Intellia Therapeutics (NTLA Quick QuoteNTLA - Free Report) has made meaningful progress with the development of its in vivo programs. The company is currently advancing two late-stage in vivo candidates — lonvo-z (or, NTLA-2002) for hereditary angioedema (HAE) and nex-z (or, NTLA-2001) for transthyretin (ATTR) amyloidosis.

Last month, Intellia completed enrollment in the phase III HAELO study lonvo-z, its novel in vivo CRISPR gene editing therapy, for treating patients with HAE. Top-line data from the same is expected in the first half of 2026.

Despite the current treatment options that require chronic administration for disease control, HAE attacks still occur. Lonvo-z aims to prevent HAE attacks by suppressing the plasma kallikrein activity.

Intellia remains on track to submit a potential biologics license application for lonvo-z for treating HAE in the second half of 2026. Upon potential approval, the company plans to launch lonvo-z in the United States in the first half of 2027.

Intellia is developing another in vivo genome-editing candidate, nex-z, in collaboration with Regeneron Pharmaceuticals (REGN Quick QuoteREGN - Free Report) . Nex-z is being evaluated in two late-stage studies, MAGNITUDE and MAGNITUDE-2, for ATTR amyloidosis with cardiomyopathy (ATTR-CM) and ATTR amyloidosis with polyneuropathy (ATTRv-PN), respectively.

The collaboration with REGN for nex-z is a boost for Intellia, as it provides the latter with resources to support the development of the candidate.

Earlier this year, Intellia dosed the first patient in the phase III MAGNITUDE 2 study evaluating nex-z for the treatment of patients with ATTRv-PN. Enrollment in this study is expected to be completed by the first half of 2026.

If the data from the MAGNITUDE-2 study is found to be positive, it will enable global regulatory filings for nex-z.

NTLA's Pipeline Setback & Competition in the Target Market

Earlier this year, Intellia suffered a major setback with nex-z development after it reported that a participant in the MAGNITUDE study experienced grade 4 liver transaminase elevations, indicating a notable increase in liver enzymes. Though the company noted that this adverse event appeared to have resolved without hospitalization or medical intervention, it had raised concerns about the gene therapy’s safety in the long run.

While Intellia’s pipeline of innovative CRISPR-based therapies looks promising, the development of these remains a complex affair. Upon successful development and potential approval, the candidates are likely to face stiff competition from companies that are also using the CRISPR/Cas9 gene editing technology to address various diseases in specific areas.

CRISPR Therapeutics (CRSP Quick QuoteCRSP - Free Report) is the first and only company in the world to market a CRISPR/Cas9-based therapy. The company’s one-shot gene therapy, Casgevy, was approved in late 2023 and early 2024 across the United States and Europe for two blood disorder indications — sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT).

CRSP has developed Casgevy in partnership with large biotech, Vertex Pharmaceuticals, which is responsible for global development and commercialization.

Meanwhile, Beam Therapeutics (BEAM Quick QuoteBEAM - Free Report) is developing its lead ex-vivo genome-editing candidate, BEAM-101, in the phase I/II BEACON study for treating SCD.

Beam Therapeutics is also developing in vivo therapies targeting AATD-associated liver disease and glycogen storage disease Ia indications.