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Beam Therapeutics Inc.

For the period ended Sep 30, 2026. Filed Nov 3, 2026.

View on SEC.gov

About 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, 2026 · Filed Nov 3, 2026

BEAM$26.41+2.1%Stored close · as of Nov 6, 2026

What changed in Management’s Discussion and Analysis?

Read the current filing

Previous report: 10-Q

Period ended 2026-06-30; filed 2026-08-04.

0001193125-26-331541

Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations

Current report: 10-Q

Period ended 2026-09-30; filed 2026-11-03.

0001193125-26-444455

Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations

The 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 201 previous paragraphs and 202 current paragraphs.

121 unchanged; 56 reworded; 20 added; 19 removed; 5 previous and 5 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 (20)

Current report

In September 2026, we announced updated safety and efficacy data from the trial. The data included 29 patients treated with a single dose of BEAM-302 in the dose-escalation portion of the trial, including Part A (three patients treated with 15 mg, three patients treated with 30 mg, six patients treated with 60 mg, and nine patients treated with 75 mg) and Part B (three patients treated with 30 mg and five patients treated with 60 mg). Data from patients in the multi-dose cohort (three patients) and the Part A expansion cohort (six patients) were not included in this data. As of the June 24, 2026, data cutoff date, treatment with single-dose BEAM-302 was well tolerated with an acceptable safety profile in both Parts A and B. Mild-to-moderate infusion-related reactions, or IRRs, were the most common drug-related treatment-emergent adverse event, or TEAE, occurring in 41% of all patients. Transient and predominantly Grade 1 alanine transaminase, or ALT, and/or aspartate aminotransferase, or AST, elevations were also observed. One Part B patient with underlying AATD-related liver disease treated with 60 mg of BEAM-302 experienced transient Grade 3 ALT/AST elevations that were not accompanied by bilirubin increases and did not require treatment.

Current report

Increased total AAT in circulation was functional. Functional AAT levels increased in a dose-dependent manner up to 60 mg and were durable through follow-up.

Current report

Human neutrophil elastase activity levels, another measure of AAT function, decreased in a dose proportionate manner with BEAM-302 treatment, with more than 80% of patients in the Part A 60 mg cohort having at least one human neutrophil elastase activity measurement below the lower limit of quantification.

Current report

Mutant Z-AAT was durably and significantly reduced after treatment with BEAM-302, with an 84% reduction in circulating Z-AAT in both Part A and Part B 60 mg cohorts.

Current report

BEAM-302 also led to a significant decrease in circulating Z-AAT protein aggregates, referred to as Z-polymers, from baseline in the 60 mg cohort. Circulating Z-polymers have been shown to correlate with liver disease severity and to amplify lung inflammation by inducing neutrophil recruitment.

Current report

Evidence of AAT protein production under normal physiologic control was observed during an upper respiratory tract infection in a patient in the Part A 60 mg cohort.

Current report

In October 2026, we announced initial safety and efficacy data from the trial. As of the August 17, 2026 data cutoff date, six patients had been treated with a single intravenous infusion of BEAM-301, including three patients treated with 20 mg and three patients treated with 40 mg. BEAM-301 demonstrated an acceptable safety profile that was consistent with known risks associated with LNP-based therapies, with no dose-limiting toxicities, Grade 4 or 5 TEAEs or treatment discontinuations due to TEAEs reported. Dose-dependent improvements in time to fasting hypoglycemia were observed. Median time to fasting hypoglycemia increased from 2.9 hours at baseline to 4.3 hours at Day 36 in the 20 mg cohort, and from 2.5 hours at baseline to 15.0 hours at Day 36 in the 40 mg cohort. Two of three patients in the 40 mg cohort achieved the protocol-defined maximum controlled fasting duration of 15 hours, consistent with restored endogenous glucose production. During the 15-hour fasting challenges, blood glucose levels were generally maintained within the euglycemic range. The patient in the 40 mg cohort who did not reach the 15-hour maximum was compound heterozygous for R83C and had a higher body mass index. Reductions in prescribed daily starch intake were also observed following treatment with BEAM-301, with two of three patients in the 40 mg cohort achieving a 100% reduction in daily starch requirements and no longer requiring starch supplementation at their latest reported assessments.

Current report

Based on the safety and clinical findings observed to date, we are enrolling patients in a 60 mg cohort of the ongoing open-label Phase 1/2 dose-exploration study and plan to engage with the FDA regarding a potential path to a regulatory approval submission.

Current report

A decrease of $14.5 million of in-process research and development expenses;

Current report

A decrease of $3.0 million in stock-based compensation driven by the expense recognized on additional one-time stock awards granted in prior years for which there was no equivalent expense in the three months ended September 30, 2026.

Current report

General and administrative expenses were $31.5 million and $26.7 million for the three months ended September 30, 2026 and 2025, respectively. The increase of $4.8 million was primarily due to the following:

Current report

Comparison of the nine months ended September 30, 2026 and 2025

Current report

The decrease of $0.6 million was primarily due to the following:

Current report

A decrease of $14.5 million of in-process research and development expenses; and

Current report

A decrease of $11.3 million in stock-based compensation driven by the expense recognized on additional one-time stock awards granted in prior years for which there was no equivalent expense in the nine months ended September 30, 2026.

