[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]

Core Viewpoint - Taysha Gene Therapies, Inc. (TSHA) is anticipated to report a year-over-year increase in earnings despite lower revenues, which could significantly influence its near-term stock price depending on the actual results compared to estimates [1][2]. Earnings Expectations - The upcoming earnings report is expected to show a quarterly loss of $0.08 per share, reflecting a year-over-year change of +20% [3]. - Revenues are projected to be $2.1 million, representing a decline of 38.4% from the same quarter last year [3]. Estimate Revisions - The consensus EPS estimate has been revised 2.22% lower in the last 30 days, indicating a reassessment by analysts [4]. - The Most Accurate Estimate aligns with the Zacks Consensus Estimate, resulting in an Earnings ESP of 0%, suggesting no recent differing analyst views [10][11]. Earnings Surprise Prediction - The Zacks Earnings ESP model indicates that a positive or negative reading can predict the deviation of actual earnings from consensus estimates, with a strong predictive power for positive readings [7][8]. - Taysha Gene Therapies currently holds a Zacks Rank of 3, making it challenging to predict an earnings beat conclusively [11]. Historical Performance - In the last reported quarter, Taysha Gene Therapies was expected to post a loss of $0.08 per share but actually reported a loss of $0.07, resulting in a surprise of +12.50% [12]. - Over the past four quarters, the company has exceeded consensus EPS estimates three times [13]. Conclusion - While Taysha Gene Therapies does not appear to be a strong candidate for an earnings beat, investors should consider other factors when making investment decisions ahead of the earnings release [16].

Financial Data and Key Metrics Changes - Research and development expenses increased to $66 million for the full year ended December 31, 2024, compared to $56.8 million for the previous year, driven by good manufacturing practices and additional clinical trial activities [29] - General and administrative expenses decreased to $29 million for the full year ended December 31, 2024, from $30 million in 2023, primarily due to a decrease in issuance costs [30] - Net loss for the full year ended December 31, 2024, was $89.3 million or $0.36 per share, compared to a net loss of $111.6 million or $0.96 per share for the previous year [30] - As of December 31, 2024, the company had $139 million in cash and cash equivalents, expected to support planned operating expenses into the fourth quarter of 2026 [31] Business Line Data and Key Metrics Changes - The TSHA-102 program is advancing with a focus on generating critical clinical data across two REVEAL Phase 1/2 trials for Rett syndrome [10][11] - Dosing of ten patients in Part A of the REVEAL trials has been completed, with both high and low doses demonstrating an encouraging safety profile [12][13] Market Data and Key Metrics Changes - The company is focused on the Rett syndrome market, which has a high unmet need due to the progressive nature of the disease and the significant burden on caregivers [24] Company Strategy and Development Direction - The strategy involves utilizing Part A of the trials to generate a dataset that informs the pivotal Part B trial, focusing on objective measures that capture improvements in core features of Rett syndrome [13][20] - The company aims to bring TSHA-102 to patients as expeditiously and safely as possible, with ongoing discussions with the FDA to solidify the regulatory pathway [16][28] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the differentiated gene therapy candidate, anticipating a transformative year ahead with key milestones expected in the first half of 2025 [32][33] - The FDA discussions have been constructive, with a focus on functional outcomes that are clinically meaningful to patients and caregivers [46][49] Other Important Information - The company has completed dosing in both the high and low dose cohorts, with ongoing evaluations of functional gains across multiple domains [15][27] - The FDA has been open-minded regarding trial design and endpoints, emphasizing the importance of clinically meaningful outcomes [63][64] Q&A Session Summary Question: Can you speak to the number of people involved in FDA discussions? - Management indicated that discussions typically involve 15 to 20 FDA representatives, including senior officials, and have not been impacted by recent changes in administration [38][39] Question: Any changes in FDA's expectations regarding functional gains? - Management stated that FDA discussions have remained consistent, focusing on clinically meaningful objective improvements across key domains [44][49] Question: What differentiates TSHA-102 from competitors in the gene therapy space? - Management highlighted the use of self-complementary technology in TSHA-102, which allows for quicker protein production and potentially faster clinical effects compared to competitors [56][57] Question: What is the timeline for reaching alignment with the FDA on endpoints? - Management expressed optimism about ongoing discussions and aims to provide a regulatory update coinciding with clinical data updates in the first half of 2025 [84][85] Question: What is the safety profile of the high dose patients? - Management reported no treatment-related serious adverse events or dose-limiting toxicities observed in the ten patients dosed, indicating a favorable safety profile [75][103]

