The study medication is called NTLA-2001, and it is an investigational medicine. Investigational means NTLA-2001 has not been approved by any health or regulatory authority and can only be used in clinical research studies.NTLA-2001 is given as a 1-time intravenous (IV) infusion into the arm or hand.
NTLA-2001 is a gene editing system known as CRISPR/Cas9. It is designed to find and “knockout” (disable) the TTR gene. This means it changes a person’s DNA, so the gene stops making the TTR protein.
CRISPR/Cas9 is a powerful gene-editing tool that can add, remove, or alter sections of DNA, to fight specific diseases or mutations within an organism’s genetic code. CRISPR is a sort of genetic memory system that is engineered to target specific sequences of DNA, which can then be edited by Cas9. The Cas9 system allows us to cut DNA at specific points and edit the code using the stored DNA sequences. Click here to learn more about CRISPR and NTLA-2001.
Genes are made of DNA. All your genes together make up your genome, which acts like an instruction manual for your body that tells it how to work. Your genes are the pages within that instruction manual, and the DNA are the individual letters that make up the words. Genes tell your body to make different proteins to help it function.
Proteins are the building blocks for every tissue and organ in your body, including the heart.
A pathological gene variant or mutation is an error in a gene that causes the gene to produce an abnormal version of that a protein, resulting in incorrect amounts and/or function of that protein.
Participants in the MAGNITUDE study will be assigned at random to receive NTLA-2001 or a placebo, which has no active ingredients. You are 2 times more likely to receive NTLA-2001 than to receive a placebo. The study will be double-blind, which means neither you nor the study doctor will know if you are getting NTLA-2001 or a placebo.
The study medication is called NTLA-2001, and it is an investigational medicine. Investigational means NTLA-2001 has not been approved by any health or regulatory authority and can only be used in clinical research studies.
NTLA-2001 is given as a 1-time intravenous (IV) infusion into the arm or hand.
NTLA-2001 is a gene editing system known as CRISPR/Cas9. It is designed to find and “knockout” (disable) the TTR gene. This means it changes a person’s DNA, so the gene stops making the TTR protein. Click here to learn more about CRISPR and NTLA-2001.
Participants in the MAGNITUDE study will be assigned at random to receive NTLA-2001 or a placebo, which has no active ingredients. You are 2 times more likely to receive NTLA-2001 than to receive a placebo. The study will be double-blind, which means neither you nor the study doctor will know if you are getting NTLA-2001 or a placebo.
The study medication is called NTLA-2001, and it is an investigational medicine. Investigational means NTLA-2001 has not been approved by any health or regulatory authority and can only be used in clinical research studies.
NTLA-2001 is given as a 1-time intravenous (IV) infusion into the arm or hand.
NTLA-2001 is a gene editing system known as CRISPR/Cas9. It is designed to find and “knockout” (disable) the TTR gene. This means it changes a person’s DNA, so the gene stops making the TTR protein. Click here to learn more about CRISPR and NTLA-2001.
Participants in the MAGNITUDE study will be assigned at random to receive NTLA-2001 or a placebo, which has no active ingredients. You are 2 times more likely to receive NTLA-2001 than to receive a placebo. The study will be double-blind, which means neither you nor the study doctor will know if you are getting NTLA-2001 or a placebo.
The study medication is called NTLA-2001, and it is an investigational medicine. Investigational means NTLA-2001 has not been approved by any health or regulatory authority and can only be used in clinical research studies.
NTLA-2001 is given as a 1-time intravenous (IV) infusion into the arm or hand.
NTLA-2001 is a gene editing system known as CRISPR/Cas9. It is designed to find and “knockout” (disable) the TTR gene. This means it changes a person’s DNA, so the gene stops making the TTR protein. Click here to learn more about CRISPR and NTLA-2001.
Participants in the MAGNITUDE study will be assigned at random to receive NTLA-2001 or a placebo, which has no active ingredients. You are 2 times more likely to receive NTLA-2001 than to receive a placebo. The study will be double-blind, which means neither you nor the study doctor will know if you are getting NTLA-2001 or a placebo.
