Overcome Delivery difficulties CRISPR Therapeutics is expected to enter clinical

Source: to overcome Delivery difficulties CRISPR Therapeutics is expected to enter clinical

features conference:new drug R & D“push the new”project Roadshow

The development of the CRISPR/Cas9 gene editing therapy bio-pharmaceutical company Intellia Therapeutics announced that a preclinical study results recently published in the Cell Reports Journal. In this pre-clinical trial achieved excellent results, is also expected to boost the CRISPR/Cas9 technology into the human bodyclinical trials.

go thyroxine protein amyloidosis（ATTR）is composed of a TTR gene in about 136 differentgeneticmutation, one of the causes of the chronic progressive and debilitating diseases. By these mutations cause abnormal protein deposition may affect the peripheral and autonomic nervous system, resulting in patients in the 20-year-old around the time of the occurrence of various symptoms.

Intellia in vivo site-directed projects use of lipidNanoparticles LNPs, will CRISPR/Cas9 composition delivered to the liver. The company in vivo targeting ATTR of the research project with Regeneron Pharmaceuticals common development. Intellia is designed to achieve the liver cells of the mutant TTR gene specific DNA implementation of the knock-out editing. These genes can lead to the heart, nerves and other tissue destructive transthyretin deposition.

this pre-clinical edit research aims to explore the use of LNP the CRISPR/Cas9 portion of the delivery to the mouse liver, mediated by the liver cells within the target DNA editing. Research in the LNP, the Cas9 mRNA and targeting the mouse TTR gene chemical synthesis of the gRNA of a common formulation, through a tail vein injection into mice in vivo. The other study evaluated in the guide mode different factors on the Edit of the impact, the chemical modification degree and a dose-response relationship between the efficiency of editing. By four months, the study evaluated the liver within the editing of the durability, and the measurement of the Cas9 mRNA and sgRNA pharmacokinetics（PK parameter.

studies show that lipidNanoparticles delivery of Cas9 mRNA and the sgRNA resulted in mouse liver, transthyretin levels decreased 97%, and maintain at least 12 months. The paper also reported the use Intellia’s LNP delivery system, a three-day period in mice in vivo detected CRISPR/Cas9 section. The researchers further showed that Intellia’s LNP technology to more advanced rodents, such as rats also have a similar stability and effective delivery of results.

“these data show that our proprietary lipidNanoparticle technology by a single dose to achieve the TTR gene significant and lasting edit,”Intellia platform and delivery technologies, Senior Vice President David Morrissey Dr. said:“Our lipid nanoparticle system is a transient expression system, that the CRISPR/Cas9 to edit the target gene, and then from the cell and cleared. This can minimize the cells CRISPR/Cas9 residence time, and decrease with CRISPR/Cas9-related security issues. If desired, the LNP also allows us to re-administration, to achieve the desired therapeutic effect. This document provides details about by far the most effective systemic CRISPR/Cas9 delivery method, which will be further support this year we raised the IND of the project and future livergenetictreatment of disease.”

we expect this technology to be able to successfully enter clinical practice, an early for us to bring new gene editing therapies. (bio ValleyBioon.com

Published at Mon, 05 Mar 2018 04:08:25 +0000