Vertex Advances Alpha-1 Antitrypsin Deficiency Program – Vertex Pharmaceuticals (NASDAQ:VRTX)

  • Vertex Pharmaceuticals Inc. VRTX advanced its research program targeting alpha-1 antitrypsin deficiency (AATD), a rare genetic disorder characterized by defective protein folding that can lead to liver and lung disease.
  • Vertex announced that the FDA has cleared the investigational new drug (IND) application for VX-634, allowing the company to initiate a first-in-man clinical trial for this small-molecule AAT corrector in healthy volunteers. .
  • Also see: Vertex Pharma’s Flagship Cystic Fibrosis Drug Gains FDA Approval for Infants.
  • Additionally, Vertex will initiate a 48-week Phase 2 study of VX-864, a first-generation AAT corrector, to assess the impact of longer-term treatment on polymer clearance from the liver. and the resulting functional AAT (fAAT) levels. plasma.
  • In line with its portfolio approach for all programs, Vertex is offering additional new wave AAT correctors, with the next molecules expected to enter the clinic starting in 2023.
  • Price action: VRTX shares are up 1.33% at $298.47 when last checked on Tuesday.

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