PRECISION BIOSCIENCES INC
For the period ended Jun 30, 2025. Filed Aug 7, 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.
Jun 30, 2025 · Filed Aug 7, 2025
DTIL$10.60+7.4%Stored close · as of Nov 6, 2026
What changed in Management’s Discussion and Analysis?
Read the current filingPrevious report: 10-Q
Period ended 2025-03-31; filed 2025-05-15.
0000950170-25-071991
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsCurrent report: 10-Q
Period ended 2025-06-30; filed 2025-08-07.
0001628280-25-038594
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 135 previous paragraphs and 131 current paragraphs.
73 unchanged; 30 reworded; 24 added; 29 removed; 3 previous and 4 current paragraphs rearranged.
Tables are not compared: 3 previous and 5 current tables excluded. Dates, whitespace, punctuation and numbering changes are ignored; changes to financial figures remain significant.
A changed paragraph is not evidence that risk increased or decreased. An unchanged paragraph does not establish that risk stayed the same.
Added paragraphs (24)
Current report
In May 2025, we presented initial safety data for PBGENE-HBV at the European Association for the Study of the Liver Congress as well as safety data for the sentinel patient in cohort 2 during the American Society of Gene and Cell Therapy (“ASGCT”) annual meeting.
Current report
On August 6, 2025, we announced Phase 1 safety and efficacy data for Cohort 1, the lowest dose level in the ELIMINATE-B trial. Cohort 1 consisted of three patients each of whom received three planned administrations of 0.2 mg/kg of PBGENE-HBV dosed approximately eight weeks apart. The primary objective is to characterize the safety of PBGENE-HBV. PBGENE-HBV was well-tolerated in all three patients in Cohort 1. Across Cohort 1, no patient experienced above a Grade 2 treatment-related adverse event, a serious adverse event, or dose-limiting toxicity. No clinically significant lab abnormalities were observed, including liver enzymes and platelets.
Current report
PBGENE-HBV demonstrated a substantial HBsAg reduction in all three patients in Cohort 1 with best response reductions of 56%, 69% and 47% compared to baseline HBsAg levels (ranging between 562-11,813 IU/mL) in patients one, two and three, respectively. One of three patients (33%) in Cohort 1 achieved a durable HBsAg reduction of approximately 50% from baseline that was maintained as of the data cutoff-date (July 28, 2025), which was seven months after initial dosing. This data provides evidence of the ability of PBGENE-HBV to drive a durable antiviral response by editing the viral DNA at the source of chronic Hepatitis B infection. There was no association between baseline HBsAg and efficacy in Cohort 1.
Current report
We also announced initial safety data from Cohort 2 (0.4 mg/kg) in the ELIMINATE-B study. As of the data cutoff, one patient received three dose administrations with two weeks of follow-up, and two patients received one dose administration with four weeks of follow-up. In these patients, no adverse events above Grade 2 were observed. No serious adverse events or dose limiting toxicities were observed, and no cumulative adverse effects were observed. There were no clinically significant elevations of liver transaminases. One additional patient did not complete their dose due to a transient infusion-related serious adverse event that resolved within minutes. The Data Monitoring Committee determined that this transient reaction was not dose-related or dose-limiting. Given the favorable safety profile of Cohorts 1 and 2, the Data Monitoring Committee recently recommended initiation of Cohort 3.
Current report
We are on track to complete dosing of all three patients across all dose administrations in Cohort 2 and commence dosing Cohort 3. We expect to provide a data update later in 2025.
Current report
The FDA granted PBGENE-DMD Rare Pediatric Disease designation in June 2025 for the treatment of DMD followed by Orphan Drug Designation in July 2025, highlighting the significant unmet need for new therapeutic options for those living with DMD.
Current report
In July 2025, we announced new preclinical data building upon previous data shared at ASGCT in May 2025. These data demonstrated the ability of PBGENE-DMD to target the key muscle types involved in the progression of DMD as well as significant, durable functional improvement in a humanized DMD mouse model. PBGENE-DMD showed a three-fold increase in dystrophin-positive muscle cells between three and nine months in the quadricep, gastrocnemius (calf), heart, and diaphragm. In the gastrocnemius specifically, up to 85% of cells were dystrophin-positive. This broad increase in dystrophin-positive cells, combined with the increased dystrophin protein detected in tissues, further validates the improved muscle function that was observed over time and may be attributable to edited satellite cells.
Current report
We are advancing the final U.S. investigational new drug (“IND”)-enabling toxicology studies with an anticipated IND and/or clinical trial application (“CTA”) filing targeted by the end of 2025 with initial clinical data expected in 2026.
Current report
As we advance the ELIMINATE-B clinical trial and prepare to file an IND and/or CTA for the PBGENE-DMD program, we are closely managing our operating costs. We have also extended our expected cash runway to the second half of 2027 to enable meaningful clinical data readouts for both programs. In July 2025, we initiated an operating efficiency program, including reductions in early research, manufacturing, and general and administrative operating expenses, which are aimed at reducing our annual operating expenses.
Current report
Imugene, our clinical stage partner developing azer-cel for oncology indications, announced updated clinical data in July 2025. In the updated data in patients diagnosed with relapsed/refractory diffuse large B-cell lymphoma (“DLBCL”), two additional patients achieved a Complete Response and three additional patients achieved a Partial Response. Imugene reported that, as a result of the new data, the best overall response rate for azer-cel in DLBCL reached 75%, and the Complete Response rate reached 55%. Based on the updated response rate and maturing durability data, as well as having been awarded FDA Fast Track Designation for DLBCL, Imugene expects to request an end of Phase 1 meeting with the FDA in the fourth quarter of 2025 to present the data and discuss designs for a pivotal/registrational trial for azer-cel.
