TSHA earnings

Reaffirmed FDA alignment on TSHA-102 BLA pathway, including pivotal trial design, endpoints and potential to submit for approval based on REVEAL pivotal trial 6-month interim analysis, following recent breakthrough therapy Type B meeting Further advanced dosing in the REVEAL pivotal trial, with enrollment in the ASPIRE trial ongoing across multiple sites; on track to complete dosing in REVEAL and ASPIRE in Q2 2026 Maintained favorable tolerability profile with no treatment-related SAEs or DLTs in REVEAL Phase 1/2 and REVEAL pivotal trials as of May 2026 data cutoff; plan to present longer-term safety and efficacy data from Part A of REVEAL Phase 1/2 trials in Q2 2026 Initiated BLA-enabling P

DALLAS, April 29, 2026 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (NASDAQ:TSHA) (Taysha or the Company), 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), today announced that it will report its financial results for the first quarter ended March 31, 2026, and host a corporate update conference call and webcast on Wednesday, May 6, 2026, at 8:30 AM Eastern Time. Participants may access the live webcast of the conference call from the Events and Presentations page of Taysha's website at ir.tayshagtx.com. An archived replay of the webcast will be available on t

Dosed multiple Rett syndrome patients in REVEAL pivotal trial of TSHA-102, with enrollment advancing across multiple sites; on track to complete dosing in Q2 2026 Received FDA clearance to initiate ASPIRE trial in three patients aged 2 to <4 years with inclusion of ≥3 months of safety data in planned BLA submission to support potential for broad label; on track to complete dosing in Q2 2026 Maintained favorable tolerability profile with no treatment-related SAEs or DLTs in REVEAL Phase 1/2 and REVEAL pivotal trials as of March 2026 data cutoff; longer-term safety and efficacy data from Part A of REVEAL Phase 1/2 trials expected in Q2 2026 Reached written alignment with FDA on proposed PPQ

DALLAS, March 12, 2026 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (NASDAQ:TSHA) (Taysha or the Company), 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), today announced that it will report its financial results for the full-year ended December 31, 2025, and host a corporate update conference call and webcast on Thursday, March 19, 2026, at 8:30 AM Eastern Time. Participants may access the live webcast of the conference call from the Events and Presentations page of Taysha's website at ir.tayshagtx.com. An archived replay of the webcast will be available on

TSHA-102 granted Breakthrough Therapy designation by FDA Finalized FDA alignment on REVEAL pivotal trial protocol and SAP, including 6-month interim analysis that may expedite BLA submission, which was enabled by the rigorous developmental milestone evaluation in Part A REVEAL Phase 1/2 trials showing an unprecedented response rate Dosing of first patient in REVEAL pivotal trial scheduled for Q4 2025, with enrollment of additional patients expected to continue at multiple sites this quarter Presented new supplemental analysis of Part A REVEAL data reinforcing the broad and consistent, multi-domain impact of TSHA-102 on activities of daily living at the CNS Annual Meeting TSHA-102

DALLAS, Oct. 28, 2025 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (NASDAQ:TSHA) (Taysha or the Company), 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), today announced that it will report its financial results for the third quarter ended September 30, 2025, and host a corporate update conference call and webcast on Tuesday, November 4, 2025, at 8:30 AM Eastern Time. Conference Call DetailsTuesday, November 4, at 8:30 AM Eastern Time / 7:30 AM Central TimeToll Free: 800-245-3047International: 203-518-9765Conference ID: TAYSHAWebcast: https://ir.tayshagtx.com

Commenced site activation for REVEAL pivotal trial in accordance with previously aligned upon key design elements, following receipt of No Objection Letter (NOL) from Health Canada and feedback from the FDA; patient enrollment anticipated to begin in Q4 2025 High dose and low dose TSHA-102 continue to be generally well tolerated with no treatment-related SAEs or DLTs in the 12 patients treated in Part A of REVEAL Phase 1/2 trials as of August 2025 data cutoff Part A data from REVEAL Phase 1/2 trials presented at IRSF Scientific Meeting showed 100% response rate for pivotal trial primary endpoint of gain/regain of ≥ one developmental milestone, corroborated by improvements in key secondary

DALLAS, Aug. 05, 2025 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (NASDAQ:TSHA) (Taysha or the Company), 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), today announced that it will report its financial results for the second quarter ended June 30, 2025, and host a corporate update conference call and webcast on Tuesday, August 12, 2025, at 8:30 AM Eastern Time. Conference Call DetailsTuesday, August 12, at 8:30 AM Eastern Time / 7:30 AM Central TimeToll Free: 877-407-0792International: 201-689-8263Conference ID: 13754869Webcast: https://ir.tayshagtx.com/new

