Home » Results of Phase 1 of the World’s First Human CRISPR Gene Editing Trial Announced-Scientific Exploration

Results of Phase 1 of the World’s First Human CRISPR Gene Editing Trial Announced-Scientific Exploration

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Recently, Regeneron Pharmaceuticals and Intellia Therapeutics reported that the in-development CRISPR in vivo genome editing therapy NTLA-2001 has obtained positive results in a phase 1 clinical trial and has the potential to cure transthyretin amyloidosis (ATTR).

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Source: Science News

Source: Science News

On June 26, the research results were published in NEJM Magazine, thesis titled CRISPR-Cas9 In vivo Gene Editing for Transthyretin Amyloidosis

Image source: NEJM

Picture source:NEJM

In ATTR patients, a specific mutation in the TTR gene causes the liver to produce misfolded transthyretin. NTLA-2001 is delivered through non-viral lipid nanoparticles (LNP), which can specifically knock out the TTR gene in the liver, thereby reducing the expression of TTR protein.

The clinical trial enrolled 6 ATTR patients (4 males and 2 females), of which 3 received NTLA-2001 at a dose of 0.1 mg/kg, and the other 3 received the same therapy at a dose of 0.3 mg/kg.

Among the 3 people who received larger doses of treatment, 2 people’s TTR levels dropped by 80-96%, and current standard treatments for the disease usually reduce this indicator by an average of 81%.

In terms of safety, as of 28 days after receiving treatment, NTLA-2001 showed good safety, and no serious adverse events and liver problems were found. And the therapeutic dose of genetic NTLA-2001 did not produce off-target effects.

This research is the first to successfully treat hereditary diseases through blood injection of CRISPR, and may even become the first method to completely cure this type of disease. (Source: Science News,NEJM

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