Core Insights - Taysha Gene Therapies, Inc. reported a quarterly loss of $0.07 per share, which was better than the Zacks Consensus Estimate of a loss of $0.08, representing an earnings surprise of 12.50% [1] - The company generated revenues of $2.02 million for the quarter ended December 2024, exceeding the Zacks Consensus Estimate by 6.42%, but down from $3.6 million in the same quarter last year [2] - The stock has underperformed the market, losing about 1.7% since the beginning of the year compared to the S&P 500's gain of 1.3% [3] Earnings Outlook - The current consensus EPS estimate for the upcoming quarter is -$0.08 on revenues of $2.63 million, and for the current fiscal year, it is -$0.37 on revenues of $9.73 million [7] - The estimate revisions trend for Taysha Gene Therapies is mixed, resulting in a Zacks Rank 3 (Hold), indicating expected performance in line with the market [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, suggesting potential challenges ahead [8] - Empirical research indicates a strong correlation between near-term stock movements and trends in earnings estimate revisions, which could impact Taysha's stock performance [5]

Clinical Development - TSHA-102 is in clinical development for Rett syndrome, a rare neurodevelopmental disorder, with no approved disease-modifying therapies currently available[17]. - The REVEAL Phase 1/2 trials have completed dosing of 10 patients, with no treatment-related serious adverse events or dose-limiting toxicities reported as of February 17, 2025[19]. - Positive longer-term clinical data from the REVEAL trials indicate sustained clinical improvements in both adolescent/adult and pediatric cohorts[29]. - Improvements in motor skills, communication, and autonomic function were observed in adult patients as early as four weeks post-treatment, with effects persisting over time[31]. - The maximum tolerated dose established in Part A of the REVEAL trials will be administered during the pivotal Part B phase[26]. - The company aims to report safety and efficacy data from the REVEAL trials in the first half of 2025[19]. - TSHA-102 demonstrated improvements in multiple efficacy measures as early as four weeks post-treatment, with sustained improvements observed at week 52 for patient one and week 25 for patient two[34]. - In the pediatric trial, TSHA-102 was well-tolerated with no treatment-related serious adverse events (SAEs) or dose-limiting toxicities (DLTs) reported as of February 17, 2025[35]. - Patient one showed a CGI-S score improvement from 5 (markedly ill) at baseline to 2 (minimally improved) by week 12, while patient two improved from a score of 4 (moderately ill) to 1 (very much improved) by week 8[37]. - Patient one improved hand function, able to hold an object for 3 minutes compared to 12 seconds at baseline, while patient two showed improved gait and stability when walking[37]. Regulatory Designations - TSHA-102 has received multiple designations from regulatory authorities, including orphan drug designation and Fast Track Designation from the FDA[20]. - TSHA-118 has received orphan drug designation and fast track designation from the FDA for the treatment of CLN1 disease[46]. - TSHA-105 has also received orphan drug designation and rare pediatric disease designation from the FDA for SLC13A5 deficiency[50]. - Orphan drug designation is granted for drugs intended to treat rare diseases affecting fewer than 200,000 individuals in the U.S., providing seven years of exclusivity upon FDA approval[120]. - Fast track designation allows for expedited review of products intended to treat serious conditions and may include rolling review of BLA sections[112]. - Breakthrough therapy designation provides intensive FDA interaction and guidance, expediting development for products showing substantial improvement over existing therapies[113]. Financial Overview - As of December 31, 2024, the company had incurred net losses of $89.3 million and $111.6 million for the years ended December 31, 2024 and 2023, respectively, with an accumulated deficit of $602.3 million[192]. - The company has financed operations with $671.0 million in gross proceeds from equity financings, including pre-IPO private placements and public offerings, but has not generated any revenue from product sales[192]. - The company had cash and cash equivalents of $139.0 million as of December 31, 2024, which is expected to fund operating expenses into the fourth quarter of 2026[200]. - The company expects to incur significant expenses and operating losses over the next several years as it conducts clinical trials and seeks regulatory approval for product candidates[199]. - The company may require additional capital to achieve its business objectives, and its ability to raise capital may be adversely impacted by global economic conditions[202]. - The company currently has no committed external source of funds and may need to raise additional capital through equity offerings or debt financing, which could dilute stockholder ownership[209]. Intellectual Property and Licensing - The company has in-licensed five U.S. patents, 14 foreign patents, and 54 pending foreign patent applications as of February 24, 2024, to protect its proprietary technology[87]. - The company in-licenses patents related to TSHA-102, with expected expiration dates ranging from 2038 to 2041, depending on the specific patent[87][88]. - The company issued 2,179,000 shares of common stock to UT Southwestern as part of the license agreement[69]. - The company paid a one-time upfront license fee of $3.0 million to Abeona for the CLN1 Agreement in fiscal year 2020[73]. - The company is obligated to pay up to $26.0 million in regulatory-related milestones and up to $30.0 million in sales-related milestones per licensed product under the Abeona CLN1 Agreement[73]. Market and Economic Challenges - The company faces significant uncertainty regarding coverage and reimbursement for its pharmaceutical products, which depend on third-party payors' decisions[168]. - The company may encounter challenges in obtaining adequate coverage and reimbursement for products administered under physician supervision due to higher associated prices[170]. - The Inflation Reduction Act of 2022 introduces price negotiation for certain high-expenditure drugs under Medicare, impacting the company's pricing strategies[176]. - Legislative changes have resulted in aggregate reductions of Medicare payments to providers, which will remain in effect through 2032 unless further action is taken[175]. - Increased scrutiny over drug pricing and reimbursement methodologies may lead to further regulatory challenges for the company[176]. Clinical Trial Regulations - The FDA requires the submission of an IND application before initiating clinical trials, which must become effective for trials to begin[102]. - Human clinical trials are conducted in three phases: Phase 1 focuses on safety and dosage, Phase 2 evaluates efficacy and side effects, and Phase 3 confirms efficacy and safety in a larger population[105]. - The FDA may suspend clinical trials if patients are exposed to unacceptable health risks or if the trial is unlikely to meet its objectives[104]. - Regulatory agencies require extensive monitoring and auditing of clinical activities and data throughout all phases of clinical development[106]. Company Operations and Risks - The company has a limited operating history and no history of commercializing products, making it difficult for investors to assess future viability[197]. - The company relies on collaboration with UT Southwestern for preclinical research and development, and any failure in this collaboration could materially harm its business[191]. - The company anticipates that negative public opinion and increased regulatory scrutiny of gene therapy may adversely impact the development of its product candidates[193]. - The company faces risks related to the development of its product candidates, including the need for significant investment and successful completion of clinical trials[214]. - The company may need to relinquish rights to technologies or product candidates if it raises additional funds through collaborations or licensing arrangements[210].

