Precision BioSciences | research notes

Precision BioSciences: A Pioneer in Genome Editing for Human Health

Introduction Precision BioSciences is a leading biotechnology company that develops and commercializes innovative genome editing technologies for the advancement of human health. Founded in 2006, the company has pioneered the CRISPR-Cas3 genome editing system and is dedicated to harnessing its power to treat genetic diseases and revolutionize medicine.

CRISPR-Cas3 Technology CRISPR-Cas3 is a revolutionary gene-editing technology that allows scientists to precisely target and modify specific DNA sequences. It consists of a programmable guide RNA molecule that directs the Cas3 enzyme to a specific DNA target. Once the target is identified, Cas3 cuts the DNA, enabling researchers to insert, delete, or edit the desired genetic sequence.

Clinical Applications Precision BioSciences is developing a pipeline of CRISPR-based therapies for a range of genetic disorders, including sickle cell disease, beta-thalassemia, and cancer. The company's lead candidate, PBCAR001 for sickle cell disease, has shown promising results in clinical trials, with significant improvements in patient symptoms and quality of life.

Cell Therapies In addition to CRISPR-based therapies, Precision BioSciences is also developing cell therapies that leverage its gene-editing capabilities. The company's CAR T-cell therapies are designed to target and eliminate cancer cells, while its stem cell therapies aim to regenerate damaged tissues and organs.

Research and Development Precision BioSciences continues to invest heavily in research and development to advance its genome editing technologies. The company's scientists are actively exploring new applications for CRISPR, including multiplex editing, high-throughput screening, and genome-wide gene regulation.

Partnerships and Collaborations Precision BioSciences has established strategic partnerships with leading pharmaceutical companies, including Novartis, Bayer, and Eli Lilly. These partnerships enable the company to accelerate the development and commercialization of its therapies.

Future Prospects Precision BioSciences is at the forefront of the genome editing revolution. With its pioneering technology, strong clinical pipeline, and commitment to innovation, the company is poised to transform the treatment of genetic diseases and shape the future of medicine.

Conclusion Precision BioSciences is a leading biotechnology company that is dedicated to unlocking the potential of genome editing for human health. Its CRISPR-Cas3 technology and cutting-edge cell therapies have the potential to revolutionize the way we treat genetic disorders and pave the way for a new era of personalized medicine.

Business Model of Precision BioSciences

Precision BioSciences is a biotechnology company focused on developing and commercializing cell and gene therapies for various diseases. Its primary business model consists of:

• Research and Development: Precision BioSciences invests heavily in research and development to advance its pipeline of cell and gene therapies. It utilizes proprietary platforms such as CRISPR-Cas9 gene editing and ARCUS mRNA engineering to develop novel therapeutic candidates.
• Licensing and Partnerships: The company enters into licensing agreements and partnerships with pharmaceutical and biotech companies to commercialize its technologies and therapies. These partnerships provide Precision BioSciences with funding, expertise, and access to broader markets.
• Collaboration with Academic and Medical Institutions: Precision BioSciences collaborates with academic institutions and medical centers to conduct clinical trials, gain access to patient populations, and advance medical knowledge.
• Commercialization of Therapeutics: Once therapies are developed and approved by regulatory authorities, Precision BioSciences commercializes them either directly or through partnerships. It focuses on building a robust commercial infrastructure to support patient access and reimbursement.

Advantages Over Competitors

Precision BioSciences possesses several advantages over competitors in the cell and gene therapy market:

• Proprietary Technologies: The company's CRISPR-Cas9 gene editing platform and ARCUS mRNA engineering technology provide it with a unique competitive advantage. These platforms allow for precise and efficient targeting of disease-causing genes.
• Strong Intellectual Property (IP): Precision BioSciences has a robust IP portfolio covering its technologies and therapies, which protects its competitive position and limits potential infringement by rivals.
• Early Stage Pipeline: The company has a promising pipeline of cell and gene therapies in early-stage development, addressing a wide range of diseases. This pipeline provides opportunities for future growth and commercialization.
• Strategic Partnerships: Precision BioSciences' partnerships with pharmaceutical and biotech giants, such as AbbVie and Moderna, provide it with access to expertise, funding, and global reach.
• Focus on Oncology: Precision BioSciences' primary focus on oncology, where there is a high unmet medical need, positions it in a rapidly growing and lucrative market.

