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  • Directly injected gene scissor therapy receives PT3 approval
  • by Lee, Hye-Kyung | translator Alice Kang | Oct 25, 2024 05:49am
MFDS approves 'NTLA-2001' trial for ATTR patients
An in vivo gene scissor therapy that is injected directly into the body will enter Phase III clinical trials in Korea.

On the 23rd, the Ministry of Food and Drug Safety (MFDS) approved a Phase III clinical trial to evaluate the efficacy and safety of ¡®NTLA-2001¡¯ in participants suffering from Transthyretin Amyloidosis with Cardiomyopathy (ATTR-CM).

'NTLA-2001,¡¯ which is an investigational new drug by U.S. Intellia Therapeutics, works by delivering the ¡®guide RNA¡¯ that guides the gene editing ¡®Cas9 mRNA¡¯ to the target gene in a Lipid Nano Particle (LNP) to liver cells through intravenous injection to eliminate the target gene in the liver.

LNPs basically bind to ApoE in the blood and enter the liver ma
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