Financial Performance - Total revenues for Q3 2025 were less than $0.1 million, a decrease from $0.6 million in Q3 2024, primarily due to reduced billable efforts under the Novartis Agreement[17] - Revenue for Q3 2025 was $13,000, a significant decrease from $576,000 in Q3 2024, representing a decline of approximately 97.7%[29] - Net loss for Q3 2025 was $21.8 million, or ($1.84) per share, compared to a net loss of $16.4 million, or ($2.25) per share, in Q3 2024[20] - The net loss for Q3 2025 was $21,772,000, compared to a net loss of $16,425,000 in Q3 2024, indicating an increase in losses of approximately 32.5%[29] - Total operating expenses for Q3 2025 were $20,680,000, slightly down from $21,851,000 in Q3 2024, a decrease of about 5.4%[29] Cash and Assets - As of September 30, 2025, the company had approximately $71.2 million in cash, cash equivalents, and restricted cash, with a cash runway expected to extend into the second half of 2027[16] - Cash, cash equivalents, and restricted cash as of September 30, 2025, were $71,212,000, down from $108,468,000 at the end of 2024, a decrease of about 34.4%[31] - Total assets decreased to $93,510,000 as of September 30, 2025, from $136,388,000 at the end of 2024, a decline of approximately 31.4%[31] - Total stockholders' equity fell to $16,634,000 as of September 30, 2025, down from $56,393,000 at the end of 2024, a decrease of about 70.5%[31] Research and Development - Research and development expenses increased to $13.4 million in Q3 2025 from $13.1 million in Q3 2024, driven by the PBGENE-DMD program[18] - The company commenced dosing in Cohort 3 of the ELIMINATE-B trial during Q3 2025, with additional data readouts planned for early 2026[7] - The investigational new drug (IND) filing for PBGENE-DMD is anticipated by the end of 2025, with Phase 1 initiation expected in the first half of 2026[7] - The company anticipates an IND filing for PBGENE-DMD by the end of 2025 and expects to initiate a Phase 1 clinical trial in 2026[26] - The design of PBGENE-DMD targets over 60% of patients with DMD, aiming for durable functional improvement with a one-time lower dose of AAV[26] Clinical Trials and Milestones - PBGENE-HBV has shown a durable HBsAg reduction of approximately 50% from baseline in one patient, ongoing seven months post-treatment[6] - The company received an $8 million milestone payment from Imugene on October 31, 2025, related to the azer-cel program[13] - The company has been granted Breakthrough Therapy designation by the FDA for PBGENE-HBV, which is designed to eliminate the root cause of chronic Hepatitis B[21] - The company aims to complete enrollment for the OTC-HOPE clinical trial by late 2025, with data expected in the first half of 2026[26]

Core Insights - Precision BioSciences, Inc. is a clinical stage gene editing company focused on developing in vivo gene editing therapies for diseases with high unmet needs [1] - The company will publish its financial results for the third quarter of 2025 and provide a business update on November 3, 2025 [1] Company Overview - Precision BioSciences utilizes its proprietary ARCUS® platform for gene editing [1] - The company is dedicated to improving life through innovative gene editing solutions [1]