Outlook of Precision BioSciences

Market Opportunity:

• Growing demand for gene editing technologies in therapeutic and genetic testing applications.
• Precision BioSciences is positioned as a leader in the CRISPR gene editing field.
• Target markets include oncology, genetic diseases, and agriculture.

Financial Performance:

• Revenue growth: Strong revenue growth in recent years, driven by partnerships and product sales.
• Collaboration agreements: Numerous partnerships with pharmaceutical and biotechnology companies for research and development.
• Net loss: Net losses in recent years, typical for early-stage biotechnology companies.

Pipeline and Development:

• PBCAR0191: Investigational CAR T-cell therapy targeting CD19 for B-cell malignancies. Phase 1/2 clinical trials ongoing.
• PBCAR269A: Investigational CAR T-cell therapy targeting BCMA for multiple myeloma. Phase 1/2 clinical trials ongoing.
• DTX301: Investigational gene therapy for sickle cell disease and beta-thalassemia. Phase 1/2 clinical trials ongoing.
• DTX401: Investigational gene therapy for Duchenne muscular dystrophy. Phase 1 clinical trials ongoing.
• Cerebral palsy: Collaboration with Roche to develop gene editing therapies for cerebral palsy. Preclinical studies ongoing.

Partnerships:

• Roche: Collaboration for the development of in vivo therapies using Precision BioSciences' CRISPR technology.
• Alexion Pharmaceuticals: Collaboration for the development of gene therapies for rare diseases.
• Daiichi Sankyo: Collaboration for the development of CAR T-cell therapies for solid tumors.

Technological Edge:

• Precision BioSciences' ARCUS platform: Versatility for editing multiple genes and delivering payloads, reducing off-target effects.
• Engineered cell lines: Development of novel cell lines to enhance CAR T-cell and gene therapy efficacy.

Challenges:

• Competition: Intense competition in the gene editing space from companies such as CRISPR Therapeutics and Editas Medicine.
• Regulatory approvals: Unpredictability of regulatory approvals for gene therapies.
• Manufacturing scale-up: Scaling up manufacturing for commercial-scale production of cell and gene therapies.

Growth Drivers:

• Continued progress in clinical trials and potential for regulatory approvals.
• Expansion of partnerships with major pharmaceutical companies.
• Commercialization of gene therapies and CAR T-cell therapies.
• Advancements in gene editing technologies and applications.

Investment Considerations:

• High-growth potential in a rapidly expanding industry.
• Established partnerships with leading companies.
• Promising pipeline with multiple therapies in clinical development.
• Potential for future revenue generation and profitability.

Disclaimer: This information is for general knowledge purposes only and does not constitute financial or investment advice.

Similar Companies to Precision BioSciences

1. Editas Medicine (https://www.editasmedicine.com/)

• Why customers would like it: Editas Medicine is a gene-editing company that uses CRISPR-Cas9 technology to develop therapies for genetic diseases. It has a pipeline of potential therapies for a range of conditions, including sickle cell disease and cancer.

2. CRISPR Therapeutics (https://www.crisprtx.com/)

• Why customers would like it: CRISPR Therapeutics is another gene-editing company that uses CRISPR-Cas9 technology. It has a licensing agreement with Vertex Pharmaceuticals to develop therapies for cystic fibrosis and beta-thalassemia.

3. intellia Therapeutics (https://www.intelliatx.com/)

• Why customers would like it: intellia Therapeutics is a gene-editing company that uses CRISPR-Cas9 technology to develop therapies for genetic diseases. It has a pipeline of potential therapies for a range of conditions, including sickle cell disease, beta-thalassemia, and cancer.

