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).

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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.

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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.

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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.