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]

DALLAS, June 26, 2024 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a clinicalstage biotechnology company focused on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system (CNS), today announced the pricing of an underwritten public offering of 14,361,113 shares of its common stock at a price to the public of $2.25 per share and, in lieu of common stock to certain investors, pre-funded warrants to purchase 18,972,221 shares of ...

Taysha Gene Therapies (NASDAQ:TSHA) stock is taking a beating on Wednesday after the gene therapy company announced a proposed public offering for its shares. What we do know is that it also intends to offer pre-funded warrants for certain investors. On top of that, underwriters have a 30-day option to acquire an additional 15% of shares included in the offering. How This Affects TSHA Stock On top of that, the pricing of the offering may upset investors. It's expected to be discounted compared to the compan ...

Exhibit 99.2 We are a clinical-stage biotechnology company focused on advancing AAV-based gene therapies for the treatment of severe monogenic diseases of the central nervous system, or CNS. Our lead clinical program TSHA-102 is in development for the treatment of Rett syndrome, a rare neurodevelopmental disorder with no approved disease-modifying therapies that address the genetic root cause of the disease. With a singular focus on developing transformative medicines, we aim to address severe unmet medical ...

Core Viewpoint - Taysha Gene Therapies, Inc. has initiated an underwritten public offering of up to $75 million in common stock and pre-funded warrants, subject to market conditions [1][2]. Company Overview - Taysha Gene Therapies is a clinical-stage biotechnology company focused on AAV-based gene therapies for severe monogenic diseases of the central nervous system [6]. - The company's lead clinical program, TSHA-102, is in development for Rett syndrome, a rare neurodevelopmental disorder with no approved disease-modifying therapies [6]. - Taysha aims to address severe unmet medical needs and improve the lives of patients and caregivers through transformative medicines [6]. Offering Details - The public offering includes a 30-day option for underwriters to purchase an additional 15% of the shares offered [1]. - Jefferies and Goldman Sachs & Co. LLC are acting as joint book-running managers for the offering, with Cantor also serving as a book-running manager [2].

Jefferies and Goldman Sachs & Co. LLC are acting as joint book-running managers for the offering, and Cantor is also serving as a book-running manager for the proposed offering. About Taysha Gene Therapies time to time with the SEC. These forward-looking statements speak only as of the date hereof, and Taysha disclaims any obligation to update these statements except as may be required by law. DALLAS, June 25, 2024 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a clinicalstage biotechnology ...

Durable improvements across consistent clinical domains in both adult and pediatric patients, including motor skills, communication/socialization, autonomic function, seizures, and an encouraging safety profile seen across adult (up to 52 weeks) and pediatric (up to 22 weeks) patients with different genetic mutation severity Initial data from first two pediatric patients showed improvements across multiple efficacy measures and clinical domains, with early evidence of developmental gains (patient one: impro ...

Company to host webcast on Tuesday, June 18, 2024, at 8:00 AM Eastern Time Date/Time: Tuesday, June 18, 2024, at 8:00 AM Eastern Time Registration: https://lifescievents.com/event/taysha-2/ The live webcast and replay will be available through Taysha's website here. About Taysha Gene Therapies Media Contact: Carolyn Hawley Taysha-Hosted Webcast Details Oral Presentation Title: TSHA-102 gene therapy for Rett syndrome: First-cohort data from the REVEAL Adolescent/Adult and Pediatric studies Date/Time: Wednesd ...

DALLAS, June 07, 2024 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a clinicalstage biotechnology company focused on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system (CNS), today announced that, on June 3, 2024, the Compensation Committee of Taysha's Board of Directors granted three new employees, in the aggregate, options to purchase 360,000 shares of the Company's common stock in connection with their employment. The ...

Financial Data and Key Metrics Changes - The net loss for the three months ended March 31, 2024, was $24.1 million or $0.10 per share, compared to a net loss of $17.6 million or $0.28 per share for the same period in 2023, indicating an increase in losses [90] - Research and development expenses were $20.7 million for the three months ended March 31, 2024, compared to $12.5 million for the same period in 2023, reflecting increased clinical trial activities [108] - As of March 31, 2024, the company had $124 million in cash and cash equivalents, which is expected to support planned operating expenses and capital requirements into 2026 [110] Business Line Data and Key Metrics Changes - The company is focused on the TSHA-102 program, which is in clinical evaluation for the treatment of Rett Syndrome, with ongoing Phase I/II REVEAL trials for both adolescent/adult and pediatric populations [21][24] - Encouraging longer-term data was reported for the first two adult patients treated in the low-dose cohort, showing sustained improvements across key efficacy measures [26][28] - The second pediatric patient has been dosed in the low-dose cohort, with initial data expected to be reported in mid-2024 [29][86] Market Data and Key Metrics Changes - Rett Syndrome affects an estimated 15,000 to 20,000 patients in the United States, European Union, and the United Kingdom, with no approved disease-modifying therapies currently available [42] - The company received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA, which facilitates closer collaboration and expedited development processes [31][70] Company Strategy and Development Direction - The company aims to generate critical longer-term clinical data across a broad range of ages and stages of patients with Rett Syndrome, guiding the next phase of studies [22][51] - The strategy includes expanding clinical trials into pediatric populations and evaluating the high dose of TSHA-102 across different age groups [51][62] - The company is committed to working closely with the FDA and other regulatory agencies to advance the TSHA-102 program [51] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism regarding the therapeutic potential of TSHA-102, highlighting the sustained improvements observed in adult patients and the potential for similar outcomes in pediatric patients [41][82] - The management acknowledged the high unmet medical need in Rett Syndrome and the importance of their gene therapy approach in addressing this challenge [42][70] - Future updates are anticipated, including initial clinical data from the pediatric trial and further insights from the completed low-dose cohort of the adolescent and adult trial [71][92] Other Important Information - The company is currently enrolling patients in both the adolescent/adult and pediatric trials, with a focus on diverse genetic backgrounds to evaluate the clinical impact of TSHA-102 [63][69] - The independent data monitoring committee approved the request to proceed to an early dose escalation in the adolescent and adult trial [25] Q&A Session Summary Question: What are the expectations for younger patients in the trial? - Management indicated that while the pediatric population is heterogeneous, they expect to see consistent clinical impacts across multiple domains similar to adult patients, with a focus on gross and fine motor function, socialization, and seizure improvement [95][113] Question: Can you comment on the RMAT designation and the data provided to the FDA? - The RMAT designation was granted based on data from two adult patients and one pediatric patient, indicating significant clinical impact and unmet medical needs [115][119] Question: How is Astellas involved in data sharing? - The company provides quarterly updates to Astellas, and they are involved in regulatory interactions, ensuring transparency regarding data generation and strategic aspects of the program [120] Question: When can we expect initial pediatric data? - Initial pediatric data is expected to be presented at the Rett conference on June 19, with plans for a webcast for investors [125] Question: What are the advantages of intrathecal administration compared to competitors? - Management highlighted that preclinical and clinical data support the effectiveness of TSHA-102 via intrathecal administration, showing significant impacts on various clinical domains [130]