Current report
(Loss) Gain from Equity Method Investment
Current report
The direct expense decrease of $6.3 million for PBGENE-HBV was due to lower manufacturing and toxicology expenses as the program transitioned to the clinic in the fall of 2024. The increase in expense of $4.0 million for PBGENE-DMD was a result of the program's acceleration in the second quarter of 2025. PBGENE-3243 had a $1.0 million decrease in expense as we have paused development. Platform development and early-stage research expenses decreased over the comparison period as a result of a $0.6 million decrease in employee-related costs and a $0.5 million decrease in depreciation and amortization.
Current report
General and administrative expenses were $9.1 million for the three months ended June 30, 2025, compared to $8.5 million for the three months ended June 30, 2024. The increase of $0.6 million was primarily due to a $0.6 million increase in employee-related costs, partially offset by decreases in other general and administrative expenses.
Current report
Loss from Equity Method Investment
Current report
Loss from equity method investment was $0.7 million during the three months ended June 30, 2025 compared to $1.0 million during the three months ended June 30, 2024, which represented our proportionate share of Elo's net loss in each period.
Current report
Loss on changes in fair value was $2.5 million for the three months ended June 30, 2025 resulting from the change in fair value of the iECURE investment in the current period. Gain on changes in fair value was $0.7 million for the three months ended June 30, 2024, which was primarily attributable to the increase in fair value of the Imugene Convertible Note for that period.
Current report
Interest Income
Current report
Comparison of the Six Months Ended June 30, 2025 and June 30, 2024
Current report
Revenue
Current report
Revenue for the six months ended June 30, 2025 was less than $0.1 million, compared to $67.5 million for the six months ended June 30, 2024. The decrease of $67.4 million in revenue during the six months ended June 30, 2025 was primarily the result of $52.7 million of revenue recognized in the prior period which represented all remaining deferred revenue related to the Prevail Therapeutics Agreement at its conclusion in April 2024. Additionally contributing to the decrease in revenue was a $9.5 million decrease as a result of revenue recognized under the TG License Agreement and a license agreement with Caribou Biosciences, Inc. during the six months ended June 30, 2024 and a $5.3 million decrease in revenue recognized under the Novartis Agreement as we near the completion of our preclinical work plan.
Current report
Research and development expenses for the six months ended June 30, 2025 were $26.4 million, compared to $30.6 million for the six months ended June 30, 2024. The decrease of $4.2 million was primarily due to a $7.8 million decrease in our direct expenses in PBGENE-HBV and a $2.7 million decrease in platform development and early-stage research expenses. These expense decreases were partially offset by a $6.3 million increase in our direct expenses in PBGENE-DMD.
Current report
The direct expense decrease of $7.8 million for PBGENE-HBV was due to lower manufacturing and toxicology expenses as the program transitioned to the clinic in the fall of 2024. These decreases were partially offset by the $6.3 million increase in PBGENE-DMD development due to the program's acceleration in the second quarter of 2025. Platform development and early-stage research expenses also decreased by $2.7 million in expense over the comparison period. The primary factors for the decrease were a $1.1 million decrease in depreciation and amortization, a $0.7 million decrease in employee-related costs, and a $0.5 million decrease in license fees and other research and development costs.
Current report
Interest expense was $0.7 million for the six months ended June 30, 2025 compared to $1.1 million for the six months ended June 30, 2024. The decrease of $0.4 million of interest expense was primarily driven by lower interest rates during the six months ended June 30, 2025 as compared to the six months ended June 30, 2024.
Current report
Interest income was $2.4 million during the six months ended June 30, 2025 compared to $3.5 million during the six months ended June 30, 2024. The $1.1 million decrease in interest income was the result of lower interest rates and a lower cash balance during the six months ended June 30, 2025 as compared to the six months ended June 30, 2024.
Removed paragraphs (29)
Previous report
In February 2025, we announced initial results from the first administration of PBGENE-HBV in cohort 1, the lowest dose level of the ELIMINATE-B trial. PBGENE-HBV was safe and well tolerated in all three participants in cohort 1 after the first administration of a 0.2 mg/kg dose. In addition to safety, PBGENE-HBV demonstrated a substantial reduction in Hepatitis B surface antigen (“HBsAg”) in two of the three participants following the first administration at the lowest dose level.
Previous report
In March 2025, the U.S. Food and Drug Administration (“FDA”) cleared PBGENE-HBV to commence Phase 1 clinical trials in the United States, and in April 2025, the FDA granted Fast Track designation to PBGENE-HBV for chronic Hepatitis B. Throughout 2025, we plan to share updates on the full cohorts including antiviral efficacy at different dose levels as each cohort is completed following three dose administrations and appropriate follow up. Also, in April 2025, the U.K. Medicines and Healthcare products Regulatory Agency (“MHRA”) cleared PGENE-HBV for a Phase 1 trial, making it the fifth country to clear a clinical trial application (“CTA”) or investigational new drug (“IND”) for PBGENE-HBV.
Previous report
On May 8, 2025, we presented initial safety for PBGENE-HBV at the European Association for the Study of the Liver Congress (“EASL”), which indicated translation of PBGENE-HBV nonclinical pharmacokinetic and safety data from non-human primates into the clinic. These data support pre-planned repeat dosing as well as dose escalation of PBGENE-HBV with the goal of moving appropriate dose and schedule into Phase 2 expansion.
