Intellia Therapeutics, Inc.
For the period ended Mar 31, 2026. Filed May 11, 2026.
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A company's report for each of its first three quarters. The financial statements are not audited. It shows how the business has moved since the last annual report, and what management says changed.
Mar 31, 2026 · Filed May 11, 2026
NTLA$11.35+2.3%Stored close · as of Nov 6, 2026
What changed in Management’s Discussion and Analysis?
Read the current filingPrevious report: 10-Q
Period ended 2025-09-30; filed 2025-11-06.
0001193125-25-269680
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsCurrent report: 10-Q
Period ended 2026-03-31; filed 2026-05-11.
0001193125-26-215740
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsThe baseline is this issuer’s latest earlier reporting period of the same form that was filed before the current report. A previous 10-Q is a different quarter, not a year-over-year comparison. Amendments are excluded.
Compared 126 previous paragraphs and 105 current paragraphs.
53 unchanged; 32 reworded; 20 added; 34 removed; 7 previous and 0 current paragraphs rearranged.
Tables are not compared: 5 previous and 3 current tables excluded. Dates, whitespace, punctuation and numbering changes are ignored; changes to financial figures remain significant.
A changed paragraph is not evidence that risk increased or decreased. An unchanged paragraph does not establish that risk stayed the same.
Added paragraphs (20)
Current report
Intellia Therapeutics, Inc. (“we,” “us,” “our,” “Intellia,” or the “Company”) is a leading biopharmaceutical company, focused on revolutionizing medicine leveraging CRISPR gene editing and other core technologies. Our mission is to transform the lives of people with severe diseases by developing and commercializing potentially curative treatments. With deep scientific, technical and clinical development experience, we aim to reset the standard for medicine by durably treating the root causes of disease.
Current report
For over a decade, we have applied our proprietary technologies and expertise, including CRISPR-based gene editing technologies, oligonucleotides, and lipid nanoparticles (“LNPs”), to develop novel, first-in-class product candidates. This includes the development of lonvoguran ziclumeran (“lonvo-z,” previously referred to as NTLA-2002) for the treatment of hereditary angioedema (“HAE”) and nexiguran ziclumeran (“nex-z,” previously referred to as NTLA-2001) for the treatment of transthyretin (“ATTR”) amyloidosis. These lead product candidates are the firstin vivo genome editing product candidates to advance into Phase 3 clinical development. These systemically administered CRISPR-based candidates are designed to address diseases with high unmet need with a single intravenous (“IV”) infusion that is administered in an outpatient setting. In April 2026, we reported positive topline data from the global Phase 3 HAELO clinical trial of lonvo-z in HAE, and preparations are underway for the planned commercial launch of lonvo-z in the first half of 2027.
Current report
In April 2026, we announced positive topline results from the global Phase 3 HAELO clinical trial. The trial met its primary endpoint. For the six-month efficacy evaluation period (weeks 5 to 28), a one-time infusion of lonvo-z reduced attacks by 87% versus placebo, with a mean monthly attack rate of 0.26 in the lonvo-z arm compared with 2.10 in the placebo arm (p<0.0001). The trial met all of its key secondary endpoints with statistical significance (p<0.0001). These included a 62% rate of patients who were entirely attack free and therapy free in the lonvo-z arm for the six-month efficacy evaluation period, compared with 11% of patients in the placebo arm. Favorable safety and tolerability data were observed for lonvo-z. The most common treatment emergent adverse events (“TEAEs”) during the primary observation period (infusion through week 28) were infusion-related reactions, headache and fatigue. All TEAEs reported as of the data cutoff (February 10, 2026) were mild or moderate (Grade 1 or Grade 2) and there were no serious adverse events observed in the lonvo-z arm. As of the data cutoff, all patients who received lonvo-z at baseline or in crossover after week 28 remained free from long-term prophylaxis therapies.
Current report
In April 2026, we announced the initiation of a rolling biologics license application (“BLA”) to the U.S. Food and Drug Administration (“FDA”) to seek regulatory approval for lonvo-z. We expect to complete our BLA submission to the FDA in the second half of 2026 to support a potential U.S. launch of lonvo-z in the first half of 2027.
Current report
In January 2026, we announced that the FDA lifted the clinical hold on the IND for MAGNITUDE-2 for patients with ATTRv-PN. In March 2026, we announced that the FDA lifted the clinical hold on the IND for MAGNITUDE for patients with ATTR-CM. Patient screening activities are advancing in both trials.
Current report
In November 2025, we presented positive follow-up data from the ongoing Phase 1 clinical trial of nex-z in patients with ATTR-CM at the American Heart Association Scientific Sessions. The data were as of an August 23, 2025, data cut-off date. Across all 36 patients, a one-time treatment of nex-z led to consistent and durable reductions in serum TTR, regardless of baseline levels, through the latest follow-up. Among the nine patients who reached 36 months of follow-up, the mean serum TTR reduction was 87%. Patients dosed with nex-z continued to show evidence of disease stabilization or improvement as evidenced by multiple markers of cardiomyopathy, including N-terminal pro-B-type natriuretic peptide, high sensitivity Troponin T, 6-minute walk test, Kansas City Cardiomyopathy Questionnaire and echocardiographic measures. The most commonly reported treatment-related adverse events were infusion-related reactions (“IRRs”) and transaminase elevations. Additionally, data from a post-hoc analysis was presented from a cohort of 1,792 ATTR-CM patients from the National Amyloidosis Center (“NAC”) who were followed contemporaneously and whose baseline characteristics were matched to those of the Phase 1 nex-z population. The analysis showed patients receiving a one-time treatment with nex-z had an all-cause mortality rate of 3.9 per 100 patient-years, while the matched cohort had an all-cause mortality rate of 12.7 per 100 patient-years. Nex-z was generally well tolerated across all patients in the Phase 1 clinical trial.
Current report
We plan to complete enrollment in MAGNITUDE-2 in the second half of 2026.
