On November 5, 2026 Intellia Therapeutics, Inc. (Nasdaq: NTLA), a leading biopharmaceutical company focused on revolutionizing medicine leveraging CRISPR gene editing and other core technologies, reported business updates and financial results for the third quarter ended September 30, 2026.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"This is a defining period for Intellia as we advance our late-stage pipeline and prepare for a transformative year ahead," said John Leonard, M.D., Intellia President and Chief Executive Officer. "We have assembled the teams and capabilities needed to support a successful launch in HAE while also continuing to advance nex-z’s Phase 3 clinical trials and our pipeline expansion efforts. Looking to 2027, we are working toward a historic milestone: the potential approval of lonvo-z as the world’s first in vivo CRISPR-based therapy. As a one-time treatment, we believe it could transform the HAE treatment paradigm while also delivering significant value to patients, physicians, payers and the overall healthcare system."

Lonvoguran Ziclumeran (Lonvo-z) for Hereditary Angioedema (HAE)

Designed as a one-time treatment that is administered in an outpatient setting, lonvo-z is an in vivo CRISPR gene editing candidate that is intended to inactivate the kallikrein B1 (KLKB1) gene to permanently lower kallikrein and bradykinin levels and to eliminate HAE attacks and the need for lifelong treatment.

•
In September, the U.S. Food and Drug Administration (FDA) accepted the Biologics License Application (BLA) for lonvo-z and granted the BLA Priority Review with a Prescription Drug User Fee Act (PDUFA) target action date of March 10, 2027. Additionally, FDA advised that it is not currently planning to hold an advisory committee to discuss the application. If approved, lonvo-z would be the world’s first in vivo CRISPR-based therapy and the only one-time treatment for HAE.
•
Intellia significantly advanced its launch readiness in recent months, completing the deployment of its field organization and deepening its engagement with HAE treatment centers and payers in preparation for a planned commercial launch in the first quarter of 2027.

Nexiguran Ziclumeran (Nex-z) for Transthyretin (ATTR) Amyloidosis

Nex-z is an in vivo CRISPR-based therapeutic candidate designed to inactivate the TTR gene in the liver, thereby preventing the production of transthyretin (TTR) protein. Nex-z offers the possibility of halting and reversing disease by driving a rapid, deep, consistent and potentially lifelong reduction in TTR protein after a one-time treatment. Intellia leads the development and commercialization of nex-z in collaboration with Regeneron Pharmaceuticals, Inc. (Regeneron).

•
Intellia has made significant progress advancing enrollment in its MAGNITUDE Phase 3 clinical trial in ATTR amyloidosis with cardiomyopathy (ATTR-CM).
•
During the third quarter, the company completed enrollment in its MAGNITUDE-2 Phase 3 clinical trial in hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN), exceeding its targeted randomization of 60 patients.
•
During the third quarter, Intellia began providing investigators with human leukocyte antigen (HLA) genotyping results for patients enrolled or entering screening in the ongoing nex-z Phase 3 trials.

Other Recent Updates

•
In September, Intellia entered into a non-dilutive senior secured term loan facility with OrbiMed, a leading global healthcare investment firm, for up to $400 million. The transaction provides Intellia with greater financial and operational flexibility as it advances toward several key milestones, including the planned U.S. approval and commercial launch of lonvo-z in HAE.
•
In October, the company announced the appointment of Jing L. Marantz, M.D., Ph.D., as Executive Vice President and Chief Medical Officer. Dr. Marantz is an accomplished drug development leader with more than 25 years of experience in the biopharmaceutical industry that includes the successful advancement of several now-approved rare disease therapies. In this role, she is leading the company’s translational, development, regulatory and medical affairs functions.

Upcoming Events

The company will participate in the following upcoming events:

•
November 6-9 – American Heart Association Scientific Sessions in Chicago, IL
•
November 12 – Intellia investor webcast regarding lonvo-z in HAE
•
November 12-16 – American College of Allergy, Asthma & Immunology 2026 Annual Scientific Meeting in Phoenix, AZ

•
November 15-18 – International Symposium on Amyloidosis in Montevideo, Uruguay
•
November 17 – Jefferies London Healthcare Conference in London, England
•
December 7-9 – 23rd Global Cardiovascular Clinical Trialists Forum in Washington, D.C.

Third Quarter 2026 Financial Results

•
Cash Position: Cash, cash equivalents and marketable securities were $614.8 million as of September 30, 2026, compared to $605.1 million as of December 31, 2025. The company’s existing cash resources are expected to fund its operations at least into 2028 and well beyond lonvo-z’s anticipated U.S. approval and commercial launch in HAE in the first quarter of 2027. This guidance excludes all potential commercial revenues from lonvo-z and potential future funding from the company’s term loan facility.
•
Collaboration Revenue: Collaboration revenue was $7.2 million for the third quarter of 2026, compared to $13.8 million for the third quarter of 2025. The decrease is primarily due to a reduction in revenue from Regeneron.
•
R&D Expenses: Research and development (R&D) expenses were $83.3 million for the third quarter of 2026, compared to $94.7 million for the third quarter of 2025. The decrease was primarily driven by lower external costs related to lonvo-z, lower stock-based compensation and lower research materials and contracted services costs, partially offset by higher headcount-related expenses. Stock-based compensation expense included in R&D expenses was $7.4 million for the third quarter of 2026.
•
G&A Expenses: General and administrative (G&A) expenses were $46.1 million for the third quarter of 2026, compared to $30.5 million for the third quarter of 2025. The increase was primarily driven by consulting costs associated with the continued buildout of the company’s commercial infrastructure and higher legal, facility-related and compensation expenses. Stock-based compensation expense included in G&A expenses was $7.0 million for the third quarter of 2026.
•
Net Loss: Net loss was $127.5 million for the third quarter of 2026, compared to $101.3 million for the third quarter of 2025. Net loss for the third quarters of 2026 and 2025 included an unrealized loss of $10.4 million and an unrealized gain of $3.4 million, respectively, due to changes in the fair value of investments.

(Press release, Intellia, NOV 5, 2026, View Source [SID1234674312])