Vertex Pharmaceuticals
NEWS
During the past two years, fundraising and investment in biotechnology have reached record levels.
With a handful of drugs for cystic fibrosis already approved, Vertex Pharmaceuticals has turned its attention to expanding its pipeline through collaborations and acquisitions the past few years.
With this milestone, the field is now wide open for the next in vivo CRISPR success, and Editas is hoping that it will be in the ocular disease space.
The company is eager to diversify, putting Phase II clinical trials in sickle-cell disease, beta-thalassemia, kidney diseases, and pain management.
Yao has more than 15 years of experience in the cell and gene therapy fields. He also established and directed the Cell Therapy R&D business unit, with a focus on CAR-T development.
The data showed that both experimental therapies restored function in the cystic fibrosis transmembrane conductance regulator gene.
Shares of Vertex fell more than 13% in premarket trading after it announced that it will not advance a novel small molecule corrector of the Z-AAT protein into a Phase III study.
June is turning out to be a busy month for PDUFA dates for the U.S. Food and Drug Administration. Here’s a look at this week’s dates.
“The recent progress of cystic fibrosis drugs has been amazing and, in my case, miraculous,” said Kelly Peters, who lives with cystic fibrosis. “The new drugs are not a cure, but they feel pretty close.”
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