First CRISPR Clinical Trial Gets Red Light from US Regulator


The CRISPR-Cas9 genome editing system, a technique that allows scientists to alter the DNA of different organisms with high speed and precision, has been recognized as a tool that has the distinct ability to alter the course of human evolution and consequently transform in a fundamental way our power over nature. But since its discovery six years ago, progress towards the method’s eventual application to the clinic has been slow. Now, another setback has appeared with the U.S. Food and Drug Administration (FDA) placing a clinical hold on CRISPR Therapeutics (NASDAQ:CRSP) and its joint venture partner Vertex Pharmaceuticals’ (NASDAQ:VRTX) plans to start a Phase 1/2 trial-testing of a CRISPR treatment in sickle cell disease in the US.

The stem cell therapy to be trialed is called CTX001. The treatment involves extracting stem cells from a patient’s bone marrow and genetically editing them with CRISPR in the lab. The idea is that once infused back into the patient, the edited cells would raise levels of fetal hemoglobin in red blood cells. It should be noted that the lab results have been promising enough for the scientists to move towards human trials.

The FDA however, and without providing any details, has ordered both biotechs to not proceed with the study. At this stage, the clinical hold on the trial could be based on anything from a bureaucratic holdup to concerns over CRISPR’s specificity or editing efficiency

Meanwhile, Vertex and CRISPR Therapeutics said they are expecting to receive minutes from the regulator in the coming weeks. Once that information is received, both companies hope to have a better understanding of what it’ll take to get their study back on track.

After the news came out in after-hours trading on Wednesday/May 25, CRISPR Therapeutics, a name that has been on a triple-digit tear in recent months, traded down almost 20% to $60, while shares of Vertex remained largely unchanged. Although CRSP have recovered somewhat, they remain 12% lower than Wednesday’s close. Ticker currently trades at $67 and change.

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