Risk Factors Update Summary

• The company's net loss increased significantly from $121.8 million in 2022 to $198.1 million in 2023.
• The company opted out all European patents from the UPC, potentially impacting patent protection.
• Proceeds from the IPO in October 2022 were $180.2 million, with net proceeds of $180.2 million.
• The company may seek rare orphan disease designation for some product candidates.
• Cash, cash equivalents, and investments were $121.7 million in 2023, compared to $135.2 million in 2022.
• Legislation may shorten the 10-year marketing exclusivity period for certain orphan medicines.
• Accumulated deficit increased from $293 million in 2022 to $491 million in 2023.
• The company may need to hire additional personnel to comply with requirements.
• The company transitioned from research to early preclinical development for potential product candidates.
• The company faces risks related to artificial intelligence use and potential security breaches.
• The company identified four areas of focus: blood, liver, eye, and neuromuscular indications.
• The company will be required to furnish a report on internal control over financial reporting.
• The company's cash and cash equivalents will fund operations into the third quarter of 2025.
• The company faces risks related to health epidemics and widespread disease outbreaks.