Clinical Trials - TSHA-102 clinical program has completed dosing of 10 patients in Part A of the REVEAL trials, with 6 in the high dose cohort and 4 in the low dose cohort[5] - The pivotal trial design update for TSHA-102 is anticipated in the first half of 2025[7] - Clinical data from both high and low dose cohorts in the REVEAL trials is expected in the first half of 2025[8][9] Financial Performance - Net loss for the full year 2024 was $89.3 million, or $0.36 per share, compared to a net loss of $111.6 million, or $0.96 per share, in 2023[12] - Research and development expenses for the full year 2024 were $66.0 million, an increase of 16.2% from $56.8 million in 2023[10] - Total operating expenses for 2024 were $99.8 million, up from $87.9 million in 2023, reflecting increased clinical trial activities[20] Cash and Assets - Cash and cash equivalents as of December 31, 2024, were $139.0 million, expected to support operations into Q4 2026[12] - Total assets decreased to $160.4 million in 2024 from $172.7 million in 2023, primarily due to a reduction in cash and cash equivalents[22] Administrative Expenses - General and administrative expenses decreased to $29.0 million in 2024 from $30.0 million in 2023, a reduction of 3.3%[11] Warrant Liability - The company reported a change in fair value of warrant liability resulting in a gain of $16,000 in 2024, compared to a loss of $34.7 million in 2023[20]

High dose and low dose of TSHA-102 continue to be generally well tolerated with no treatment-related SAEs or DLTs in all pediatric, adolescent and adult patients treated (high dose, n=6; low dose, n=4) across both REVEAL trials as of February 2025 data cutoff Completed dosing of the 10 patients in Part A of both REVEAL trials; maturing dataset continues to support advancement toward pivotal Part B trial Productive ongoing discussions with the FDA to solidify regulatory pathway for TSHA-102; update on pivota ...