Current report
In order to treat—and potentially cure—a broader range of severe diseases, we and our collaboration partners are advancing the development of additional clinical and preclinical product candidates. Throughout our history, we have leveraged collaborations to expand and accelerate the development of product candidates. Our current relationships include our collaboration with Regeneron Pharmaceuticals, Inc. (“Regeneron”) and other research collaborations focused on the development of product candidates. See Note 7, “Collaborations and Other Arrangements” of our condensed consolidated financial statements appearing elsewhere in this Quarterly Report on Form 10-Q for additional information related to the terms of the agreements between us and our collaborators.
Current report
Collaboration Revenue
Current report
Research and Development
Current report
General and Administrative
Current report
Collaboration Revenue
Current report
Collaboration revenue decreased by $1.6 million during the three months ended March 31, 2026, as compared to the three months ended March 31, 2025. The decrease in collaboration revenue is primarily due to a reduction in revenue under the license and collaboration agreement with AvenCell Therapeutics, Inc. (the “AvenCell LCA”), as well as the achievement of a development milestone under the 2016 Regeneron Agreement in March of 2025, partially offset by an increase in revenue related to the ATTR Co/Co.
Current report
Research and Development
Current report
Research and development expenses decreased by $27.7 million during the three months ended March 31, 2026, as compared to the three months ended March 31, 2025.
Current report
a $5.0 million decrease in stock-based compensation driven by lower grant date fair values of share-based awards granted in the current period.
Current report
General and Administrative
Current report
General and administrative expenses increased by $5.8 million during the three months ended March 31, 2026, as compared to the three months ended March 31, 2025. This increase is primarily related to increased legal expenses and consulting costs associated with the Company’s continued buildout of the commercial function, partially offset by a decrease in stock-based compensation.
Current report
Net cash provided by investing activities
Current report
Net cash provided by financing activities of $33.6 million during the three months ended March 31, 2026 is related to net proceeds from at-the-market offerings. There was no cash provided by financing activities during the three months ended March 31, 2025.
Removed paragraphs (34)
Previous report
Intellia Therapeutics, Inc. (“we,” “us,” “our,” “Intellia,” or the “Company”) is a leading clinical-stage gene editing company, focused on revolutionizing medicine with CRISPR-based therapies. CRISPR is a gene editing technology which is also sometimes referred to as CRISPR/Cas or CRISPR/Cas9 when referring to the use of CRISPR technology with the Cas9 enzyme. Since our inception, Intellia has focused on leveraging gene editing technology to develop novel, first-in-class medicines that address important unmet medical needs and advance the treatment paradigm for patients. To harness the full potential of gene editing, Intellia continues to expand the capabilities of its CRISPR-based platform with novel editing and delivery technologies. To fully realize the transformative potential of CRISPR-based technologies, we are building a full-spectrum gene editing company, by leveraging our modular platform, to advancein vivoandex vivo therapies for diseases with high unmet need by pursuing two primary approaches. Forin vivo applications to address genetic diseases, we deploy CRISPR as the therapy. Ourin vivo programs use CRISPR to enable precise editing of disease-causing genes directly inside the human body. In addition, we are advancingex vivo applications to address immuno-oncology and autoimmune diseases, where CRISPR is used as the tool to create the engineered cell therapy. For ourex vivo programs, CRISPR is used to engineer human cells outside the body. Intellia’s deep scientific, technical and clinical development experience, along with its people and our robust intellectual property (“IP”) portfolio, have enabled us to unlock broad therapeutic applications of CRISPR and related technologies to create new classes of genetic medicine.
Previous report
For over a decade, we have been applying novel technologies, such as CRISPR-based gene editing technologies and lipid nanoparticle (“LNP”) delivery technologies, to developin vivoandex vivo product candidates. Ourin vivo product candidates address genetic diseases by deploying our technologies, including CRISPR/Cas9 delivered by LNPs, as the therapy for diseases with high unmet need. For ourex vivo product candidates, we apply our technologies to create engineered cell therapies to address immuno-oncology and autoimmune diseases. This experience has enabled us to develop first-in-class therapeutic applications of CRISPR/Cas9 and our other technologies, opening a new frontier in genetic medicine.
Previous report
Treating—and potentially curing—a broad range of severe diseases requires the application of multiple technologies. With our proprietary technologies at the core of our platform, we continue to research and develop new gene editing and delivery technologies to expand our therapeutic opportunities, furthering progress on the frontier of genetic medicine and generating additional development candidates. We continue to advance our platform’s modular solutions and research efforts on genome editing technologies as well as delivery and cell engineering capabilities to generate additional development candidates. Our mission is to transform the lives of people with severe diseases by developing potentially curative genome editing treatments.
Previous report
Our leadin vivo product candidates, lonvo-z for the treatment of HAE and nex-z for the treatment of ATTR amyloidosis, are the first CRISPR-based therapy candidates to be administered systemically, via intravenous (“IV”) infusion, for precision editing of a gene in a target tissue in humans.
Previous report
In addition, we are advancingex vivo programs, wholly owned and in collaboration with partners, to develop product candidates for the treatment of immuno-oncology and autoimmune diseases, and multiplein vivo programs, also wholly owned and in collaboration with partners, to address diseases with significant unmet medical need by delivering gene editing therapeutics to organs outside the liver.
Previous report
We presented three-year follow-up data from the Phase 1 portion of the ongoing Phase 1/2 study after receiving a single dose of lonvo-z in an oral presentation at the European Academy of Allergy and Clinical Immunology (“EAACI”) Congress 2025 on June 15, 2025 in Glasgow, United Kingdom. In the Phase 1 portion of the study, a one-time dose of 25 mg (N=3), 50 mg (N=4) or 75 mg (N=3) of lonvo-z was administered via IV infusion and plasma kallikrein protein levels were measured along with HAE attacks. At the time of the February 12, 2025 data cutoff, all 10 patients were attack-free and treatment-free for a median of nearly two years. With up to three years of follow-up, a single dose of lonvo-z led to a mean reduction in monthly HAE attack rate of 98% over the study period, compared to pre-treatment baseline. For all 10 patients, deep, dose-dependent and durable reductions in plasma kallikrein protein continued to be observed through the latest assessment. Across all three dose levels, lonvo-z was generally well tolerated and showed a safety profile consistent with earlier data presented at EAACI in 2024. The most frequent adverse events during the study period were infusion-related reactions (“IRRs”). IRRs were mostly Grade 1 and resolved with all patients receiving the full dose. With up to three years of follow-up, no treatment-emergent serious adverse events were observed, and no treatment-related adverse events were observed during the period following 28 days after dosing.
