Crispr Therapeutics Ag 10-K Filing

Ticker: CRSP · Form: 10-K · Filed: Feb 11, 2025 · CIK: 1674416

Sentiment: neutral

Filing Stats: 4,383 words · 18 min read · ~15 pages · Grade level 17 · Accepted 2025-02-11 16:10:29

Filing Documents

Business

Business 1 Item 1A.

Risk Factors

Risk Factors 42 Item 1B. Unresolved Staff Comments 90 Item 1C. Cybersecurity 90 Item 2.

Properties

Properties 91 Item 3.

Legal Proceedings

Legal Proceedings 92 Item 4. Mine Safety Disclosures 92 PART II Item 5. Market for Registrant's Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities 93 Item 6. Reserved 96 Item 7.

Management's Discussion and Analysis of Financial Condition and Results of Operations

Management's Discussion and Analysis of Financial Condition and Results of Operations 96 Item 7A.

Quantitative and Qualitative Disclosures About Market Risk

Quantitative and Qualitative Disclosures About Market Risk 107 Item 8.

Financial Statements and Supplementary Data

Financial Statements and Supplementary Data 107 Item 9. Changes in and Disagreements with Accountants on Accounting and Financial Disclosure 107 Item 9A.

Controls and Procedures

Controls and Procedures 107 Item 9B. Other Information 110 Item 9C. Disclosure Regarding Foreign Jurisdictions that Prevent Inspections 110 PART III Item 10. Directors, Executive Officers and Corporate Governance 111 Item 11.

Executive Compensation

Executive Compensation 111 Item 12.

Security Ownership of Certain Beneficial Owners and Management and Related Stockholder Matters

Security Ownership of Certain Beneficial Owners and Management and Related Stockholder Matters 111 Item 13. Certain Relationships and Related Transactions, and Director Independence 111 Item 14. Principal Accountant Fees and Services 111 PART IV Item 15. Exhibits and Financial Statement Schedules 112 Item 16. Form 10-K Summary 115 i Risk Factor Summary Our business is subject to a number of risks and uncertainties of which you should be aware before making an investment decision in our business. These risks are discussed more fully in the "Risk Factors" section of this Annual Report on Form 10-K. These risks include, but are not limited to, the following: We have incurred significant operating losses since our inception and anticipate that we will incur continued losses for the foreseeable future. We will need to raise substantial additional funding, which will dilute our shareholders. If we are unable to raise capital when needed, we would be forced to delay, reduce or eliminate some of our product development programs or commercialization efforts. If we are unable to advance our product candidates to clinical development, obtain regulatory approval and ultimately commercialize our product candidates, or experience significant delays in doing so, our business will be materially harmed. Our CRISPR/Cas9 gene editing product candidates are based on a relatively new gene editing technology, which makes it difficult to predict the time and cost of development and of subsequently obtaining regulatory approval, if at all. There have only been a limited number of clinical trials of product candidates based on gene editing technology. The U.S. Food and Drug Administration, or FDA, the National Institutes of Health, the Medicines and Healthcare products Regulatory Agency, or MHRA and the European Medicines Agency, or EMA, have demonstrated caution in their regulation of gene therapy treatments, and ethical and legal concerns about

BUSINESS

BUSINESS Overview Our mission is to create transformative gene-based medicines for serious human diseases. We are a leading gene editing company focused on the development of CRISPR-based therapeutics, including by using CRISPR/Cas9 technology. CRISPR/Cas9 is a revolutionary technology for gene editing, the process of precisely altering specific sequences of genomic DNA. We aim to apply this technology to disrupt, delete, correct and insert genes to treat genetic diseases and to engineer advanced cellular therapies. We have advanced this technology from discovery to an approved medicine with unparalleled speed, culminating in the landmark first approval of a CRISPR-based therapy, CASGEVY (exagamglogene autotemcel [exa-cel]), in 2023 with our collaborators at Vertex Pharmaceuticals Incorporated, or Vertex. The use of CRISPR/Cas9 for gene editing was co-invented by one of our scientific founders, Dr. Emmanuelle Charpentier, the Acting and Founding Director of the Max Planck Unit for the Science of Pathogens in Berlin, Germany. Dr. Charpentier and her collaborators published work elucidating the mechanism by which the Cas9 endonuclease, a key component of CRISPR/Cas9, can be programmed to cut double-stranded DNA at specific locations. Dr. Charpentier and her collaborator, Dr. Jennifer Doudna of the University of California, Berkeley, shared the 2020 Nobel Prize in Chemistry for their groundbreaking work. We acquired exclusive rights to intellectual property encompassing CRISPR/Cas9 and related technologies from Dr. Charpentier and continue to strengthen our intellectual property estate through our own research and additional in-licensing efforts, furthering our leadership in gene editing therapeutics. We have established a portfolio of therapeutic programs spanning four core franchises: hemoglobinopathies, CAR T, in vivo approaches and type 1 diabetes. Depending on the program, we take either an ex vivo approach, in which we edit cells outside of the human body

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