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; IND amendment submission expected in Q2 2025 High dose and low dose of TSHA-102 continue to be generally well tolerated with no treatment-related SAEs or DLTs in the pediatric, adolescent and adult patients treated (high dose, n=6; low dose, n=4) across both REVEAL trials as of April 10, 2025,

DALLAS, May 08, 2025 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (NASDAQ:TSHA), 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), today announced that it will report its financial results for the first quarter ended March 31, 2025, and host a corporate update conference call and webcast on Thursday, May 15, 2025, at 8:30 AM Eastern Time. Conference Call DetailsThursday, May 15, at 8:30 AM Eastern Time / 7:30 AM Central TimeToll Free: 877-407-0792International: 201-689-8263Conference ID: 13753490Webcast: https://ir.tayshagtx.com/news-events/events-presentations

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 pivotal trial design expected in H1 2025 Clinical data from cohort two (high dose) and cohort one (low dose) of both REVEAL trials expected in H1 2025 Conference call and live webcast today

DALLAS, Feb. 19, 2025 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (NASDAQ:TSHA), 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), today announced that it will report its financial results for the full-year ended December 31, 2024, and host a corporate update conference call and webcast on Wednesday, February 26, 2025, at 8:30 AM Eastern Time. Conference Call DetailsWednesday, February 26, at 8:30 AM Eastern Time / 7:30 AM Central TimeToll Free: 877-407-0792International: 201-689-8263Conference ID: 13751800Webcast: https://ir.tayshagtx.com/news-events/events-p

High dose TSHA-102 was generally well tolerated with no SAEs or DLTs in two adolescent/adult patients and one pediatric patient as of data cutoff; IDMC approved continued enrollment in cohort two (high dose) across both REVEAL trials; eight patients dosed to date (low dose=4, high dose=4) Advanced discussions with the FDA on trial design, endpoints and potential use of established natural history dataset for Part B of REVEAL trials, and aligned on a meeting cadence to expedite the development plan for TSHA-102 following initial RMAT Type B meeting FDA approved use of pivotal TSHA-102 product in REVEAL trials based on successful demonstration of analytical comparability; Company released pi

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 first (pediatric trial) patient in cohort two, which is scheduled for Q3 2024   Cohort two (n=3) and cohort one (n=2) data from both trials (adolescent/adult and pediatric) expected in 1H 2

DALLAS, Aug. 05, 2024 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (NASDAQ:TSHA), 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), today announced that it will report its financial results for the second quarter ended June 30, 2024, and host a corporate update conference call and webcast on Monday, August 12, 2024, at 8:30 AM Eastern Time. Conference Call DetailsMonday, August 12, 2024, at 8:30 AM Eastern Time / 7:30 AM Central TimeToll Free:877-407-0792International:201-689-8263Conference ID:13747741Webcast:https://ir.tayshagtx.com/news-events/events-presenta

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 Longer-term data from both adult patients showed sustained and new improvements across multiple efficacy measures and clinical domains following the completion of steroid taper (patient one: sat unassisted for first time in over a decade, normalized sleep, stabilized seizures; patient two: improved hand stereotypies and breathing, seizure-free for 8.5 months at 25% lowe

Completed dosing in cohort one (low dose, 5.7x1014 total vg) of REVEAL Phase 1/2 adolescent and adult trial with longer-term data supporting the safety profile and durable response of TSHA-102; enrolled first patient in cohort two (high dose, 1x1015 total vg) with dosing scheduled for Q2 2024 Dosed second pediatric patient in cohort one (low dose, 5.7x1014 total vg) of REVEAL Phase 1/2 pediatric trial in Q1 2024 FDA granted Regenerative Medicine Advanced Therapy designation following review of available safety and efficacy data from the first three patients dosed with the low dose of TSHA-102 across both REVEAL trials (adolescent/adult and pediatric) Initial data f

DALLAS, May 07, 2024 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of severe monogenic diseases of the central nervous system (CNS), today announced that it will report its financial results for the first quarter ended March 31, 2024, and host a corporate update conference call and webcast on Tuesday, May 14, 2024, at 4:30 PM Eastern Time. Conference Call DetailsTuesday, May 14, at 4:30 PM Eastern Time / 3:30 PM Central TimeToll Free: 877-407-0792International: 201-689-8263Conference ID: 13745689Webcast: https://ir.tayshagtx.com/news-events/events-pres

