Core Insights - Taysha Gene Therapies, Inc. reported promising preliminary data from its TSHA-102 clinical program for Rett syndrome, showing durable improvements and a favorable safety profile in both adult and pediatric patients [1][2][4] - The company successfully dosed the first patient in the high dose cohort of its adolescent/adult trial and received approval to proceed with additional dosing in both adolescent/adult and pediatric trials [1][2][5] - Taysha completed a public follow-on offering, raising $76.8 million, which extends its cash runway into Q4 2026 [1][3][10] Clinical Program Updates - TSHA-102 demonstrated sustained improvements in clinical domains such as motor skills and communication in patients from cohort one of the trials [4][6] - The first patient in cohort two (high dose) was dosed, and initial assessments indicated no serious adverse events or dose-limiting toxicities [5][6] - Data from both cohorts of the adolescent/adult and pediatric trials are expected in the first half of 2025 [1][7] Financial Highlights - Research and development expenses decreased to $15.1 million in Q2 2024 from $19.8 million in Q2 2023, primarily due to a prior milestone fee [8] - General and administrative expenses increased to $7.3 million in Q2 2024 from $6.0 million in Q2 2023, attributed to higher stock-based compensation and consulting fees [9] - The net loss for Q2 2024 was $20.9 million, or $0.09 per share, compared to a net loss of $24.6 million, or $0.38 per share, in Q2 2023 [10][16] Cash Position - As of June 30, 2024, Taysha had $172.7 million in cash and cash equivalents, supporting operations into Q4 2026 [10][17] Company Overview - Taysha Gene Therapies focuses on AAV-based gene therapies for severe monogenic diseases, with TSHA-102 as its lead program targeting Rett syndrome [12][14] - The company has received multiple designations from regulatory authorities, including Regenerative Medicine Advanced Therapy and Fast Track designations from the FDA [12]
Taysha Gene Therapies Reports Second Quarter 2024 Financial Results and Provides Corporate Update