Previous report
In preclinical data presented at the Muscular Dystrophy Association (“MDA”) in March 2025 and additional new data presented at the American Society of Gene and Cell Therapy (“ASGCT”) on May 14, 2025, PBGENE-DMD demonstrated the ability to target the key muscle types involved in the progression of DMD and significant, durable functional improvement in a humanized DMD mouse model. PBGENE-DMD restored the body’s ability to produce a near full length functional dystrophin protein across multiple muscles, including cardiac tissue and various key skeletal muscle groups. In addition, PBGENE-DMD edited satellite muscle stem cells, believed to be critical for long-term durability and sustained functional improvement.
Previous report
Based on these compelling data, the significant unmet need in DMD, and the clear regulatory guidance established for new therapeutics for DMD, we believe that PBGENE-DMD holds significant potential for the majority of patients with DMD. As a result, we are prioritizing and accelerating the development of PBGENE-DMD as our second wholly owned clinical program and target filing an IND and/or CTA for PBGENE-DMD in 2025 with clinical data expected in 2026.
Previous report
New preclinical data presented at ASGCT on May 14, 2025 highlight the ability of an ARCUS nuclease to eliminate mutant mtDNA and achieve therapeutically meaningful heteroplasmy shiftsin vivo. Reductions of mutant mtDNA exceed the baseline cited in the literature to be sufficient to alleviate the clinical symptoms in patients with m.3243-associated disease.
Previous report
To accelerate development of PBGENE-DMD and maintain operational capability to deliver PBGENE-HBV and PBGENE-DMD data through Phase 1 clinical results, we plan to pause development of PBGENE-3243 and will stage future development of PBGENE-3243 alone or with partners following completion of the Phase 1 ELIMINATE-B HBV trial and after the PBGENE-DMD program reaches the clinic.
Previous report
TG Therapeutics
Previous report
On January 7, 2024, we entered into a license agreement (the “TG License Agreement”) with TG Cell Therapy, Inc. (“TG Subsidiary”) and its parent company TG Therapeutics, Inc. (“TG Parent” and, together with TG Subsidiary, “TG Therapeutics”), pursuant to which we granted TG Subsidiary certain exclusive and non-exclusive license rights to develop, manufacture, and commercialize azercabtagene zapreleucel (“azer-cel”) for autoimmune diseases and other indications outside of cancer. We received a deferred cash payment of $2.5 million from TG Therapeutics on January 6, 2025 in exchange for 220,712 shares of our common stock at a price of $11.33, which represented a 100% premium to the VWAP of our common stock for the 30 trading days prior to the date of payment. We recognized no revenue under the TG License Agreement during the three months ended March 31, 2025, and we recognized $7.0 million of revenue under the TG License Agreement in the three months ended March 31, 2024.
Previous report
Imugene
Previous report
On August 15, 2023 we entered into an asset purchase agreement with Imugene Limited, and its wholly-owned subsidiary Imugene (USA) Inc. (“Imugene US” and together with Imugene Limited, “Imugene”). Additionally, we entered into a license agreement with Imugene (the “Imugene License Agreement”) on August 15, 2023, pursuant to which we granted Imugene US certain exclusive and non-exclusive license rights to develop, manufacture, and commercialize oncological applications of our allogeneic chimeric antigen receptor (“CAR”) T therapy, azer-cel, and up to three additional research product candidates directed to targets that Imugene US may nominate prior to the fifth anniversary of the effective date of the Imugene License Agreement, pursuant to the terms of the Imugene License Agreement.
Previous report
As of March 31, 2025, management has constrained all variable consideration related to milestone payments in the Imugene License Agreement given the level of uncertainty associated with achievement of the milestone payments. Accordingly, no revenue was recognized under the Imugene License Agreement during the three months ended March 31, 2025.
Previous report
Novartis Pharma AG
Previous report
On June 14, 2022, we entered into a collaboration and license agreement (the “Novartis Agreement”) with Novartis Pharma AG (“Novartis”), which became effective on June 15, 2022, to collaborate, discover and developin vivo gene editing products incorporating our custom ARCUS nucleases for the purpose of seeking to research and develop potential treatments for certain hemoglobinopathies, including sickle cell disease and beta thalassemia. During the three months ended March 31, 2025 and 2024, we recognized revenue under the Novartis Agreement of less than $0.1 million and $4.5 million, respectively. Deferred revenue related to the Novartis Agreement amounted to $26.2 million and $26.3 million as of March 31, 2025 and December 31, 2024, respectively, of which $5.7 million and $3.0 million, respectively, was included in current liabilities within the condensed balance sheets.
Previous report
Prevail Therapeutics, Inc.
Previous report
On November 19, 2020, we entered into a development and license agreement with Eli Lilly and Company (“Lilly”) to collaborate, discover and developin vivo gene editing products incorporating ARCUS nucleases for the research and development of potentialin vivo therapies for genetic disorders, which was subsequently assigned to Prevail Therapeutics Inc., a wholly-owned subsidiary of Lilly (“Prevail”), effective November 1, 2022 and amended on June 30, 2023 (the “Prevail Agreement”). For the three months ended March 31, 2025, we did not recognize any revenue under the Prevail Agreement as it was terminated in July 2024, and we subsequently exercised our right to the return of our three programs. We recognized revenue of $4.5 million for the three months ended March 31, 2024. We have no deferred revenue related to the Prevail Agreement as of March 31, 2025 or December 31, 2024.
Previous report
iECURE
Previous report
Research and Development Expenses
Previous report
General and Administrative Expenses
Previous report
As partial consideration for the assets acquired by Imugene in connection with the asset purchase agreement (the “Imugene Purchase Agreement”), Imugene issued convertible notes to us pursuant to the terms and conditions set forth in a convertible note subscription deed (collectively, the “Imugene Convertible Note”) in an aggregate principal amount of $13 million. The Imugene Convertible Note matured on August 30, 2024 and resulted in payment to us in cash and ordinary shares of Imugene Limited.
