Group 1 - The article discusses Taysha Gene Therapies, Inc. (NASDAQ: TSHA) and its recent developments, particularly focusing on initial data related to Rett Syndrome and upcoming catalysts in 2024 [2]. - The author operates the Biotech Analysis Central service, which provides in-depth analysis of various pharmaceutical companies and includes a library of over 600 biotech investing articles [2]. - The service offers a model portfolio of more than 10 small and mid-cap stocks, along with live chat and analysis to assist healthcare investors in making informed decisions [2].

Core Insights - Taysha Gene Therapies Inc. announced details of its pivotal Part B trial design for TSHA-102 after receiving alignment from the FDA [1] - The company revealed clinical data from Part A of the REVEAL Phase 1/2 trials for TSHA-102 in treating Rett syndrome, a rare genetic disorder [2] Trial Design and Data - The pivotal Part B trial will include a single-arm, open-label design with an intended enrollment of 15 female patients aged 6 years and older [6] - The primary endpoint will assess the gain or regain of developmental milestones, while safety will be evaluated in a younger cohort aged 2-6 years [6] - The FDA advised the company to submit the trial protocol as an amendment to its IND application, streamlining the process [6] Clinical Efficacy - Efficacy data showed that 100% of patients gained at least one developmental milestone across key functional domains post-treatment with TSHA-102 [7] - A total of 22 developmental milestones were achieved among 10 patients, with significant early gains observed [7] - The high-dose cohort demonstrated a 100% responder rate 25% faster than the low-dose cohort, indicating accelerated functional benefits [7] Safety Profile - Both high and low doses of TSHA-102 were generally well tolerated, with no serious adverse events or dose-limiting toxicities reported [7] - All treatment-emergent adverse events related to TSHA-102 were mild to moderate in severity [7] Financial Activity - Taysha Gene Therapies priced an underwritten public offering of 46.9 million shares at $2.75 per share, raising $200 million in gross proceeds [4] - The company's stock saw a 9% increase, reaching $2.66 [5]

Core Insights - Taysha Gene Therapies announced positive clinical data for TSHA-102, a gene therapy for Rett syndrome, showing a 100% responder rate in patients aged 6-21 years, with all patients gaining or regaining at least one developmental milestone post-treatment [1][2][5] - The FDA has provided written alignment on the pivotal Part B trial design for TSHA-102, which is expected to begin in Q3 2025 [1][4][12] - The analysis of natural history data indicates that patients aged six years and older are in a developmental plateau, with a likelihood of gaining new milestones being exceedingly low without treatment [1][6] Company Overview - Taysha Gene Therapies is a clinical-stage biotechnology company focused on AAV-based gene therapies for severe monogenic diseases of the CNS, with TSHA-102 as its lead program targeting Rett syndrome [8][10] - TSHA-102 is designed as a one-time treatment that delivers a functional form of the MECP2 gene to address the genetic root cause of Rett syndrome [8][9] Clinical Trial Details - The pivotal Part B trial will be a single-arm, open-label study with an intended enrollment of 15 patients, assessing developmental milestone gain/regain [1][6] - The primary endpoint of the trial is the gain or regain of at least one defined developmental milestone, with independent central raters evaluating outcomes based on video evidence [6] - Safety data from the ongoing REVEAL Phase 1/2 trials indicated no treatment-related serious adverse events or dose-limiting toxicities [1][5][6] Efficacy and Safety Findings - In the REVEAL Part A trial, 100% of patients treated with TSHA-102 achieved developmental milestones, with high-dose cohorts showing faster and more pronounced improvements compared to low-dose cohorts [1][5][6] - Improvements were observed across multiple clinician-assessed outcome measures, including the Revised Motor Behavior Assessment and Clinician Global Impression – Improvement [6] Future Plans - The company plans to submit the pivotal trial protocol and statistical analysis plan as an amendment to the IND application within the current quarter [2][12] - Taysha anticipates initiating pivotal trial activities in Q3 2025, following FDA guidance [1][12]

