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• A gene-editing company like CRISPR Therapeutics, but using a different proprietary platform.
• Like Allogene Therapeutics for 'off-the-shelf' cancer immunotherapies.

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• ARCUS Gene Editing Platform: A proprietary gene-editing technology utilized to develop therapies for genetic and infectious diseases.
• In Vivo Gene Editing Programs: Therapeutic candidates in various stages of development that employ the ARCUS platform to directly correct genetic defects or combat infectious diseases within the patient.

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Precision BioSciences (DTIL) primarily sells its gene editing technology and drug development services to other companies in the pharmaceutical and biotechnology sectors, rather than directly to individuals. Its major customers are strategic partners and collaborators who license its ARCUS gene editing platform or engage in co-development agreements for therapeutic programs.

The company's major customer companies include:

• Eli Lilly and Company (Symbol: LLY) - Through its wholly-owned subsidiary, Prevail Therapeutics, Eli Lilly is collaborating with Precision BioSciences on in vivo gene editing programs for neurological disorders. This collaboration is a significant source of revenue and strategic focus for DTIL.
• Imugene Limited (Symbol: ASX: IMU) - Imugene is collaborating with Precision BioSciences to develop novel allogeneic CAR T cell therapies for the treatment of solid tumors.

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Michael Amoroso, President, Chief Executive Officer

Michael Amoroso joined Precision BioSciences as President, Chief Executive Officer, and Director in October 2021. He is a biotech CEO with significant operational experience in cell and gene therapies. Prior to Precision BioSciences, he served as President and Chief Executive Officer of Abeona Therapeutics, Inc., a gene and cell therapy company, where he also held roles as Chief Commercial Officer and Chief Operating Officer. Mr. Amoroso's background also includes serving as Senior Vice President and Chief Commercial Officer at Kite Pharma, Inc. from 2018 to 2020, and various senior executive positions at biopharmaceutical companies such as Eisai Inc., Celgene Corporation, and Aventis (now Sanofi S.A.). He earned his Executive M.B.A. from the Stern School of Business, New York University.

Alex Kelly, Chief Financial Officer

Alex Kelly was appointed Chief Financial Officer of Precision BioSciences in May 2021, after joining the company in October 2020 as Chief Corporate Affairs Officer and serving as Interim CFO since December 2020. Before Precision BioSciences, he was Executive Vice President, Corporate Affairs and Chief Communications Officer at Allergan, where he supported transformations including Allergan's merger with AbbVie. Notably, Mr. Kelly served as Senior Vice President, Chief Integration Officer for Actavis, where he led the integration efforts for the acquisitions of Forest Labs (2014) and Allergan (2015). He also led integration efforts at Forest Labs when it acquired Aptalis Pharma. His career includes senior communications and investor relations roles at Bausch + Lomb, Merck, Schering-Plough, Novartis, Pharmacia, and Pharmacia & Upjohn.

Cindy Atwell, Chief Development and Business Officer

Cindy Atwell was promoted to Chief Development and Business Officer in January 2025. She joined Precision BioSciences in 2019 and previously served as the company's Chief Business Officer since 2022. Ms. Atwell brings over twenty years of multi-disciplinary experience in the biopharma industry, with prior roles in business development and clinical research at companies such as Halozyme, AbbVie, and Amylin.

Jeff Smith, Ph.D., Chief Research Officer

Jeff Smith is a co-founder of Precision BioSciences, which he helped establish in 2006. He was promoted to Chief Research Officer in September 2022, having previously served as Chief Technology Officer. Dr. Smith was instrumental in building Precision BioSciences from its inception and in optimizing the proprietary ARCUS gene editing platform for therapeutic use.

Cassie Gorsuch, Ph.D., Chief Scientific Officer

Cassie Gorsuch was promoted to Chief Scientific Officer in January 2025. In this role, she oversees non-clinical development and gene therapy discovery efforts for Precision BioSciences.

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The key risks to Precision BioSciences (DTIL) are primarily related to the inherent uncertainties of clinical-stage biotechnology development, the company's financial health, and the competitive landscape of novel gene-editing technologies.

