Intellia Therapeutics and Regeneron Pharmaceuticals have reported positive interim data from a Phase I clinical trial of in vivo CRISPR/Cas9 genome editing therapy, NTLA-2001, to treat transthyretin (ATTR) amyloidosis.
The open-label, international, multicentre, two-part trial is designed to assess NTLA-2001 in adult patients with ATTR amyloidosis with cardiomyopathy (ATTR-CM) or hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN).
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The findings comprise data from 12 adult ATTR-CM patients with New York Heart Association (NYHA) Class I-III heart failure.
Trial subjects were given single, intravenous doses of 0.7mg/kg and 1mg/kg NTLA-2001.
The trial analysed the variation from baseline in serum transthyretin (TTR) protein concentration in these participants.
NTLA-2001 offered a quick and profound decline in serum TTR by day 28 of 93% and 92% at 0.7mg/kg and 1mg/kg doses, respectively.
Additionally, these serum TTR declines lasted throughout the observation time with subjects followed up for two to six months.
NTLA-2001 was also found to be well tolerated at both dosages tested in the trial.
Transient infusion reactions were found to be the only treatment-related adverse event, which was reported in two subjects.
The findings back the potential of the therapy as a single-dose treatment for permanently inactivating the TTR gene and lowering the disease-causing protein in ATTR-CM patients.
An investigational candidate, NTLA-2001 is given systemically, or through a vein, for editing genes inside the body.
Intellia is carrying out this trial under a multi-target partnership with Regeneron. Under the deal, Intellia is leading the development and commercialisation of NTLA-2001.
Intellia president and CEO John Leonard said: “Together with the previously reported data from the polyneuropathy arm of this landmark study, these results strongly suggest that NTLA-2001 could serve as a single-dose treatment regardless of disease manifestation.
“Given the similarly robust TTR reductions observed at the two doses tested, we have selected a fixed dose comparable to the 0.7mg/kg level for evaluation across both arms in the ongoing dose-expansion portion of the study.”
In February this year, the companies reported positive interim data from the Phase I trial of NTLA-2001 for ATTR amyloidosis.
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