Risk Factors Update Summary

• Transition from gene therapy to biotechnology focus. This shift may impact product development strategies.
• Entered a second collaboration agreement with Neurocrine for gene therapy products, including GBA1 Program. This collaboration may lead to additional responsibilities and expenses.
• Net income increased from $132.3 million to $261 million. This significant rise is noteworthy.
• Pfizer exercised an option for a capsid for a rare neurological disease, leading to a payment of $25 million.
• Signed the 2023 Novartis Collaboration Agreement for AAV gene therapy products. Novartis will solely develop and commercialize products under the Novartis SMA and HD Programs.
• Added reference to "Internal Revenue" with potential impact on tax implications.
• Accumulated deficit decreased from $393 million to $261 million. This reduction is positive.
• Proceeds from public offering in January 2024 totaled $93.5 million. This affects financial resources.
• Completed a private placement and public offering of common stock, totaling 2,145,002 shares and 7,777,778 shares respectively.
• Increased mention from Section 104 to Section 110, indicating a specific regulatory change.
• Cash, cash equivalents, and marketable securities increased from $118 million to $230 million.
• Increased NOL carryforwards to $175 million federal and $166 million state, with state NOLs expiring from 2033 to 2041.
• Upfront payment received in January 2024 of $80 million. This payment impacts financial stability.
• Potential legal challenges from Touchlight regarding intellectual property rights related to TRACER Capsids.