Current report

General and administrative expenses were $97.9 million and $81.5 million for the nine months ended September 30, 2026 and 2025, respectively. The increase of $16.4 million was primarily due to the following:

Current report

A decrease of $5.6 million in stock-based compensation driven by the expense recognized on additional one-time stock awards granted in prior years for which there was no equivalent expense in the nine months ended September 30, 2026.
Removed paragraphs (19)

Previous report

In March 2026, we announced updated safety and efficacy data from the trial. As of the February 10, 2026 data cutoff date, 29 patients had been treated with BEAM-302 in Part A (15 mg, n=3; 30 mg, n=3; 60 mg, n=6; 75 mg, n=9; 2x 60 mg, n=3) and Part B (30 mg, n=3; 60 mg, n=2) and followed for up to 18 months.

Previous report

Data from 26 patients treated with a single-dose of BEAM-302 support a well-tolerated safety profile up to 75 mg that is consistent across Part A and Part B. Adverse events, or AEs, were mild to moderate, with no serious AEs reported and no dose-limiting toxicities as of the data cutoff. Transient Grade 1 and Grade 2 infusion-related reactions, or IRRs, and Grade 1 asymptomatic alanine transaminase, or ALT, and aspartate aminotransferase, or AST, elevations were observed. In the multi-dose cohort (n=3), following the second dose of BEAM-302, patients experienced Grade 2 IRRs, one patient had Grade 4 ALT and Grade 3 AST elevations, and one patient had a Grade 2 ALT elevation. All ALT/AST elevations were asymptomatic and did not require treatment. No bilirubin increases were observed in any patient.

Previous report

After treatment with a single dose of BEAM-302 in Part A, the steady-state 2 circulating total AAT mean (LC-MS)3 was 16.1 µM in the 60 mg cohort (follow-up ranging from 5-12 months) and 14.4 µM in the 75 mg cohort (follow-up ranging from 2-9 months). In the multi-dose cohort, patients achieved a mean of 16.5 µM total AAT at Day 84, 28 days after the second 60 mg dose.

Previous report

Across all cohorts, increased total AAT in circulation was functional as demonstrated by a neutrophil elastase inhibition assay.

Previous report

Mutant Z-AAT was durably and significantly reduced after treatment with BEAM-302. The steady-state mean reduction in Z-AAT was 84% in the 60 mg cohort and 79% in the 75 mg cohort. In the multi-dose cohort, the mean reduction in Z-AAT was 80% at Day 84.

Previous report

Evidence of dynamic induction of AAT expression was observed during a respiratory infection around Month 8 in a patient in the 60 mg Part A cohort. During the infection, total AAT levels increased from steady-state levels of 15.9 µM to 29.5 µM while maintaining consistent AAT composition of 95% M-AAT.

Previous report

In Part B patients with AATD-associated liver disease, single doses of 30 mg and 60 mg BEAM-302 demonstrated consistent efficacy comparable to results observed in Part A patients without liver disease.

Previous report

1 One patient dosed at 60 mg in Part B was not efficacy evaluable at the time of data cutoff.

Previous report

3 Circulating AAT levels were measured using a liquid chromatography–mass spectrometry, or LC–MS, assay. LC-MS is a quantitative method preferred by regulatory authorities to assess specificity of AAT detection.

Previous report

The decrease of $6.7 million was primarily due to the following:

Previous report

A decrease of $3.6 million in stock-based compensation driven by the expense recognized on additional one-time stock awards granted in prior years for which there was no equivalent expense in the three months ended June 30, 2026.

Previous report

General and administrative expenses were $31.9 million and $26.9 million for the three months ended June 30, 2026 and 2025, respectively. The increase of $5.1 million was primarily due to the following:

Previous report

A decrease of $1.5 million in stock-based compensation driven by the expense recognized on additional one-time stock awards granted in prior years for which there was no equivalent expense in the three months ended June 30, 2026.

Previous report

The decrease of $1.0 million was primarily due to the following:

Previous report

A decrease of $8.3 million in stock-based compensation driven by the expense recognized on additional one-time stock awards granted in prior years for which there was no equivalent expense in the six months ended June 30, 2026.

