Intellia Therapeutics, Inc.
For the period ended Jun 30, 2025. Filed Aug 7, 2025.
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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.
Jun 30, 2025 · Filed Aug 7, 2025
What changed in Management’s Discussion and Analysis?
Read the current filingPrevious report: 10-Q
Period ended 2025-03-31; filed 2025-05-08.
0000950170-25-066914
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsCurrent report: 10-Q
Period ended 2025-06-30; filed 2025-08-07.
0000950170-25-104987
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 107 previous paragraphs and 117 current paragraphs.
61 unchanged; 34 reworded; 12 added; 12 removed; 0 previous and 10 current paragraphs rearranged.
Tables are not compared: 3 previous and 5 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 (12)
Current report
Lonvo-z Clinical Program
Current 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. The most commonly reported treatment-related adverse events were IRR, which were mild or moderate, and did not result in any discontinuations. Observed liver enzyme abnormalities were not considered serious, were asymptomatic and resolved spontaneously without medical intervention or sequelae.
Current 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. Nex-z was generally well tolerated across all patients and at all dose levels tested. Treatment-related adverse events were consistent with those described for the cardiomyopathy population.
Current report
In September 2025, we will present interim Phase 1 extended data in a symposium at the 5 th International ATTR Amyloidosis Meeting for Patients and Doctors in Baveno, Italy.
Current report
Research and development expenses decreased by $17.2 million during the three months ended June 30, 2025, as compared to the three months ended June 30, 2024.
Current report
a $1.4 million increase in external costs related to the development of lonvo-z, primarily due to an increase in spend on drug components;
Current report
General and administrative expenses decreased by $4.6 million during the three months ended June 30, 2025, as compared to the three months ended June 30, 2024. This 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.
Current report
Comparison of Six Months Ended June 30, 2025 and 2024
Current report
Collaboration revenue decreased by $5.0 million to $30.9 million during the six months ended June 30, 2025, as compared to $35.9 million during the six months ended June 30, 2024. The decrease in collaboration revenue during the six months ended June 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 six months ended June 30, 2024, offset in part by cost reimbursements related to our collaboration with Regeneron in the six months ended June 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.
Current report
Research and development expenses decreased by $20.6 million to $205.5 million during the six months ended June 30, 2025, as compared to $226.1 million during the six months ended June 30, 2024.
Current report
Net cash provided by (used in) investing activities
Current report
Net cash provided by financing activities of $101.2 million during the six months ended June 30, 2024 includes $96.4 million in net proceeds from at-the-market offerings, $3.1 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.
Removed paragraphs (12)
Previous report
NTLA-2002 Clinical Program
Previous report
Collaboration Revenue
Previous report
Research and Development
Previous report
General and Administrative
Previous report
Other Income (Expense), Net
Previous report
Collaboration Revenue
Previous report
Research and Development
Previous report
Research and development expenses decreased by $3.4 million during the three months ended March 31, 2025, as compared to the three months ended March 31, 2024.
Previous report
General and Administrative
Previous report
Other Income (Expense), Net
Previous report
Net cash provided by investing activities
Previous report
There was no cash provided by financing activities during the three months ended March 31, 2025.
Reworded paragraphs (34)
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 successfully complete our global Phase 3 study for ATTR amyloidosis with cardiomyopathy (“ATTR-CM”), the ability to complete our global Phase 3 study for hereditary ATTR amyloidosis with polyneuropathy (“ATTRv-PN”), the ability to file a biologics license application (“BLA”) or comparable marketing application within a certain time period, or the success of such program;
Current 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 successfully complete our global Phase 3 study for ATTR amyloidosis with cardiomyopathy (“ATTR-CM”), the ability to complete our global Phase 3 study for hereditary ATTR amyloidosis with polyneuropathy (“ATTRv-PN”), the ability to file a biologics license application (“BLA”) or comparable marketing application for nex-z for ATTRv-PN by 2028 and potentially launch nex-z in 2029, or the success of such program;
Previous wording
our ability to execute our clinical study strategy for NTLA-2002, our program for the treatment of hereditary angioedema (“HAE”), including the ability to successfully complete our global Phase 3 study, generate results supporting the potential of NTLA-2002 to achieve lifelong control of HAE attacks after a single dose, the ability to file a BLA or comparable marketing application within a certain time period, or the success of such program;
Current 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 Phase 3 study, generate results supporting the potential of lonvo-z to achieve lifelong control of HAE attacks after a single dose, the ability to file a BLA or comparable marketing application in the second half of 2026 and potentially launch lonvo-z in 2027, or the success of such program;
Previous wording
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. Intellia’s deep scientific, technical and clinical development experience, along with its people, is helping set the standard for a new class of medicine. 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 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.