Previous report
Interest Expense
Previous report
Loss on Disposal of Assets
Previous report
March 31, 2024 and a $4.5 million decrease in revenue recognized under the Prevail Agreement during the three months ended March 31, 2025 compared to the three months ended March 31, 2024 following conclusion of the collaboration in April 2024.
Previous report
Research and Development Expenses
Previous report
The direct expense increases of $2.3 million for PBGENE-DMD and $1.0 million for PBGENE-3243 were driven by development costs to continue advancing these programs. These increases were partially offset by a $1.5 million decrease in PBGENE-HBV development costs, which was driven by the program’s conclusion of non-clinical work and the program’s transition to the clinic. Contributing to the decrease in platform development and development expenses was a $0.6 million decrease in depreciation and amortization, a $0.3 million decrease in licensing fees and other research and development costs, and a $0.3 million decrease in laboratory supplies and services expenses.
Previous report
General and Administrative Expenses
Previous report
Gain from Equity Method Investment
Previous report
Interest Expense
Previous report
Loss on Disposal of Assets
Reworded paragraphs (30)
Previous wording
We are leveraging our proprietary ARCUS genome editing platform to advancein vivo gene editing programs that go beyond gene knockouts in the liver and carry out more sophisticated edits such as gene insertions, gene excision, and gene elimination, unlocking a broader potential for ARCUS in vivo gene editing in human therapeutics.
Current wording
We are leveraging our proprietary ARCUS genome editing platform to advancein vivo gene editing programs that go beyond gene knockouts in the liver and carry out more sophisticated edits such as gene insertions, gene excision, and gene elimination, unlocking a broader potential for ARCUSin vivo gene editing in human therapeutics.
Previous wording
PBGENE-HBV is our wholly owned in vivo gene editing program under investigation in a global first-in-human clinical trial, which is designed to be a potentially curative treatment for chronic Hepatitis B (“HBV”) infection. PBGENE-HBV is the first and only potentially curative gene editing program to enter the clinic that is specifically designed to eliminate cccDNA and inactivate integrated HBV DNA, the root cause chronic Hepatitis B. The ELIMINATE-B trial is investigating PBGENE-HBV at multiple ascending dose levels with three dose administrations per dose level in patients with chronic Hepatitis B.
Current wording
PBGENE-HBV is our wholly owned in vivo gene editing program under investigation in a global first-in-human clinical trial, which is designed to be a potentially curative treatment for chronic Hepatitis B infection. PBGENE-HBV is the first and only potentially curative gene editing program to enter the clinic that is specifically designed to eliminate cccDNA and inactivate integrated HBV DNA, the root cause chronic Hepatitis B. The ELIMINATE-B trial is investigating PBGENE-HBV at multiple ascending dose levels with three dose administrations per dose level in patients with chronic Hepatitis B. The ELIMINATE-B trial is recruiting patients in the U.S., Moldova, Hong Kong, New Zealand and the U.K. and six patients have been treated to date.
Previous wording
PBGENE-DMD is our development program for the treatment of Duchenne muscular dystrophy (“DMD”). DMD is a genetic disease caused by mutations in the dystrophin gene that prevent production of the dystrophin protein and affects approximately 15,000 patients in the United States alone. There are currently no approved therapies with curative intent that can drive durable and significant functional improvements over time. PBGENE-DMD is designed to improve function over time and addresses more than 60% of patients afflicted with DMD by employing two complementary ARCUS nucleases delivered in a single AAV to excise exons 45-55 of the dystrophin gene with the aim of restoring a near-full length functional dystrophin protein within the body that more closely resembles normal dystrophin as opposed to synthetic, truncated dystrophin approaches with minimal functional benefit.
Current wording
DMD is a genetic disease caused by mutations in the dystrophin gene that prevent production of the dystrophin protein and affects approximately 15,000 patients in the United States alone. There are currently no approved therapies with curative intent that can drive durable and significant functional improvements over time. PBGENE-DMD is designed to improve function for more than 60% of patients afflicted with DMD by employing two complementary ARCUS nucleases delivered in a single AAV to excise exons 45-55 of the dystrophin gene. The aim of this approach is to restore a near-full length functional dystrophin protein within the body that more closely resembles normal dystrophin as opposed to synthetic, truncated dystrophin approaches with minimal functional benefit.
Previous wording
The PBGENE-3243 is a first of its kind potential treatment for m.3243 associated mitochondrial disease that is designed to specifically target and eliminate mutant m.3243G mitochondrial DNA (“mtDNA”), thereby eliminating the root cause of the disease. Currently, there are no curative treatments for m.3243-associated mitochondrial disease, which affects approximately 20,000 people in the U.S. alone and an even larger prevalent population globally. PBGENE-3243 is designed to alleviate muscular myopathy symptoms, providing a significant quality of life and functional improvement for patients.
Current wording
PBGENE-3243 is a potential treatment for m.3243 associated mitochondrial disease that is designed to specifically target and eliminate mutant m.3243G mitochondrial DNA (“mtDNA”), thereby eliminating the root cause of the disease. We have paused development of PBGENE-3243 to prioritize our two lead programs, PBGENE-HBV and PBGENE-DMD.