Previous report

General and administrative expenses were $66.4 million and $54.8 million for the six months ended June 30, 2026 and 2025, respectively. The increase of $11.6 million was primarily due to the following:
Reworded paragraphs (56)

Previous wording

BEAM-302 is a liver-targeting LNP formulation of base editing reagents designed to offer a one-time treatment to correct the E342K point mutation (PiZZ genotype) predominantly responsible for the severe form of alpha-1 antitrypsin deficiency, or AATD. AATD is an inherited genetic disorder that can cause early onset emphysema and liver disease. The most severe form of AATD arises when a patient has a point mutation in both copies of the SERPINA1 gene at amino acid 342 position (E342K, also known as the PiZ mutation or the “Z” allele). This point mutation causes Alpha-1 antitrypsin, or AAT, protein to misfold, accumulating inside liver cells rather than being secreted, resulting in very low levels (10%-15%) of circulating AAT. In addition to resulting in lower levels, the PiZ AAT protein variant is also less enzymatically effective compared to wildtype AAT protein. As a consequence, the lung is left unprotected from neutrophil elastase, resulting in progressive, destructive changes in the lung, such as emphysema, which can result in the need for lung transplants. The mutant AAT protein also accumulates in the liver, causing liver inflammation and cirrhosis, which can ultimately cause liver failure or cancer requiring patients to undergo a liver transplant. It is estimated that approximately 100,000 individuals in the United States have two copies of the Z allele. There are currently no curative treatments for patients with AATD.

Current wording

BEAM-302 is an investigational liver-targeting LNP formulation of base editing reagents designed to offer a one-time treatment to correct the E342K point mutation (PiZZ genotype) predominantly responsible for the severe form of alpha-1 antitrypsin deficiency, or AATD. AATD is an inherited genetic disorder that can cause early onset emphysema and liver disease. The most severe form of AATD arises when a patient has a point mutation in both copies of the SERPINA1 gene at amino acid 342 position (E342K, also known as the PiZ mutation or the “Z” allele). This point mutation causes Alpha-1 antitrypsin, or AAT, protein to misfold, accumulating inside liver cells rather than being secreted, resulting in very low levels (10%-15%) of circulating AAT. In addition to resulting in lower levels, the PiZ AAT protein variant is also less enzymatically effective compared to wildtype AAT protein. As a consequence, the lung is left unprotected from neutrophil elastase, resulting in progressive, destructive changes in the lung, such as emphysema, which can result in the need for lung transplants. The mutant AAT protein also accumulates in the liver, causing liver inflammation and cirrhosis, which can ultimately cause liver failure or cancer requiring patients to undergo a liver transplant. It is estimated that approximately 100,000 individuals in the United States have two copies of the Z allele. There are currently no curative treatments for patients with AATD.

Previous wording

We are conducting a Phase 1/2 open label, dose exploration and dose expansion clinical trial of BEAM-302 for the treatment of AATD. The trial will evaluate the safety, tolerability, pharmacodynamics, pharmacokinetics and efficacy of BEAM-302. Part A of the trial is designed to evaluate AATD patients with lung disease, and Part B will evaluate AATD patients with mild to moderate liver disease with or without lung disease.

Current wording

We are conducting a Phase 1/2 open label, dose exploration and dose expansion clinical trial of BEAM-302 for the treatment of AATD. The trial will evaluate the safety, tolerability, pharmacodynamics, pharmacokinetics and efficacy of BEAM-302. Part A of the trial is designed to evaluate AATD patients with lung disease, and Part B is designed to evaluate AATD patients with mild to severe liver disease with or without lung disease. As of August 17, 2026, 38 patients had been treated with BEAM-302 across Parts A and B.

Previous wording

Treatment with BEAM-302 led to rapid and durable increases of total and functional AAT, decreases in mutant Z-AAT, and new production of corrected M-AAT. Key data from 28 efficacy evaluable patients 1 include the following:

Current wording

Treatment with BEAM-302 led to rapid and durable increases of total and functional AAT, decreases in mutant Z-AAT, and new production of corrected M-AAT. Data was consistent with the topline results announced in March 2026. Key data from the 29 efficacy evaluable patients as of the June 24, 2026, data cutoff date include the following:

Previous wording

2 Steady state is defined as the period beginning on a patient's Day 28 visit and lasting until that patient's Month 12 visit (or until that patient's last visit, if earlier than twelve months).

Current wording

A single dose of 60 mg of BEAM-302 led to sustained increases in total AAT above the 11 µM protective threshold. After treatment with a single dose of BEAM-302 in Part A, the steady-state circulating total AAT mean and median were 14.4 µM and 15.2 µM, respectively, compared to 5.0 µM at baseline. In the Part B 60 mg cohort, the post-treatment steady-state circulating total AAT mean and median were 13.5 µM and 13.8 µM, respectively, compared to 4.7 µM at baseline. Steady state is defined as the period beginning on a patient’s day 28 visit and lasting until that patient’s last visit. Circulating total AAT levels were measured using turbidimetry.

Previous wording

Following treatment with BEAM-302, newly produced corrected M-AAT comprised the majority of AAT in circulation. The steady-state mean proportion of M-AAT was 94% in the 60 mg cohort and 91% in the 75 mg cohort. In the multi-dose cohort, the mean proportion of M-AAT was 93% at Day 84.

Current wording

Following treatment with BEAM-302, newly produced corrected M-AAT comprised the majority of AAT in circulation. The proportion of M-AAT at steady-state was 93% following treatment with 60 mg in both Part A and Part B, exceeding the approximately 80% M-AAT proportion associated with the MZ genotype.

