Directly injected gene scissor therapy receives PT3 approval

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 mainly through ApoE receptors on the surface of liver cells, but there have been problems with toxicity due to excessive accumulation.

 

However, NTLA-2001 offers enhanced biodegradability by incorporating ester links into its lipid structure and achieved a half-life of one-quarter that of conventional LNPs in animal studies.

 

NTLA-2001 has achieved positive results in a global Phase I study as a treatment for hereditary transthyretin amyloidosis (hATTR) and is on track to become the first-in-class Crispr-based therapy.

 

ATTR is a disease in which mutations in the transthyretin gene cause the liver to produce misfolded transthyretin protein, leading to neurological damage and heart muscle abnormalities.

 

There are reportedly around 50,000 patients worldwide, and with an average life expectancy of 2-15 years after symptom onset, there is much interest in its cure.

 

Meanwhile, in April 2016, the US-based Regeneron signed a collaboration agreement with Intellia Therapeutics to jointly develop next-generation gene therapies using CRISPR gene editing technology.

 

Under the agreement, Intellia received an upfront payment of USD 75 million (KRW 104.6 billion) and is eligible to receive additional payments based on future performance.

 

In 2020, Regeneron secured the rights to commercialize NTLA-2001 through an additional USD 100 million (KRW 139.5 billion) license agreement.

 

Current U.S.

 

FDA-approved treatments for ATTR include Pfizer's Vyndaqel (tafamidis meglumine) and Vyndamax (tafamidis), Alnylam’s Onpattro (patisiran) and Amvuttra (vutrisiran), and Ionis' Tegsedi (inotersen).