Current wording
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 wording
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. Our deep scientific, technical and clinical development experience have enabled us to develop first-in-class therapeutic applications of CRISPR/Cas9 and our other technologies, opening a new frontier in genetic medicine.
Current wording
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 wording
Our leadin vivo product candidates, nex-z for the treatment of ATTR amyloidosis and NTLA-2002 for the treatment of HAE, 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.
Current wording
Our leadin vivo product candidates, nex-z for the treatment of ATTR amyloidosis and lonvo-z for the treatment of HAE, 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 wording
NTLA-2002 is a wholly owned, investigationalin vivo CRISPR-based therapy designed to knock out thekallikrein B1 (“KLKB1”)gene in the liver, with the goal of achieving lifelong control of HAE attacks after a single dose. 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 therapy designed to knock out thekallikrein B1 (“KLKB1”)gene in the liver, with the goal of lifelong control of HAE attacks after a single dose. It also aims to eliminate the significant treatment burden associated with currently available HAE therapies.
Previous wording
In October 2024, we announced the initiation of the Phase 3 HAELO study of NTLA-2002. HAELO is a global, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of NTLA-2002 in 60 adults with Type I or Type II HAE. Patients will be randomized 2:1 to receive a single 50 mg infusion of NTLA-2002 or placebo. Patients randomized to the placebo arm will be eligible for optional crossover to NTLA-2002 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. We expect to complete enrollment in the third quarter of 2025 and remain on track to submit a biologics license application (“BLA”) in the second half of 2026.
Current 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. Recruitment ended earlier than expected during the second quarter and we now expect to complete randomization during the third quarter of 2025. We are on track to submit a BLA in the second half of 2026 and potentially launch lonvo-z in 2027 in the U.S.
Previous wording
We will present new data from the ongoing Phase 1/2 study in an oral presentation at the European Academy of Allergy and Clinical Immunology (“EAACI”) Congress 2025 on Sunday, June 15 in Glasgow, United Kingdom. The presentation will include longer-term durability data for patients in the Phase 1 part of the Phase 1/2 study.
Current wording
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 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 wording
In addition, we expect to present longer-term data from the ongoing Phase 1/2 study in 2025, which will include patients in the Phase 2 portion who initially received a 25 mg dose or placebo and were subsequently given the 50 mg dose of NTLA-2002 selected for the Phase 3 study.
Current wording
We expect to present additional data from the ongoing Phase 1/2 study in the second half of 2025.
Previous wording
Nex-z is an investigational CRISPR-based therapy designed to inactivate the TTR gene in liver cells, thereby preventing the production of TTR protein for the treatment of ATTR amyloidosis. 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 single dose.
Current wording
Nex-z is an investigationalin vivo CRISPR-based therapy designed to inactivate the TTR gene in liver cells, thereby preventing the production of transthyretin (“TTR”) protein for the treatment of ATTR amyloidosis. 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 single dose.
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. Patients will be randomized 2:1 nex-z:placebo, with a single 55 mg infusion of nex-z administered. The MAGNITUDE trial is currently enrolling and we anticipate enrollment to exceed 550 total patients by the end of 2025.
Current 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. Patients will be randomized 2:1 nex-z:placebo, with a single 55 mg infusion of nex-z administered. Enrollment in the global Phase 3 MAGNITUDE trial is progressing ahead of our projections and we are tracking to enroll at least 650 patients cumulatively by the end of 2025. We are amending the MAGNITUDE study to expand total enrollment to approximately 1,200 patients from 765 patients, subject to health authority review. Expanding the patient number in the study would provide a more robust dataset, particularly in the stabilizer stratum, which we believe will be very important to patients, clinicians, and payers. This change has no expected impact on previously projected enrollment or financial runway. We expect to complete enrollment in MAGNITUDE in early 2027.
Previous wording
We expect to present longer-term data from both ATTR-CM and ATTRv-PN patients in the Phase 1 study in the second half of 2025, which will include updated measures of clinical efficacy and safety.
Current wording
We expect to present longer-term data from ATTR-CM patients in the Phase 1 study in the second half of 2025. The data will include updated measures of clinical efficacy and safety.