Previous wording

Based on feedback from the FDA, we intend to pursue an accelerated approval pathway for BEAM-302 based on a primary endpoint of AAT biomarkers evaluated over 12 months, with 60 mg as the selected dose. To support a future BLA submission, we anticipate enrolling approximately 50 additional patients with AATD-associated lung disease, with or without liver disease, in a pivotal expansion of the ongoing open-label Phase 1/2 trial. In July of 2026, we dosed the first patient in the pivotal cohort.

Current wording

Based on feedback from the FDA, we intend to pursue an accelerated approval pathway for BEAM-302 based on a primary endpoint of AAT biomarkers evaluated over 12 months, with 60 mg as the selected dose. To support a future BLA submission, we anticipate enrolling approximately 50 additional patients with AATD-associated lung disease, with or without liver disease, in an expansion of the ongoing open-label Phase 1/2 trial. Enrollment and dosing of patients in this global pivotal cohort are ongoing and we expect to complete dosing in this pivotal cohort in the first half of 2027.

Previous wording

BEAM-304 is a liver-targeting LNP formulation of base editing reagents designed to correct disease-causing mutations responsible for phenylketonuria, or PKU. PKU is an autosomal recessive disorder caused by mutations in the phenylalanine hydroxylase, or PAH, gene that prevents the body from metabolizing the amino acid phenylalanine, or Phe. Elevated levels of Phe may result in severe neurological and neurocognitive impairments. Patients are generally identified via newborn screening, with the standard of care involving a Phe-restricted diet, as well as medicines that manage Phe levels. Initially, we plan to develop BEAM-304 for the treatment of the two most prevalent variants found in PKU patients in the United States, with ongoing research effort to address the majority of the remaining mutations. In preclinical studies, administration of BEAM-304 resulted in the normalization of Phe levels in mice at therapeutically relevant doses, even when consuming a standard diet. In June of 2026, we announced that the FDA cleared our investigational new drug (IND) application for BEAM-304, and we have initiated clinical start-up activities for an open-label, dose-ascending, Phase 1/2 trial. The trial will initially evaluate safety, tolerability, and reduction of blood Phe levels in PKU patients with the R408W mutation, one of the most prevalent disease-causing mutations among patients with PKU in the United States, with a goal of establishing clinical proof of concept for base editing in PKU. We believe that learnings from this trial have the potential to provide a predictable path to accelerated development of BEAM-304 for additional mutations, including as a result of novel FDA frameworks for platform medicines.

Current wording

BEAM-304 is an investigational liver-targeting LNP formulation of base editing reagents designed to correct disease-causing mutations responsible for phenylketonuria, or PKU. PKU is an autosomal recessive disorder caused by mutations in the phenylalanine hydroxylase, or PAH, gene that prevents the body from metabolizing the amino acid phenylalanine, or Phe. Elevated levels of Phe may result in severe neurological and neurocognitive impairments. Patients are generally identified via newborn screening, with the standard of care involving a Phe-restricted diet, as well as medicines that manage Phe levels. Initially, we plan to develop BEAM-304 for the treatment of the two most prevalent variants found in PKU patients in the United States, with ongoing research effort to address the majority of the remaining mutations. In preclinical studies, administration of BEAM-304 resulted in the normalization of Phe levels in mice at therapeutically relevant doses, even when consuming a standard diet. In June 2026, we announced that the FDA cleared our investigational new drug, or IND, application for BEAM-304, and we have initiated clinical start-up activities for an open-label, dose-ascending, Phase 1/2 trial. The trial will initially evaluate safety, tolerability, and reduction of blood Phe levels in PKU patients with the R408W mutation, one of the most prevalent disease-causing mutations among patients with PKU in the United States, with a goal of establishing clinical proof of concept for base editing in PKU. We believe that learnings from this trial have the potential to provide a predictable path to accelerated development of BEAM-304 for additional mutations, including as a result of novel FDA frameworks for platform medicines. In October 2026, we nominated a third development candidate within the BEAM-304 program, targeting a third undisclosed PKU mutation. We are engaged in clinical start-up activities for the planned Phase 1/2 trial of BEAM-304 and we expect to initiate dosing in the Phase 1/2 trial in early 2027.

Previous wording

BEAM-301 is a liver-targeting LNP formulation of base editing reagents designed to correct the R83C mutation, the most prevalent disease-causing mutation for, and the mutation which results in the most severe form of, glycogen storage disease Ia, or GSDIa. GSDIa is an autosomal recessive disorder caused by mutations in the G6PC gene that disrupts a key enzyme, G6Pase, critical for maintaining glucose homeostasis. Inhibition of G6Pase activity results in low fasting blood glucose levels that can result in seizures and be fatal. Patients with this mutation typically require ongoing corn starch administration, without which they may enter into hypoglycemic shock within one to three hours.

