Vertex Moves Its Alpha-1 Antitrypsin Deficiency Program Forward

 

• Vertex Pharmaceuticals Inc 
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   has advanced its investigational program targeting alpha-1 antitrypsin deficiency (AATD), a rare genetic disease characterized by a protein folding defect that can lead to liver and lung disease.
• Vertex announced that the FDA had cleared the Investigational New Drug (IND) Application for VX-634, enabling the company to initiate a first-in-human clinical trial for this small molecule AAT corrector in healthy volunteers. 
• 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 resultant levels of functional AAT (fAAT) in the plasma. 
• Consistent with its portfolio approach for all programs, Vertex is bringing forward additional next-wave AAT correctors, with the next molecules expected to enter the clinic starting in 2023.

https://www.benzinga.com/general/biotech/22/10/29221738/vertex-moves-its-alpha-1-antitrypsin-deficiency-program-forward