Vertex receives IND clearance for mRNA cystic fibrosis therapy
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The US FDA has cleared an IND application from Vertex Pharmaceuticals (NASDAQ:VRTX) for VX-522, an mRNA candidate for cystic fibrosis ("CF").
The therapy is intended for the ~5K CF patients who do not benefit from a cystic fibrosis transmembrane conductance regulator (CFTR) modulator.
VX-522 is delivered via inhalation of a CFTR mRNA encapsulated by a lipid nanoparticle.
Vertex (VRTX) anticipates beginning a single ascending dose trial in the coming weeks.
Read why Seeking Alpha contributor ONeil Trader calls Vertex (VRTX) a buy.