Current wording

BEAM-301 is an investigational liver-targeting LNP formulation of base editing reagents designed to correct the R83C mutation, the most prevalent disease-causing mutation for, and the mutation which results in the most severe form of, glycogen storage disease Ia, or GSDIa. GSDIa is an autosomal recessive disorder caused by mutations in the G6PC gene that disrupts a key enzyme, G6Pase, critical for maintaining glucose homeostasis. Inhibition of G6Pase activity results in low fasting blood glucose levels that can result in seizures and be fatal. Patients with this mutation typically require ongoing corn starch administration, without which they may enter into hypoglycemic shock within one to three hours.

Previous wording

We are conducting a Phase 1/2 clinical trial of BEAM-301 at a select number of sites in the United States. The trial is an open-label, multi-cohort, single-ascending dose evaluation of BEAM-301 for the treatment of GSDIa in patients with the R83C mutation. Key endpoints of the trial include safety and tolerability, time to hypoglycemia during fasting, and changes from baseline in corn starch supplementation. Dosing is complete in the first cohort, and dosing has been initiated in the second cohort. We expect to report initial data from the trial in 2026.

Current wording

We are conducting a Phase 1/2 clinical trial of BEAM-301 at a select number of sites in the United States. The trial is an open-label, multi-cohort, single-ascending dose evaluation of BEAM-301 for the treatment of GSDIa in patients with the R83C mutation. Key endpoints of the trial include safety and tolerability, time to hypoglycemia during fasting, and changes from baseline in corn starch supplementation.

Previous wording

We were party to a research collaboration agreement, or the Apellis Agreement, with Apellis Pharmaceuticals, Inc., or Apellis, focused on the use of our base editing technology to discover new treatments for complement system-driven diseases. Under the terms of the Apellis Agreement, we conducted preclinical research on six base editing programs that target specific genes within the complement system in various organs, including the eye, liver, and brain. Apellis had an exclusive option to license any or all of the six programs and would assume responsibility for subsequent development. In 2025, Apellis notified us of its decision to opt-in to the base editing program directed to FcRN. As a result of Apellis' decision to opt-in to the program, we received a cash opt-in fee of $3.8 million during the year ended December 31, 2025. We may elect to enter into a 50-50 U.S. co-development and co-commercialization agreement with Apellis with respect to one program licensed under the collaboration. In May 2026, Biogen Inc. announced that it had completed its acquisition of Apellis.

Current wording

We are party to a research collaboration agreement, or the Apellis Agreement, with Apellis Pharmaceuticals, Inc., or Apellis, which was acquired by Biogen Inc., or Biogen, in May 2026. The Apellis Agreement is focused on the use of our base editing technology to discover new treatments for complement system-driven diseases. Under the terms of the Apellis Agreement, we conducted preclinical research on six base editing programs that target specific genes within the complement system in various organs, including the eye, liver, and brain. Apellis had an exclusive option to license any or all of the six programs and would assume responsibility for subsequent development. In 2025, Apellis notified us of its decision to opt-in to the base editing program directed to FcRN. As a result of Apellis’ decision to opt-in to the program, we received a cash opt-in fee of $3.8 million during the year ended December 31, 2025. In September 2026, Biogen notified us of its decision to terminate its rights with respect to the FcRN base editing program, effective December 2026.

Previous wording

We are party to a license agreement, or the Verve Agreement, with Verve Therapeutics, Inc., or Verve, pursuant to which we granted Verve exclusive worldwide licenses under our base editing technologies for human therapeutic applications against a total of three liver-mediated, cardiovascular disease targets, which consist of PCSK9, ANGPTL3 and an undisclosed target. In October 2023, we entered into a transfer and delegation agreement, or the Lilly Agreement, with Eli Lilly and Company, or Lilly, pursuant to which Lilly acquired certain assets and other rights under the Verve Agreement, including our opt-in rights to co-develop and co-commercialize each of Verve’s base editing programs. In addition, Lilly acquired the right to receive any future milestone or royalty payments payable to us under the Verve Agreement. Under the terms of the Lilly Agreement, we received a $200.0 million payment and are eligible to receive up to $350.0 million in potential future development-stage payments upon the completion of certain clinical, regulatory and alliance events, of which $50.0 million has been received through June 30, 2026. The Company recognized $25.0 million of revenue related to the achievement of a milestone event during the six months ended June 30, 2026.

Current wording

We are party to a license agreement, or the Verve Agreement, with Verve Therapeutics, Inc., or Verve, pursuant to which we granted Verve exclusive worldwide licenses under our base editing technologies for human therapeutic applications against a total of three liver-mediated, cardiovascular disease targets, which consist of PCSK9, ANGPTL3 and an undisclosed target. In October 2023, we entered into a transfer and delegation agreement, or the Lilly Agreement, with Eli Lilly and Company, or Lilly, pursuant to which Lilly acquired certain assets and other rights under the Verve Agreement, including our opt-in rights to co-develop and co-commercialize each of Verve’s base editing programs. In addition, Lilly acquired the right to receive any future milestone or royalty payments payable to us under the Verve Agreement. Under the terms of the Lilly Agreement, we received a $200.0 million payment and are eligible to receive up to $350.0 million in potential future development-stage payments upon the completion of certain clinical, regulatory and alliance events, of which $50.0 million has been received through September 30, 2026. We recognized $25.0 million of revenue related to the achievement of a milestone event during the nine months ended September 30, 2026.