Previous report
On November 8, 2025, at the American College of Allergy, Asthma & Immunology Annual Scientific Meeting (“ACAAI”), longer-term clinical data will be presented from all patients who received a 50 mg dose of lonvo-z in our ongoing Phase 1/2 clinical trial in an oral session.
Previous report
More than 650 patients with ATTR-CM are currently enrolled in MAGNITUDE, and 47 patients with ATTRv-PN are enrolled in MAGNITUDE-2. To date, Grade 4 liver transaminase elevations have been reported in less than one percent of all patients enrolled in MAGNITUDE and no Grade 4 liver transaminase elevations have been reported in MAGNITUDE-2.
Previous report
We recently mandated that all MAGNITUDE and MAGNITUDE-2 clinical sites increase their monitoring of patient laboratory values in the weeks after dosing. We continue to consult with clinical investigators and other experts to investigate the transaminase elevations and consider potential additional risk mitigation strategies while awaiting the FDA’s formal clinical hold letter. Given the clinical hold, we have suspended our milestone guidance for nex-z pending regulatory alignment. We plan to provide an update once we have finalized a plan with regulators on the path forward.
Previous report
In March 2025, we announced that the FDA granted Regenerative Medicine Advanced Therapy (“RMAT”) designation to nex-z for the treatment of ATTR-CM.
Previous report
In May 2025, we presented Phase 1 wildtype vs. variant ATTR-CM data at the Heart Failure 2025 Meeting in Belgrade, Serbia. The data showed that nex-z reduced TTR production and showed promise for treating both wild-type (“ATTRwt”) and variant (“ATTRv”) ATTR-CM with a favorable safety profile. Absolute TTR levels dropped from 222.4 to 16.5 μg/mL (ATTRwt) and 132.0 to 16.6 μg/mL (ATTRv). Functional capacity and clinical biomarkers were favorably impacted in both patient groups. Evidence of stability or improvement in disease progression markers were observed across both populations at similar rates. In this Phase 1 data, the most commonly reported treatment-related adverse events were IRR, which were mild or moderate, and did not result in any discontinuations. And observed liver enzyme abnormalities were not considered serious, were asymptomatic and resolved spontaneously without medical intervention or sequelae.
Previous report
The MAGNITUDE Phase 3 clinical trial is now on hold following reports of Grade 4 liver transaminase elevations and increased total bilirubin, as noted above.
Previous report
On November 10, 2025, at the 2025 American Heart Association Scientific Sessions (“AHA”), longer-term data will be presented in a late breaker oral session from our ongoing Phase 1 clinical trial in patients with ATTR-CM.
Previous report
In May 2025, we presented positive two-year follow-up Phase 1 data in an oral presentation at the 2025 Peripheral Nerve Society (“PNS”) Annual Meeting in Edinburgh, United Kingdom. Across patients who received a one-time dose of 0.3 mg/kg or higher (n=33), the mean serum TTR reduction by Day 28 was 90% (corresponding mean absolute serum TTR level of 23.8 µg/mL), with levels remaining virtually unchanged through at least 24 months. Among the 18 patients with 24 month mNIS+7 endpoint assessments, 13 showed improvements of ≥ 4 points, which is considered to be a clinically meaningful threshold. Most of the patients in the cohort who had progressed on patisiran improved, and only a single patient among the 18 had a deterioration of ≥ 4. In this Phase 1 data, nex-z was generally well tolerated across all patients and at all dose levels tested. And treatment-related adverse events were consistent with those described for the cardiomyopathy population.
Previous report
The MAGNITUDE-2 Phase 3 clinical trial is now on hold following reports of Grade 4 liver transaminase elevations and increased total bilirubin, as noted above.
Previous report
To accelerate the development and commercialization of CRISPR/Cas9-based products in multiple therapeutic areas, we have formed, and intend to seek other opportunities to form, strategic alliances with collaborators who can augment our leadership in CRISPR/Cas9 therapeutic development. See Note 7, “Collaborations and Other Arrangements” of our condensed consolidated financial statements appearing elsewhere in this Quarterly Report on Form 10-Q for additional information related to the terms of the agreements between us and our collaborators.
Previous report
Collaboration revenue increased by $4.7 million during the three months ended September 30, 2025, as compared to the three months ended September 30, 2024. The increase in collaboration revenue is primarily due to cost reimbursements related to our collaboration with Regeneron.
Previous report
Research and development expenses decreased by $28.6 million during the three months ended September 30, 2025, as compared to the three months ended September 30, 2024.
Previous report
a $2.9 million decrease in external costs related to the development of nex-z, primarily due to the timing of clinical trial expenses;
Previous report
a $1.8 million decrease in external costs related to the development of NTLA-3001 as we continue to wind down the program as part of our strategic restructuring in 2025;
Previous report
a $12.0 million decrease in stock-based compensation driven by our reduced workforce and lower stock price.
Previous report
General and administrative expenses remained consistent during the three months ended September 30, 2025, as compared to the three months ended September 30, 2024.
Previous report
Comparison of Nine Months Ended September 30, 2025 and 2024
Previous report
Collaboration revenue decreased by $0.3 million to $44.7 million during the nine months ended September 30, 2025, as compared to $45.0 million during the nine months ended September 30, 2024. The decrease in collaboration revenue during the nine months ended September 30, 2025 is primarily due to the recognition of $21.0 million of previously eliminated intra-entity profit under the license and collaboration agreement with AvenCell in the nine months ended September 30, 2024, offset in part by cost reimbursements related to our collaboration with Regeneron in the nine months ended September 30, 2025. Refer to Note 7 to our condensed consolidated financial statements appearing elsewhere in this Quarterly Report on Form 10-Q for further details.
Previous report
Research and development expenses decreased by $49.2 million to $300.2 million during the nine months ended September 30, 2025, as compared to $349.4 million during the nine months ended September 30, 2024.
