Intellia propels into 2024 with CRISPR pipeline updates
05 Jan 2024
Phase 3Gene TherapyIND
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Source: Pharmaceutical Technology
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).
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 diseasealpha-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.
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.
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