Previous wording

In December 2025, Bristol-Myers Squibb Company completed an acquisition of Orbital, or the Acquisition. At the closing of the Acquisition, we received $255.1 million in closing cash consideration, plus the right to receive up to approximately $26.3 million in additional cash consideration upon the release, if any, of certain escrows. During the three months ended June 30, 2026, we received an additional $0.5 million related to the release of a portion of the escrow associated with the Acquisition. We may receive additional cash consideration in future periods upon the release of the remaining escrow amounts, if any, in accordance with the terms of the Acquisition.

Current wording

In December 2025, Bristol-Myers Squibb Company completed an acquisition of Orbital, or the Acquisition. At the closing of the Acquisition, we received $255.1 million in closing cash consideration, plus the right to receive up to approximately $26.3 million in additional cash consideration upon the release, if any, of certain escrows. During the nine months ended September 30, 2026, we received an additional $0.5 million related to the release of a portion of the escrow associated with the Acquisition. We may receive additional cash consideration in future periods upon the release of the remaining escrow amounts, if any, in accordance with the terms of the Acquisition.

Previous wording

We are a clinical-stage company, and our programs are at a preclinical or clinical stage of development. To date, we have not generated any revenue from product sales and do not expect to generate revenue from the sale of products in the near future. Our revenue to date has been primarily derived from license and collaboration agreements with partners. Since inception we have incurred significant operating losses. Our net losses for the six months ended June 30, 2026 and 2025 were $217.0 million and $210.5 million, respectively. As of June 30, 2026, we had an accumulated deficit of $1.9 billion. We expect to continue to incur significant expenses and increasing operating losses in connection with ongoing development activities related to our internal programs and collaborations as we continue our preclinical and clinical development of product candidates; advance additional product candidates toward clinical development; operate our cGMP facility in North Carolina; further develop our base editing platform; continue to make investments in delivery technology for our base editors; conduct research activities as we seek to discover and develop additional product candidates; maintain, expand, enforce, defend and protect our intellectual property portfolio; and continue to hire research and development, clinical, technical operations and commercial personnel. In addition, we expect to continue to incur the costs associated with operating as a public company.

Current wording

We are a clinical-stage company, and our programs are at a preclinical or clinical stage of development. To date, we have not generated any revenue from product sales and do not expect to generate revenue from the sale of products in the near future. Our revenue to date has been primarily derived from license and collaboration agreements with partners. Since inception we have incurred significant operating losses. Our net losses for the nine months ended September 30, 2026 and 2025 were $351.4 million and $323.0 million, respectively. As of September 30, 2026, we had an accumulated deficit of $2.0 billion. We expect to continue to incur significant expenses and increasing operating losses in connection with ongoing development activities related to our internal programs and collaborations as we continue our preclinical and clinical development of product candidates; advance additional product candidates toward clinical development; operate our cGMP facility in North Carolina; further develop our base editing platform; continue to make investments in delivery technology for our base editors; conduct research activities as we seek to discover and develop additional product candidates; maintain, expand, enforce, defend and protect our intellectual property portfolio; build our commercial function; and continue to hire research and development, clinical, technical operations and commercial personnel. In addition, we expect to continue to incur the costs associated with operating as a public company.

Previous wording

We have not generated any revenue to date from product sales and do not expect to do so in the near future. During the six months ended June 30, 2026 and 2025, we recognized $32.2 million and $15.9 million of license and collaboration revenue, respectively, which is primarily related to our collaboration and license agreements with Lilly and Apellis.

Current wording

We have not generated any revenue to date from product sales and do not expect to do so in the near future. During the nine months ended September 30, 2026 and 2025, we recognized $32.5 million and $25.6 million of license and collaboration revenue, respectively, which is primarily related to our collaboration and license agreements with Lilly and Apellis.

Previous wording

the cost to obtain licenses to intellectual property, such as those with Harvard University, or Harvard, The Broad Institute, Inc., or Broad Institute, Editas Medicine, Inc., or Editas, and Bio Palette Co., Ltd., or Bio Palette, and related future payments should certain success, development and regulatory milestones be achieved;

Current wording

the cost to obtain licenses to intellectual property, such as those with Harvard University, or Harvard, The Broad Institute, Inc., or Broad Institute, Editas Medicine, Inc., or Editas, and Kobe University (with respect to rights previously licensed from Bio Palette Co., Ltd., or Bio Palette), and related future payments should certain success, development and regulatory milestones be achieved;

Previous wording

Change in fair value of contingent consideration liabilities consists of remeasurement of the fair value of the milestone payments associated with our contingent consideration liabilities from acquisitions.

Current wording

Change in fair value of contingent consideration liabilities consists of gains or losses resulting from the remeasurement of the fair value of the milestone payments associated with our contingent consideration liabilities from acquisitions.

