U.S. regulator Food and Drug Administration put on hold first major clinical trial to use CRISPR on humans in U.S.

Briefing

U.S. regulator Food and Drug Administration put on hold first major clinical trial to use CRISPR on humans in U.S.

June 5, 2018

Briefing
  • Regulatory Oversight – U.S. Food and Drug Administration (FDA) put on hold clinical trial by biotechnology companies CRISPR Therapeutics and Vertex Pharmaceuticals to use CRISPR to treat sickle cell disease in adult humans
  • Sickle Cell Anemia – Presence of abnormal hemoglobin in red blood cells impairs transport of oxygen around body, resulting to chronic pain, blindness, and stroke
  • CTX001 – CRISPR gene editing method that modifies blood stem cells so body can produce more fetal haemoglobin, facilitating better oxygen transfer
  • Additional Requirements – FDA requires more answers to questions regarding clinical trial before obtaining regulatory approval
  • First in U.S. – Should it receive approval, clinical trial will be first in U.S. to use CRISPR on humans, feat already achieved by China in 2016

Accelerator
Biotechnology and Bioinformatics

Market Disruption
Innovation, Competitiveness and Trade

Sector
Healthcare/Health Sciences

Organization
CRISPR Therapeutics

Source

Original Publication Date
May 31, 2018

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