Financial Performance - Taysha Gene Therapies reported a quarterly loss of $0.08 per share, consistent with the Zacks Consensus Estimate, compared to a loss of $0.10 per share a year ago [1] - The company posted revenues of $2.3 million for the quarter ended March 2025, exceeding the Zacks Consensus Estimate by 9.62%, but down from $3.41 million year-over-year [2] - Over the last four quarters, Taysha Gene Therapies has surpassed consensus EPS estimates two times and topped consensus revenue estimates two times [2][1] Stock Performance - Taysha Gene Therapies shares have increased approximately 37% since the beginning of the year, significantly outperforming the S&P 500's gain of 0.2% [3] - The stock currently holds a Zacks Rank 3 (Hold), indicating it is expected to perform in line with the market in the near future [6] Earnings Outlook - The current consensus EPS estimate for the upcoming quarter is -$0.08 on revenues of $1.93 million, and for the current fiscal year, it is -$0.34 on revenues of $7.96 million [7] - The estimate revisions trend for Taysha Gene Therapies is mixed, which may change following the recent earnings report [6] Industry Context - The Medical - Generic Drugs industry, to which Taysha Gene Therapies belongs, is currently ranked in the bottom 24% of over 250 Zacks industries, indicating potential challenges ahead [8]

Financial Data and Key Metrics Changes - Research and development expenses decreased to $15.6 million for Q1 2025 from $20.7 million in Q1 2024, a reduction of $5.1 million primarily due to good manufacturing practices batch activities [22] - General and administrative expenses increased to $8.2 million for Q1 2025 from $7.1 million in Q1 2024, an increase of $1.1 million mainly due to higher compensation and legal fees [22] - Net loss for Q1 2025 was $21.5 million or $0.08 per share, compared to a net loss of $24.1 million or $0.10 per share in Q1 2024 [23] Business Line Data and Key Metrics Changes - The TATIA-102 gene therapy program for Rett syndrome has shown significant regulatory and clinical progress, with alignment from the FDA on key elements of the pivotal trial design [8][11] - The REVEAL Phase 1/2 trials have reported clinical improvements in patients, including functional gains in fine motor, gross motor, and socialization skills [10][18] Market Data and Key Metrics Changes - The company has maintained ongoing discussions with the FDA, which have been productive and supportive of the development approach for TATIA-102 [11][20] - The potential market opportunity for TATIA-102 is significant, given the unmet medical need in the Rett syndrome patient population [16] Company Strategy and Development Direction - The company is focused on advancing the TATIA-102 gene therapy program towards registration, with plans to submit the pivotal trial protocol and statistical analysis plan in the current quarter [11][24] - The strategy includes leveraging existing clinical sites for rapid enrollment in the pivotal trial, with a focus on minimizing the time to market [66] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the regulatory pathway and the potential for TATIA-102 to provide meaningful benefits to patients with Rett syndrome [13][24] - The company anticipates initiating site activities for the pivotal trial as early as summer 2025, with potential dosing in Q4 2025 or Q1 2026 [93] Other Important Information - The company plans to provide updates on the pivotal trial design and clinical data at the International Rett Syndrome Foundation's scientific meeting in June 2025 [13][24] - The company has $116.6 million in cash and cash equivalents as of March 31, 2025, which is expected to support operations into Q4 2026 [23] Q&A Session Summary Question: What signals can potentially point to a dose response? - Management indicated that temporal aspects and qualitative metrics like RMBA and CGI could help assess dose response, with expectations of differentiation at higher doses [26][27] Question: Can you frame the bar for success regarding recent regulatory feedback? - Management emphasized the importance of natural history analysis and alignment with the FDA on trial design and endpoints, which will provide insights into the probability of success [32][35] Question: Is there any specific feedback from the FDA post-protocol submission? - Management confirmed that they have reached alignment with the FDA on key aspects of the trial design, minimizing the likelihood of significant further comments [41][42] Question: What will the primary endpoint include? - The primary endpoint is expected to be objective and clinically meaningful, focusing on gains of function or restoration of lost function [45][49] Question: How does the aligned design compare with the initial proposal? - Management stated that the FDA endorsed the design proposed by the company, with no significant pushback during discussions [52][56] Question: What is the timeline for Part B enrollment and study completion? - Management indicated that if the protocol submission goes smoothly, site activities could begin shortly after, with potential dosing starting in late 2025 or early 2026 [93] Question: Can you characterize the confidence in TATIA-102's safety? - Management reported no treatment-emergent adverse events or dose-limiting toxicities to date, indicating a favorable safety profile [105]

