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INTELLIA THERAPEUTICS Corporate
Overview January 2026 Exhibit 99.1
Intellia Therapeutics' Legal
Disclaimer This presentation contains "forward-looking statements" of Intellia Therapeutics, Inc. ("Intellia", "we" or "our") within the meaning of the Private Securities Litigation Reform Act of 1995.
These forward-looking statements include, but are not limited to, express or implied statements about Intellia's beliefs and expectations regarding: our ability to successfully develop and commercialize nexiguran ziclumeran
("nex-z"), also known as NTLA-2001, for the treatment of transthyretin ("ATTR") amyloidosis and lonvoguran ziclumeran ("lonvo-z"), also known as NTLA-2002, for the treatment of hereditary angioedema
("HAE") to address the significant unmet needs of patients and prescribers in ATTR amyloidosis and HAE, respectively; our ability to achieve upcoming objectives, including presenting topline data from the Phase 3 HAELO of lonvo-z by
mid-2026, submitting a biologics license application for lonvo-z for the treatment of HAE in the second half of 2026, successfully launching lonvo-z for the treatment of HAE in the U.S. in the first half of 2027, and resolving the clinical holds on
the MAGNITUDE and MAGNITUDE-2 trials of nex-z for the treatment of ATTR amyloidosis and finalizing a plan with regulators on the path forward; our ability to optimize the impact of our collaborations on our development programs, including our
collaboration with Regeneron Pharmaceuticals, Inc. ("Regeneron") and our co-development program for ATTR amyloidosis, and to advance additional development candidates; our expectations regarding our uses of capital, expenses, and ability to fund
operations into mid-2027; the potential commercial opportunities for our product candidates, including the value and market potential for our product candidates, including the potential of nex-z and lonvo-z to be single-dose treatments administered
in an outpatient setting, the potential of nex-z to transform the standard of care for ATTR amyloidosis, provide patients with consistently rapid, deep and durable TTR reduction, stability or improvement in the disease for most patients, improved
quality of life, and reduced cardiovascular events and mortality, and represent a meaningful opportunity for significant revenues and healthcare system savings, and the potential of lonvo-z to eliminate HAE attacks and burdensome ongoing
therapy for most patients; and our ability to leverage our in vivo and ex vivo technology for pipeline expansion efforts and collaborations. Any forward-looking statements in this presentation are based on management's current expectations and
beliefs of future events, and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties
include, but are not limited to: risks related to the ability to develop and commercialize any one or more of Intellia's product candidates successfully, including risks related to our ability to present a topline data readout from the HAELO
study by mid-2026, generate data to support lonvo-z's potential to be a one-time treatment for HAE, address the clinical hold on the MAGNITUDE and MAGNITUDE-2 trials of nex-z for ATTR amyloidosis and to resume
those clinical trials; risks related to Intellia's ability to protect and maintain its intellectual property position; risks related to Intellia's relationship with third parties, including our contract manufacturers, licensors and
licensees; risks related to the ability of our licensors to protect and maintain their intellectual property position; uncertainties related to the authorization, initiation and conduct of preclinical and clinical studies and other development
requirements for our product candidates, including uncertainties related to regulatory approvals to conduct clinical trials; risks related to the results of preclinical studies or clinical studies not being predictive of future results in connection
with future studies; the risk that clinical trial results will not be positive; risks related to the development and advancement of in vivo and ex vivo technologies for pipeline expansion and collaborations; risks related to Intellia's future
financial condition and our ability to fund our operations; risks related to Intellia's collaborations with Regeneron or our other collaborations not continuing or not being successful; and risks related to Intellia's ability to execute its
strategic plans, including completing pivotal clinical trials and commercial launch of its product candidates. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause Intellia's actual
results to differ from those contained in the forward-looking statements, see the section entitled "Risk Factors" in Intellia's most recent Annual Report of Form 10-K and Quarterly Report on Form 10-Q as well as discussions of
potential risks, uncertainties, and other important factors in Intellia's other filings with the Securities and Exchange Commission. All information in this presentation is as of the date on its cover page, and Intellia undertakes no duty to
update this information unless required by law.
