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Vertex and Moderna Launch First-In-Human Inhaled mRNA Candidate for Cystic Fibrosis

A novel collaboration breaks ground with an inhaled mRNA therapy entering early clinical testing for the first time—a potentially game-changing advance for cystic fibrosis treatment.

Vertex and Moderna Bring mRNA into the Lungs

On 5 January 2025, Vertex Pharmaceuticals and Moderna confirmed they have initiated a first-in-human trial of an inhaled mRNA therapy targeting cystic fibrosis, a landmark in expanding mRNA’s reach beyond vaccines. This inhalable treatment aims to deliver mRNA directly to the lungs, offering a precise therapeutic payload to the tissue most affected by the disease. In replacing defective CFTR protein functionality at the source, this approach could, if successful, represent a truly transformative option for patients. The collaboration underscores mutual strengths—Vertex’s deep clinical expertise in cystic fibrosis, paired with Moderna’s mRNA development capabilities—creating a powerful dynamic for innovation. It also diversifies Moderna’s clinical portfolio and signals the broader feasibility of pulmonary mRNA therapies.

Why It Matters for Innovation—and What Comes Next

This crossover into pulmonary-targeted mRNA not only stretches the therapeutic boundaries of the technology class but pivots its application toward a chronic, life-shortening inherited disease. If initial safety and delivery assessments go well, this program could catalyse a wave of organ-specific mRNA initiatives—particularly in areas where conventional therapies fall short. Next steps will include further dose escalation, pharmacokinetic studies, and early signs of biological activity in the clinical cohort. Beyond clinical relevance, this marks a new chapter in delivery science—and underscores how platform flexibility may soon define the pace of therapeutic breakthroughs.

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