Previous wording
In partnership with iECURE, Inc. (“iECURE”), an ARCUS-mediated gene insertion approach is being pursued as a potential treatment option for neonatal onset ornithine transcarbamylase (“OTC”) deficiency. In January 2025, iECURE reported clinical efficacy and safety data in the first patient dosed showing a complete clinical response from three months post-exposure to the end of study at six months, as defined by the study protocol. The patient is now more than one year old and is eating age-appropriate levels of protein for a child of this age. Clinical data from the first patient in the ongoing OTC-HOPE trial was presented at the 2025 ACMG Annual Clinical Genetics Meeting in March 2025 and additional data has been accepted for presentation at ASGCT 2025. The OTC-HOPE study is ongoing in the U.K., the U.S., Australia, and Spain, and iECURE expects to complete enrollment in 2025 and anticipates complete data from the trial in the first half of 2026.
Current wording
In partnership with iECURE, Inc. (“iECURE”), an ARCUS-mediated gene insertion approach is being pursued as a potential treatment option for neonatal onset ornithine transcarbamylase (“OTC”) deficiency. In January 2025, iECURE reported clinical efficacy and safety data in the first patient dosed showing a complete clinical response from three months post-exposure to the end of study at six months, as defined by the study protocol. The patient is now more than one year of age and is eating appropriate levels of protein for a child of his age. iECURE has reported that a second infant with severe OTC deficiency was dosed in the first half of 2025. The OTC-HOPE study is ongoing in the U.K., the U.S., Australia, and Spain, and iECURE expects to complete enrollment in 2025 and anticipates complete data from the trial in the first half of 2026.
Previous wording
Research and development activities are central to our business model. We expect that our research and development expenses will increase over the long term and will comprise a larger percentage of our total expenses as we progress development of our product candidates.
Current wording
Research and development activities are central to our business model. While we expect our costs to decrease in the short-term for 2026 and 2027 as a result of our cost reduction initiatives, we expect that our research and development expenses will increase over the long term and will comprise a larger percentage of our total expenses as we progress development of our product candidates.
Previous wording
Gain from equity method investment represents our proportionate share of the net loss of our equity method investee, Elo Life Systems, Inc. (“Elo”).
Current wording
Gain from equity method investment represents a gain from dilution from our proportionate share of our equity method investee, Elo Life Systems, Inc. (“Elo”), resulting from its Series A-2 financing offset by our proportionate share of net loss of Elo.
Previous wording
In August 2021, we entered into a development and license agreement with iECURE (the “iECURE DLA”) under which iECURE was to advance our PBGENE-PCSK9 candidate through preclinical activities as well as a Phase 1 clinical trial in order to gain access to a license to use our PCSK9-directed ARCUS nuclease to insert genes into the PCSK9 locus to develop treatments for four pre-specified rare genetic diseases, including OTC deficiency (the “PCSK9 License”). Simultaneously with the entry into the iECURE DLA, we and iECURE entered into an equity issuance agreement (the “iECURE Equity Agreement”), pursuant to which iECURE issued us common stock in iECURE as additional consideration for the PCSK9 license. Additionally, we are eligible to receive milestone and mid-single digit to low double digit royalty payments on sales of iECURE products developed with ARCUS.
Current wording
As part of the consideration received in connection to the development and license agreement with iECURE, we entered into an equity issuance agreement with iECURE (the “iECURE Equity Agreement”), pursuant which iECURE issued us common stock in iECURE.
Previous wording
We adjust the carrying value of the iECURE equity to fair value each reporting period with any changes in fair value recorded to other income (expense). There was no change in the fair value of the iECURE equity for the three months ended March 31, 2025 and three months ended March 31, 2024.
Current wording
We adjust the carrying value of the iECURE investment under the iECURE Equity Agreement to its fair value each reporting period with any changes in fair value recorded to other (expense) income.
Previous wording
Gain (Loss) on Changes in Fair Value
Current wording
Gain (Loss) on Change in Fair Value of Warrant Liability
Previous wording
Revenue for the three months ended March 31, 2025 was less than $0.1 million, compared to $17.6 million for the three months ended March 31, 2024. The decrease of $17.6 million in revenue during the three months ended March 31, 2025 was primarily the result of $8.5 million in revenue recognized under the TG License Agreement and a license agreement with Caribou Biosciences, Inc. during the three months ended March 31, 2024, a $4.5 million decrease in revenue recognized under the Novartis Agreement as we near the completion of our pre-clinical workplan during the three months ended March 31, 2025 compared to the three months ended
Current wording
Revenue for the three months ended June 30, 2025 was less than $0.1 million, compared to $49.9 million for the three months ended June 30, 2024. The decrease of $49.9 million in revenue during the three months ended June 30, 2025 was primarily the result of $48.2 million of revenue recognized in the prior period which represented all remaining deferred revenue related to the Prevail Therapeutics Agreement at its conclusion in April 2024. Additionally contributing to the decrease in revenue was a $0.9 million decrease in revenue recognized under the TG License Agreement during the three months ended June 30, 2024, and a $0.8 million decrease in revenue recognized under the Novartis Agreement as we near the completion of our preclinical work plan during the three months ended June 30, 2025 compared to the three months ended June 30, 2024.
Previous wording
Research and development expenses for the three months ended March 31, 2025 were $13.6 million, compared to $13.3 million for the three months ended March 31, 2024. The increase of $0.3 million was primarily due to a $3.2 million increase in our direct expenses in PBGENE-DMD and PBGENE-3243 programs, partially offset by a $1.5 million decrease in our direct expense in PBGENE-HBV and a reduction in platform development and unallocated expenses of $1.5 million.
Current wording
Research and development expenses for the three months ended June 30, 2025 were $12.8 million, compared to $17.2 million for the three months ended June 30, 2024. The decrease of $4.5 million was primarily due to a $7.3 million decrease in our direct expenses in PBGENE-HBV and PBGENE-3243 programs, partially offset by a $4.0 million increase in our direct expenses in PBGENE-DMD. Additionally contributing to the decrease was a reduction in platform development and early-stage research expenses of $1.2 million.