Our Vision: Transform the Lives of
Patients Leveraging Gene Editing Technology Treat patients at the root cause of their disease 1x Single dose treatment with potential lifelong benefit Reduce burden for the patient and the healthcare system Best outcomes for
Intellia is Leading a New Era of
Medicine 3 Phase 3 Trials in hereditary angioedema (HAE) and transthyretin amyloidosis (ATTR-CM and ATTRv-PN) Patients Dosed with Intellia's investigational in vivo CRISPR-based therapies 600+ Working diligently to bring the first-ever in
vivo CRISPR-based therapies to patients living with severe diseases 4+ Publications in 5 AUG 5 2021 CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis JAN 31 2024 CRISPR-Cas9 In Vivo Gene Editing of KLKB1 for Hereditary Angioedema OCT 24
2024 CRISPR-Based Therapy for Hereditary Angioedema NOV 16 2024 CRISPR-Cas9 Gene Editing with Nexiguran Ziclumeran for ATTR Cardiomyopathy SEP 25 2025 Nexiguran Ziclumeran Gene Editing in Hereditary ATTR with Polyneuropathy Years of Follow-Up in
earliest dosed patients CRISPR: clustered regularly interspaced short palindromic repeats; Cas9: CRISPR-associated protein 9; ATTR-CM: ATTR amyloidosis cardiomyopathy; ATTRv-PN: hereditary ATTR amyloidosis polyneuropathy; KLKB1: kallikrein
Pipeline With Significant, Near-Term
Opportunities PROGRAM APPROACH Research and Preclinical Early-Stage Clinical Late-Stage Clinical PARTNERS In Vivo: CRISPR is the therapy Lonvo-z*: Hereditary Angioedema Knockout Nex-z**: Transthyretin Amyloidosis Knockout Hemophilia B**** Insertion
Research Programs for Other Targets Various Ex Vivo: CRISPR creates the therapy Research Programs Allogeneic and other *** LEAD LEAD *** Lead refers to lead development and commercial party. * Lonvo-z (lonvoguran ziclumeran), formerly referred to as
NTLA-2002. ** Nex-z (nexiguran ziclumeran), formerly referred to as NTLA-2001; Regeneron shares in approximately 25% of worldwide development costs and commercial profits for the ATTR program and has an option to enter into a co-promotion agreement
for the U.S. commercialization. *** Intellia is advancing both wholly owned and partnered programs. **** Hemophilia B is being advanced by Regeneron - Intellia is eligible for milestones and royalties. CRISPR: clustered regularly interspaced
short palindromic repeats.
Two Late-Stage Assets: Breakthrough
Profiles and Blockbuster Potential Hereditary Angioedema (HAE) Potential to be the first to offer lifelong freedom from attacks and prophylaxis with a single dose Phase 3 trial fully enrolled with 80 patients RMAT, ODD, PRIME designation
Expect to present Phase 3 topline results by mid-2026 BLA submission planned in second half 2026 Worldwide prevalence: ~150,0001 HAE market expected to reach $6.3B2 by 2030 Transthyretin Amyloidosis (ATTR) Potential to be the first to stabilize or
reverse disease progression with a single dose Phase 3 trials currently on clinical hold RMAT & ODD designation >90% patients enrolled in polyneuropathy Phase 3 >50% patients enrolled in cardiomyopathy Phase 3 Worldwide prevalence:
250,000 to 500,0004-7 ATTR market expected to reach $16.8B3 by 2030 Target Indication Unique Proposition Program Status Total Market Lonvo-z Nex-z 1. Zuraw et al. NEJM, 2008. 2. Evaluate Pharma Consensus Analyst forecasts (Oct. 2025). 3.