Previous 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. We expect enrollment to be completed in 2026.
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. Enrollment is ahead of schedule and we now expect enrollment to be completed in the first half of 2026 to support submission of a BLA or comparable marketing application for nex-z for ATTRv-PN by 2028 and potentially launch nex-z in 2029.
Previous wording
During the three months ended March 31, 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 and six months ended June 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.
Previous wording
Collaboration revenue decreased by $12.3 million during the three months ended March 31, 2025, as compared to the three months ended March 31, 2024. The decrease in collaboration revenue is primarily due to a reduction in revenue under the AvenCell license and collaboration agreement (the “AvenCell LCA”).
Current wording
Collaboration revenue increased by $7.3 million during the three months ended June 30, 2025, as compared to the three months ended June 30, 2024. The increase in collaboration revenue is primarily due to cost reimbursements related to our collaboration with Regeneron.
Previous wording
a $9.5 million increase in external costs related to the development of nex-z, our lead product candidate, primarily due to an increase in spend on contracted services and drug components;
Current wording
a $9.3 million increase in external costs related to the development of nex-z, our lead product candidate, primarily due to an increase in spend on contracted services and drug components;
Previous wording
a $1.7 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;
Current wording
a $1.4 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 wording
a $5.5 million decrease in employee-related expenses driven primarily by a workforce reduction in January 2025, offset in part by severance expense recorded in the first quarter of 2025;
Current wording
a $9.4 million decrease in employee-related expenses driven primarily by a workforce reduction in January 2025;
Previous wording
a $3.1 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
an $8.0 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 $2.9 million increase in facility-related expenses primarily related to rent expense allocated to research and development; and
Current wording
a $2.7 million increase in facility-related expenses primarily related to rent expense allocated to research and development; and
Previous wording
a $7.5 million decrease in stock-based compensation driven by our reduced workforce and lower stock price.
Current wording
an $11.4 million decrease in stock-based compensation driven by our reduced workforce and lower stock price.
Previous wording
The decrease in other income, net of $0.1 million is primarily related to a $4.0 million decrease in interest income, offset in part by a $3.9 million decrease in expense due to the change in fair value of our investments.
Current wording
The increase in other income, net of $16.7 million is primarily related to a $21.7 million decrease in expense due to the change in fair value of our investments, offset in part by a $5.0 million decrease in interest income.
Previous wording
a $1.9 million increase in external costs related to the development of NTLA-2002, primarily due to an increase in spend on drug components and contracted services;
Current wording
a $3.3 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 wording
General and administrative expenses decreased by $2.1 million during the three months ended March 31, 2025, as compared to the three months ended March 31, 2024. This decrease was primarily related to lower employee-related expenses due to a workforce reduction in January 2025 and lower stock-based compensation, partially offset by increases related to severance expenses recorded in the first quarter of 2025.
Current wording
General and administrative expenses decreased by $6.7 million during the six months ended June 30, 2025, as compared to the six months ended June 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 wording
As of March 31, 2025, we had $707.1 million in cash, cash equivalents and marketable securities.
Current wording
As of June 30, 2025, we had $630.5 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 March 31, 2025 we have issued 14,522,533 shares of our common stock under the 2022 Sale Agreement, as amended. During the three months ended March 31, 2025, we did not issue any shares of our common stock under the 2022 Sale Agreement, as amended. As of March 31, 2025, $249.1 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 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 June 30, 2025 we have issued 15,981,790 shares of our common stock under the 2022 Sale Agreement, as amended. During the three and six months ended June 30, 2025, we issued 1,459,257 shares of our common stock, in a series of sales, at an average price of $9.64 per share, in accordance with the 2022 Sale Agreement, as amended, for aggregate net proceeds of $13.7 million, after payment of cash commissions. As of June 30, 2025, $0.3 million of these proceeds are included in “Prepaid expenses and other current assets” on our condensed consolidated balance sheet, representing offerings with trade dates in June 2025 that were settled in July 2025. As of June 30, 2025, $235.1 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 recently announced strategic reorganization in January 2025, as we focus resources on high value programs within our pipeline, such as NTLA-2002 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, 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.
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 March 31, 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 the first half of 2027, 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 June 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 the first half of 2027 and into the anticipated first commercial launch, 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
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, further reduced by accretion of investment discounts of $2.3 million and net changes in operating assets and liabilities of $58.8 million. Included in the net cash used in operating activities is approximately $51.0 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 $21.8 million, $2.1 million in net adjustments to the fair value of our investment in Kyverna and depreciation expense of $2.5 million.