Previous report
a $6.7 million increase in external costs related to the development of lonvo-z, primarily due to an increase in spend on drug components and contracted services;
Previous report
a $4.9 million decrease in external costs related to NTLA-3001 as we continue to wind down the program as part of our strategic restructuring in 2025;
Previous report
a $30.9 million decrease in stock-based compensation driven by our reduced workforce and lower stock price.
Previous report
General and administrative expenses decreased by $6.7 million during the nine months ended September 30, 2025, as compared to the nine months ended September 30, 2024. This decrease was primarily related to a decrease in stock-based compensation, offset in part by increased expenses related to the ongoing buildout of our commercial infrastructure.
Previous report
Other Income, Net
Previous report
The increase in other income, net of $17.7 million is primarily related to a $32.1 million change to income from expense related to the fair value of our investments, offset in part by a $14.5 million decrease in interest income.
Previous report
Net cash provided by (used in) investing activities
Previous report
Net cash provided by financing activities of $129.5 million during the nine months ended September 30, 2025 includes $128.2 million in net proceeds from at-the-market offerings, $1.2 million in cash received from the issuance of shares through our employee stock purchase plan and $0.1 million in cash received from the exercise of stock options.
Previous report
Net cash provided by financing activities of $183.4 million during the nine months ended September 30, 2024 includes $176.9 million in net proceeds from at-the-market offerings, $4.8 million in cash received from the exercise of stock options and $1.7 million in cash received from the issuance of shares through our employee stock purchase plan.
Reworded paragraphs (32)
Previous wording
our ability to execute our clinical study strategy for lonvoguran ziclumeran (“lonvo-z,” also referred to as NTLA-2002), our program for the treatment of hereditary angioedema (“HAE”), including the ability to successfully complete our global HAELO Phase 3 study, generate results supporting the potential of lonvo-z to achieve lifelong control of HAE attacks after a one-time treatment, report topline data from the HAELO Phase 3 trial by mid-2026, the ability to file a biologics license application (“BLA”) or comparable marketing application in the second half of 2026 and potentially launch lonvo-z in the U.S. in the first half of 2027, or the success of such program;
Current wording
our ability to execute our planned commercial launch of lonvoguran ziclumeran (“lonvo-z,” previously referred to as NTLA-2002), our program for the treatment of hereditary angioedema (“HAE”), including the ability to successfully complete the filing of a biologics license application (“BLA”) or comparable marketing application for lonvo-z in the second half of 2026, receive approval to market lonvo-z, and launch lonvo-z in the U.S. in the first half of 2027, or the success of such program;
Previous wording
our ability to execute our clinical study strategy for nexiguran ziclumeran (“nex-z,” also referred to as NTLA-2001), our program for the treatment of transthyretin (“ATTR”) amyloidosis, including the ability to resume dosing and screening in the MAGNITUDE and MAGNITUDE-2 Phase 3 clinical trials by resolving the clinical hold placed by the U.S. Food and Drug Administration (“FDA”) on the Investigational New Drug (“IND”) applications for these trials and the ability to successfully complete these studies, or the success of such program;
Current wording
our ability to execute our clinical study strategy for nexiguran ziclumeran (“nex-z,” previously referred to as NTLA-2001), our program for the treatment of transthyretin (“ATTR”) amyloidosis, including the ability to resume dosing in the MAGNITUDE and MAGNITUDE-2 Phase 3 clinical trials following removal of the clinical hold placed by the U.S. Food and Drug Administration (“FDA”) on their Investigational New Drug (“IND”) applications, the ability to complete enrollment in MAGNITUDE-2 in the second half of 2026, and the ability to successfully complete this study, or the success of such program;
Previous wording
estimates of our expenses, future revenues, capital requirements and our needs for additional financing;
Current wording
estimates of our expenses, future revenues, capital requirements and our needs for additional financing, including our ability to fund our ongoing operating expenses and capital expenditure requirements at least into 2028;
Previous wording
Our strategy is to develop and commercialize our product candidates and further our gene editing technology to develop new product candidates, expanding the application of gene editing medicine. All of our revenue to date has been collaboration revenue. Since our inception and through September 30, 2025, we have funded our operations through our initial public offering (“IPO”) and concurrent private placements, follow-on public offerings, at-the-market offerings and the sale of convertible preferred stock, as well as through our collaboration agreements.
Current wording
Our strategy is to (1) advance the clinical development of our lead CRISPR-based product candidates, (2) build and leverage a fully integrated commercial-stage infrastructure, (3) expand our pipeline utilizing our expertise and proprietary technologies, and (4) collaborate with partners to expand our technology reach and maximize our return on investment. All of our revenue to date has been collaboration revenue. Since our inception and through March 31, 2026, we have funded our operations through our initial public offering (“IPO”), private placements, follow-on public offerings, at-the-market offerings, the sale of convertible preferred stock and through our collaboration agreements.
Previous wording
Our leadin vivo programs are the only Phase 3 genome editing product candidates designed to address unmet need in patients with HAE and ATTR amyloidosis. Ourin vivo research efforts focus on pursuing additional targets within the liver and expanding delivery technology to enable product candidates for diseases outside the liver.
Current wording
We are the first company to advancein vivo genome editing product candidates into Phase 3 clinical development. These candidates are being developed for patients with HAE and ATTR amyloidosis. Our research efforts focus on pursuing additional targets within the liver and expanding our targets and delivery technology for diseases outside the liver.
Previous wording
Lonvo-z is a wholly owned, investigationalin vivo CRISPR-based therapy designed to inactivate thekallikrein B1 (“KLKB1”)gene in the liver that offers the possibility of dramatically reducing or eliminating HAE attacks by driving consistent, deep and potentially lifelong reduction in kallikrein levels after a one-time treatment. It also aims to eliminate the significant treatment burden associated with currently available HAE therapies.
Current wording
Lonvo-z is a wholly owned, investigationalin vivo CRISPR-based therapeutic candidate designed to inactivate thekallikrein B1 (“KLKB1”)gene in the liver, drive consistent, deep and potentially lifelong reduction in kallikrein levels, and dramatically reduce or eliminate HAE attacks via a one-time treatment. It also aims to eliminate the significant burdens associated with currently available HAE therapies.