Financial Data and Key Metrics Changes - Research and development expenses decreased to $15.6 million for Q1 2025 from $20.7 million in Q1 2024, a reduction of $5.1 million primarily due to lower GMP batch activities [18] - General and administrative expenses increased to $8.2 million for Q1 2025 from $7.1 million in Q1 2024, an increase of $1.1 million mainly due to higher compensation and legal fees [18] - Net loss for Q1 2025 was $21.5 million or $0.08 per share, compared to a net loss of $24.1 million or $0.10 per share in Q1 2024 [19] Business Line Data and Key Metrics Changes - The TATIA-one hundred two program has shown significant regulatory and clinical progress, supporting a clear path to registration [7][10] - The REVEAL Phase one/two trials for TATIA-one hundred two have reported clinical improvements in patients, including functional gains in fine motor, gross motor, and socialization skills [9][16] Market Data and Key Metrics Changes - The company has maintained ongoing discussions with the FDA, which have been productive and supportive of the development approach for TATIA-one hundred two [10][82] - The company is preparing to engage with EMA regarding requirements for potential expansion into European markets, indicating a significant patient population opportunity [66] Company Strategy and Development Direction - The company aims to finalize the protocols and statistical analysis plan for the pivotal Part B trial of TATIA-one hundred two, with an expected submission of the IND amendment in the current quarter [11][103] - The focus remains on advancing TATIA-one hundred two towards registration, with a clear path established through alignment with the FDA on trial design and endpoints [10][32] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the potential of TATIA-one hundred two to provide meaningful benefits to patients with Rett syndrome, emphasizing the importance of early clinical improvements [11][17] - The company anticipates initiating site activities for the pivotal trial as early as summer 2025, with potential dosing beginning in Q4 2025 or Q1 2026 [90][103] Other Important Information - The company has received written alignment from the FDA on key elements of the pivotal trial design, which eliminates the need for a formal end-of-phase meeting [10][56] - The upcoming IRSF Scientific Meeting will feature presentations related to TATIA-one hundred two, providing further updates on clinical data and trial design [11][103] Q&A Session Summary Question: What signals can potentially point to a dose response given the heterogeneity of Rett syndrome? - Management indicated that temporal aspects and qualitative metrics like RMBA and CGI could be used to assess dose response, with expectations of differentiation at higher doses [22][23] Question: Can you frame the bar for success in light of recent regulatory feedback? - Management highlighted the importance of natural history analysis and alignment with the FDA on trial design, endpoints, and statistical significance requirements [30][32] Question: Is there any specific feedback from the FDA post the thirty-day period on the study protocol? - Management confirmed that they have reached alignment with the FDA on key aspects of the trial design, minimizing the likelihood of significant further comments [39][40] Question: What will be the primary endpoint of the trial? - The primary endpoint is expected to be objective and clinically meaningful, focusing on gains of function or restoration of lost function [44][95] Question: How does the aligned design compare with the initial proposal? - Management stated that the design endorsed by the FDA was consistent with the company's initial proposal, with no significant pushback from the agency [50][52] Question: What is the timeline for Part B enrollment and study completion? - Management anticipates initiating site activities and potentially beginning dosing in Q4 2025 or Q1 2026 following the IND amendment [90][103] Question: Can you characterize the level of confidence in TATIA-one hundred two's safety? - Management reported no treatment-related serious adverse events or dose-limiting toxicities, indicating a strong safety profile for the therapy [100][101]

[PART I. FINANCIAL INFORMATION](index=3&type=section&id=PART%20I.%20FINANCIAL%20INFORMATION) [Condensed Consolidated Financial Statements (Unaudited)](index=3&type=section&id=Item%201.%20Condensed%20Consolidated%20Financial%20Statements%20(Unaudited)) This section presents Taysha Gene Therapies' unaudited condensed consolidated financial statements for Q1 2025, covering balance sheets, operations, and cash flows with notes [Condensed Consolidated Balance Sheets](index=3&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) Total assets decreased to **$138.4 million** from **$160.4 million** as of March 31, 2025, primarily due to reduced cash and cash equivalents Condensed Consolidated Balance Sheet Highlights (in thousands) | Account | March 31, 2025 | December 31, 2024 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $116,593 | $139,036 | | Total current assets | $120,708 | $142,130 | | **Total assets** | **$138,362** | **$160,364** | | **Liabilities & Equity** | | | | Total current liabilities | $22,557 | $26,227 | | Term loan, net | $42,453 | $43,942 | | **Total liabilities** | **$83,303** | **$88,839** | | **Total stockholders' equity** | **$55,059** | **$71,525** | | **Total liabilities and stockholders' equity** | **$138,362** | **$160,364** | [Condensed Consolidated Statements of Operations](index=4&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations) Q1 2025 revenue was **$2.3 million** with a net loss of **$21.5 million**, an improvement from Q1 2024's **$24.1 million** net loss, driven by reduced R&D Condensed Consolidated Statements of Operations (in thousands, except per share data) | Metric | Q1 2025 | Q1 2024 | | :--- | :--- | :--- | | Revenue | $2,302 | $3,411 | | Research and development | $15,565 | $20,657 | | General and administrative | $8,158 | $7,084 | | Total operating expenses | $23,723 | $27,741 | | Loss from operations | ($21,421) | ($24,330) | | **Net loss** | **($21,529)** | **($24,061)** | | Net loss per common share, basic and diluted | ($0.08) | ($0.10) | [Condensed Consolidated Statements of Cash Flows](index=7&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) Net cash used in operating activities increased to **$22.0 million** in Q1 2025, with total cash decreasing by **$22.4 million** to **$119.2 million** Condensed Consolidated Statements of Cash Flows Highlights (in thousands) | Cash Flow Activity | Q1 2025 | Q1 2024 | | :--- | :--- | :--- | | Net cash used in operating activities | ($22,020) | ($19,798) | | Net cash used in investing activities | ($371) | ($140) | | Net cash used in financing activities | ($52) | ($22) | | **Net decrease in cash, cash equivalents and restricted cash** | **($22,443)** | **($19,960)** | | Cash, cash equivalents and restricted cash at end of period | $119,193 | $126,580 | [Notes to Condensed Consolidated Financial Statements](index=8&type=section&id=Notes%20to%20Condensed%20Consolidated%20Financial%20Statements) Notes detail the company's business, accounting policies, liquidity, Astellas collaboration, term loan, stock compensation, and legal matters - The company is a clinical-stage biotechnology firm focused on AAV-based gene therapies for severe monogenic diseases of the central nervous system[24](index=24&type=chunk) - As of March 31, 2025, the company had cash and cash equivalents of **$116.6 million**, which management believes is sufficient to fund planned operations for at least the next twelve months[28](index=28&type=chunk) - Revenue is derived from the Astellas Option Agreement for the Rett syndrome program, with **$2.3 million** recognized in Q1 2025 from Rett research and development activities[65](index=65&type=chunk) - The company is a nominal defendant in a consolidated stockholder derivative lawsuit and has received subpoenas from the SEC, both related to its August 2023 private placement, with possible losses currently inestimable[131](index=131&type=chunk)[132](index=132&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=41&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses advancing TSHA-102 for Rett syndrome, Q1 2025 financial results with decreased R&D and revenue, and liquidity into Q4 2026 [Overview](index=41&type=section&id=Overview) Taysha is a clinical-stage biotech focused on AAV-based gene therapies, with lead program TSHA-102 for Rett syndrome in Phase 1/2 trials and key regulatory designations - The company's primary focus is advancing TSHA-102, a gene therapy for Rett syndrome, which is currently in REVEAL Phase 1/2 trials for both adolescent/adult and pediatric patients[142](index=142&type=chunk)[143](index=143&type=chunk) - TSHA-102 has received several key regulatory designations, including Orphan Drug, Rare Pediatric Disease, Fast Track, and Regenerative Medicine Advanced Therapy (RMAT) from the FDA, as well as ILAP designation in the U.K[145](index=145&type=chunk)[146](index=146&type=chunk) - As of March 31, 2025, the company had an accumulated deficit of **$623.8 million** and expects to incur significant operating losses for the foreseeable future[150](index=150&type=chunk) [Results of Operations](index=51&type=section&id=Results%20of%20Operations) Q1 2025 revenue decreased to **$2.3 million**, R&D expenses fell by **$5.1 million** to **$15.6 million**, and G&A expenses rose to **$8.2 million** Comparison of Results of Operations (in thousands) | Line Item | Q1 2025 | Q1 2024 | Change | | :--- | :--- | :--- | :--- | | Revenue | $2,302 | $3,411 | ($1,109) | | Research and development | $15,565 | $20,657 | ($5,092) | | General and administrative | $8,158 | $7,084 | $1,074 | | **Net loss** | **($21,529)** | **($24,061)** | **$2,532** | - The **$5.1 million** decrease in R&D expenses was primarily driven by the completion of GMP batch manufacturing activities for TSHA-102 in the prior year period, partially offset by increased headcount[183](index=183&type=chunk) [Liquidity and Capital Resources](index=52&type=section&id=Liquidity%20and%20Capital%20Resources) As of March 31, 2025, cash and cash equivalents totaled **$116.6 million**, expected to fund operations into Q4 2026, with funding from equity, Astellas, and a **$40.0 million** term loan - The company's cash and cash equivalents of **$116.6 million** as of March 31, 2025, are expected to provide a cash runway into the fourth quarter of 2026[188](index=188&type=chunk)[202](index=202&type=chunk) - In November 2023, the company secured a **$40.0 million** term loan from Trinity Capital, which is interest-only for 36 months (extendable to 48) and matures in November 2028[189](index=189&type=chunk) - The company will require substantial additional capital to fund the research, development, manufacturing, and potential commercialization of its product candidates[200](index=200&type=chunk)[202](index=202&type=chunk) [Quantitative and Qualitative Disclosures About Market Risk](index=61&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) As a smaller reporting company, Taysha is exempt from providing quantitative and qualitative disclosures about market risk - As a smaller reporting company, Taysha is exempt from providing quantitative and qualitative disclosures about market risk[220](index=220&type=chunk) [Controls and Procedures](index=61&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded that disclosure controls and procedures were effective as of March 31, 2025, with no material changes to internal control over financial reporting - The CEO and CFO concluded that the company's disclosure controls and procedures were effective as of March 31, 2025[221](index=221&type=chunk) - No changes in internal control over financial reporting occurred during the quarter that materially affected, or are reasonably likely to materially affect, internal controls[222](index=222&type=chunk) [PART II. OTHER INFORMATION](index=62&type=section&id=PART%20II.%20OTHER%20INFORMATION) [Legal Proceedings](index=62&type=section&id=Item%201.%20Legal%20Proceedings) The company is a nominal defendant in a stockholder derivative lawsuit and received SEC subpoenas related to its August 2023 Private Placement, with potential losses inestimable - The company is involved in a consolidated stockholder derivative lawsuit alleging breach of fiduciary duty by certain directors and officers related to the August 2023 Private Placement, with litigation currently stayed until September 30, 2025[225](index=225&type=chunk) - The SEC is conducting an investigation and issued subpoenas for materials related to the company's August 2023 PIPE and certain public offerings, with the company completing its production of materials in April 2025[226](index=226&type=chunk) [Risk Factors](index=62&type=section&id=Item%201A.%20Risk%20Factors) This section highlights risks from healthcare legislative and regulatory reforms, including the ACA and IRA, and increased scrutiny on drug pricing, potentially impacting profitability - The company's business is subject to risks from healthcare reform measures, such as the ACA and the Inflation Reduction Act (IRA), which could delay approvals, restrict activities, and affect profitability[229](index=229&type=chunk)[231](index=231&type=chunk) - There is heightened governmental scrutiny on pharmaceutical pricing, which has led to legislation and executive orders designed to increase transparency and control costs, potentially creating downward pressure on prices for any approved products[234](index=234&type=chunk) - Changes in regulations in key markets, such as the EU's new Clinical Trials Regulation (CTR), could impose additional costs, lengthen review times, and impact development plans[237](index=237&type=chunk) [Unregistered Sales of Equity Securities and Use of Proceeds](index=66&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) The company reported no unregistered sales of equity securities, no use of proceeds from registered securities, and no issuer purchases during the period - There were no unregistered sales of equity securities during the quarter[241](index=241&type=chunk) [Defaults Upon Senior Securities](index=66&type=section&id=Item%203.