The companies have announced the first clinical data supporting the safety of in vivo CRISPR genome editing in humans.
NTLA-2001 is an investigational in vivo CRISPR therapy for transthyretin amyloidosis, currently being tested in an ongoing phase I clinical study. Interim data from the study, which involves a single intravenous infusion of the therapy, have now been published – and the results are promising.
NTLA-2001 is being tested in people with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN), and by day 28, no serious adverse events were observed in the first six patients, and the therapy was generally well tolerated.
NTLA-2001 is intended to inactivate the TTR gene in liver cells in order to prevent production of misfolded transthyretin (TTR) protein. Treatment with NTLA-2001 led to reductions in serum TTR, with mean reductions of 52% among three patients in the lower dose group, and 87% among the three patients in the higher dose group, including one patient with a 96% reduction.
“These are the first ever clinical data suggesting that we can precisely edit target cells within the body to treat genetic disease with a single intravenous infusion of CRISPR. The interim results support our belief that NTLA-2001 has the potential to halt and reverse the devastating complications of ATTR amyloidosis with a single dose,” said Intellia President and Chief Executive Officer John Leonard. “Solving the challenge of targeted delivery of CRISPR/Cas9 to the liver, as we have with NTLA-2001, also unlocks the door to treating a wide array of other genetic diseases with our modular platform, and we intend to move quickly to advance and expand our pipeline. With these data, we believe we are truly opening a new era of medicine.”