1. Clinical Trial Success and Regulatory Approval: As a clinical-stage biotechnology company, Precision BioSciences' success is heavily dependent on the ability to successfully develop product candidates through lengthy, expensive, and uncertain clinical trials, and subsequently obtain regulatory approval. Any safety or efficacy concerns during preclinical studies or clinical trials could significantly limit prospects for regulatory approval, leading to a material adverse effect on the business. The historical failure rate for product candidates in the biotechnology industry is high, and promising early results do not guarantee success in later stages.
2. Financial Health and Need for Capital: Precision BioSciences faces significant financial challenges, including a history of steady losses, negative revenue growth, and substantial cash burn. The company's financial health is rated poorly, with indicators like an Altman Z-Score placing it in the distress zone and a high probability of bankruptcy. While the company has extended its cash runway, there remains a risk of future capital raises through mechanisms like secondary offerings, which could lead to further shareholder dilution.
3. Competition and Novel Technology Risk: The biotechnology sector is characterized by rapid technological change and intense competition. Precision BioSciences' ARCUS gene editing technology is novel, making it challenging to predict the timeline, cost, and ultimate success of product candidate development. Competitors may develop more effective or safer treatments, or achieve regulatory approval before Precision BioSciences, which could negatively impact the company's financial condition and ability to commercialize its products. Additionally, adverse public perception regarding genome editing technologies could hinder development and commercial adoption.

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The rapid advancement and clinical translation of next-generation gene editing technologies, particularly Prime Editing and Base Editing, by competing companies represent clear emerging threats. These technologies, championed by firms such as Beam Therapeutics (Base Editing) and Verve Therapeutics (Base Editing), offer the potential for enhanced precision and reduced off-target effects by modifying individual DNA bases or performing "search and replace" operations without inducing double-strand breaks. In contrast, Precision BioSciences' proprietary ARCUS platform operates by creating targeted double-strand breaks in the genome. Should these newer platforms demonstrate superior safety, efficacy, or broader applicability in clinical settings, they could potentially displace existing nuclease-based gene editing approaches, including ARCUS, for a range of therapeutic indications, particularly for in vivo gene editing where precision and minimization of unintended genetic alterations are paramount.

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Precision BioSciences (DTIL) is a clinical-stage biotechnology company primarily focused on developing in vivo gene editing therapies utilizing its proprietary ARCUS® genome editing platform. The company also has an allogeneic CAR T cell therapy, azer-cel, which has been out-licensed for development in both cancer and autoimmune diseases.

Addressable Markets for Main Products and Services:

• Allogeneic CAR T Cell Therapies (e.g., azer-cel, PBCAR19B): The global allogeneic CAR T cell therapy market was valued at approximately $1.2 billion in 2024 and is projected to reach $13.5 billion by 2033, demonstrating a Compound Annual Growth Rate (CAGR) of 31.8%. The broader global CAR T cell therapy market, which includes both autologous and allogeneic approaches, was valued at USD 4.534 billion in 2024 and is predicted to grow to approximately USD 23.247 billion by 2034, with a CAGR of 18.10% from 2025 to 2034.
• Hereditary Angioedema (HAE) Therapeutics (via in vivo gene editing partnerships): The global hereditary angioedema therapeutics market was estimated at USD 3.3 billion in 2022 and is projected to reach USD 5.8 billion by 2030, with a CAGR of 7.4%. Another report valued the global market at USD 4.1 billion in 2023. The market was also reported at USD 2.9 billion in 2024, with a projected growth to $5.2 billion by 2030 at a CAGR of 8.6%. North America held the largest revenue share in this market, accounting for 73.5% in 2022 and 46.8% in 2023.
• Phenylketonuria (PKU) Treatment (via in vivo gene editing partnerships): The global phenylketonuria market was valued at USD 1.27 billion in 2024 and is projected to reach USD 2.44 billion by 2032, with a CAGR of 8.51% during the forecast period of 2025 to 2032. Another estimate places the global market size at USD 1.17 billion in 2023, expected to reach USD 2.02 billion by 2032, growing at a CAGR of 6.3%. The U.S. phenylketonuria treatment market size was valued at USD 280.6 million in 2023 and is anticipated to reach USD 502.5 million by 2032. Europe is identified as the fastest-growing region in the global PKU market.
• Chronic Hepatitis B Virus (HBV) (PBGENE-HBV): Null
• m.3243 Associated Mitochondrial Myopathy (PBGENE-3243/PBGENE-PMM): Null
• Duchenne Muscular Dystrophy (PBGENE-DMD): Null
• Sickle Cell and Beta Thalassemia (PBGENE-NVS): Null
• Ornithine Transcarbamylase (OTC) Deficiency (iECURE-OTC): Null

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Precision BioSciences (DTIL) is a clinical-stage gene editing company whose future revenue growth over the next 2-3 years is primarily expected to be driven by the advancement of its proprietary ARCUS® platform and its therapeutic candidates, as well as strategic partnerships.