Previous wording
In October 2024, we announced the initiation of the Phase 3 HAELO study of lonvo-z. HAELO is a global, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of lonvo-z in 60 adults with Type I or Type II HAE. Patients will be randomized 2:1 to receive a single 50 mg infusion of lonvo-z or placebo. Patients randomized to the placebo arm will be eligible for optional crossover to lonvo-z at week 28. The primary endpoint is the number of HAE attacks from week 5 through week 28. In January 2025, we announced that the first patient had been dosed in the global Phase 3 study. In September 2025, we announced that enrollment was completed. Patients in this trial are receiving a 50 mg dose of lonvo-z. We expect to report topline data from the HAELO Phase 3 trial by mid-2026, submit a BLA to the FDA in the second half of 2026 and continue preparing for an anticipated U.S. commercial launch of lonvo-z in the first half of 2027.
Current wording
HAELO is our Phase 3 clinical study of lonvo-z for the treatment of HAE. HAELO is a global, randomized, double-blind, placebo-controlled study that was designed to evaluate the efficacy and safety of lonvo-z planned for 60 adults with Type I or Type II HAE. Patients were randomized 2:1 to receive a single 50 mg infusion of lonvo-z or placebo. Patients randomized to the placebo arm are eligible for optional crossover to lonvo-z at week 28. The primary endpoint is the number of HAE attacks from week 5 through week 28. In January 2025, we announced that the first patient had been dosed in the global Phase 3 study. In September 2025, we announced that enrollment was completed. In total, 80 patients were enrolled in the trial and received a one-time 50 mg dose of lonvo-z or placebo.
Previous wording
Nex-z is an investigationalin vivo CRISPR-based therapy 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 deep, consistent and potentially lifelong reduction in TTR protein after a single dose.
Current wording
Nex-z is an investigationalin vivo CRISPR-based therapeutic candidate designed to inactivate the TTR gene in the liver, thereby preventing the production of TTR protein. Delivered with ourin vivo LNP technology, nex-z offers the possibility of halting and reversing the disease by driving a deep, consistent and potentially lifelong reduction in TTR protein after a one-time treatment.
Previous wording
On October 29, 2025, the FDA placed a clinical hold on the IND applications for the MAGNITUDE and MAGNITUDE-2 Phase 3 clinical trials for patients with ATTR amyloidosis with cardiomyopathy (“ATTR-CM”) and hereditary ATTR amyloidosis with polyneuropathy (“ATTRv-PN”), respectively. This action followed a report of Grade 4 liver transaminase elevations and increased total bilirubin in a patient who was dosed with nex-z in the MAGNITUDE trial. This patient passed away on November 5, 2025, and an investigation is underway to evaluate the case.
Current wording
On October 29, 2025, the FDA placed a clinical hold on the investigational new drug (“IND”) applications for the MAGNITUDE and MAGNITUDE-2 Phase 3 clinical trials for patients with ATTR amyloidosis with cardiomyopathy (“ATTR-CM”) and hereditary ATTR with polyneuropathy (“ATTRv-PN’), respectively. This action followed a report of Grade 4 liver transaminase elevations and increased total bilirubin in a patient who was dosed with nex-z in the MAGNITUDE trial. This patient passed away on November 5, 2025. It was reported by the principal investigator that the patient died due to septic shock secondary to a perforated duodenal ulcer. The patient’s complicated clinical course also included acute liver injury and its treatment with corticosteroids. An autopsy report supported the clinical diagnoses.
Previous wording
In 2024, we initiated the pivotal Phase 3 MAGNITUDE trial. The MAGNITUDE trial is a randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of nex-z in adults with ATTR-CM. The primary endpoint of the study is a composite of cardiovascular (“CV”)-related mortality and events. MAGNITUDE-2 is an international, randomized, double-blind, placebo-controlled study to evaluate the efficiency and safety of nex-z in approximately 1,200 adults with ATTR-CM. Patients will be randomized 2:1 nex-z:placebo, with a single 55 mg infusion of nex-z administered.
Current wording
In 2024, we initiated the pivotal Phase 3 MAGNITUDE trial. The MAGNITUDE trial is an international, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of nex-z in approximately 1,200 adults with ATTR-CM. The primary endpoint of the study is a composite of cardiovascular (“CV”)-related mortality and events. Patients are randomized 2:1 nex-z:placebo, with a single 55 mg infusion of nex-z administered.
Previous wording
In November 2024, we announced that the FDA had cleared our nex-z IND application to initiate the MAGNITUDE-2 pivotal Phase 3 trial for ATTRv-PN. MAGNITUDE-2 is an international, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of nex-z in 50 adults with ATTRv-PN. Patients will be randomized 1:1 to receive a single 55 mg infusion of nex-z or placebo. Patients randomized to the placebo arm will be eligible for optional crossover to receive nex-z following month 18. The primary endpoints are the change from baseline in modified Neuropathy Impairment Score +7 (mNIS+7) at month 18 and serum TTR at day 29. The mNIS+7 scale is a validated measure specifically designed to assess and quantify polyneuropathy impairment, including muscle weakness, muscle stretch reflexes, sensory loss and autonomic impairment. In April 2025, we announced that the first patient was randomized and dosed with nex-z in the global Phase 3 MAGNITUDE-2 study.
Current wording
In November 2024, we announced that the FDA had cleared our nex-z Investigational New Drug (“IND”) application to initiate the MAGNITUDE-2 pivotal Phase 3 trial for ATTRv-PN. MAGNITUDE-2 is an international, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of nex-z in 50 adults with ATTRv-PN. Patients will be randomized 1:1 to receive a single 55 mg infusion of nex-z or placebo. Patients randomized to the placebo arm will be eligible for optional crossover to receive nex-z following month 18. The primary endpoints are the change from baseline in modified Neuropathy Impairment Score +7 (mNIS+7) at month 18 and serum TTR at day 29. The mNIS+7 scale is a validated measure specifically designed to assess and quantify polyneuropathy impairment, including muscle weakness, muscle stretch reflexes, sensory loss and autonomic impairment. In April 2025, we announced that the first patient was randomized and dosed with nex-z in the global Phase 3 MAGNITUDE-2 study.