Previous wording
Loss from change in fair value of the warrant liability was $0.8 million for the three months ended March 31, 2025 compared to a gain of $10.4 million for the three months ended March 31, 2024, which represents the mark-to-market fair value adjustment to the outstanding warrants issued in connection with the March 2024 Public Offering.
Current wording
Gain from change in fair value of the warrant liability was $0.8 million for the three months ended June 30, 2025 compared to a gain of $7.8 million for the three months ended June 30, 2024, which represents the mark-to-market fair value adjustment to the outstanding warrants issued in connection with the March 2024 Public Offering.
Previous wording
Interest income was $1.3 million during the three months ended March 31, 2025 compared to $1.7 million during the three months ended March 31, 2024. The $0.4 million decrease in interest income was the result of lower interest rates during the three months ended March 31, 2025 as compared to the three months ended March 31, 2024.
Current wording
Interest income was $1.1 million during the three months ended June 30, 2025 compared to $1.8 million during the three months ended June 30, 2024. The $0.7 million decrease in interest income was the result of lower interest rates and a lower cash balance during the three months ended June 30, 2025 as compared to the three months ended June 30, 2024.
Previous wording
General and administrative expenses were $8.6 million for the three months ended March 31, 2025 compared to $8.4 million for the three months ended March 31, 2024. The increase of $0.2 million was primarily due to a $0.5 million increase in employee-related costs, partially offset by decreases in depreciation and amortization and other operating expenses, specifically taxes and insurance.
Current wording
General and administrative expenses were $17.7 million for the six months ended June 30, 2025, compared to $17.0 million for the six months ended June 30, 2024. The increase of $0.7 million was primarily due to a $1.1 million increase in employee-related costs, partially offset by decreases in depreciation and amortization and other operating expenses, specifically subscription software.
Previous wording
Gain from equity method investment was $1.3 million during the three months ended March 31, 2025 which was primarily driven by a $2.3 million gain on dilution from our proportionate share of Elo’s proceeds from a Series A-2 financing in such period, partially offset by our proportionate share of Elo’s net loss of $0.9 million. Gain from equity method investment was $1.7 million during the three months ended March 31, 2024, which was primarily the result of a $2.7 million gain recorded from our proportionate share of Elo’s proceeds from a Series A-2 financing in such period, partially offset by our proportionate share of Elo’s loss of $1.0 million.
Current wording
Gain from equity method investment was $0.7 million during the six months ended June 30, 2025 which was primarily driven by a $2.3 million gain on dilution from our proportionate share of Elo’s proceeds from a Series A-2 financing in such period, partially offset by our proportionate share of Elo’s net loss of $1.6 million during the period. Gain from equity method investment was $0.8 million during the six months ended June 30, 2024 which was primarily driven by a $2.7 million gain on dilution from our proportionate share of Elo’s proceeds from a Series A-2 financing in such period, partially offset by our proportionate share of Elo’s net loss of $1.9 million during the period.
Previous wording
Gain on changes in fair value was less than $0.1 million for the three months ended March 31, 2025, which was primarily attributed to an increase during such period in the fair value of the Imugene Convertible Note and the ordinary shares issued thereunder. Loss on changes in fair value was $0.3 million for the three months ended March 31, 2024, which was primarily attributable to a decrease during such period in the fair value of the Imugene Convertible Note.
Current wording
Loss on changes in fair value was $2.4 million for the six months ended June 30, 2025 due to the change in fair value of the iECURE investment in the current period. Gain on changes in fair value was $0.3 million for the six months ended June 30, 2024, which was primarily attributable to a increase during such period in the fair value of the Imugene Convertible Note.
Previous wording
Loss on disposal of assets was less than $0.1 million during each of the three months ended March 31, 2025 and 2024.
Current wording
Loss on disposal of assets was less than $0.1 million during the six months ended June 30, 2025 and $0.3 million during the six months ended June 30, 2024.
Previous wording
We have incurred significant operating losses since our inception and have not generated any revenue from the sale of products. Our ability to generate any product revenue or product revenue sufficient to achieve profitability will depend on the successful development and eventual commercialization of one or more of our product candidates or the product candidates of our collaborators or other licensees for which we may receive milestone payments or royalties. As of March 31, 2025, we had an accumulated deficit of $503.0 million.
Current wording
We have historically incurred significant operating losses and have not generated any revenue from the sale of products. Our ability to generate any product revenue or product revenue sufficient to achieve profitability will depend on the successful development and eventual commercialization of one or more of our product candidates or the product candidates of our collaborators or other licensees for which we may receive milestone payments or royalties. As of June 30, 2025, we had an accumulated deficit of $526.5 million.
Previous wording
As of March 31, 2025, we had a total cash, cash equivalents and restricted cash balance of $99.8 million. Pursuant to our July 31, 2024 amended and restated loan and security agreement with Banc of California (the “2024 Loan and Security Agreement”) pursuant to which Banc of California provided us with a term loan with a principal amount of $22.5 million (the “2024 Term Loan”), we are not entitled to borrow any additional amounts under the 2024 Term Loan and are required to maintain an aggregate balance in a cash security account with Banc of California (the “Cash Security Account”) at least equal to the outstanding principal amount of the 2024 Term Loan then outstanding.