FactSet Consensus Analyst forecasts (Dec. 2025). 4. Hawkins et al. Annals of Medicine, 2015. 5. Maurer et al. Circulation: Heart Failure, 2019. 6. Nativi-Nicolau et al. ESC Heart Failure, 2021. 7. Gillmore et al. American Heart Association
Scientific Sessions, 2022. B: billion; BLA: Biologics License Application; ODD: Orphan Drug Designation; PRIME: Priority Medicine; RMAT: Regenerative Medicine Advanced Therapy.
Key 2025 Accomplishments and Upcoming
Objectives Lonvo-z HAE Presented longer-term data from Phase 1/2 clinical trial Completed enrollment of 80 patients in global Phase 3 HAELO trial Present topline Phase 3 HAELO data by mid-2026 Planned BLA submission in second half of 2026 Planned
U.S. commercial launch in first half of 2027 Nex-z ATTR Presented longer-term data from Phase 1 clinical trials in cardiomyopathy and polyneuropathy Enrolled >650 CM patients in the global Phase 3 MAGNITUDE trial Enrolled 47 PN patients in the
global Phase 3 MAGNITUDE-2 trial Resolve clinical holds on MAGNITUDE and MAGNITUDE-2 ATTR: transthyretin amyloidosis; BLA: biologics license application; HAE: hereditary angioedema
Lonvoguran Ziclumeran (lonvo-z) for
Hereditary Angioedema
Hereditary Angioedema (HAE): Currently
a Life-Long Genetic Condition with Significant Burden Rare, genetic and life-threatening disease Patients have unpredictable, recurrent, painful and potentially life-threatening swelling attacks1,2 Average age of diagnosis is 20 years old3 Symptoms
often begin in the first decade of life and typically worsen in puberty4,5 Attacks can be triggered by stress, trauma, infection, fatigue and hormones2 Approximately 7,000 patients treated in the U.S.6 Despite available treatments, significant unmet
need persists Many patients only achieve partial clinical control7,8,9 Patients make lifestyle modifications to manage fear and anxiety10 Treatment burden negatively affects patients, especially those taking injectable medications11 Insurance delays
and denials associated with maintaining access have significant impacts on individuals with HAE12 1. Zuraw et al. NEJM, 2008. 2. Busse and Christiansen, NEJM, 2020. 3. Farkas et al. Allergy, 2017. 4. Norris et al. Allergy Asthma Proc., 2022. 5.
Pancholy et al. Curr Opin. Pediatr., 2019. 6. Castaldo et al. Ann Allergy Asthma Immunol, 2025. 7. Banerji et al. JAMA, 2018. 8. Zuraw et al. Allergy Clin. Immunol, 2021. 9. Longhurst et al. NEJM, 2017. 10. Bork et al. Allergy Asthma Clin. Immunol,
2021. 11. Radojicic et al. Allergy Asthma Proc., 2021. 12. Arora et al. JACI In Practice, 2023. KIM Living with HAE "The fear is always there - a tickle in your throat, and you think, Do I have a cold, or is this a
HAE: Significant Market Opportunity
- Ripe for Disruptive Innovation 1. Evaluate Pharma Consensus Analyst forecasts (October 2025). 2. Castaldo et al. Allergy Asthma Immunol, 2025. 3. Mendivil et al. Allergy, Asthma & Clinical Immunology, 2023. 4. Lumry et al. ACAAI poster,
2022. B: billion; HAE: hereditary angioedema. 7,000 APPROX. Global HAE market projected to double from 2024 to 2030 patients treated2 U.S. represents 68% of the global HAE market value1 Sizable Addressable Patient Population in the U.S. ~60 % on
prophylaxis3,4 2024 2030 $3.1B $6.3B Rapidly Growing Global Market1
Lonvo-z Has the Potential to be a