Previous wording
In September 2025, we presented positive longer-term follow-up data from the Phase 1 clinical trial in an oral presentation at the 5 th International ATTR Amyloidosis Meeting for Patients and Doctors in Baveno, Italy. The results were simultaneously published in the New England Journal of Medicine.
Current wording
In September 2025, we presented updated data from our Phase 1 clinical trial of nex-z in patients with ATTRv-PN at the 5 th International ATTR Amyloidosis Annual Meeting for Patients and Doctors in Baveno, Italy. The results were simultaneously published in the New England Journal of Medicine. Deep, durable and consistent TTR reductions continued to be observed. Across the 33 patients who received a one-time dose of 0.3 mg/kg or higher, the mean serum TTR reduction at 24 months was 92% and among the 12 patients who had reached 36 months of follow-up, the mean serum TTR reduction was 90%. Favorable trends indicating stability or improvement were observed in most patients with ATTRv-PN after a single dose of nex-z as evidenced by multiple clinical and biomarker measures, including Neuropathy Impairment Score, modified Neuropathy Impairment Score +7, modified body mass index, Norfolk Quality of Life-Diabetic Neuropathy questionnaire, neurofilament light chain, and polyneuropathy disability score. Nex-z was generally well tolerated as of the data cutoff date across all patients and at all dose levels tested. The most commonly reported treatment-related adverse events were infusion-related reactions, which were mild or moderate and did not result in any discontinuations. As previously reported, three participants had Grade 3 or greater liver enzyme elevations that were not considered serious, were asymptomatic and resolved spontaneously without medical intervention or sequelae.
Previous wording
General and administrative expenses consist primarily of compensation and benefits, including stock-based compensation, for our executive, finance, legal, human resources, business development and support functions. Also included in general and administrative expenses are allocated facility-related costs not otherwise included in research and development expenses, travel expenses and professional fees for auditing, tax and legal services, including intellectual property-related legal services, and other consulting fees and expenses.
Current wording
General and administrative expenses consist primarily of compensation and benefits, including stock-based compensation, for our executive, finance, legal, human resources, business development and support functions. Also included in general and administrative expenses are allocated facility-related costs not otherwise included in research and development expenses, adjustments related to impairment or accelerated amortization of long-lived assets, ongoing expenses related to the buildout of our commercial infrastructure, travel expenses and professional fees for auditing, tax and legal services, including IP-related legal services, and other consulting fees and expenses.
Previous wording
During the three and nine months ended September 30, 2025 and 2024, other income (expense), net consists of interest income earned on our cash, cash equivalents, restricted cash equivalents and marketable securities and change in the fair value of our investments.
Current wording
During the three months ended March 31, 2026 and 2025, other income (expense), net consists of interest income earned on our cash, cash equivalents, restricted cash equivalents and marketable securities and change in the fair value of our investments.
Previous wording
a $15.8 million increase in external costs related to the development of nex-z, primarily due to an increase in spend on contracted services and drug components;
Current wording
a $2.9 million decrease in external costs related to the development of nex-z, one of our lead product candidates, primarily due to a decrease in spend on contracted services and drug components;
Previous wording
a $3.4 million increase in external costs related to the development of lonvo-z, primarily due to an increase in spend on drug components and contracted services;
Current wording
a $2.5 million decrease in external costs related to the development of lonvo-z, one of our lead product candidates, primarily due to a decrease in spend on drug components;
Previous wording
a $7.7 million decrease in employee-related expenses driven primarily by a workforce reduction in January 2025;
Current wording
a $7.0 million decrease in employee-related expenses driven primarily by decreased consulting costs and severance expense related to the Company's workforce reduction in January 2025;
Previous wording
a $9.2 million decrease in research materials and contracted services primarily driven by a decrease in internal research and development spend, contracted services and drug components;
Current wording
a $7.6 million decrease in research materials and contracted services primarily driven by a decrease in internal research and development spend, contracted services and drug components;
Previous wording
a $1.6 million increase in facility-related expenses primarily related to rent expense allocated to research and development; and
Current wording
a $2.4 million decrease in facility-related expenses primarily related to rent expense allocated to research and development; and
Previous wording
The increase in other income, net of $1.1 million is primarily related to a $6.5 million change to income from expense related to the fair value of our investments, offset in part by a $5.4 million decrease in interest income.
Current wording
The decrease in other income, net of $2.2 million is primarily related to a $3.4 million decrease in interest income, partially offset by a $1.2 million decrease in expense due to the change in fair value of our investment.
Previous wording
Since our inception through September 30, 2025, we have funded our operations through our collaboration agreements, our initial public offering and concurrent private placements, follow-on public offerings, at-the-market offerings and the sale of convertible preferred stock.
Current wording
Since our inception through March 31, 2026, we have funded our operations through an initial public offering, private placements, follow-on public offerings, at-the-market offerings, the sale of convertible preferred stock and through our collaboration agreements.
Previous wording
As of September 30, 2025, we had $669.9 million in cash, cash equivalents and marketable securities.
Current wording
As of March 31, 2026, we had $517.2 million in cash, cash equivalents and marketable securities.
Previous wording
In March 2022, we entered into an Open Market Sale Agreement (the “2022 Sale Agreement”) with Jefferies LLC (“Jefferies”), under which Jefferies is able to offer and sell, from time to time in “at-the-market” offerings, shares of our common stock having aggregate gross proceeds of up to $400.0 million. In February 2024, we entered into an amendment to the 2022 Sale Agreement (the “2022 Sale Agreement, as amended”) to increase the size of the at-the-market offering program from $400.0 million to $750.0 million. We agreed to pay cash commissions of up to 3.0% of the gross proceeds of sales of common stock under the 2022 Sale Agreement, as amended. Through September 30, 2025 we have issued 26,313,157 shares of our common stock under the 2022 Sale Agreement, as amended. During the nine months ended September 30, 2025, we issued 11,790,624 shares of our common stock, in a series of sales, at an average price of $11.15 per share, in accordance with the 2022 Sale Agreement, as amended, for aggregate net proceeds of $128.2 million, after payment of cash commissions. As of September 30, 2025, $117.7 million in shares of common stock remain eligible for sale under the 2022 Sale Agreement, as amended.
