The first systemic in vivo CRISPR therapy has entered clinical trials

Professors Emmanuelle Charpentier and Jennifer Doudna, who discovered CRISPR's gene editing system, won the Nobel Prize in Chemistry this year.
co-founder of Intellya Therapeutics is Professor Jennifer Doudna.
company is working to develop innovative therapies using CRISPR gene editing technology through gene editing in and outside the body.
NTLA-2001 is an innovative gene-editing therapy in the body that packages CRISPR gene-editing systems that target TTR genes through lipid nanoparticles (LNP).
ATTR patients with a specific mutation in the TTR gene cause the liver to produce misfolded thyroxine protein.
NTLA-2001 is delivered via non-viral LNP, the TTR gene can be specifically removed by the liver, thereby reducing the expression of the TTR protein.
in non-human primate models, the therapy reduced the expression level of TTR protein by more than 95 percent after a treatment and lasted at least a year.
NTLA-2001 reduces TTR protein levels by 95% in animal models and maintains them for at least a year (Photo source: Resources: 2) Reference: s1. Intellia Therapeutics Doses First Patient in LandmarkR/Cas9 Clinical Trial of NTLA-2001 for The Treatment of Transthyretin Amyloidos. Retrieved November 9, 2020, from [2] Intellia Corporate Overview. Retrieved November 9, 2020, from。