Previous wording

License and collaboration revenue was $0.5 million and $8.5 million for the three months ended June 30, 2026 and 2025, respectively. The decrease in revenue of $8.0 million is due to a change in the level of research activities on our license and collaboration programs.

Current wording

License and collaboration revenue was $0.3 million and $9.7 million for the three months ended September 30, 2026 and 2025, respectively. The decrease in revenue of $9.4 million is due to changes in the level of research activities on our license and collaboration programs.

Previous wording

Research and development expenses were $95.1 million and $101.8 million for the three months ended June 30, 2026 and 2025, respectively. The following table summarizes our research and development expenses for the three months ended June 30, 2026 and 2025 (in thousands):

Current wording

Research and development expenses were $110.1 million and $109.8 million for the three months ended September 30, 2026 and 2025, respectively. The following table summarizes our research and development expenses for the three months ended September 30, 2026 and 2025 (in thousands):

Previous wording

An increase of $1.0 million of other expenses driven primarily by milestone activity.

Current wording

An increase of $15.9 million of other expenses driven by milestone activity;

Previous wording

An increase of $4.3 million of employee related expenses due to the increase in research and development employees from 401 as of June 30, 2025 to 420 as of June 30, 2026; and

Current wording

An increase of $4.6 million of employee related expenses due to the increase in research and development employees from 397 as of September 30, 2025 to 429 as of September 30, 2026; and

Previous wording

A decrease of $8.4 million in external research and development expenses driven by a decrease of $9.0 million in outsourced services, primarily due to the timing of manufacturing and clinical activities, partially offset by an increase in lab supply expenses of $0.6 million due to an increase in research activities when compared to the prior year period; and

Current wording

A decrease of $3.0 million in external research and development expenses driven by a decrease of $4.0 million in outsourced services, primarily due to the timing of manufacturing and clinical activities, partially offset by an increase in lab supply expenses of $1.0 million due to an increase in research activities when compared to the prior year period; and

Previous wording

An increase of $8.1 million in employee related costs due to the growth in general and administrative employees from 108 as of June 30, 2025 to 120 as of June 30, 2026 and expenses related to consultants;

Current wording

An increase of $5.6 million in employee related costs due to the growth in general and administrative employees from 113 as of September 30, 2025 to 128 as of September 30, 2026 and expenses related to consultants; and

Previous wording

During the three months ended June 30, 2026 and 2025, we recorded $4.2 million of other expense and $1.3 million of other income, respectively, primarily related to the change in fair value of success payment liabilities, driven by changes in the price of our common stock over the related periods.

Current wording

During the three months ended September 30, 2026 and 2025, we recorded $4.7 million of other income and $2.6 million of other expense, respectively, primarily related to the change in fair value of success payment liabilities, driven by changes in the price of our common stock over the related periods.

Previous wording

During the three months ended June 30, 2026 and 2025, we recorded $0.3 million and $4.4 million of other income, respectively, as a result of changes in the fair value of our investment in corporate equity securities.

Current wording

During the three months ended September 30, 2026 and 2025, we recorded $1.0 million of other expense and $4.9 million of other income, respectively, as a result of changes in the fair value of our investment in Prime common stock and the sale of Verve's common stock.

Previous wording

During the six months ended June 30, 2026 and 2025, we recorded $0.3 million of other income and less than $0.1 million of other expense, respectively, related to the change in fair value of the milestone payments related to our contingent consideration liabilities.

Current wording

During the three months ended September 30, 2026 and 2025, we recorded $2.9 million of other expense and $1.0 million of other income, respectively, related to the change in fair value of the milestone payments related to our contingent consideration liabilities.

Previous wording

Interest and other income (expense), net was $7.5 million and $12.3 million of income for the three months ended June 30, 2026 and 2025, respectively.

Current wording

Interest and other income (expense), net was $6.1 million and $10.9 million of income for the three months ended September 30, 2026 and 2025, respectively.

Previous wording

License and collaboration revenue was $32.2 million and $15.9 million for six months ended June 30, 2026 and 2025, respectively. The increase in revenue of $16.3 million is driven primarily by the recognition of $25.0 million of revenue related to a milestone achieved under the Lilly Agreement during the six months ended June 30, 2026, partially offset by a decrease in revenue due to the level of research activities on our license and collaboration programs.

Current wording

License and collaboration revenue was $32.5 million and $25.6 million for nine months ended September 30, 2026 and 2025, respectively. The increase in revenue of $6.9 million is driven primarily by the recognition of $25.0 million of revenue related to a milestone achieved under the Lilly Agreement during the nine months ended September 30, 2026, partially offset by a decrease in revenue due to the level of research activities on our license and collaboration programs.

