Intellia Therapeutics hopes to later gain approval of NTLA-2001 as a one-time tretament for transthyretin amyloidosis with cardiomyapthy. Image credit: Shutterstock / Panuwach.

Intellia Therapeutics has dosed the first patient in a pivotal trial of its in vivo clustered regularly interspaced short palindromic repeat (CRISPR) candidate NTLA-2001.

The US-based biotech has begun dosing in the global, Phase III study with its one-time gene editing treatment for patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM).

Go deeper with GlobalData

Data Insights

The gold standard of business intelligence.

Find out more

Related Company Profiles

View all

The in vivo CRISPR-Cas9 candidate acts by inactivating the transthyretin (TTR) gene to prevent the production of the TTR protein, which causes the disease.

The MAGNITUDE trial (NCT06128629) will be a randomised, double-blind, placebo-controlled study evaluating the efficacy and safety of NTLA-2001 in 765 patients. Patients will be randomised on a 2:1 ratio to receive a single 55mg infusion of the candidate or a placebo.

The first patient was dosed at Richmond Pharmacology in the UK. The trial will take place across three other sites, notably two in New Zealand and one in the US.

The primary endpoint of the study is cardiovascular (CV) mortality and CV-related events at 18 months. Secondary endpoints will evaluate change in serum TTR and change in Kansas City Cardiomyopathy Questionnaire Overall Summary Score (KCCQ-OS) at 18 months.

How well do you really know your competitors?

Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.

Company Profile – free sample

Thank you!

Your download email will arrive shortly

Not ready to buy yet? Download a free sample

We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below form

By GlobalData
Visit our Privacy Policy for more information about our services, how we may use, process and share your personal data, including information of your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address.

Intellia Therapeutics has partnered with Regeneron for the development of NTLA-2001.

GlobalData’s annual consensus forecast for NTLA-2001 projects that sales will reach $1.4bn in 2029.

GlobalData is the parent company of the Clinical Trials Arena.

CRISPR landscape

December 2023 marked an important moment in the history of CRISPR therapies after Vertex Pharmaceuticals’ and CRISPR Therapeutics’ exagamglogene autotemcel (exa-cel) was the first CRISPR-based therapy to receive approval from the US Food and Drug Administration (FDA).

The landscape is still evolving with many companies at discovery and pre-clinical stages. There are some candidates in clinical trials but 88% of them are in the early stages.

Several CRISPR candidates are further ahead in the pipeline, including Vor BioPharma’s VOR33, Shanghai Vitalgen BioPharma’s VGB-Ex01 and EdiGene ET-02.

Although expected to be one of the greatest pharmaceutical advances of the decade, there are potential risks and challenges associated with CRISPR gene editing including ethical and practical difficulties in conducting trials, concerns about safety and long-term adverse events of CRISPR therapies, potential off-target effects, and unintended modifications.

In 2020, the Royal Swedish Academy of Sciences awarded the Nobel Prize in Chemistry to two leading CRISPR researchers, Jennifer Doudna and Emmanuelle Charpentier.