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.

成人和儿科患者在一致的临床领域取得了持久的改善，包括运动技能，沟通/社会化，自主神经功能，癫痫发作，以及在成人（长达52周）和儿科（长达22周）患者中看到的令人鼓舞的安全性。不同的基因突变严重程度。

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% lower anti-seizure medication) .

来自两名成年患者的长期数据显示，在完成类固醇减量后，多种疗效指标和临床领域都有持续和新的改善（患者一：十多年来首次无人辅助坐着，睡眠正常，癫痫发作稳定；患者二：改善手部刻板印象和呼吸，抗癫痫药物降低25%，无癫痫发作8.5个月）。

Initial data from first two pediatric patients showed improvements across multiple efficacy measures and clinical domains, with early evidence of developmental gains (patient one: improved hand function, grasp and gross motor coordination, gained visual reception and receptive language skills; patient two: gained ability to stand up from chair and walk up a stair, increase in seizure-free days).

来自前两名儿科患者的初步数据显示，在多种疗效指标和临床领域都有所改善，早期证据表明发育有所改善（患者一：改善了手部功能，抓握和粗大运动协调，获得了视觉接收和接受语言技能；患者二：获得了从椅子上站起来爬楼梯的能力，增加了无癫痫发作的天数）。

IDMC approved Company’s request for early advancement to cohort two (high dose) in the REVEAL pediatric trial; dosing expected in Q3 2024 following IDMC review of initial safety data from the first high dose patient in the adolescent and adult trial

IDMC批准了该公司在REVEAL儿科试验中提前进入第二组（高剂量）的请求；在IDMC审查了青少年和成人试验中第一位高剂量患者的初始安全性数据后，预计2024年第三季度的剂量

Company will host webcast today at 8:00 AM Eastern Time

该公司将于东部时间今天上午8:00主持网络广播

DALLAS, June 18, 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 positive longer-term clinical data from the ongoing REVEAL Phase 1/2 adolescent and adult trial and initial clinical data from the REVEAL Phase 1/2 pediatric trial evaluating TSHA-102 in Rett syndrome..

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“We are highly encouraged by the safety profile and broad clinical response observed across multiple domains in both the adult and pediatric patients with different genetic mutation severity treated with the low dose of TSHA-102,” said Sean P. Nolan, Chairman and Chief Executive Officer of Taysha. “The longer-term follow up data indicate a durable response with sustained and new improvements across multiple clinical domains in both adult patients, and importantly, both pediatric patients showed initial improvements across consistent clinical domains, with early evidence of developmental gains following treatment with TSHA-102.

Taysha董事长兼首席执行官肖恩·诺兰（Sean P.Nolan）表示：“在使用低剂量TSHA-102治疗的具有不同基因突变严重程度的成年和儿科患者中，在多个领域观察到的安全性和广泛的临床反应使我们深受鼓舞。”。“长期随访数据表明，两名成年患者在多个临床领域都有持续和新的改善，这是一种持久的反应，重要的是，两名儿科患者在一致的临床领域都表现出初步的改善，早期证据表明TSHA-102治疗后发育有所改善。

We believe these improvements in adult and pediatric patients further reinforce the potential of TSHA-102 to be transformative for a broad range of patients with Rett syndrome.”.

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Elsa Rossignol, M.D., FRCP, FAAP, Associate Professor in Neuroscience and Pediatrics at the Université de Montréal and Principal Investigator of the REVEAL adolescent and adult trial at the CHU Sainte-Justine added, “TSHA-102 was well-tolerated in both adult patients treated, with no serious adverse events or dose-limiting toxicities as of week 52 and week 36 post-treatment for the first and second patient, respectively.

