Intellia to present data on CRISPR therapy for ATTR amyloidosis

CAMBRIDGE, Mass. - Intellia Therapeutics, Inc. (NASDAQ:NTLA), which has seen its stock surge 34.75% in the past week and commands a market capitalization of $1.67 billion, announced Monday it will present longer-term data from its ongoing Phase 1 trial of investigational nex-z for hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN) at an upcoming medical conference.

The presentation, scheduled for September 25 at the 5th International ATTR Amyloidosis Meeting in Baveno, Italy, will include disease-relevant measures with up to three years of patient follow-up. According to InvestingPro data, 11 analysts have recently revised their earnings expectations upward for the upcoming period, suggesting growing confidence in the company’s clinical programs.

Dr. Julian Gillmore from the National Amyloidosis Centre at UCL Division of Medicine will present the 24-month follow-up data from the study evaluating the safety and efficacy of nex-z, which is based on CRISPR/Cas9 gene editing technology.

Nex-z is designed to inactivate the TTR gene that produces the transthyretin protein. According to the press release, interim Phase 1 data has shown that administration of nex-z led to consistent reductions in TTR levels.

ATTR amyloidosis is a rare, progressive and fatal disease where transthyretin proteins misfold and build up as amyloid in the body, causing complications in multiple tissues including the heart and nerves. The hereditary form occurs when a person is born with mutations in the TTR gene.

The company estimates there are approximately 50,000 people worldwide living with ATTRv amyloidosis and between 200,000 and 500,000 people with the wild-type form of the disease.

Intellia is developing nex-z in collaboration with Regeneron Pharmaceuticals, Inc. as part of a multi-target discovery and development partnership.

In other recent news, Intellia Therapeutics has completed enrollment in its global Phase 3 HAELO study for hereditary angioedema (HAE) treatment, achieving this milestone in just nine months. The study involves approximately 60 participants, with nearly half from the United States, and aims to evaluate the efficacy and safety of their CRISPR-based gene editing therapy. Intellia plans to release topline data in the first half of 2026 and anticipates submitting a biologics license application in the second half of 2026, with a potential U.S. launch in early 2027.

On the financial front, H.C. Wainwright has raised its price target for Intellia Therapeutics to $30 while maintaining a Buy rating, citing the rapid enrollment in the Phase 3 trial as a positive sign. Meanwhile, Citizens JMP reiterated a Market Perform rating, also highlighting the swift enrollment as an indicator of significant interest in the treatment. However, Guggenheim has significantly lowered its price target to $14 from $45, reflecting adjusted peak sales projections for Intellia’s clinical programs. Guggenheim now estimates peak sales of approximately $330 million for NTLA-2002, a decrease from the previous $380 million projection.

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