May 30, 2018
FDA halts
one of the first human CRISPR studies before it begins
A trial
planning to use the gene-editing tool CRISPR on sickle-cell patients has
been put on hold because of unspecified questions from US regulators.
Background: CRISPR
Therapeutics, which is developing the therapy, sought approval from the US Food
and Drug Administration in April to begin the study. The therapy involves
extracting stem cells from a patient’s bone marrow and editing them with CRISPR
in the lab. The idea is that the edited cells, once infused back into the
patient, would give rise to healthy red blood cells.
FDA
hold: But according to a statement on Wednesday
from CRISPR Therapeutics, the FDA ordered the company not to proceed with its
study until it answers questions about its treatment (the company didn’t
provide any further details about what those questions are, however).
The FDA
did not immediately respond to a request for comment.
Planned
trials: CRISPR Therapeutics says a European trial for the inherited
blood disorder beta thalassemia, which
involves a similar procedure, won’t be affected by the FDA’s order. The company
plans to begin that study in the second half of the year.
Separately, the first CRISPR trial in the US,
conducted by the University of Pennsylvania, is currently enrolling patients.
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