Current wording
As of June 30, 2025, we had a total cash, cash equivalents and restricted cash balance of $84.8 million. Pursuant to our July 31, 2024 amended and restated loan and security agreement with Banc of California (the “2024 Loan and Security Agreement”) pursuant to which Banc of California provided us with a term loan with a principal amount of $22.5 million (the “2024 Term Loan”), we are not entitled to borrow any additional amounts under the 2024 Term Loan and are required to maintain an aggregate balance in a cash security account with Banc of California (the “Cash Security Account”) at least equal to the outstanding principal amount of the 2024 Term Loan then outstanding.
Previous wording
Our cash, cash equivalents, and restricted cash totaled $99.8 million and $137.8 million as of March 31, 2025 and 2024, respectively.
Current wording
Our cash, cash equivalents, and restricted cash totaled $84.8 million and $123.6 million as of June 30, 2025 and June 30, 2024, respectively.
Previous wording
Cash used in operating activities during the three months ended March 31, 2025 was $19.1 million compared to $19.0 million during the three months ended March 31, 2024. The $0.1 million increase in cash used in operating activities was primarily driven by a $29.2 million decrease in net income offset by increases of $10.0 million in non-cash adjustments and $19.0 million in changes in operating assets and liabilities.
Current wording
Cash used in operating activities during the six months ended June 30, 2025 was $39.3 million compared to $33.9 million during the six months ended June 30, 2024. The $5.5 million increase in cash used in operating activities was primarily driven by an $85.4 million decrease in net income offset by increases of $19.1 million in non-cash adjustments and $60.9 million in changes in operating assets and liabilities.
Previous wording
The increase in non-cash adjustments was primarily driven by the net change in fair value of warrants for the three months ended March 31, 2025 compared to the three months ended March 31, 2024.
Current wording
The increase in non-cash adjustments year over year was primarily driven by the net change in fair value of warrants during the six months ended June 30, 2024 due to the decrease in the warrant liability which resulted in a negative balance within non-cash adjustments for the period. For the six months ended June 30, 2025, the net change in fair value of warrants was slightly positive within non-cash adjustments given the incremental increase in the warrant liability for the current period. Also contributing to the increase in non-cash adjustments year over year was the change in fair value of the iECURE investment in the current period.
Previous wording
The change in cash used in operating assets and liabilities is primarily driven by the net changes of deferred revenue as a result of the conclusion of the Prevail collaboration and a decrease in revenue recognized under the Novartis collaboration for the three months ended March 31, 2025 compared to the three months ended March 31, 2024. Additionally driving the change in cash used in operating assets and liabilities is the net change of the contract asset as a result of the TG Therapeutics deferred share payment in three months ended March 31, 2025 and net changes in prepaid expenses.
Current wording
The change in cash used in operating assets and liabilities is primarily driven by the net changes of deferred revenue as a result of the conclusion of the Prevail collaboration and a decrease in revenue recognized under the Novartis collaboration for the six months ended June 30, 2025 compared to the six months ended June 30, 2024. Additionally driving the change in cash used in operating assets and liabilities is the net change of the contract asset as a result of the TG Therapeutics deferred share payment in January 2025 as well as the and net change in accounts payable across the comparison period.
Previous wording
Cash used in investing activities primarily relates to cash expenditures to acquire leasehold additions, equipment, software and intangible assets. Net cash used in investing activities during the three months ended March 31, 2025 was $0.3 million, compared to an amount less than $0.1 million used in investing activities in the three months ended March 31, 2024. The increase in cash used in investing activities was the result of a $0.3 million increase in intangible asset purchases during the three months ended March 31, 2025.
Current wording
Cash used in investing activities primarily relates to cash expenditures to acquire leasehold additions, equipment, software and intangible assets. Net cash used in investing activities during the six months ended June 30, 2025 was $0.3 million, compared to an amount less than $0.1 million used in investing activities in the six months ended June 30, 2024. The increase in cash used in investing activities was primarily the result of a $0.3 million increase in intangible asset purchases during the six months ended June 30, 2025.
Previous wording
Net cash provided by financing activities during the three months ended March 31, 2025 was $10.7 million, compared to $40.1 million during the three months ended March 31, 2024. The $29.4 million decrease in cash provided by financing activities during the three months ended March 31, 2025 was primarily due to the Company receiving $29.6 million in net proceeds from an underwritten offering of common stock and warrants in the three months ended March 31, 2024, whereas the Company did not receive any net proceeds from underwritten offerings during the three months ended March 31, 2025.
Current wording
Net cash provided by financing activities during the six months ended June 30, 2025 was $16.0 million, compared to $40.8 million during the six months ended June 30, 2024. The $24.8 million decrease in cash provided by financing activities during the six months ended June 30, 2025 was primarily due to the Company receiving $37.5 million in net proceeds from an underwritten offering of common stock and warrants in the six months ended June 30, 2024, whereas the Company did not receive any net proceeds from underwritten offerings during the six months ended June 30, 2025. The decrease was partially offset by a $12.1 million increase in net proceeds from the issuance of common stock through the ATM facility between the comparison periods.
Previous wording
We will continue to have funding requirements in connection with the continuation of our research and development efforts, potential IND and CTA submissions, potential clinical trials, and expected growth in ourin vivo portfolios.
Current wording
We will continue to have funding requirements in connection with the continuation of our research and development efforts, potential IND and/or CTA submissions, potential clinical trials, and expected growth in ourin vivo portfolios.