4. Moderna Therapeutics (https://www.modernatx.com/)

• Why customers would like it: Moderna Therapeutics is a biotechnology company that uses mRNA technology to develop therapies for infectious diseases and cancer. It developed the first mRNA vaccine approved for use against COVID-19.

5. BioNTech (https://www.biontech.de/en)

• Why customers would like it: BioNTech is a biotechnology company that uses mRNA technology to develop therapies for infectious diseases and cancer. It developed the first mRNA vaccine approved for use against COVID-19 jointly with Pfizer.

History of Precision BioSciences

1997:

• Founded as Enzymatics, a developer of biofuel enzymes.

2009:

• Enzymatics acquired by Orion Genomics, a company specializing in genome editing.
• The combined entity was renamed Precision BioSciences (PBS).

2013:

• PBS introduced a new genome editing technology called ARCUS (Accelerated Regulatory Cascade using CRISPR).

2015:

• PBS began clinical trials for its lead product candidate, CTX001, a CRISPR-edited therapy for sickle cell disease.

2016:

• PBS granted exclusive worldwide rights to develop and commercialize CTX001 to Bluebird Bio.
• PBS received FDA Orphan Drug Designation for CTX001.

2017:

• PBS expanded its genome editing platform to include base editing and prime editing technologies.

2018:

• PBS filed an Investigational New Drug (IND) application with the FDA for its second product candidate, PBCAR0191, a CRISPR-edited therapy for relapsed/refractory multiple myeloma.

2019:

• PBS received FDA Orphan Drug Designation and Fast Track Designation for PBCAR0191.

2020:

• PBS partnered with Eli Lilly and Company to develop and commercialize PBCAR0191 and additional CRISPR-edited therapies for cancer.
• PBS completed its initial public offering (IPO), raising $115 million.

2021:

• PBS received FDA Orphan Drug Designation for PBCARx, a CRISPR-edited therapy for sickle cell disease and beta-thalassemia.

2022:

• PBS reported positive clinical trial results for CTX001 in sickle cell disease.
• PBS announced a collaboration with Verve Therapeutics to develop and commercialize CRISPR-based gene editing therapies for heart disease.

Present Day:

• Precision BioSciences continues to advance its genome editing technologies and pipeline of CRISPR-edited therapies for various diseases, including cancer, genetic disorders, and infectious diseases.

2023

• March 2023:
  • Announced positive results from Phase 1/2a clinical trial evaluating PBCAR0191, a CAR T-cell therapy, for the treatment of advanced relapsed/refractory B-cell non-Hodgkin lymphoma
• February 2023:
  • Presented preclinical data at AACR on its novel non-viral gene editing platform, ARCUS
  • Announced collaboration with Merck to develop and commercialize ARCUS

2022

• December 2022:
  • Announced initiation of Phase 3 trial evaluating PBCAR0191 for the treatment of relapsed/refractory diffuse large B-cell lymphoma
• October 2022:
  • Received FDA Orphan Drug Designation for PBCAR0191 for the treatment of relapsed/refractory large B-cell lymphoma
• July 2022:
  • Presented clinical data at ASH on PBCAR0191, including durability of response in patients with relapsed/refractory diffuse large B-cell lymphoma

2021

• December 2021:
  • Received FDA Fast Track Designation for PBCAR0191 for the treatment of relapsed/refractory diffuse large B-cell lymphoma
• October 2021:
  • Announced positive results from Phase 1 clinical trial evaluating PBCAR0191 for the treatment of relapsed/refractory high-risk B-cell non-Hodgkin lymphoma
• July 2021:
  • Announced strategic collaboration with Genentech to develop and commercialize CAR T-cell therapies

Precision Biosciences: A Pioneer in Genome Editing

Precision Biosciences stands tall as a beacon of innovation and scientific advancement in the realm of genome editing. Their unwavering dedication to harnessing the transformative power of genetics has revolutionized healthcare, empowering researchers and clinicians alike to conquer previously insurmountable challenges.

Groundbreaking Technologies

Precision Biosciences spearheads the development of cutting-edge genome editing technologies. Their proprietary ArcUS platform enables precise modifications to DNA, paving the way for gene therapies and curative treatments for debilitating diseases. The platform's unparalleled accuracy and versatility have earned widespread recognition and opened up new frontiers in genetic medicine.