One-Time Treatment for Hereditary Angioedema First and only investigational therapy that targets the KLKB1 gene, to reduce production of kallikrein protein at its source Observations from Phase 1/2 trial: Consistently rapid, deep and durable
kallikrein reductions Freedom from HAE attacks and ongoing therapy for most patients Well-tolerated safety profile Lonvo-z is being developed as a one-time IV infusion administered in an outpatient setting Based on interim lonvo-z Phase 1 data as of
data cutoff February 12, 2025. Studies are ongoing to assess this investigational product. IV: intravenous; KLKB1: kallikrein B1. KIM Living with HAE
Pooled Analysis of Phase 1/2
Clinical Data: Deep Kallikrein Reduction Remained Stable After a One-Time Treatment with Lonvo-z 50 mg Data cutoff: August 29, 2025. One month = 30.4375 days. This presentation includes data for an investigational product not yet approved by
regulatory authorities. BL: baseline; mg: milligram; SD: standard deviation. (N) 32 32 32 30 29 27 26 23 18 14 14 11 9 BL
Most Patients were Attack-Free
After a One-Time Treatment with Lonvo-z 50 mg; Attack Rate was Low and Stable with Up to 3 Years of Follow-Up Pooled Phase 1/2 Analysis Data cutoff date: August 29, 2025. The monthly attack rate is calculated as number of attacks that occurred
during each 28-day interval. Timepoints with data from at least 3 patients are presented. a. One patient had no attacks during the screening period. This patient was receiving long-term prophylaxis until 19 days prior to lonvo-z infusion. 1 month =
28 days. This presentation includes data for an investigational product not yet approved by regulatory authorities. BL: baseline; mg: milligram; SE: standard error. (N) 32 32 32 32 31 31 30 30 28 27 27 26 22 18 17 14 14 14 14 14 14 14 14 13 12 10 9
Pooled Analysis of Phase 1/2
Clinical Data: After Becoming Attack-Free and LTP-Free for 6 Months, All Patients Maintained Their Response Data cutoff date: August 29, 2025. Phase 1 eligibility was determined by historical attack period. a. Patient IDs align with prior
Phase 1 and Phase 2 publications. b. Baseline is defined as the screening period (50 mg initial dose or 25 mg to 50 mg) or for PBO to 50 mg as the time from informed consent to 50 mg infusion or start of any LTP, whichever occurred first. c. Patient
had 0.9 attacks per month in the 3 months prior to screening. This presentation includes data for an investigational product not yet approved by regulatory authorities. BL: baseline; LTP: long-term prophylaxis; mg: milligram; PBO: placebo; Pt:
patient. Patient IDa Phase-Initial Dose-Pt # Baseline Monthly Attack Rateb 1.2 1-50-04 0c 1-50-02 6.5 1-50-01 0.7 1-50-03 1.0 2-50-04 0.9 2-50-03 2.1 2-50-08 7.8 2-50-10 1.1 2-PBO-23 1.0 2-50-11 3.7 2-PBO-25 4.3 2-PBO-24 6.4 2-25-19 7.2 1-25-02 3.3
2-50-06 1.9 2-50-01 3.1 2-25-18 2.3 2-25-20 5.2 2-PBO-26 1.4 2-25-17 3.1 2-25-13 1.5 2-PBO-27 2.8 2-PBO-22 6.8 2-25-21 1.4 2-25-14 2.9 2-25-16 4.5 2-25-15 2.8 2-50-02 5.4 2-50-07 4.8 2-50-05 2.9 1-25-03 8.7 2-50-09 Months after lonvo-z 50 mg
infusion 24 patients have been attack-free and LTP-free for 7-32 months and remain so at last follow-up 7 patients have been attack-free and LTP-free for <6 months and remain so at last follow-up 59% reduction from BL in monthly attack rate
Mild Moderate Severe LTP initiation LTP withdrawal Attack-free and LTP-free: 6 months <6 months Attack:
A One-Time Treatment of Lonvo-z 50