Summary of Precision BioSciences FY Conference Call Company Overview - **Company**: Precision BioSciences (NasdaqCM:DTIL) - **Technology**: Proprietary gene editing technology called Arcus, which is a nuclease-based system distinct from CRISPR Cas technologies [4][5] Key Points Discussed Industry Context - **Gene Editing Technologies**: The session focused on advancements in genome editing technologies, particularly the applications of gene insertion and targeting viral genomes [1][2] - **Participants**: Included executives from Precision BioSciences, eCure, Harbor Site, and Seamless Therapeutics, discussing their respective technologies and programs [2] Precision BioSciences' Arcus Technology - **Unique Features**: Arcus nucleases create staggered overhang cuts, allowing for high-efficiency gene insertion in both dividing and non-dividing cells [30] - **Clinical Applications**: The technology is being utilized in partnership with eCure for clinical gene insertion, specifically targeting conditions like ornithine transcarbamylase (OTC) deficiency [5][28] Clinical Data and Outcomes - **OTC Deficiency Treatment**: A patient treated at six and a half months of age showed significant improvement, with a 50% reduction in ammonium levels and no hyperammonemic events post-treatment [36][37] - **Regulatory Approval**: The program has received clearance to operate in multiple regions, including the US, UK, Spain, and Australia [32] Comparison with Traditional Gene Therapy - **Gene Insertion vs. Gene Addition**: Traditional gene addition approaches can lead to dilution of therapeutic effects in actively dividing tissues, whereas gene insertion ensures that the therapeutic gene is integrated into the genome, providing sustained expression [18][19] - **Targeting Multiple Mutations**: The ability to insert large DNA fragments addresses diseases with multiple mutations, which traditional gene editing systems cannot effectively target [12][15] Market Potential and Competitive Landscape - **Hepatitis B Program**: Precision BioSciences is pursuing a hepatitis B program aimed at eliminating cccDNA, which is crucial for curing chronic infections [54][56] - **Commercial Viability**: The potential market for hepatitis B treatments is significant, with a large patient population and a clear unmet need for effective therapies [62] Regulatory Outlook - **Positive Regulatory Interactions**: The company has had favorable interactions with regulatory agencies, indicating a supportive environment for gene editing technologies [81][83] - **Flexibility in Development**: Regulators are showing a willingness to adapt traditional trial designs to accommodate the unique challenges of rare diseases [86] Future Directions - **Expansion Beyond Rare Diseases**: There is a focus on applying gene editing technologies to larger patient populations and various indications beyond rare genetic disorders [94][95] - **Technological Advancements**: Continuous improvements in gene editing technologies and cost efficiencies are expected to enhance the commercial viability of these therapies [95] Additional Insights - **Investor Sentiment**: Investors are increasingly interested in technologies that address unmet medical needs and have clear commercial potential [76][78] - **Long-term Impact**: The ability to treat young patients with severe genetic disorders could have significant societal and economic benefits [90] This summary encapsulates the key discussions and insights from the Precision BioSciences FY Conference Call, highlighting the company's innovative approach to gene editing and its implications for future therapies.

Summary of Precision BioSciences Conference Call Company Overview - **Company**: Precision BioSciences - **Industry**: Biotechnology, specifically focusing on gene editing therapies for liver diseases, including hepatitis B virus (HBV) and hepatocellular carcinoma [2][4] Key Points and Arguments Arcus Gene Editing Platform - **Technology**: Precision BioSciences utilizes a proprietary gene editing platform called Arcus, which is distinct from CRISPR Cas9 technology [4][5] - **Unique Features**: - **Cut**: Arcus creates a four base pair three-prime overhang, enhancing gene insertion efficiency [8] - **Size**: Arcus is the smallest gene editor at approximately 1,000 bases, facilitating better delivery via lipid nanoparticle (LNP) and adeno-associated virus (AAV) technologies [10][11] - **Simplicity**: Arcus consists of a single protein that recognizes and cuts DNA, contrasting with CRISPR systems that require multiple components [12][13] Focus on Hepatitis B Virus (HBV) - **Unmet Need**: Current functional cure rates for HBV are only 1-2%, highlighting a significant unmet medical need [15][46] - **Differentiation**: The unique features of the Arcus platform make it particularly suitable for targeting covalently closed circular DNA (cccDNA) in HBV, which is crucial for achieving a complete cure rather than just viral suppression [17][22] Clinical Trials and Efficacy - **ILLUMINATE B Study**: A Phase 1 study testing the safety and antiviral response of PBG and HBV in patients on nucleoside analogs [24] - **Design**: Patients receive three administrations of PBG and HBV while continuing their nucleoside analog therapy to prevent reactivation [26] - **Cohort Data**: Initial data from Cohort 1 showed good tolerability and substantial S antigen reductions of 45-70% [31] - **Safety**: No greater than grade two adverse events (AEs) were observed, with infusion-related reactions being the most common [30] Future Directions - **Biopsy Data**: Liver biopsies will be conducted to assess the effects of PBG and HBV on cccDNA and integrated DNA levels [42][43] - **Dosing Interval**: The current dosing interval is eight weeks, but there is potential to shorten this based on safety data [36][38] - **Upcoming Milestones**: Key milestones include presentations at the AASLD liver meeting, completion of Cohorts 2 and 3, and potential adjustments to dosing intervals [49][51] Broader Implications - **Treatment Landscape**: The current treatment landscape for HBV has been underwhelming, with no effective means to eliminate cccDNA, which is essential for achieving a complete cure [46][47] - **Field Excitement**: The innovative approach of targeting the root cause of HBV infection has generated excitement within the field, as evidenced by the opportunity for a late breaker oral presentation at an upcoming conference [47][48] Additional Important Insights - **Long-term Goals**: The ultimate aim is to achieve complete viral clearance and eliminate the risk of reactivation, moving beyond the current focus on functional cures [22][27] - **Patient-Centric Approach**: The design of the trial considers patient safety and aims to optimize treatment duration and efficacy [38][51]