Data from first adult patient in REVEAL Phase 1/2 trial showed TSHA-102 (low dose, 5.7x1014 total vg) was well-tolerated with no treatment-emergent SAEs as of 35-week assessment, with sustained improvement across key efficacy measures at decreased steroid levels and new improvement in RSBQ at month six Data from second adult patient showed TSHA-102 (low dose, 5.7x1014 total vg) was well-tolerated with no treatment-emergent SAEs as of 19-week assessment, with sustained improvement across key efficacy measures, significantly reduced seizure events and new improvement in R-MBA at week 12 Principal Investigator observed sustained and new improvements across multiple clinical domains following

DALLAS, March 14, 2024 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (NASDAQ:TSHA) (Taysha or the Company), a clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of severe monogenic diseases of the central nervous system (CNS), today announced that it will report its financial results for the full-year ended December 31, 2023, provide corporate and clinical updates and host a corporate update conference call on Tuesday, March 19, 2024, at 4:30 PM Eastern Time. Conference Call DetailsTuesday, March 19, at 4:30 PM Eastern Time / 3:30 PM Central TimeToll Free: 877-407-0792International: 201-689-8263Conference ID: 13744574W

Data from first adult patient in REVEAL Phase 1/2 trial showed TSHA-102 was well-tolerated with no treatment-emergent SAEs as of 20-week assessment with sustained improvement across key efficacy measures and new improvement in R-MBA, PGI-I and hand function, a hallmark characteristic of Rett syndrome at week 12 Data from second adult patient showed TSHA-102 was well-tolerated with no treatment-emergent SAEs as of six-week assessment with improvement across key efficacy measures, including CGI-I, R-MBA, PGI-I and RSBQ at week four Notable differences in genetic mutation and phenotypic expression reported between patient one and two; Principal Investigator (PI) observed improvements in both

DALLAS, Nov. 07, 2023 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS), today announced that it will report its financial results for the third quarter ended September 30, 2023, and host a corporate update conference call and webcast on Tuesday, November 14, 2023, at 4:30 PM Eastern Time. Conference Call DetailsTuesday, November 14, at 4:30 PM Eastern Time / 3:30 PM Central TimeToll Free: 877-407-0792International: 201-689-8263 Conference ID: 13741244 Webcast: https://ir.tayshagtx.com/news-events

Available clinical data from the two adult patients dosed with TSHA-102 in the first cohort (low dose) to be discussed during upcoming quarterly earnings call following Independent Data Monitoring Committee (IDMC) review Dosing of third adult patient and completion of enrollment in the low-dose cohort expected in the fourth quarter of 2023 Dosing of first pediatric Rett syndrome patient expected in the first quarter of 2024 DALLAS, Sept. 26, 2023 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous s

Data from first adult patient dosed in REVEAL Phase 1/2 trial showed TSHA-102 was well-tolerated with no treatment-emergent serious adverse events (SAEs) as of six-week assessment and improvement in key efficacy measures, including Clinical Global Impression – Improvement (CGI-I), Clinical Global Impression – Severity (CGI-S) and Rett Syndrome Behavior Questionnaire (RSBQ), four weeks post-treatment Principal Investigator (PI) observed clinical improvement in multiple domains, including autonomic function (sleep and breathing), vocalization, as well as gross motor skills (gained ability to sit unassisted for three minutes) and fine motor skills (gained ability to hold objects), supported by

Independent Data Monitoring Committee recommended REVEAL Phase 1/2 trial continuation and proceeding with dosing of second patient based on encouraging initial clinical data from the first adult with Rett syndrome dosed with investigational gene therapy TSHA-102 Initial clinical update from the first patient dosed with TSHA-102 planned for forthcoming quarterly earnings call Dosing of second patient expected in the third quarter of 2023 DALLAS, July 31, 2023 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous

Screening completed and dosing scheduled for first potential subject in the Phase 1/2 REVEAL trial in Rett syndrome; dosing of first adult patient with TSHA-102 expected in Q2 2023; initial available Phase 1/2 clinical data, primarily on safety, expected in Q2 2023 Clinical Trial Application (CTA) submission to United Kingdom (UK) MHRA for TSHA-102 in pediatric patients with Rett syndrome expected in mid-2023; Investigational New Drug (IND) application to United States (U.S.) Food and Drug Administration (FDA) in Rett syndrome anticipated in H2 2023 New preclinical data for TSHA-102 in Rett syndrome to be presented during a poster presentation at the upcoming American Society of Gene a

