Intellia Therapeutics, Inc.
For the period ended Sep 30, 2025. Filed Nov 6, 2025.
View on SEC.govAbout this filing
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.
Sep 30, 2025 · Filed Nov 6, 2025
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 2025-06-30; filed 2025-08-07.
0000950170-25-104987
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsCurrent 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 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 117 previous paragraphs and 126 current paragraphs.
65 unchanged; 45 reworded; 15 added; 6 removed; 1 previous and 1 current paragraphs rearranged.
Tables are not compared: 5 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 (15)
Current 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.
Current report
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 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.
Current 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.
Current 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.
Current 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.
Current 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.
Current report
Other Income (Expense), Net
Current 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.
Current 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;
Current 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.
Current report
Other Income, Net
Current report
Comparison of Nine Months Ended September 30, 2025 and 2024
Current 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.
Current report
Other Income, Net
Removed paragraphs (6)
Previous report
We expect to present additional data from the ongoing Phase 1/2 study in the second half of 2025.
Previous report
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 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.
Previous report
Comparison of Six Months Ended June 30, 2025 and 2024
Previous 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.
Previous report
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;
Reworded paragraphs (45)
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 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;
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 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;
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 for nex-z for ATTRv-PN by 2028 and potentially launch nex-z in 2029, 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 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;
Previous wording
Our leadin vivo programs are the only Phase 3 genome editing product candidates designed to address unmet need in patients with ATTR amyloidosis and HAE. 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
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.
Previous 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.
Current wording
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 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.
Current 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.
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. 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.
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. 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.
Previous 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.
Current 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.
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. 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.
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. 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.
Previous wording
In March 2025, we announced that the U.S. Food and Drug Administration (“FDA”) granted Regenerative Medicine Advanced Therapy (“RMAT”) designation to nex-z for the treatment of ATTR-CM.
Current wording
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 wording
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 wording
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 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.
Current 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.
Previous wording
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 wording
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 wording
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 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.
Previous wording
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. We have existing collaboration agreements with Regeneron, AvenCell Therapeutics, Inc. (“AvenCell”), SparingVision SAS (“SparingVision”), Kyverna Therapeutics, Inc. (“Kyverna”), ONK Therapeutics, Ltd. (“ONK”) and ReCode Therapeutics, Inc. (“ReCode”). 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 wording
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 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.
Current 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.
Previous 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.
Current wording
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 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;
Current 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;
Previous 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;
Current wording
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 wording
a $9.4 million decrease in employee-related expenses driven primarily by a workforce reduction in January 2025;
Current wording
a $7.7 million decrease in employee-related expenses driven primarily by a workforce reduction in January 2025;
Previous 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;
Current 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;
Previous wording
a $2.7 million increase in facility-related expenses primarily related to rent expense allocated to research and development; and
Current wording
a $1.6 million increase in facility-related expenses primarily related to rent expense allocated to research and development; and
Previous wording
an $11.4 million decrease in stock-based compensation driven by our reduced workforce and lower stock price.
Current wording
a $12.0 million decrease in stock-based compensation driven by our reduced workforce and lower stock price.
Previous 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.
Current 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.
Previous wording
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 wording
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 wording
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 wording
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 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;
Current 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;
Previous wording
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 wording
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 wording
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 wording
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 wording
a $15.0 million decrease in employee-related expenses driven primarily by a workforce reduction in January 2025;
Current wording
a $22.7 million decrease in employee-related expenses driven primarily by a workforce reduction in January 2025;
Previous wording
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 wording
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 wording
a $5.6 million increase in facility-related expenses primarily related to rent expense allocated to research and development; and
Current wording
a $7.2 million increase in facility-related expenses primarily related to rent expense allocated to research and development; and
Previous wording
an $18.9 million decrease in stock-based compensation driven by our reduced workforce and lower stock price.
Current wording
a $30.9 million decrease in stock-based compensation driven by our reduced workforce and lower stock price.
Previous 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.
Current wording
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 wording
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.
Current wording
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 wording
As of June 30, 2025, we had $630.5 million in cash, cash equivalents and marketable securities.
Current wording
As of September 30, 2025, we had $669.9 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 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.
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 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.
Previous 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 equity financings and collaboration arrangements. We receive cost reimbursements from Regeneron related to our collaboration agreements with them. Additionally, we are eligible to earn milestone payments and royalties, in each case, on a per-product basis under our collaborations with SparingVision, ONK and ReCode, on a per-target basis under our collaboration with Regeneron, and upon achievement of certain events 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
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.
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 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.
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 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.
Previous 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.
Current 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.
Previous 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.
Current 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.
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Current report — wording carried from 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
Rearranged previous paragraphs (1)
Previous report — wording carried elsewhere
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.