Lipid nanoparticles (LNP) can play an important role in gene editing therapy. IntelliaTherapeutics and Regeneron recently announced that their co-development program, ntra-2001, a systemic CRISPR/Cas9 therapy, has achieved positive results in a phase I clinical trial. A single dose of NTLA2001 resulted in an average 87% decrease in serum transthyroxine protein level (TTR), with a maximum reduction of 96% at day 28.CRISPR/Cas9 is a gene-editing tool that makes permanent, precisely targeted changes to a patient's chromosome and fixes potential genetic mutations.Ntra-2001 is a CRISPR/ CAS9-based treatment for hereditary transthyroxine protein-mediated amyloidosis with polyneuropathy (ATTRV-PN).

According to Intellia Therapeutics, NTRA-2001 is a targeted delivery of LNP in humans that selectively knocks out disease-causing genes and restores necessary genetic function through targeted insertion. Three of the six patients treated in the Phase I trial received a dose of 0.1mg/kg ntLA-2001 and the other three received a dose of 0.3mg/kg NTLA2001.At day 28, TTR decreased by an average of 52% in patients receiving 0.1mg/kg and 87% in patients receiving 0.3mg/kg, with a 97% reduction in one patient.

As revealed in IntelliaTherapeutics' patent, LNP contains amine lipids for encapsulation and in vivo escape, neutral and helper lipids for stabilization, and cloaking lipids. In general, LNPS used on CRISPR/Cas9 include DSPC, cholesterol, PE2K-DMG and other liposomes, which are mostly similar to those used for LNPS of mRNA vaccines.

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Reference

1. Intellia Therapeutics. (2021).Source: retrieve Intellia Therapeutics: https://www.intelliatx.com/crisprcas9/types-of-edits-2/

2.IntelliaTherapeutics(NTLA.us) and Regentium (RegN.us) announce the results of the first CRISPR clinical trial.Retrieve source: baidu: https://baijiahao.baidu.com/s?id=1703680441595581467&wfr=spider&for=pc