DALLAS, May 04, 2023 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS), today announced that it will report its financial results for the first quarter ended March 31, 2023, and host a corporate update conference call and webcast on Thursday, May 11, 2023, at 4:30 PM Eastern Time. Conference Call DetailsThursday, May 11, at 4:30 PM Eastern Time / 3:30 PM Central TimeToll Free: 855-327-6837International: 631-891-4304Conference ID: 10021767https://ir.tayshagtx.com/news-events/events-presentations  Abo

Initiated screening of first potential subject in Phase 1/2 REVEAL trial in Rett syndrome; dosing of first adult patient with TSHA-102 expected in H1 2023; submitted protocol amendment to allow for younger patients; initial available Phase 1/2 clinical data, primarily on safety, expected in H1 2023 Clinical Trial Application (CTA) submission to United Kingdom (UK) MHRA for TSHA-102 in pediatric patients with Rett syndrome expected in mid-2023; Investigational New Drug (IND) application to United States (U.S.) Food and Drug Administration (FDA) in Rett syndrome anticipated in H2 2023 FDA feedback for TSHA-120 in giant axonal neuropathy (GAN) suggests consideration of alternative clinical t

DALLAS, March 15, 2023 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic rare diseases of the central nervous system (CNS), today announced that it will report its financial results for the fourth quarter and full-year ended December 31, 2022, and host a corporate update conference call and webcast on Tuesday, March 28, 2023, at 4:30 PM Eastern Time. Conference Call DetailsTuesday, March 28, at 4:30 PM Eastern Time / 3:30 PM Central TimeToll Free:877-407-0792International:201-689-8263Conference ID:13736479Webcast:https://ir.ta

Type B end-of-Phase 2 meeting with U.S. Food and Drug Administration (FDA) provided additional clarity for TSHA-120 for the treatment of giant axonal neuropathy (GAN) ultra-rare disease program - FDA acknowledged MFM32 as an acceptable endpoint with a recommendation to dose additional patients in a double-blind, placebo-controlled design to support Biologics License Application (BLA) submission Organizational and business review by new management with operational, structural and personnel changes implemented to enhance execution Dosing of first adult patient with Rett syndrome from ongoing trial in Canada expected in H1 2023; update of initial available clinical data anticipated in H1 2023

DALLAS, Nov. 02, 2022 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced that it will report its financial results for the third quarter ended September 30, 2022, and host a corporate update conference call and webcast on Tuesday, November 8, 2022, at 8:00 AM Eastern Time. Conference Call DetailsTuesday, November 8, at 8:00 AM Eastern Time / 7:00 AM Central TimeToll Free:855-327-6387International:631-891-4304Conference ID:100

DALLAS, Oct. 24, 2022 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced that it will host a conference call and webcast with slides to discuss Astellas Pharma's strategic investment to support the development of two of Taysha's AAV-based gene therapy programs, TSHA-102 for Rett syndrome and TSHA-120 for giant axonal neuropathy (GAN), on Tuesday, October 25, 2022, at 8:00 AM Eastern Time. Conference Call DetailsTuesday, Oc

TSHA-120 treated patients in GAN demonstrated durable improvement and recoverability of sensory nerve amplitude potential (SNAP), a definitive clinical endpoint, compared to natural history TSHA-120 commercial grade and clinical trial material considered comparable across all key quality attributes as assessed by an extensive panel of release assays and next-generation sequencing Positive feedback from MHRA supports regulatory strategy and manufacturing approach including potency assay matrix; additional regulatory feedback, including from FDA, expected by year-end 2022 Preclinical data for TSHA-102 in Rett syndrome demonstrated near normalization of survival as well as normalization of b

DALLAS, Aug. 09, 2022 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced that it will report its financial results for the second quarter ended June 30, 2022, and host a corporate update conference call and webcast on Thursday, August 11, 2022, at 8:00 AM Eastern Time. Conference Call DetailsThursday, August 11, at 8:00 AM Eastern Time / 7:00 AM Central TimeToll Free:877-407-0792International:201-689-8263Conference ID:1373084

Initiated clinical development of TSHA-102 for Rett Syndrome under recently approved Clinical Trial Application (CTA) with preliminary Phase 1/2 data expected by year-end 2022 Received Orphan Drug Designation from the European Commission for TSHA-120 for giant axonal neuropathy (GAN) and recently completed commercially representative GMP batch; regulatory update expected in mid-2022 Existing cash and cash equivalents, along with full access to the term loan facility, is expected to fund operating expenses and capital requirements into the fourth quarter of 2023 DALLAS, May 16, 2022 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-stage gene thera

