Risk Factors Update Summary

• Received first-ever marketing approval of CRISPR-based gene editing therapy, CASGEVY, in 2023 and subsequent approval in 2024. This may impact future marketing approvals.
• Added internal manufacturing capabilities and own cell therapy manufacturing facility to reduce reliance on outside vendors.
• Enhanced product liability claims section with detailed scenarios and potential consequences, emphasizing financial risks.
• Cash, cash equivalents, and marketable securities decreased from $2,379.1 million in 2022 to $1,695.7 million in 2023.
• Increased shareholder "say on pay" vote frequency from biennial to annual.
• Highlighted risks related to intellectual property, including opposition proceedings and potential impacts on patent protection.
• Addressed risks related to evolving European privacy laws, emphasizing GDPR compliance challenges and potential penalties.
• Research focus shifted to developing next-generation product candidates, investing substantially in CRISPR/Cas9 gene editing.
• Expanded product liability risks, including potential lawsuits and claims, with increased commercialization.
• Noted the importance of obtaining and protecting patents in the biopharmaceutical industry amidst legal complexities.
• Transitioned from a company capable of supporting clinical development to developing, manufacturing, and commercializing product candidates.
• Limited operating history may hinder the successful advancement of product candidates to clinical development and regulatory approval.
• Uncertainty in obtaining regulatory approvals for product candidates may impact operations and commercialization efforts.
• Reliance on Vertex for manufacturing and commercialization of CASGEVY may affect revenues and financial results.