The US Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for VX-522, an experimental messenger RNA (mRNA) therapy being developed by Vertex Pharmaceuticals and Moderna to treat the underlying cause of cystic fibrosis (CF) in patients who cannot benefit from a CF transmembrane conductance regulator (CFTR) modulator.

“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,” Reshma Kewalramani, MD, chief executive officer and president of Vertex, said in a press release.

VX-522 is composed of a CFTR mRNA encapsulated by a lipid nanoparticle. It is delivered to lung cells via inhalation, where the mRNA is used to produce functional copies of the CFTR protein.

Read more about experimental therapies for CF

“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,” Stéphane Bancel, chief executive officer of Moderna, said.

Vertex plans to initiate a single ascending dose clinical trial to evaluate the safety and tolerability of VX-522 in patients with CF, aged 18 years and above, who do not respond to CFTR modulator therapy.

Reference

Vertex announces investigational new drug (IND) application for VX-522, mRNA therapy for people with cystic fibrosis, cleared by FDA. News release. Vertex Pharmaceuticals Inc; December 12, 2022.