Intellia Therapeutics declared clinical updates for its CRISPR therapeutics portfolio. Credit: rafapress via Shutterstock

Intellia Therapeutics teased its upcoming goals for several clinical programmes over the next three years, funded by a secure cash runway until 2026.

The Massachusetts-headquartered company announced its 2024 aim to complete patient enrolment for studies evaluating NTLA-2001 and NTLA-2002. Alongside this, Intellia plans to dose the first patient in the MAGNITUDE trial (NCT03748641) to investigate NTLA-2001 for treating ATTR 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 company plans to follow this with a Phase III study of NTLA-2001 in 2024, pursuing the therapy as a treatment for ATTR amyloidosis with polyneuropathy (ATTRv-PN). In a 4 January press release, Intellia forecasted a potential biologics license application (BLA) submission for NTLA-2002 in 2026.

Last year, the CRISPR-based therapeutics company ended Q4 with a $1 billion cash runway, expected to fund operations through to mid-2026. This was achieved following an expanded collaboration with Regeneron in 2023. The company plans to reorganise its operations, pausing some select exploratory research-stage programs and reducing its workforce by approximately 15%.

Amidst this, the company furthered its clinical programmes by initiating the MAGNITUDE trial, completing the dosing in a Phase I/II hereditary angioedema (HAE) trial with NTLA-2002, and submitting a clinical trial application to the US Food and Drug Administration (FDA) for NTLA-3001, a gene therapy for alpha-1 antitrypsin deficiency (AATD)-associated lung disease.

The US company has two main clinical focuses, which are looking at the development of CRISPR gene therapies in an in vivo and an ex vivo programme. As per a 4 January press release, this will continue to be a major focus for the company for the next three years.

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’s ex vivo programme is still in the early stages with an ongoing investigational new drug-enabling trial and multiple research collaborations for different conditions. However, the company is approaching later stage development in its in vivo programme with its ATTR therapy NTLA-2001. The company is also currently investigating new CRISPR therapies in three IND-enabling studies.