Core Insights - Precision BioSciences (DTIL) shares increased by 11.1% to close at $6.5, with a notable trading volume, reflecting a 25.3% gain over the past four weeks [1][2] Company Overview - The rise in stock price is linked to positive investor sentiment regarding Precision BioSciences' clinical-stage pipeline, particularly the in vivo gene editing therapy PBGENE-HBV aimed at curing chronic hepatitis B [2] - The company has a fully owned pipeline that includes several gene therapy programs in preclinical evaluation and a partnered candidate, ECUR-506, in a first-in-human phase I/II study for treating neonatal onset ornithine transcarbamylase deficiency [2] Financial Performance - Precision BioSciences is expected to report a quarterly loss of $0.31 per share, reflecting a year-over-year change of +89.1%, with revenues projected at $11.1 million, an increase of 1813.8% from the previous year [3] - The consensus EPS estimate for the upcoming quarter has remained unchanged over the last 30 days, indicating that stock price movements may not sustain without trends in earnings estimate revisions [4] Industry Context - Precision BioSciences is part of the Zacks Medical - Biomedical and Genetics industry, where another company, CervoMed Inc. (CRVO), experienced a 1.5% decline in its stock price [4] - CervoMed Inc. has a consensus EPS estimate of -$0.68, representing a year-over-year change of -23.6%, and currently holds a Zacks Rank of 4 (Sell) [5]

Core Insights - Precision BioSciences, Inc. has been selected for a late-breaking oral presentation at the Liver Meeting® 2025, highlighting its innovative gene editing capabilities [1] Company Summary - The company specializes in gene editing using its proprietary ARCUS® platform, focusing on developing in vivo gene editing therapies for diseases with high unmet medical needs [1] - The presentation will showcase new data from the PBGENE-HBV program, specifically from the first two cohorts of the Phase 1 ELIMINATE-B Trial [1]

Core Insights - Precision BioSciences, Inc. is a clinical stage gene editing company focused on developing in vivo gene editing therapies for diseases with high unmet needs [1] - The company announced the publication of a peer-reviewed manuscript in the journal Nucleic Acids Research, showcasing the efficiency of its proprietary ARCUS® platform [1] - The publication highlights the capability of the engineered homing endonuclease ARCUS for high-efficiency homology-directed insertion into the genome [1] Company Summary - Precision BioSciences utilizes its novel ARCUS® platform for gene editing, targeting diseases that currently lack effective treatments [1] - The recent publication serves to validate the technology and its potential applications in gene therapy [1] - The focus on in vivo therapies indicates a strategic direction towards addressing complex genetic disorders [1]

Core Insights - Precision BioSciences, Inc. has activated its first U.S. clinical trial site for PBGENE-HBV as part of the ELIMINATE-B Phase 1 clinical trial [1] - The clinical trial site is located at Massachusetts General Hospital in Boston, Massachusetts, and is now actively recruiting patients [1] Company Overview - Precision BioSciences is a clinical stage gene editing company that utilizes its proprietary ARCUS® platform [1] - The company focuses on developing in vivo gene editing therapies for diseases with high unmet medical needs [1]

Core Insights - Precision BioSciences, Inc. is a clinical stage gene editing company focused on developing in vivo gene editing therapies for diseases with high unmet needs [1] - The company will present late-breaking preclinical data for its product PBGENE-DMD at the 30th Annual International Congress of the World Muscle Society in Vienna, Austria, from October 7-12, 2025 [1]

Core Viewpoint - Precision BioSciences is a clinical stage gene editing company utilizing its proprietary ARCUS platform to develop in vivo gene editing therapies for diseases with high unmet needs [1][2]. Group 1: Company Overview - Precision BioSciences focuses on gene editing and is currently in the clinical stage of development [1]. - The company leverages its novel ARCUS platform, which is designed to address diseases that have significant unmet medical needs [1]. Group 2: Technology Differentiation - The ARCUS platform distinguishes itself from other gene editing technologies through its unique cutting mechanism, smaller size, and simpler structure [2]. - These characteristics enable ARCUS nucleases to achieve more defined and intended therapeutic outcomes [2].