DALLAS, May 11, 2022 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced that it will report its financial results for the first quarter ended March 31, 2022, and host a corporate update conference call and webcast on Monday, May 16, 2022, at 8:00 AM Eastern Time. Conference Call DetailsMonday, May 16, at 8:00 AM Eastern Time / 7:00 AM Central TimeToll Free: 877-407-0792International: 201-689-8263Conference ID: 13729044Webc

Strategic pipeline prioritization initiatives focused on giant axonal neuropathy (GAN), with feedback on registration pathway by mid-2022, and Rett syndrome, with preliminary clinical data expected by year-end 2022 under recently approved Clinical Trial Application (CTA) Rett syndrome affects over 350,000 patients worldwide; GAN has an estimated addressable patient population of 5,000 worldwide Activities for other ongoing clinical programs will be minimized and all additional research and development will be paused to increase operational focus and efficiency Pipeline prioritization initiatives, existing cash and financing under current debt facility expected to extend cash runway into

Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced that it will report its financial results for the fourth quarter and full-year ended December 31, 2021, and host a corporate update conference call and webcast on Thursday, March 31, 2022, at 8:00 AM Eastern Time. Conference Call Details Thursday, March 31 at 8:00 AM Eastern Time / 7:00 AM Central Time Toll Free: 877-407-0792 International: 201-689-8263 Conference ID:

Efficacy data for high dose cohort demonstrated clinically meaningful and statistically significant improvement in MFM32 by Year 1 compared to natural history (n=3) Long-term durability data across all therapeutic dose cohorts demonstrated a 10-point improvement in mean change in MFM32 by Year 3 compared to estimated natural history decline of 24 points (n=5) Biopsy data in five of six patient samples analyzed to date confirmed active regeneration of nerve fibers following treatment with TSHA-120 (n=6) TSHA-120 was safe and well-tolerated supported by 53 patient-years of clinical data Conference call and live webcast today at 8:00 AM Eastern Time Taysha Gene Therapies, Inc. (NASDAQ:TSH

Patient 1 with Sandhoff disease realized normalization of Hex A enzyme activity by Month 1, achieving 58-fold above the presumed asymptomatic level of 5% of normal identified by natural history at Month 3 Patient 2 with Tay-Sachs disease achieved Hex A enzyme activity 5-fold above the presumed asymptomatic level of 5% of normal identified by natural history at Month 1 First-ever data supporting bicistronic vector approach in humans, TSHA-101 is designed to deliver both HEXA and HEXB genes in the endogenous ratio Conference call and live webcast today at 8:00 AM Eastern Time Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-stage gene therapy company focused on develo

Anticipate clinical safety and MFM32 functional data for TSHA-120 from the highest dose cohort of 3.5x1014 total vg in GAN in December 2021 Expect preliminary clinical safety data and Hex A enzyme activity in the plasma and cerebral spinal fluid for TSHA-101 in GM2 gangliosidosis in December 2021 Anticipate preliminary clinical data for the first-generation construct in CLN7 disease including safety data from first patient ever dosed intrathecally at 1.0x1015 total vg in December 2021 On track to initiate Phase 1/2 trials for TSHA-118 in CLN1 disease and TSHA-102 in Rett syndrome by year-end 2021 Completed five successful concurrent GMP campaigns for multiple programs to date, sufficien

Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced that it will report its financial results for the third quarter ended September 30, 2021, and host a corporate update conference call and webcast on Wednesday, November 10, 2021, at 8:00 AM Eastern Time. Conference Call Details Wednesday, November 10, at 8:00 AM Eastern Time / 7:00 AM Central Time Toll Free: 877-407-0792 International: 201-689-8263 Conference ID: 1372

Positive pre-IND/CTA feedback obtained from several key regulatory agencies for TSHA-102 in Rett syndrome, TSHA-101 in GM2 gangliosidosis and TSHA-118 in CLN1 disease, paving the way for multiple anticipated IND/CTA filings in the second half of 2021 Entered into a non-dilutive term loan agreement with Silicon Valley Bank that provides up to $100 million in tranches at an attractive interest rate that lowers overall cost of capital, strengthens the balance sheet and offers additional financial and operational flexibility; assuming full drawdown, cash runway expected to be sufficient to achieve potential regulatory approval for TSHA-120 in GAN without the need for additional financing Reit

Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced that it will report its financial results for the second quarter ended June 30, 2021, and host a corporate update conference call and webcast on Monday, August 16, 2021, at 8:00 AM Eastern Time. Conference Call Details Monday, August 16, at 8:00 AM Eastern Time / 7:00 AM Central Time Toll Free:   877-407-0792 International:   201-689-8263 Conference ID: