Core Insights - Taysha Gene Therapies, Inc. reported progress in the development of TSHA-102, a gene therapy for Rett syndrome, with positive clinical data and FDA engagement [1][2][3] Clinical Development - TSHA-102 was well tolerated in patients, with no serious adverse events (SAEs) or dose-limiting toxicities (DLTs) reported in both adolescent/adult and pediatric cohorts [1][4] - The FDA approved the pivotal product for use in REVEAL trials based on successful analytical comparability [2][3] - Clinical data from both low and high dose cohorts of the REVEAL trials are expected to be reported in the first half of 2025 [1][5] Regulatory Engagement - Advanced discussions with the FDA regarding trial design, endpoints, and the use of a natural history dataset for Part B of the REVEAL trials have been established [2][3] - The company has aligned with the FDA on a meeting cadence to expedite the development plan for TSHA-102 [2][3] Financial Performance - Research and development expenses for Q3 2024 were $14.9 million, an increase from $11.8 million in Q3 2023, primarily due to GMP batch activities [5][6] - General and administrative expenses decreased to $7.9 million in Q3 2024 from $8.6 million in Q3 2023 [6] - The net loss for Q3 2024 was $25.5 million, significantly reduced from a net loss of $117.1 million in Q3 2023 [7] Cash Position - As of September 30, 2024, Taysha had $157.7 million in cash and cash equivalents, expected to support operations into Q4 2026 [8] 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 [10][12]

Core Insights - Taysha Gene Therapies, Inc. is advancing AAV-based gene therapies for severe monogenic diseases of the central nervous system (CNS) and has presented biodistribution data from studies on AAV9 gene therapy vectors in non-human primates (NHPs) [1][2][5] - The analysis indicates that intrathecal (IT) administration is an effective, safe, and minimally invasive method for delivering gene therapies targeting CNS diseases, including Rett syndrome [1][2][3] Company Overview - Taysha Gene Therapies is a clinical-stage biotechnology company focused on developing AAV-based gene therapies for severe CNS diseases, with its lead program TSHA-102 targeting Rett syndrome [5] - TSHA-102 is designed as a one-time treatment to deliver a functional form of the MECP2 gene to address the genetic root cause of Rett syndrome [3][4] - The company has received multiple designations from regulatory agencies, including Regenerative Medicine Advanced Therapy and Fast Track designations from the FDA [3] Research Findings - The analysis of five NHP studies showed that both IT and intra-cisterna magna (ICM) administration resulted in comparable biodistribution of AAV9 vectors across brain and spinal cord regions [1][2] - The findings support the clinical potential of IT administration for outpatient use in both children and adults, reinforcing Taysha's clinical development strategy [2] Rett Syndrome Context - Rett syndrome is a rare neurodevelopmental disorder primarily affecting females, characterized by loss of communication and motor functions, with no approved disease-modifying therapies currently available [4] - It is estimated that Rett syndrome affects between 15,000 and 20,000 patients in the U.S., EU, and U.K. due to mutations in the MECP2 gene [4]

Core Viewpoint - Taysha Gene Therapies, Inc. (TSHA) has received an upgrade to a Zacks Rank 1 (Strong Buy), indicating a positive outlook based on rising earnings estimates, which significantly influence stock prices [1][4]. Earnings Estimates and Revisions - The Zacks Consensus Estimate for Taysha Gene Therapies indicates an expected loss of -$0.35 per share for the fiscal year ending December 2024, reflecting a year-over-year change of 63.5% [9]. - Over the past three months, the Zacks Consensus Estimate for the company has increased by 16.8%, showcasing a trend of rising earnings estimates [9]. Zacks Rating System - The Zacks rating system is based on changes in earnings estimates, which are crucial for predicting near-term stock price movements [2][3]. - The system classifies stocks into five groups, with Zacks Rank 1 stocks historically generating an average annual return of +25% since 1988 [8]. - Taysha Gene Therapies' upgrade to Zacks Rank 1 places it in the top 5% of Zacks-covered stocks, indicating strong potential for market-beating returns in the near term [11]. Market Implications - Rising earnings estimates and the corresponding rating upgrade suggest an improvement in Taysha Gene Therapies' underlying business, likely leading to increased buying pressure and a higher stock price [6][4]. - The influence of institutional investors, who adjust their valuations based on earnings estimates, contributes to stock price movements, reinforcing the importance of earnings revisions [5].

Financial Data and Key Metrics Changes - Research and development expenses decreased to $15.1 million for Q2 2024 from $19.8 million in Q2 2023, primarily due to a milestone fee payable in the prior year [18] - General and administrative expenses increased to $7.3 million in Q2 2024 from $6 million in Q2 2023, attributed to higher noncash stock-based compensation and consulting fees [19] - 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 [19] - As of June 30, 2024, the company had $172.7 million in cash and cash equivalents, with expectations to support operations into Q4 2026 [19] Business Line Data and Key Metrics Changes - The TSHA-102 program for Rett Syndrome showed promising safety and efficacy data from the low dose cohort in ongoing clinical trials [7][11] - The company initiated the high dose cohort and expanded its pediatric trial into Canada, indicating progress in clinical evaluations [7][10] Market Data and Key Metrics Changes - Rett Syndrome affects an estimated 15,000 to 20,000 patients in the U.S., EU, and UK, with no approved disease-modifying therapies currently available [8] Company Strategy and Development Direction - The company aims to develop a transformative therapeutic option for all patients suffering from Rett Syndrome, focusing on clinical trial execution and data collection [8][21] - The strategy includes advancing to high dose cohorts based on encouraging preliminary data from low dose cohorts, which is expected to inform regulatory discussions [12][21] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the safety profile and clinical effects of TSHA-102, emphasizing the potential to address significant unmet medical needs in Rett Syndrome [20][21] - The company plans to report safety and efficacy data from both low and high dose cohorts in the first half of 2025, indicating a commitment to transparency and data-driven decision-making [14][21] Other Important Information - The company completed a public follow-on offering, resulting in net proceeds of $76.8 million, which is expected to extend the cash runway into Q4 2026 [13] Q&A Session Summary Question: How will the high-dose data be measured for dose response? - Management indicated that preclinical data showed improvements at high doses and that consistent effects across clinical domains are expected [22] Question: What is the status of regulatory discussions? - The company has an upcoming Type B meeting with regulatory authorities to align on trial design and data sharing [26] Question: Will the timeline for data impact the timeline for FDA meetings? - Management stated that they aim to complete dosing and natural history analysis before engaging with the FDA, with flexibility in the timeline [42] Question: What are the baseline characteristics of high-dose patients? - Management noted that baseline characteristics are consistent with Rett Syndrome, with common clinical features expected across patients [48] Question: Are there any updates on safety events related to the immunosuppressive regimen? - Management confirmed that there have been no new significant adverse events reported and that discussions regarding the dosing stagger are ongoing [52]

Taysha Gene Therapies, Inc. (TSHA) came out with a quarterly loss of $0.09 per share versus the Zacks Consensus Estimate of a loss of $0.10. This compares to loss of $0.38 per share a year ago. These figures are adjusted for non-recurring items. This quarterly report represents an earnings surprise of 10%. A quarter ago, it was expected that this company would post a loss of $0.11 per share when it actually produced a loss of $0.10, delivering a surprise of 9.09%. Over the last four quarters, the company ha ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2024 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ___________ to ___________ Commission File Number: 001-39536 Taysha Gene Therapies, Inc. (Exact Name of Registrant as Specified in its Charter) Delaware 84-31995 ...

Exhibit 99.1 Taysha Gene Therapies Reports Second Quarter 2024 Financial Results and Provides Corporate Update Presented cohort one (low dose) data from both trials of TSHA-102 at 2024 IRSF Rett Syndrome Scientific Meeting that demonstrated durable improvements across consistent clinical domains and an encouraging safety profile in adult (up to 52 weeks) and pediatric (up to 22 weeks) patients TSHA-102 was generally well tolerated with no SAEs or DLTs as of initial six-week assessment in first patient in co ...

Presented cohort one (low dose) data from both trials of TSHA-102 at 2024 IRSF Rett Syndrome Scientific Meeting that demonstrated durable improvements across consistent clinical domains and an encouraging safety profile in adult (up to 52 weeks) and pediatric (up to 22 weeks) patients TSHA-102 was generally well tolerated with no SAEs or DLTs as of initial six-week assessment in first patient in cohort two (high dose) of adolescent/adult trial; IDMC approved dosing of second (adolescent/adult trial) and fir ...

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 ...