%20Defaults%20Upon%20Senior%20Securities) This item is not applicable as the company has not defaulted upon any senior securities - Not applicable[244](index=244&type=chunk) [Mine Safety Disclosures](index=66&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) This item is not applicable to the company's operations - Not applicable[245](index=245&type=chunk) [Other Information](index=66&type=section&id=Item%205.%20Other%20Information) No other information was reported for the period - None[246](index=246&type=chunk) [Exhibits](index=67&type=section&id=Item%206.%20Exhibits) This section lists exhibits filed with the quarterly report, including corporate governance documents, incentive plan amendments, and officer certifications - The exhibits filed with this report include certifications from the Principal Executive Officer and Principal Financial Officer as required by the Sarbanes-Oxley Act[248](index=248&type=chunk)

Exhibit 99.1 Taysha Gene Therapies Reports First Quarter 2025 Financial Results and Provides Corporate Update Obtained written alignment from the FDA on key elements of TSHA-102 pivotal Part B trial design and next steps on enabling study initiation The FDA advised the Company to submit pivotal Part B trial protocol and associated statistical analysis plan as an amendment to the IND application, eliminating the need for formal end-of-phase meeting and potentially expediting study initiation and registration ...

Core Insights - Taysha Gene Therapies has made significant regulatory and clinical progress with its TSHA-102 program for Rett syndrome, having reached alignment with the FDA on key elements of the pivotal Part B trial design [3][4] - The FDA has advised the company to submit the pivotal Part B trial protocol and statistical analysis plan as an amendment to the IND application, which is expected to expedite study initiation and registration [3][4] - TSHA-102 has shown to be generally well tolerated in patients, with no treatment-related serious adverse events or dose-limiting toxicities reported in the initial cohorts [4] Regulatory Progress - The company achieved alignment with the FDA on the pivotal Part B trial design for TSHA-102, following discussions under the Regenerative Medicine Advanced Therapy (RMAT) pathway [4] - The FDA's guidance allows Taysha to submit the trial protocol directly as an IND amendment, eliminating the need for a formal end-of-phase meeting, which may accelerate the process [3][4] Clinical Data - As of April 10, 2025, TSHA-102 has been administered to 10 patients (6 in the high dose cohort and 4 in the low dose cohort) across two REVEAL Phase 1/2 trials, with no serious adverse events or dose-limiting toxicities reported [4] - A comprehensive update on the pivotal trial design, natural history data analysis, and clinical data from the REVEAL trials is anticipated in Q2 2025 [4][13] Financial Highlights - For Q1 2025, research and development expenses were $15.6 million, down from $20.7 million in Q1 2024, primarily due to reduced manufacturing activities [7] - General and administrative expenses increased to $8.2 million in Q1 2025 from $7.1 million in Q1 2024, attributed to higher compensation and legal fees [8] - The net loss for Q1 2025 was $21.5 million, or $0.08 per share, compared to a net loss of $24.1 million, or $0.10 per share, in Q1 2024 [9][17] Cash Position - As of March 31, 2025, Taysha had $116.6 million in cash and cash equivalents, which is expected to support operations into Q4 2026 [9][19] Upcoming Events - Taysha will present three abstracts related to TSHA-102 at the 2025 International Rett Syndrome Foundation Scientific Meeting scheduled for June 9-11, 2025 [4][5]

Group 1 - Taysha Gene Therapies, Inc. is a clinical-stage biotechnology company focused on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system (CNS) [1][2] - The company will report its financial results for the first quarter ended March 31, 2025, and host a corporate update conference call on May 15, 2025, at 8:30 AM Eastern Time [1] - Taysha's lead clinical program, TSHA-102, is in development for Rett syndrome, a rare neurodevelopmental disorder with no approved disease-modifying therapies [2] Group 2 - The management team of Taysha has proven experience in gene therapy development and commercialization [2] - The company aims to address severe unmet medical needs and improve the lives of patients and their caregivers through transformative medicines [2] - Taysha leverages its manufacturing process and a clinically and commercially proven AAV9 capsid to translate treatments from bench to bedside [2]