The study medication is called NTLA-2001, and it is an investigational medicine. Investigational means NTLA-2001 has not been approved by any health or regulatory authority and can only be used in clinical research studies.
NTLA-2001 is given as a 1-time intravenous (IV) infusion into the arm or hand.
NTLA-2001 is a gene editing system known as CRISPR/Cas9. It is designed to find and “knockout” (disable) the TTR gene. This means it changes a person’s DNA, so the gene stops making the TTR protein. Click here to learn more about CRISPR and NTLA-2001.
Participants in the MAGNITUDE study will be assigned at random to receive NTLA-2001 or a placebo, which has no active ingredients. You are 2 times more likely to receive NTLA-2001 than to receive a placebo. The study will be double-blind, which means neither you nor the study doctor will know if you are getting NTLA-2001 or a placebo.
The study medication is called NTLA-2001, and it is an investigational medicine. Investigational means NTLA-2001 has not been approved by any health or regulatory authority and can only be used in clinical research studies.
NTLA-2001 is given as a 1-time intravenous (IV) infusion into the arm or hand.
NTLA-2001 is a gene editing system known as CRISPR/Cas9. It is designed to find and “knockout” (disable) the TTR gene. This means it changes a person’s DNA, so the gene stops making the TTR protein. Click here to learn more about CRISPR and NTLA-2001.
Participants in the MAGNITUDE study will be assigned at random to receive NTLA-2001 or a placebo, which has no active ingredients. You are 2 times more likely to receive NTLA-2001 than to receive a placebo. The study will be double-blind, which means neither you nor the study doctor will know if you are getting NTLA-2001 or a placebo.
The study medication is called NTLA-2001, and it is an investigational medicine. Investigational means NTLA-2001 has not been approved by any health or regulatory authority and can only be used in clinical research studies.
NTLA-2001 is given as a 1-time intravenous (IV) infusion into the arm or hand.
NTLA-2001 is a gene editing system known as CRISPR/Cas9. It is designed to find and “knockout” (disable) the TTR gene. This means it changes a person’s DNA, so the gene stops making the TTR protein. Click here to learn more about CRISPR and NTLA-2001.
Participants in the MAGNITUDE study will be assigned at random to receive NTLA-2001 or a placebo, which has no active ingredients. You are 2 times more likely to receive NTLA-2001 than to receive a placebo. The study will be double-blind, which means neither you nor the study doctor will know if you are getting NTLA-2001 or a placebo.
The study medication is called NTLA-2001, and it is an investigational medicine. Investigational means NTLA-2001 has not been approved by any health or regulatory authority and can only be used in clinical research studies.
NTLA-2001 is given as a 1-time intravenous (IV) infusion into the arm or hand.
NTLA-2001 is a gene editing system known as CRISPR/Cas9. It is designed to find and “knockout” (disable) the TTR gene. This means it changes a person’s DNA, so the gene stops making the TTR protein. Click here to learn more about CRISPR and NTLA-2001.
Participants in the MAGNITUDE study will be assigned at random to receive NTLA-2001 or a placebo, which has no active ingredients. You are 2 times more likely to receive NTLA-2001 than to receive a placebo. The study will be double-blind, which means neither you nor the study doctor will know if you are getting NTLA-2001 or a placebo.
The study medication is called NTLA-2001, and it is an investigational medicine. Investigational means NTLA-2001 has not been approved by any health or regulatory authority and can only be used in clinical research studies.
NTLA-2001 is given as a 1-time intravenous (IV) infusion into the arm or hand.
NTLA-2001 is a gene editing system known as CRISPR/Cas9. It is designed to find and “knockout” (disable) the TTR gene. This means it changes a person’s DNA, so the gene stops making the TTR protein. Click here to learn more about CRISPR and NTLA-2001.
Participants in the MAGNITUDE study will be assigned at random to receive NTLA-2001 or a placebo, which has no active ingredients. You are 2 times more likely to receive NTLA-2001 than to receive a placebo. The study will be double-blind, which means neither you nor the study doctor will know if you are getting NTLA-2001 or a placebo.