Previous wording
We believe that, as of the date of this Quarterly Report on Form 10-Q, existing cash and cash equivalents, expected operational receipts, including potential near-term consideration to be received from licensees, operational efficiencies gained from divestment of our historical CAR T operations, and availability of our ATM facility will be sufficient to fund our operating expenses and capital expenditure requirements into the second half of 2026. We expect our cash runway to be sufficient to achieve first-in-human Phase 1 clinical data for two wholly-ownedin vivo gene editing programs. We have based these estimates on assumptions that may prove to be imprecise, and we could utilize our available capital resources sooner than we expect. There are no assurances that we will be successful in obtaining an adequate level of financing as and when needed to finance our operations on terms acceptable to us or at all, particularly in light of current global macroeconomic conditions. If we are unable to obtain sufficient financing on a timely basis or on favorable terms, we may be required to significantly delay, alter reduce or eliminate one or more of our research or product development programs and/or commercialization efforts, or to grant rights to develop and market products or product candidates that we would otherwise prefer to develop and market ourselves. We may also be otherwise unable to execute our business plan or growth strategy, or capitalize on business opportunities as desired. Any of these events could materially adversely affect our financial condition and business prospects. Because of the numerous risks and uncertainties associated with research, development and commercialization of pharmaceutical products, it is difficult to estimate with certainty the amount of our working capital requirements. Our future funding requirements will depend on many factors, including:
Current wording
We believe that, as of the date of this Quarterly Report on Form 10-Q, existing cash and cash equivalents, expected operational receipts, including potential near-term consideration to be received from licensees, operational efficiencies gained from divestment of our historical CAR T operations, and availability of our ATM facility will be sufficient to fund our operating expenses and capital expenditure requirements into the second half of 2027. We expect our cash runway to be sufficient to enable potential commencement of a Phase 2 study for PBGENE-HBV and a potential pivotal study for PBGENE-DMD pending supportive Phase 1 data readouts. We have based these estimates on assumptions that may prove to be imprecise, and we could utilize our available capital resources sooner than we expect. There are no assurances that we will be successful in obtaining an adequate level of financing as and when needed to finance our operations on terms acceptable to us or at all, particularly in light of current global macroeconomic conditions. If we are unable to obtain sufficient financing on a timely basis or on favorable terms, we may be required to significantly delay, alter reduce or eliminate one or more of our research or product development programs and/or commercialization efforts, or to grant rights to develop and market products or product candidates that we would otherwise prefer to develop and market ourselves. We may also be otherwise unable to execute our business plan or growth strategy, or capitalize on business opportunities as desired. Any of these events could materially adversely affect our financial condition and business prospects. Because of the numerous risks and uncertainties associated with research, development and commercialization of pharmaceutical products, it is difficult to estimate with certainty the amount of our working capital requirements. Our future funding requirements will depend on many factors, including:
Previous wording
In addition to the contractual obligations and commitments as described elsewhere in this Quarterly Report on Form 10-Q with respect to leases, outstanding indebtedness, and intellectual property licenses, we also enter into contracts in the normal course of business with CMOs, universities, and other third parties for preclinical research studies, testing, manufacturing services, and other services and products for operating purposes. These contracts are generally cancelable upon written notice. There have been no material changes to our contractual obligations from those described in our Annual Report on Form 10-K for the fiscal year ended December 31, 2024.
Current wording
In addition to the contractual obligations and commitments as described elsewhere in this Quarterly Report on Form 10-Q with respect to leases, outstanding indebtedness, and intellectual property licenses, we also enter into contracts in the normal course of business with CMOs, universities, and other third parties for preclinical research studies, testing, manufacturing services, and other services and products for operating purposes. These contracts are generally cancellable upon written notice. There have been no material changes to our contractual obligations from those described in our Annual Report on Form 10-K for the fiscal year ended December 31, 2024.
Previous wording
Our critical accounting policies and estimates are described in “Item 7. Management’s Discussion and Analysis of Financial Condition and Results of Operations—Critical Accounting Policies and Use of Estimates” in our Annual Report on Form 10-K. We have reviewed those critical accounting policies and estimates for the three months ended March 31, 2025. There have been no significant changes in our critical accounting policies and estimates from those disclosed in our Annual Report on Form 10-K for the fiscal year ended December 31, 2024.
Current wording
Our critical accounting policies and estimates are described in “Item 7. Management’s Discussion and Analysis of Financial Condition and Results of Operations—Critical Accounting Policies and Use of Estimates” in our Annual Report on Form 10-K. We have reviewed those critical accounting policies and estimates for the six months ended June 30, 2025. There have been no significant changes in our critical accounting policies and estimates from those disclosed in our Annual Report on Form 10-K for the fiscal year ended December 31, 2024.
Rearranged current paragraphs (4)
Current report — wording carried from elsewhere
Gain on Change in Fair Value of Warrant Liability
Current report — wording carried from elsewhere
Loss on disposal of assets was less than $0.1 million during the three months ended June 30, 2025 compared to $0.2 million during the three months ended June 30, 2024.
Current report — wording carried from elsewhere
The following table summarizes our results of operations for the six months ended June 30, 2025 and June 30, 2024, together with the changes in those items:
Current report — wording carried from elsewhere
Loss from change in fair value of the warrant liability was less than $0.1 million for the six months ended June 30, 2025 compared to a gain of $18.2 million for the six months ended June 30, 2024, which represents the mark-to-market fair value adjustment to the outstanding warrants issued in connection with the March 2024 Public Offering.
Rearranged previous paragraphs (3)
Previous report — wording carried elsewhere
We adjust the carrying value of the ordinary shares issued under the Imugene Convertible Note to its fair value each reporting period with any changes in fair value recorded to other income (expense). As of March 31, 2025, we had sold all of our ordinary shares of Imugene Limited.
Previous report — wording carried elsewhere
Gain (Loss) on Changes in Fair Value
Previous report — wording carried elsewhere
(Loss) Gain on Change in Fair Value of Warrant Liability