mg was Well Tolerated with No Long-Term Risks Observed with Up to 3 Years of Follow-Up Pooled Phase 1/2 Analysis Data cutoff date: August 29, 2025. a. AEs that occurred after each patient received lonvo-z 50 mg are reported. b. The patient had
multiple risk factors which included recent COVID infection, ongoing history of smoking, and obesity. c. Occurred following lonvo-z 50 mg infusion in a patient previously treated with lonvo-z 25 mg. As previously reported, two patients treated with
lonvo-z 25 mg experienced transient, asymptomatic Grade 2 liver transaminase elevations, with peak values at Day 22 (Cohn, et al. NEJM., 2024) and Week 156 (Longhurst, et al. EAACI, 2025), respectively. All events resolved spontaneously without
intervention. This presentation includes data for an investigational product not yet approved by regulatory authorities. AE: adverse event; AST: aspartate aminotransferase; LTFU: long-term follow-up; mg: milligram; SAE: serious adverse event; TEAE:
treatment-emergent adverse event. Patients treated with lonvo-z 50 mg in the Phase 1/2 trial (N=32)a Reported within 28 days of infusion, n (%) Reported >28 days after infusion up to LTFU, n (%) Any TEAE ( 10% of patients) 27 (84)
30 (94) Infusion-related reaction 17 (53) 0 Fatigue 11 (34) 0 Headache 6 (19) 1 (3) Abdominal pain 2 (6) 2 (6) Nasopharyngitis 1 (3) 8 (25) Upper respiratory tract infection 1 (3) 6 (19) Arthralgia 1 (3) 4 (13) COVID-19 1 (3) 4 (13) Back pain 0 5
(16) Any SAE 0 1 (3) Pulmonary embolism 0 1 (3)b Safety of lonvo-z 50 mg after receiving the suboptimal dose (25 mg) was consistent with the overall population No clinically significant shifts in coagulation parameters Grade 1 bleeding AEs:
epistaxis (n=2) and vaginal hemorrhage (n=1) One SAE (pulmonary embolism) occurred in a patient with multiple risk factorsb one year after the infusion; the event resolved without sequelae No clinically significant shifts in liver enzymes Grade 2
AST elevation occurred in one patient (Day 1-4)c In the LTFU study (n=17), there were no SAEs or treatment-related AEs reported with lonvo-z 50 mg
Comparison of HAE Patient Survey
vs. Lonvo-z Clinical Data Most Surveyed Patients Reported They Continue to Have HAE Attacks1 Data cutoff date: August 29, 2025. 1. Busse, P et al. ACAAI poster, 2025. N=100 2. Cohn, DM, et al. Phase 1/2 pooled analysis of patients receiving a 50 mg
dose of lonvo-z presented at American College of Allergy, Asthma & Immunology Annual Scientific Meeting, Orlando, FL., 2025, November 6-10. This presentation includes data for an investigational product not yet approved by regulatory
authorities. HAE: hereditary angioedema; LTP: long term prophylaxis; mg: milligram. Only 20% reported being attack free for the past 12 months 89% of surveyed patients were on LTP therapies 76% were attack and LTP free for 12 months2 Most
Lonvo-z Patients Experienced Prolonged Disease Control2 Among the 17 patients >12 months since receiving lonvo-z 50 mg
Intellia Market Research: Most
Surveyed Patients Were Very Likely to Use "Product X" (Lonvo-z), Including Those on Leading LTPs 1. Intellia Commissioned Market Research Study Conducted with 104 US Patients and Caregivers in November and December 2025. LTP: long term
prophylaxis. Unlikely (1 or 2) Somewhat Likely (3, 4 or 5) Extremely/Very Likely (6 or 7) % "I think this treatment could bring a sense of freedom from the constant fear of attacks." Lonvo-z is an investigational product that has not