CRISPR Therapeutics, Vertex Initiate First Industry-Sponsored Trial – What’s Next?

 
• By Mandy Jackson
  
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CRISPR Therapeutics and Vertex initiated the first human study of CRISPR gene-editing technology with an industry sponsor, following an investigator-sponsored study initiated in China two years ago. These are not the first gene-editing trials, but they're important milestones for the CRISPR field.

Genetic engineering and gene manipulation concept. Hand is replacing part of a DNA molecule. 3D rendered illustration of DNA.

CRISPR gene editing has been a hot area of biotechnology for the past few years despite the absence of data in humans, but the field finally crossed a new threshold with initiation of the first industry-sponsored clinical trial, from CRISPR Therapeutics AG and partner Vertex Pharmaceuticals Inc. And, with aggressive investment in gene-editing technologies across the industry, this should be the start of a wave of clinical trials.

CRISPR Therapeutics and Vertex kicked off a 30-patient Phase I/II trial for CTX001 in transfusion-dependent beta-thalassemia, according to an Aug....

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