Current wording
In March 2022, we entered into an Open Market Sale Agreement (the “2022 Sale Agreement”) with Jefferies LLC (“Jefferies”), under which Jefferies was able to offer and sell, from time to time in “at-the-market” offerings, shares of our common stock having aggregate gross proceeds of up to $400.0 million. In February 2024, we entered into an amendment to the 2022 Sale Agreement to increase the size of the at-the-market offering program from $400.0 million to $750.0 million. In March 2026, we entered into Amendment No. 2 to the 2022 Sale Agreement (the “2022 Sale Agreement, as amended”) to increase the size of the at-the-market offering program from $750.0 million to $1,035.3 million. We agreed to pay cash commissions of up to 3.0% of the gross proceeds of sales of common stock under the 2022 Sale Agreement, as amended. Through March 31, 2026 we have issued 28,925,501 shares of our common stock under the 2022 Sale Agreement, as amended. During the three months ended March 31, 2026, we issued 2,612,344 shares of our common stock, in a series of sales, at an average price of $13.21 per share, in accordance with the 2022 Sale Agreement, as amended, for aggregate net proceeds of $33.6 million, after commissions and offering costs. As of March 31, 2026, $368.5 million in shares of common stock remain eligible for sale under the 2022 Sale Agreement, as amended.
Previous wording
Our primary uses of capital are, and we expect will continue to be, research and development research materials and contracted services, clinical trial costs, compensation and related expenses, laboratory and office facilities, research supplies, legal and regulatory expenses, patent prosecution filing and maintenance costs for our licensed IP, milestone and royalty payments and general overhead costs. During 2025, we expect our expenses to decrease compared to prior periods as a result of our strategic reorganization in January 2025, as we focus resources on high value programs within our pipeline, such as lonvo-z and nex-z, to ensure efficient execution, achieve near-term clinical milestones, and prepare for commercial launch.
Current wording
Our primary uses of capital are, and we expect will continue to be, research and development research materials and contracted services, clinical trial costs, compensation and related expenses, laboratory and office facilities, research supplies, legal and regulatory expenses, patent prosecution filing and maintenance costs for our licensed IP, commercial launch capabilities, milestone and royalty payments and general overhead costs. During 2026, we expect our research and development expenses to decrease compared to prior periods as we focus resources on high value programs within our pipeline, such as lonvo-z and nex-z, to ensure efficient execution, achieve near-term clinical milestones, and prepare for commercial launch. We expect the decrease in research and development expenses to be mostly offset by increased expenses in 2026 related to our ongoing buildout of a commercial infrastructure.
Previous wording
Regeneron related to our collaboration agreements with Regeneron. Additionally, we are eligible to earn milestone payments and royalties, in each case, on a per-product basis under our collaboration with ONK, on a per-target basis under our collaboration with Regeneron, and upon achievement of certain events under our collaboration with Kyverna, subject to the provisions of our agreements with each of them. Except for these sources of funding, we will not have any committed external source of liquidity. To the extent that we raise additional capital through the future sale of equity, the ownership interest of our stockholders will be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect the rights of our existing stockholders. If we raise additional funds through collaboration arrangements in the future, we may have to relinquish valuable rights to our technologies, future revenue streams or product candidates or grant licenses on terms that may not be favorable to us. If we are unable to raise additional funds through equity financings when needed, we may be required to delay, limit, reduce or terminate our product development or future commercialization efforts or grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves.
Current wording
Because our lead programs are in the clinical stage and the outcome of these efforts is uncertain, we cannot estimate the actual amounts necessary to successfully complete the development and commercialization of any future product candidates or whether, or when, we may achieve profitability. Until such time as we can generate substantial product revenues, if ever, we expect to fund our ongoing cash needs through financings and collaboration arrangements. We receive cost reimbursements from Regeneron related to our collaboration agreements with Regeneron. Additionally, we are eligible to earn milestone payments and royalties upon achievement of certain events under our collaboration agreements. Except for these sources of funding, we will not have any committed external source of liquidity. To the extent that we raise additional capital through the future sale of equity, the ownership interest of our stockholders will be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect the rights of our existing stockholders. If we raise additional funds through collaboration arrangements in the future, we may have to relinquish valuable rights to our technologies, future revenue streams or product candidates or grant licenses on terms that may not be favorable to us. If we are unable to raise additional funds through financings when needed, we may be required to delay, limit, reduce or terminate our product development or future commercialization efforts or grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves.
Previous wording
Based on our research and development plans and our expectations related to the progress of our programs, we expect that our cash, cash equivalents and marketable securities as of September 30, 2025, as well as research and cost reimbursement funding from our collaboration agreements, will enable us to fund our ongoing operating expenses and capital expenditure requirements into mid-2027 and through the anticipated U.S. commercial launch of lonvo-z for HAE, excluding any potential milestone payments or extension fees that could be earned and distributed under our collaboration agreements or any strategic use of capital not currently in the base case planning assumptions. We have based this estimate on current assumptions that may prove to be wrong, and we could use our capital resources sooner than we expect.
Current wording
Based on our research and development plans and our expectations related to the progress of our programs, we expect that our cash, cash equivalents and marketable securities as of March 31, 2026, the net proceeds from our April 2026 equity offering, as well as research and cost reimbursement funding from our collaboration agreements, will enable us to fund our ongoing operating expenses and capital expenditure requirements at least into 2028, excluding any potential milestone payments or extension fees that could be earned and distributed under our collaboration agreements or any strategic use of capital not currently in the base case planning assumptions. We have based this estimate on current assumptions that may prove to be wrong, and we could use our capital resources sooner than we expect.
Previous wording
Our ability to generate revenue and achieve profitability depends significantly on our success in many areas, including: developing our delivery technologies and our CRISPR/Cas9 technology platform; selecting appropriate product candidates to develop; completing research and preclinical and clinical development of selected product candidates; obtaining regulatory approvals and marketing authorizations for product candidates for which we complete clinical trials; developing a sustainable and scalable manufacturing process for product candidates; launching and commercializing product candidates for which we obtain regulatory approvals and marketing authorizations, either directly or with a collaborator or distributor; obtaining market acceptance of our product candidates; addressing any competing technological and market developments; negotiating favorable terms in any collaboration, licensing, or other arrangements into which we may enter; maintaining good relationships with our collaborators and licensors; maintaining, protecting, and expanding our portfolio of IP rights, including patents, trade secrets, and know-how; and attracting, hiring, and retaining qualified personnel.
