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CRISPR biotech Intellia Therapeutics has announced plans to reduce its workforce by 27% and halt early-stage R&D programmes as it concentrates resources on two late-stage drug candidates.
Intellia will focus on NTLA-2002 for hereditary angioedema and nexiguran ziclumeran (nex-z) for treating transthyretin (ATTR) amyloidosis. The company – which estimates an $8m charge for the layoffs and reorganisation – is in its second consecutive year of significant cuts, following a 15% workforce reduction in January 2024, alongside other research programme pauses.
Intellia’s strategic pivot comes as it seeks to transition from a research-focused biotech to a commercial-stage company, aiming to launch its first commercial therapies in 2027. The company ended 2024 with $862m in cash, which it says will fund operations through the first half of 2027, including its three ongoing Phase III clinical trials.
Intellia is prioritising NTLA-2002, a one-time CRISPR-based treatment targeting the kallikrein B1 (KLKB1) gene implicated in hereditary angioedema . The therapy is currently being investigated in the Phase III HAELO trial (NCT06634420) and could become the first approved in vivo CRISPR therapy. Preliminary Phase II results suggested NTLA-2002 could offer a “functional cure” for hereditary angioedema by reducing the frequency of life-threatening inflammatory attacks. Patient dosing in the HAELO trial is set to begin this quarter, with regulatory filings expected in 2026.
Similarly, nex-z is in late-stage trials for ATTR amyloidosis, a rare condition caused by the accumulation of misfolded transthyretin protein. Intellia is running two Phase III studies: MAGNITUDE (NCT06128629) for ATTR amyloidosis with cardiomyopathy (ATTR-CM), and MAGNITUDE-2 (NCT06672237), for patients with hereditary ATTR amyloidosis with polyneuropathy. Patient recruitment for MAGNITUDE is ongoing, with dosing for MAGNITUDE-2 expected in Q1 2025.
According to GlobalData’s Pharma Intelligence Center, NTLA-2002 will generate $534m in 2030, with nex-z set to pull in $1.2bn in the same year, if approved.
GlobalData is the parent company of Pharmaceutical Technology.
Among the programmes being discontinued is the CRISPR-based gene therapy dubbed NTLA-3001 designed to treat alpha-1 antitrypsin deficiency (AATD)-associated lung disease by correcting a mutation in the SERPINA1 gene. Preparations for Phase I/II trials of NTLA-3001 have been halted, along with other exploratory-stage projects.
In the statement accompanying the strategic reorganisation, CEO John Leonard said the decision to focus on NTLA-2002 and nex-z reflects Intellia’s need to allocate resources where it has “the greatest opportunity to create significant, near-term value,” citing a challenging market environment. As part of its transition, Intellia plans to build out a commercial leadership team by H2 2025 and increase medical education efforts to prepare for potential product launches in the coming years.