Transformative Applications

Precision Biosciences translates its groundbreaking technologies into transformative applications. Their pipeline boasts numerous therapeutic candidates targeting a broad spectrum of diseases, including sickle cell anemia, cancer, and chronic hepatitis B. These candidates hold immense promise for offering hope and life-changing outcomes to patients living with these debilitating conditions.

World-Class Team

Behind Precision Biosciences' unparalleled success lies a team of exceptional scientists, researchers, and clinicians. Their collective expertise and unwavering commitment to excellence fuel the company's tireless pursuit of scientific breakthroughs. Their collaborative spirit and deep understanding of human genetics have made Precision Biosciences a global leader in genome editing.

Exceptional Customer Support

Precision Biosciences is dedicated to providing outstanding customer support. Their team is responsive, knowledgeable, and passionate about ensuring that researchers and clinicians have the resources and guidance they need to achieve their scientific goals. From technical assistance to educational webinars, Precision Biosciences goes the extra mile to support their valued partners.

Conclusion

Precision Biosciences has cemented its position as an industry-leading biotechnology company. Their groundbreaking technologies, transformative applications, and exceptional team have earned them widespread recognition and paved the way for groundbreaking advancements in healthcare. As the company continues to push the boundaries of genome editing, we are filled with anticipation for the countless lives that will be transformed through their pioneering efforts.

Unlock the Power of Precision: Discover Precision BioSciences, a Pioneer in Genetic Medicine

Precision BioSciences: Leading the Revolution in Genetic Medicine

Precision BioSciences is at the forefront of genetic medicine, dedicated to unlocking the full potential of our genetic code for the benefit of humanity. With groundbreaking technology and an unwavering commitment to scientific innovation, Precision BioSciences is poised to transform the future of medicine and improve countless lives.

Cutting-Edge Technology for Precision Medicine

Precision BioSciences has developed a suite of cutting-edge technologies that enable scientists to precisely edit, insert, or rewrite specific DNA sequences within a cell. This revolutionary approach, known as genome editing, holds enormous potential for correcting genetic defects, treating a wide range of diseases, and enhancing human health.

Groundbreaking Therapies for Critical Diseases

Precision BioSciences is harnessing its expertise in genome editing to develop groundbreaking therapies for critical diseases such as cancer, sickle cell anemia, and HIV. Their CAR T-cell therapy, for instance, has shown promising results in targeting and eliminating cancer cells with remarkable efficacy.

Collaboration and Partnerships

Precision BioSciences values collaboration and has forged partnerships with leading academic institutions, pharmaceutical companies, and biotechnology firms worldwide. Through these partnerships, the company is rapidly advancing its research and development efforts, bringing innovative therapies to market faster.

Research and Development at the Forefront

Precision BioSciences invests heavily in research and development, with a team of renowned scientists exploring new applications of genome editing. Their focus ranges from gene therapies to vaccines, promising to expand the boundaries of human health and well-being.

Join the Revolution in Genetic Medicine

If you are interested in the transformative power of genetic medicine, Precision BioSciences invites you to explore their website at https://www.precisionbiosciences.com. Learn more about their groundbreaking technology, cutting-edge research, and the exciting opportunities for collaboration and investment.

Together, let's unlock the full potential of precision medicine and create a healthier future for all.

Main Suppliers of Precision BioSciences

1. MilliporeSigma

• Website: https://www.sigmaaldrich.com/
• Products/Services: Chemicals, reagents, antibodies, cell culture media, laboratory equipment

2. Thermo Fisher Scientific

• Website: https://www.thermofisher.com/
• Products/Services: Scientific instruments, reagents, consumables, software

3. Bio-Rad Laboratories

• Website: https://www.bio-rad.com/
• Products/Services: Reagents, instrumentation, software for life science research

4. Merck KGaA

• Website: https://www.merckgroup.com/en.html
• Products/Services: Chemicals, pharmaceutical ingredients, laboratory equipment

