Press release
Vertex Announces Investigational New Drug (IND) Application for VX-522, mRNA Therapy for People With Cystic Fibrosis, Cleared by FDA
- Novel, inhaled mRNA therapy intended for the ~5,000 people with CF who cannot benefit from CFTR modulators - - VX-522 clinical trial in people with CF to
About this update from Vertex Pharmaceuticals Incorporated
[{"type":"text","content":"\n- Novel, inhaled mRNA therapy intended for the ~5,000 people with CF who cannot benefit from CFTR modulators -\n\n- VX-522 clinical trial in people with CF to initiate in coming weeks -\n\n BOSTON--(BUSINESS WIRE)--\nVertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration has cleared its Investigational New Drug (IND) application for VX-522, a messenger ribonucleic acid (mRNA) therapy targeted at treating the underlying cause of cystic fibrosis (CF) lung disease for the approximately 5,000 people with CF who cannot benefit from a cystic fibrosis transmembrane conductance regulator (CFTR) modulator. Vertex plans to initiate a single ascending dose clinical trial for VX-522 in people with CF in the coming weeks.\nThis press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20221208005977/en/This infographic provides an outline of how we envision this investigational therapy to work and is provided for illustrative purposes only. (Graphic: Business Wire)\nVX-522 is delivered to the lung through inhalation of a CFTR mRNA encapsulated by a lipid nanoparticle. Once delivered to the target lung cells, the mRNA is designed to produce functional copies of the CFTR protein. VX-522 is the result of an exclusive research collaboration established with Moderna in 2016.\n\n“It has been our longstanding goal to bring highly effective therapies to all people with CF. Clearance of the IND represents a pivotal turning point in reaching the remaining ~5,000 people with CF who are still waiting for a medicine to treat the underlying cause of their disease,” said Reshma Kewalramani, M.D., FASN, Chief Executive Officer and President, Vertex. “The partnership with Moderna that began more than five years ago has been instrumental in achieving this milestone, and we look forward to continuing our work together.”\n\n“This partnership brings together Vertex’s scientific expertise and decades of experience in developing cystic fibrosis medicines with Moderna’s proven leadership in mRNA technologies,” said Moderna Chief Executive Officer Stéphane Bancel. “Moderna’s development of a proprietary inhalable lipid nanoparticle to deliver a functional cystic fibrosis treatment to the lungs could lead to a transformational medical achievement. We are excited by th...