Current wording
Our ability to generate revenue and achieve profitability depends significantly on our success in many areas, including: developing our delivery technologies and our CRISPR/Cas9 technology platform; selecting appropriate product candidates to develop; completing research and preclinical and clinical development of selected product candidates; obtaining regulatory approvals and marketing authorizations for product candidates for which we complete clinical trials; developing a sustainable and scalable manufacturing process for product candidates; launching and commercializing product candidates for which we obtain regulatory approvals and marketing authorizations, either directly or with a collaborator or distributor; obtaining market acceptance and pricing approvals for our product candidates; addressing any competing technological and market developments; negotiating favorable terms in any collaboration, licensing, or other arrangements into which we may enter; maintaining good relationships with our collaborators and licensors; maintaining, protecting, and expanding our portfolio of IP rights, including patents, trade secrets, and know-how; and attracting, hiring, and retaining qualified personnel.
Previous wording
Net cash used in operating activities of $263.7 million during the nine months ended September 30, 2024 primarily consists of a net loss of $390.1 million, further reduced by the non-cash recognition of $21.0 million of previously eliminated intra-entity profit recorded within “collaboration revenue” and accretion of investment discounts and premiums of $14.2 million. These decreases are offset in part by stock-based compensation of $114.7 million, $29.5 million in net adjustments to the fair value of our investments in Kyverna and AvenCell, net changes in operating assets and liabilities of $9.8 million and depreciation of $7.7 million.
Current wording
Net cash used in operating activities of $117.3 million during the three months ended March 31, 2026 primarily consists of a net loss of $96.2 million, accretion of investment discounts of $0.5 million and net changes in operating assets and liabilities of $37.2 million, partially offset by stock-based compensation of $13.5 million, depreciation expense of $2.2 million and a change in fair value of our investment in Kyverna Therapeutics, Inc. (“Kyverna”) of $0.9 million.
Previous wording
Net cash used in operating activities of $325.5 million during the nine months ended September 30, 2025 primarily consists of a net loss of $316.9 million, further reduced by accretion of investment discounts of $5.0 million, $2.7 million in net adjustments to the fair value of our investment in Kyverna and net changes in operating assets and liabilities of $74.2 million. Included in the net cash used in operating activities is approximately $65.0 million of non-recurring cash payments associated with our previously announced portfolio prioritization, workforce reduction, and real estate consolidation. These decreases are offset in part by stock-based compensation of $63.5 million, $2.5 million for the accelerated termination of a portion of the 840 Winter Lease and depreciation expense of $7.4 million.
Current wording
Net cash used in operating activities of $148.9 million during the three months ended March 31, 2025 primarily consists of a net loss of $114.3 million, accretion of investment discounts of $2.3 million and net changes in operating assets and liabilities of $58.8 million, partially offset by decreases in stock-based compensation of $21.8 million, depreciation expense of $2.5 million and a change in fair value of our investment in Kyverna of $2.1 million. Included in the net cash used in operating activities was approximately $51.0 million of non-recurring cash payments associated with a previously announced portfolio prioritization, workforce reduction, and real estate consolidation.
Previous wording
During the nine months ended September 30, 2024 our investing activities used cash of $26.0 million. The decrease is primarily due to $21.2 million of marketable securities purchased (net of maturities), and $4.8 million in cash for the purchase of property and equipment.
Current wording
During the three months ended March 31, 2026, our investing activities provided cash of $66.3 million. The increase in the three months ended March 31, 2026 is primarily due to $66.4 million in maturities of marketable securities (net of purchases).
Previous wording
During the nine months ended September 30, 2025, our investing activities provided cash of $200.5 million. The increase in the nine months ended September 30, 2025 is primarily due to $201.5 million in maturities of marketable securities (net of purchases) offset by $1.0 million in cash for the purchase of property and equipment.
Current wording
During the three months ended March 31, 2025 our investing activities provided cash of $94.3 million. The increase is primarily due to $95.1 million of marketable securities maturing (net of purchases), partially offset by $0.7 million in cash for the purchase of property and equipment.
Previous wording
There were no material changes to our contractual obligations during the nine months ended September 30, 2025 other than those described within Note 9, “Leases” in the notes to condensed consolidated financial statements. For a complete discussion of our contractual obligations, please refer to our Management’s Discussion and Analysis of Financial Condition and Results of Operations in our Annual Report for the year ended December 31, 2024.
Current wording
There were no material changes to our contractual obligations during the three months ended March 31, 2026 other than those described within Note 9, “Leases” in the notes to condensed consolidated financial statements. For a complete discussion of our contractual obligations, please refer to our Management’s Discussion and Analysis of Financial Condition and Results of Operations in our Annual Report for the year ended December 31, 2025.
Rearranged previous paragraphs (7)
Previous report — wording carried elsewhere
The following table summarizes our results of operations:
Previous report — wording carried elsewhere
The following table summarizes our research and development expenses, together with the changes in those items in dollars and the respective percentages of change:
Previous report — wording carried elsewhere
The decrease in research and development expenses for the nine months ended September 30, 2025 compared to the nine months ended September 30, 2024 was primarily attributable to:
Previous report — wording carried elsewhere
a $22.7 million decrease in employee-related expenses driven primarily by a workforce reduction in January 2025;
Previous report — wording carried elsewhere
a $20.3 million decrease in research materials and contracted services primarily driven by internal research and development spend, contracted services and drug components;
Previous report — wording carried elsewhere
a $7.2 million increase in facility-related expenses primarily related to rent expense allocated to research and development; and
Previous report — wording carried elsewhere
Because our lead programs are in the clinical stage and the outcome of these efforts is uncertain, we cannot estimate the actual amounts necessary to successfully complete the development and commercialization of any future product candidates or whether, or when, we may achieve profitability. Until such time as we can generate substantial product revenues, if ever, we expect to fund our ongoing cash needs through equity financings and collaboration arrangements. We receive cost reimbursements from