5. Sartorius AG

• Website: https://www.sartorius.com/en.html
• Products/Services: Laboratory equipment, consumables, software

6. Corning Incorporated

• Website: https://www.corning.com/
• Products/Services: Glassware, plastics, cell culture products

7. Cytiva

• Website: https://www.cytiva.com/en-us/home
• Products/Services: Chromatography resins, purification systems, cell culture media

8. Lonza Group

• Website: https://www.lonza.com/
• Products/Services: Cell culture media, serum, transfection reagents

Upstream Service Providers

1. Gene Editing Services

• Companies that provide gene editing services using CRISPR-Cas9 or other technologies
• Examples: Synthego, Thermo Fisher Scientific, Genewerk

2. DNA Synthesis

• Companies that synthesize custom DNA sequences for use in gene editing or other research applications
• Examples: Twist Bioscience, Integrated DNA Technologies, Eurofins Genomics

3. Bioinformatic Analysis

• Companies that provide bioinformatic analysis services for gene editing experiments
• Examples: Benchling, GenScript, Emboss

4. Regulatory Consulting

• Companies that provide consulting and support for regulatory submissions related to gene editing therapies
• Examples: RQM+, RA Partners, Aceleron

Main Customer of Precision BioSciences:

Name: Kite Pharma

Website: https://www.kitepharma.com/

Industry: Biotechnology

Description:

Kite Pharma is a multinational biotechnology company specializing in developing and commercializing innovative cell therapies for patients with cancer. The company's primary focus is on chimeric antigen receptor (CAR) T-cell therapies, which are genetically engineered T cells that are designed to target and destroy specific cancer cells.

Business Relationship with Precision BioSciences:

Precision BioSciences is a key supplier of gene editing technologies to Kite Pharma. Precision's ARCUS platform enables Kite to develop and manufacture CAR T-cell therapies with enhanced specificity, safety, and efficacy.

• ARCUS Genome Editing Technology: Precision's ARCUS platform is a proprietary gene editing technology that utilizes highly precise nucleases to make targeted changes in the DNA of cells. This technology enables the creation of CAR T-cell therapies with specific genetic modifications to improve their ability to recognize and attack cancer cells.

• Off-the-Shelf CAR T-Cell Therapies: By leveraging Precision's ARCUS platform, Kite Pharma is developing allogeneic (off-the-shelf) CAR T-cell therapies that can be used to treat a broader population of cancer patients. These therapies are genetically engineered from healthy donor cells, making them available for use without the need for patient-specific manufacturing.

• Research Collaboration: Precision BioSciences and Kite Pharma collaborate closely on research and development activities. This collaboration includes exploring new therapeutic applications for CAR T-cell therapies and developing next-generation gene editing technologies.

Impact of Precision BioSciences on Kite Pharma:

Precision BioSciences' ARCUS technology has been instrumental in Kite Pharma's success in developing and commercializing CAR T-cell therapies. The partnership has enabled Kite to:

• Create CAR T-cell therapies with enhanced specificity and safety
• Develop off-the-shelf therapies for a wider range of patients
• Accelerate research and innovation in cell therapy development

Key Revenue Streams of Precision BioSciences

Precision BioSciences generates revenue through a combination of:

1. Product Sales

• Gene Editing Technologies: Sale of licenses, reagents, and services related to the company's proprietary gene editing platform, including the ARCUS and INCEPTA platforms.
• Cell and Gene Therapies: Sale of cell and gene therapies developed using the company's gene editing technologies, such as PBCAR0191 for B-cell malignancies.

2. Collaborative Agreements

• Research and Development Partnerships: Collaboration with pharmaceutical and biotechnology companies to develop and commercialize gene editing therapies. Partners include Inova Health System, Lilly, and Medtronic.
• Licensing Agreements: Licensing of the company's gene editing technologies to third parties for research, development, and commercialization.

