For healthcare professionals

What is

nucresiran?

Nucresiran is an investigational medication being evaluated for people with ATTR amyloidosis with cardiomyopathy and hereditary ATTR amyloidosis with polyneuropathy.

It is a type of therapy called an RNAi therapeutic.

It is given as a subcutaneous injection (a shot under the skin).

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What is an RNAi?

DNA

Your body is made up of trillions of tiny cells. Inside each of these cells is your DNA, the information that your body uses to know how to function.

Every part of your body, like your brain and your heart, depends on the instructions stored in your DNA.

RNA

DNA can’t do it all on its own. It works with something called RNA to get the job done. DNA depends on RNA to send instructions to different parts of your body. When your DNA wants to create a protein like TTR, it uses RNA to send the message.

RNAi

RNAi therapeutics work by breaking up the messenger RNA, so the message doesn’t get delivered. In other words, an RNAi silences the RNA before it can deliver the instructions to make certain proteins, like TTR. This means RNAi therapeutics are silencer medicines.

Nucresiran is designed to prevent misfolded TTR from being made. It aims to prevent TTR amyloid deposits from building up in certain organs.

Researchers want to see if this can help reduce symptoms and improve quality of life in patients with ATTR amyloidosis.