Current wording
Net cash used in operating activities of $248.6 million during the six months ended June 30, 2025 primarily consists of a net loss of $215.6 million, further reduced by accretion of investment discounts of $3.9 million and net changes in operating assets and liabilities of $78.7 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 $43.9 million, $0.8 million in net adjustments to the fair value of our investment in Kyverna and depreciation expense of $5.0 million.
Previous wording
Net cash used in operating activities of $120.7 million during the three months ended March 31, 2024 primarily consists of a net loss of $107.4 million, further reduced by changes in operating assets and liabilities of $29.7 million, including the receipt of $5.9 million in payments from our collaboration partners during that period, and the non-cash recognition of $21.0 million of previously eliminated intra-entity profit recorded within “collaboration revenue,” offset in part by non-cash charges of $6.1 million of net adjustments to the fair value of our investments in Kyverna and AvenCell, stock-based compensation of $34.2 million and depreciation expense of $2.5 million.
Current wording
Net cash used in operating activities of $178.8 million during the six months ended June 30, 2024 primarily consists of a net loss of $254.4 million, further reduced by the non-cash recognition of $21.0 million of previously eliminated intra-entity profit recorded within “collaboration revenue,” accretion of investment discounts and premiums of $9.9 million and net changes in operating assets and liabilities of $0.1 million. These decreases are offset in part by stock-based compensation of $75.0 million, $26.4 million in net adjustments to the fair value of our investments in Kyverna and AvenCell and depreciation of $5.1 million.
Previous wording
During the three months ended March 31, 2025, our investing activities provided cash of $94.3 million. The increase in the three months ended March 31, 2025 is primarily due to $95.1 million in maturities of marketable securities (net of purchases) offset by $0.7 million in cash for the purchase of property and equipment.
Current wording
During the six months ended June 30, 2025, our investing activities provided cash of $201.2 million. The increase in the six months ended June 30, 2025 is primarily due to $202.2 million in maturities of marketable securities (net of purchases) offset by $1.0 million in cash for the purchase of property and equipment.
Previous wording
During the three months ended March 31, 2024 our investing activities provided cash of $5.9 million. The increase is primarily due to $8.4 million of marketable securities maturing (net of purchases), offset in part by $2.5 million in cash for the purchase of property and equipment.
Current wording
During the six months ended June 30, 2024 our investing activities used cash of $18.3 million. The decrease is primarily due to $14.8 million of marketable securities purchased (net of maturities), and $3.5 million in cash for the purchase of property and equipment.
Previous wording
Net cash provided by financing activities of $60.0 million during the three months ended March 31, 2024 includes $58.0 million in net proceeds from at-the-market offerings and $2.0 million in cash received from the exercise of stock options.
Current wording
Net cash provided by financing activities of $14.7 million during the six months ended June 30, 2025 includes $13.4 million in net proceeds from at-the-market offerings and $1.2 million in cash received from the issuance of shares through our employee stock purchase plan.
Previous wording
There were no material changes to our contractual obligations during the three months ended March 31, 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 six months ended June 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.
Rearranged current paragraphs (10)
Current report — wording carried from elsewhere
The following table summarizes our results of operations:
Current report — wording carried from 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:
Current report — wording carried from elsewhere
The decrease in research and development expenses for the six months ended June 30, 2025 compared to the six months ended June 30, 2024 was primarily attributable to:
Current report — wording carried from elsewhere
an $18.7 million increase in external costs related to the development of nex-z, our lead product candidate, primarily due to an increase in spend on contracted services and drug components;
Current report — wording carried from elsewhere
a $3.1 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;
Current report — wording carried from elsewhere
a $15.0 million decrease in employee-related expenses driven primarily by a workforce reduction in January 2025;
Current report — wording carried from elsewhere
an $11.1 million decrease in research materials and contracted services primarily driven by internal research and development spend, contracted services and drug components;
Current report — wording carried from elsewhere
a $5.6 million increase in facility-related expenses primarily related to rent expense allocated to research and development; and
Current report — wording carried from elsewhere
an $18.9 million decrease in stock-based compensation driven by our reduced workforce and lower stock price.
Current report — wording carried from elsewhere
The increase in other income, net of $16.6 million is primarily related to a $25.6 million decrease in expense due to the change in fair value of our investments, offset in part by a $9.0 million decrease in interest income.