1. Progression and Potential Commercialization of Wholly-Owned Clinical Programs: A significant driver of future revenue for Precision BioSciences stems from the successful advancement of its lead wholly-owned in vivo gene editing programs, PBGENE-HBV and PBGENE-DMD. PBGENE-HBV, targeting chronic Hepatitis B, is in Phase 1 trials (ELIMINATE-B) and has received FDA Fast Track designation. Positive clinical data, particularly on antiviral efficacy and safety from dose escalation studies anticipated throughout 2025 and into 2026, could lead to further development and potential commercialization or lucrative partnerships. Similarly, PBGENE-DMD for Duchenne Muscular Dystrophy is being accelerated, with an Investigational New Drug (IND) filing expected by the end of 2025, Phase 1 initiation in the first half of 2026, and initial clinical data in the second half of 2026. This program has also received Rare Pediatric Disease and Orphan Drug designations, which could accelerate its review process and potentially lead to a Priority Review Voucher upon approval.
2. Milestone Payments and Royalties from Strategic Collaborations: Precision BioSciences leverages its ARCUS platform through partnerships, which provide non-dilutive funding in the form of upfront payments, milestone payments, and future royalties. The licensing deal with TG Therapeutics for the CAR T therapy azer-cel for autoimmune diseases, announced in January 2024, includes potential milestone payments up to $288 million and high-single-digit to low-double-digit royalties on net sales, in addition to initial payments. TG Therapeutics anticipates commencing a Phase 1 clinical trial for azer-cel in 2024. Additionally, the collaboration with iECURE for ECUR-506, an ARCUS-mediated gene insertion program for ornithine transcarbamylase (OTC) deficiency, is progressing, with complete data from its first-in-human trial expected in the first half of 2026. Realization of these milestone payments and future royalties from existing and potentially new partnerships will be crucial for revenue growth.
3. Expansion and Validation of the ARCUS Gene Editing Platform: The core ARCUS genome editing platform itself is a key driver. Continued validation of its efficacy, versatility, and safety through ongoing preclinical and clinical studies can attract new collaborations and expand its application into a broader range of genetic and infectious diseases. Precision BioSciences views partnering as a cornerstone of its growth, using collaborations to deepen capabilities and access new resources and expertise. The successful development of their wholly-owned programs and progress in partnered programs will further demonstrate the platform's potential, opening doors for additional licensing agreements or research collaborations that could generate significant future revenue. The company also regained rights to certain programs, offering opportunities for internal development or new partnerships.

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Share Issuance

• In June 2022, Precision BioSciences closed an underwritten offering of 35,971,224 shares of its common stock at an offering price of $1.39 per share, resulting in gross proceeds of approximately $50 million.
• As of March 20, 2025, 921,243 shares were sold at the market.
• As part of a License Agreement with TG Therapeutics, 220,712 shares of common stock were issued to TG Therapeutics on January 6, 2025.

Inbound Investments

• In November 2020, Eli Lilly and Company made a $35 million equity investment in Precision BioSciences' common stock as part of a research collaboration, alongside an upfront cash payment of $100 million.
• Novartis made a $25 million equity investment in Precision BioSciences in 2022 and provided a $50 million upfront cash payment.
• Precision BioSciences received an $8.0 million milestone from Imugene on October 31, 2025, paid as $3.0 million cash and $5.0 million in stock.

Outbound Investments

• In December 2022, there was a $10.8 million impairment of prepaid expenses related to the iECURE PCSK9 collaboration.
• There was a $2.5 million decrease in the fair value of the iECURE equity during the nine months ended September 30, 2025.

Capital Expenditures

• As of December 31, 2024, Precision BioSciences anticipated that existing cash, cash equivalents, restricted cash, expected operational receipts, and an at-the-market (ATM) facility would extend its cash runway into the second half of 2026, supporting operating expenses and capital expenditure requirements.
• This cash runway projection was updated to the second half of 2027, as of September 30, 2025.
• The net proceeds from the June 2022 underwritten offering, totaling approximately $50 million, were intended to fund ongoing and planned research and development, as well as for working capital and general corporate purposes, which include capital expenditures.