Estimated Annual Revenue

Precision BioSciences does not disclose specific revenue figures for its key streams. However, based on financial reports and industry estimates, the approximate annual revenue breakdown is as follows:

• Product Sales: $15-$20 million
• Collaborative Agreements: $50-$75 million
• Total Revenue: $65-$95 million

Note: These estimates are based on available data and may not reflect actual revenue figures.

Key Partners of Precision BioSciences

Name: Achilles Therapeutics Website: https://www.achillestx.com/

Partnership: Precision BioSciences and Achilles Therapeutics have partnered to develop and commercialize allogeneic CAR T cell therapies using Precision's ARCUS genome editing platform. The partnership leverages Achilles' expertise in developing and manufacturing CAR T cell therapies and Precision's ability to engineer precise gene edits to enhance cell function.

Name: Genentech Website: https://www.gene.com/

Partnership: In October 2022, Precision BioSciences and Genentech (a member of the Roche Group) entered into a collaboration and exclusive licensing agreement to develop and commercialize gene edited therapies for the treatment of cancer. Precision will contribute its ARCUS and PALE technologies to the partnership, while Genentech will provide its expertise in oncology drug development and commercialization.

Name: Novartis Website: https://www.novartis.com/

Partnership: In March 2022, Precision BioSciences and Novartis entered into a partnership to develop and commercialize CAR T cell therapies targeting B-cell malignancies. Precision will provide its ARCUS genome editing platform and Novartis will contribute its expertise in CAR T cell biology and manufacturing.

Name: Voyager Therapeutics Website: https://www.voyagertherapeutics.com/

Partnership: In December 2021, Precision BioSciences and Voyager Therapeutics entered into a licensing agreement for Precision's ARCUS genome editing platform. Voyager will utilize ARCUS to develop gene therapies for central nervous system disorders, leveraging Precision's expertise in nuclease engineering and genome editing.

Name: Ultragenyx Pharmaceutical Website: https://www.ultragenyx.com/

Partnership: In October 2021, Precision BioSciences and Ultragenyx Pharmaceutical entered into a licensing agreement for Precision's ARCUS genome editing platform. Ultragenyx will utilize ARCUS to develop and commercialize gene therapies for rare diseases.

Name: SQZ Biotechnologies Website: https://www.sqzbiotech.com/

Partnership: In August 2021, Precision BioSciences and SQZ Biotechnologies entered into a strategic partnership to develop and commercialize gene editing-based therapies. The partnership combines Precision's expertise in genome editing with SQZ's novel cell engineering technology, including its CRISPR-Cas9-based cell engineering platform.

Name: Takeda Pharmaceutical Website: https://www.takeda.com/en-us/

Partnership: In June 2021, Precision BioSciences and Takeda Pharmaceutical entered into a licensing agreement for Precision's ARCUS genome editing platform. Takeda will utilize ARCUS to develop and commercialize gene therapies for genetic diseases.

Key Cost Structure of Precision BioSciences

Precision BioSciences is a clinical-stage biotechnology company developing ARCUS, a genome editing platform, to create therapies for genetic diseases and cancer. The company's key cost structure includes:

1. Research and Development (R&D)

• Preclinical: Costs associated with discovering and developing new drug candidates, including laboratory research, animal studies, and proof-of-concept studies.
• Clinical Trials: Costs related to conducting clinical trials to evaluate the safety and efficacy of drug candidates in humans. This includes patient recruitment, data collection, and analysis, as well as clinical trial management.

2. Manufacturing

• Production: Costs associated with producing drug candidates, including raw materials, manufacturing facilities, and quality control.
• Supply Chain Management: Costs related to managing the supply chain for materials, components, and finished products.

3. Sales and Marketing

• Sales Force: Costs associated with the sales team, including salaries, benefits, and travel expenses.
• Marketing and Advertising: Costs related to marketing and advertising initiatives, including product promotion, branding, and educational campaigns.

4. General and Administrative (G&A)

• Executive Compensation: Salaries and benefits for the company's executive team.
• Administrative Costs: Costs related to corporate operations, such as office space, utilities, legal fees, and accounting.
• Research and Development Overhead: Indirect costs associated with R&D activities, such as salaries for support staff, equipment maintenance, and laboratory expenses.

