FDA lifts hold on first human CRISPR trial
The US Food and Drug Administration (FDA) has allowed the first US-based human trial of CRISPR, the gene-editing technology, to proceed.
CRISPR Therapeutics and Vertex Pharmaceuticals announced the news on October 10.
Gene-editing company CRISPR Therapeutics partnered with biotechnology firm Vertex in 2015.
The strategic research collaboration was formed to discover and develop gene-editing treatments that correct defects in gene targets which cause particular diseases.
The partnership led to the development of CTX001, an investigational CRISPR therapy for patients suffering from sickle cell disease and β-thalassemia.
Sickle cell disease is a group of inherited blood disorders which affect red blood cells, and β-thalassemia is a specific disorder that reduces the production of haemoglobin.
The CTX001 therapy involves engineering cells outside of the body to produce high levels of foetal haemoglobin in them.
According to CRISPR Therapeutics and Vertex, CTX001 also has the potential to prevent sickle cell crisis, a painful and debilitating period of time where sickled red blood cells block the small blood vessels that carry blood to the bones.
The FDA put a hold on the CTX001 trial in May after the companies filed an Investigational New Drug Application (IND) to initiate it. CRISPR Therapeutics and Vertex said the FDA put the trial on hold until certain information could be produced.
The study of CTX001 in patients with β-thalassemia was not affected by the hold.
Now, the FDA has lifted the clinical hold and accepted the IND for CTX001 to treat sickle cell disease.
The collaboration between CRISPR Therapeutics and Vertex has so far resulted in Vertex gaining the exclusive rights to licence up to six new CRISPR-based treatments which have stemmed from the collaboration.
This particular study may become the first treatment to emerge from the joint research programme, and CRISPR Therapeutics and Vertex will share the research and development costs and profits equally.
News of the upcoming study follows a report by The Wall Street Journal which said that, since 2015, scientists in China have used CRISPR technology on at least 86 cancer patients—reportedly making it the first country in the world to test the gene-editing technology on humans.
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