and future royalty payments.

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