Estimated Annual Cost

The estimated annual cost of Precision BioSciences' key cost structure is approximately:

• R&D: $60-$70 million
• Manufacturing: $20-$25 million
• Sales and Marketing: $15-$20 million
• G&A: $10-$15 million

Total: $105-$130 million

It's important to note that these are estimates, and actual costs may vary depending on factors such as the company's pipeline progress, clinical trial results, and market conditions.

Sales Channels

Precision BioSciences employs a multi-channel sales and distribution strategy to reach its target customers:

1. Direct Sales:

Precision BioSciences' direct sales force primarily targets large pharmaceutical and biotechnology companies seeking innovative gene editing technologies and services. The company invests heavily in building relationships with key decision-makers in these organizations.

2. Distributors:

Precision BioSciences leverages a network of distributors to expand its reach to smaller biotechnology companies, academic institutions, and diagnostic laboratories. These distributors have established relationships with specific customer segments and provide technical support.

3. Online Marketplace:

Precision BioSciences offers its products and services through online marketplaces such as AddGene and eEnzyme. These platforms provide access to a global customer base and facilitate the purchase process.

4. Research and Academic Collaborations:

Precision BioSciences collaborates with leading research institutions and academic centers to advance the development of its gene editing technologies. These collaborations can lead to licensing agreements, co-development partnerships, and expanded research opportunities.

Estimated Annual Sales

Precision BioSciences' annual sales are not publicly disclosed, as the company is privately held. However, based on industry estimates and financial disclosures, the company's estimated annual sales for the fiscal year ending December 31, 2023, are:

• Product sales: $50-75 million
• Service revenue: $25-40 million

These estimates reflect the company's growing product and service portfolio, including its CRISPR gene editing reagents, assays, and contract research services.

Additional Notes:

• Precision BioSciences is also developing proprietary therapeutic candidates targeting cancer and genetic diseases. These candidates are currently in preclinical or clinical development stages, and their sales potential is not included in the above estimates.
• The company's sales strategy is subject to ongoing refinement and may change over time based on market conditions and customer needs.

Precision BioSciences is a clinical-stage biotechnology company developing allogeneic CAR T and TCR therapies for the treatment of cancer and infectious diseases. The company's customer segments are as follows:

• Pharmaceutical and biotechnology companies: Precision BioSciences partners with pharmaceutical and biotechnology companies to develop and commercialize its CAR T and TCR therapies. These companies typically have a large sales force and marketing reach, which can help to accelerate the commercialization of Precision BioSciences' products.
• Hospitals and clinics: Hospitals and clinics are the end users of Precision BioSciences' products. These institutions typically have a large number of patients with cancer and infectious diseases, which can provide a significant market opportunity for Precision BioSciences.
• Patients: Patients are the ultimate beneficiaries of Precision BioSciences' products. The company's goal is to develop therapies that are safe and effective, and that can improve the lives of patients with cancer and infectious diseases.

Precision BioSciences' estimated annual sales are as follows:

• 2022: $50-$70 million
• 2023: $100-$150 million
• 2024: $200-$300 million

These estimates are based on a number of factors, including the company's current pipeline of products, its partnerships with pharmaceutical and biotechnology companies, and the market potential for its therapies.

Value Proposition of Precision BioSciences

Precision BioSciences is a clinical-stage biotechnology company dedicated to developing innovative gene editing therapies based on its proprietary ARCUS genome editing platform. The company's value proposition is centered around:

1. ARCUS Genome Editing Platform:

• Precision: ARCUS enables highly precise and efficient editing of specific genes, reducing off-target effects and increasing therapeutic efficacy.
• Versatility: The platform can be applied to a wide range of genetic targets, including insertions, deletions, and point mutations in both DNA and RNA.
• Scalability: ARCUS can be scaled up for commercial manufacturing of gene editing therapies, ensuring cost-effectiveness and accessibility.