Previous wording

Research and development expenses were $199.6 million and $200.6 million for the six months ended June 30, 2026 and 2025, respectively. The following table summarizes our research and development expenses for the six months ended June 30, 2026 and 2025 (in thousands):

Current wording

Research and development expenses were $309.7 million and $310.3 million for the nine months ended September 30, 2026 and 2025, respectively. The following table summarizes our research and development expenses for the nine months ended September 30, 2026 and 2025 (in thousands):

Previous wording

A decrease of $12.8 million in external research and development expenses driven by a decrease of $14.0 million in outsourced services, primarily due to the timing of manufacturing and clinical activities, offset by an increase in lab supply expenses of $1.2 million due to an increase in research activities when compared to the prior year period; and

Current wording

A decrease of $15.8 million in external research and development expenses driven by a decrease of $18.0 million in outsourced services, primarily due to the timing of manufacturing and clinical activities, offset by an increase in lab supply expenses of $2.2 million due to an increase in research activities when compared to the prior year period;

Previous wording

An increase of other expenses of $11.9 million driven primarily by milestone and non-royalty sublicense activity;

Current wording

An increase of other expenses of $27.8 million driven primarily by milestone and non-royalty sublicense activity;

Previous wording

An increase of $8.0 million of employee related expenses due to the increase in research and development employees from 401 as of June 30, 2025 to 420 as of June 30, 2026; and

Current wording

An increase of $12.6 million of employee related expenses due to the increase in research and development employees from 397 as of September 30, 2025 to 429 as of September 30, 2026; and

Previous wording

An increase of $0.3 million in facility and information technology related expenses, including depreciation, related to our leased facilities.

Current wording

An increase of $0.7 million in facility and information technology related expenses, including depreciation, related to our leased facilities.

Previous wording

An increase of $4.9 million in employee related costs due to the growth in general and administrative employees from 108 as of June 30, 2025 to 120 as of June 30, 2026 and expenses related to consultants;

Current wording

An increase of $13.7 million in employee related costs due to the growth in general and administrative employees from 113 as of September 30, 2025 to 128 as of September 30, 2026 and expenses related to consultants;

Previous wording

During the six months ended June 30, 2026 and 2025, we recorded $1.7 million of other expense and $4.5 million of other income, respectively, related to the change in fair value of success payment liabilities, driven by changes in the price of our common stock over the related periods.

Current wording

During the nine months ended September 30, 2026 and 2025, we recorded $3.0 million and $1.9 million of other income, respectively, related to the change in fair value of success payment liabilities, driven by changes in the price of our common stock over the related periods.

Previous wording

During the six months ended June 30, 2026 and 2025, we recorded $0.4 million and $2.3 million of other income, respectively, as a result of changes in the fair value of our investment in corporate equity securities.

Current wording

During the nine months ended September 30, 2026 and 2025, we recorded $0.7 million of other expense and $7.3 million of other income, respectively, as a result of changes in the fair value of our investment in Prime common stock and the sale of Verve's common stock.

Previous wording

During the three months ended June 30, 2026 and 2025, we recorded $0.2 million and less than $0.1 million of other expense, respectively, related to the change in fair value of the milestone payments related to our contingent consideration liabilities.

Current wording

During the nine months ended September 30, 2026 and 2025, we recorded $2.5 million of other expense and $0.9 million of other income, respectively, related to the change in fair value of the milestone payments related to our contingent consideration liabilities.

Showing the first 40 of 56 pairs. Open the source sections for the rest.

Rearranged current paragraphs (5)

Current report — wording carried from elsewhere

The increase of $0.3 million was primarily due to the following:

Current report — wording carried from elsewhere

An increase of $0.4 million of facility and information technology related expenses, including depreciation, related to our leased facilities.

Current report — wording carried from elsewhere

A decrease of $1.2 million in stock-based compensation driven by the expense recognized on additional one-time stock awards granted in prior years for which there was no equivalent expense in the three months ended September 30, 2026; and

Current report — wording carried from elsewhere

A decrease of $0.3 million in legal expenses.

Current report — wording carried from elsewhere

Gain on sale of equity method investment
Rearranged previous paragraphs (5)

Previous report — wording carried elsewhere

The decrease was partially offset by the following:

Previous report — wording carried elsewhere

An increase of $6.6 million in legal expenses; and

Previous report — wording carried elsewhere

A decrease of $4.5 million in stock-based compensation driven by the expense recognized on additional one-time stock awards granted in prior years for which there was no equivalent expense in the six months ended June 30, 2026.

Previous report — wording carried elsewhere

In 2025, Bristol-Myers Squibb Company completed the Acquisition of Orbital. At the closing of the Acquisition, we held 75 million shares of Orbital common stock, which were cancelled and converted into $255.1 million in closing cash consideration, plus the right to receive up to approximately $26.3 million in additional cash consideration upon the release, if any, of certain escrows. During the six months ended June 30, 2026, we received $0.5 million of additional cash consideration upon release of certain escrows related to the Acquisition. We may receive additional cash consideration in future periods upon the release of the remaining escrow amounts, if any, in accordance with the terms of the Acquisition.

Previous report — wording carried elsewhere

As of June 30, 2026, we have sold 13,769,001 shares of common stock under the Sales Agreement at an average price of $62.75 per share for aggregate gross proceeds of $864.0 million, before deducting commissions and offering expenses payable by us. There were no shares sold under the Sales Agreement during the six months ended June 30, 2026.