2. Clinical Pipeline:

• Therapeutic Focus: Precision BioSciences is developing a pipeline of gene editing therapies targeting severe genetic diseases, such as sickle cell disease, beta-thalassemia, and muscular dystrophy.
• Early-Stage Success: The company has demonstrated clinical proof-of-concept with its lead candidate, PBCAR06, for sickle cell disease, showing significant reductions in disease severity.
• Broad Portfolio: The pipeline includes multiple candidates in clinical development, addressing a range of genetic disorders.

3. Intellectual Property:

• Proprietary Platform: ARCUS is a novel and proprietary genome editing technology protected by a substantial patent portfolio.
• IP Protection: The company's intellectual property provides a strong competitive advantage and positions it as a leader in the gene editing field.

4. Experienced Team:

• Scientific Expertise: Precision BioSciences has assembled a team of leading scientists and researchers with deep expertise in gene editing and genetic diseases.
• Industry Experience: The company's leadership has a proven track record of success in the biotechnology industry.

5. Commitment to Patients:

• Addressing Unmet Medical Needs: Precision BioSciences focuses on developing therapies for rare and severe genetic diseases, where current treatment options are limited.
• Patient-Centric Approach: The company engages with patient advocacy groups and researchers to understand disease needs and ensure that its therapies meet the expectations of patients.

Target Market:

Precision BioSciences targets the global market for genetic medicines, which is estimated to reach over $50 billion by 2025. The company primarily focuses on:

• Patients with rare and severe genetic diseases
• Pharmaceutical and biotechnology companies seeking to partner or license gene editing technologies

Competitive Landscape:

The biotechnology industry is highly competitive, with several other companies developing gene editing therapies. However, Precision BioSciences differentiates itself through its proprietary ARCUS platform, clinical pipeline, and commitment to patient-centric solutions.

Risks Associated with Precision BioSciences

Business Risks:

• Clinical Trial Risks: The success of Precision BioSciences depends heavily on the successful completion of clinical trials. Delays, adverse events, or failure to achieve desired results can significantly impact the company's financial and operational performance.
• Competition: Precision BioSciences operates in a highly competitive industry. Strong competition from established biopharmaceutical companies could limit market share and revenue growth.
• Regulatory and Policy Risks: The biotech industry is heavily regulated by government agencies. Changes in regulations or policies could impact Precision BioSciences' ability to develop and market its products.
• Intellectual Property: Precision BioSciences relies on patents and other intellectual property protections for its technologies. Challenges to the company's IP could result in loss of competitive advantage.
• Manufacturing and Supply Chain Risks: Production and distribution of Precision BioSciences' therapies require complex manufacturing and supply chain processes. Disruptions or delays in these processes could impact product availability and revenue.

Technical Risks:

• Technology Platform Limitations: Precision BioSciences' therapies use a novel gene editing platform. Technical limitations or unexpected issues with the platform could impact the effectiveness and safety of the treatments.
• Genome Editing Off-Target Effects: Gene editing techniques can lead to unintended modifications in the genome. Off-target effects could result in safety concerns or limit the efficacy of therapies.
• Vector-Related Risks: Precision BioSciences' therapies use viral vectors to deliver gene editing components. Vector-related risks include immune responses, insertional mutagenesis, and potential germline transmission.

Financial Risks:

• Research and Development Costs: Precision BioSciences incurs significant R&D expenses to develop and test new therapies. Failure to secure sufficient funding could delay or limit product development.
• Limited Revenue and Cash Flow: The company currently generates limited revenue and may face significant operating losses until its therapies are approved and commercialized.
• Debt and Financing Risks: Precision BioSciences relies on debt financing to fund operations. A high level of debt could impact the company's financial flexibility and increase interest expenses.

Other Risks:

• Regulatory Scrutiny: Gene editing technologies are subject to ongoing ethical and regulatory debates. Increased scrutiny could delay or limit the approval and commercialization of Precision BioSciences' therapies.
• Public Perception: The use of gene editing for medical treatments can be controversial. Negative public perception could impact the demand for Precision BioSciences' therapies.
• Political and Economic Volatility: Political and economic factors can affect the biotech industry and impact the company's ability to operate and generate revenue.