Elsa Rossignol，M.D.，FRCP，FAAP，蒙特利尔大学神经科学和儿科副教授，CHU Sainte-Justine的REVEAL青少年和成人试验的首席研究员补充道，“TSHA-102在两名接受治疗的成年患者中耐受性良好，第一名和第二名患者分别在治疗后第52周和第36周没有严重的不良事件或剂量限制性毒性。

It’s encouraging that we continue to see improvements across multiple clinical domains in the longer-term assessments with no diminution of effect. The first adult patient sustained improvements at week 52 post-treatment after the completion of her steroid and sirolimus taper, including regaining movement in her legs, the gained ability to sit unassisted for the first time in over a decade and gained function in her non-dominant hand.

令人鼓舞的是，在长期评估中，我们继续看到多个临床领域的改善，而效果没有减弱。。

She continues to show vastly increased interest in social communication and activities, as well as improvements in breathing dysrhythmia and normalized sleep behaviors for the first time in 20 years. The second adult patient showed sustained improvements following the completion of her steroid taper at week 25 post-treatment, including reduced hand stereotypies for the first time since regression at age three, sustained improvements in breathing dysrhythmia and a significant reduction in seizures, as she has been seizure-free for 8.5 months relative to experiencing 2-4 seizures per week pre-treatment.

她继续对社交交流和活动表现出极大的兴趣，并在20年来首次改善了呼吸节律异常和正常的睡眠行为。第二名成年患者在治疗后第25周完成类固醇减量后表现出持续改善，包括自三岁消退以来首次减少手部刻板印象，持续改善呼吸节律异常和癫痫发作显着减少，因为相对于治疗前每周发作2-4次，她已经无癫痫发作8.5个月。

Additionally, the patient showed improvement in posture and stability at week 25 post-treatment. We believe these longer-term clinical data support the durability and broad clinical benefits of TSHA-102 in adult patients with the most advanced stage of Rett syndrome.”.

此外，患者在治疗后第25周表现出姿势和稳定性的改善。我们相信这些长期的临床数据支持TSHA-102在Rett综合征最晚期的成年患者中的持久性和广泛的临床益处。”。

REVEAL Phase 1/2 Adolescent and Adult Trial (Canada and U.S.): a first-in-human, open-label, randomized, dose-escalation and dose-expansion study evaluating the safety and preliminary efficacy of TSHA-102 in adolescent and adult females aged 12 years and older with Rett syndrome due to MECP2 loss-of-function mutation.

REVEAL 1/2期青少年和成人试验（加拿大和美国）：一项首次在人体内进行的开放标签，随机，剂量递增和剂量扩展研究，评估TSHA-102在12岁及以上的青少年和成年女性中的安全性和初步疗效。由于MECP2功能丧失突变，患有Rett综合征。

TSHA-102 is administered as a single lumbar intrathecal injection. Dose escalation will evaluate two dose levels of TSHA-102 sequentially. The maximum tolerated dose (MTD) or maximum administered dose (MAD) established in Part A will then be administered during dose expansion in Part B of the study..

TSHA-102作为单次腰椎鞘内注射给药。剂量递增将依次评估TSHA-102的两个剂量水平。。。

Completed dosing in cohort one (low dose, n=2) of 5.7x1014 total vg

队列一（低剂量，n=2）完成剂量为5.7x1014总vg

Dosed first patient in cohort two (high dose, n=3) of 1x1015 total vg in the second quarter of 2024

2024年第二季度，第二组（高剂量，n=3）的第一名患者服用了1x1015总vg

Initial available safety and efficacy data from cohort two expected in the second half of 2024

预计2024年下半年第二组的初步可用安全性和有效性数据

Longer-term data from the first adult patient (20 years old; large MECP2 deletion; associated with severe phenotype) and second adult patient (21 years old; missense MECP2 mutation; associated with milder phenotype) with late motor deterioration, stage four Rett syndrome dosed with TSHA-102 in the low dose cohort:.

来自第一名成年患者（20岁；较大的MECP2缺失；与严重表型相关）和第二名成年患者（21岁；错义MECP2突变；与较轻的表型相关）的长期数据，晚期运动恶化，在低剂量队列中服用TSHA-102的第四阶段Rett综合征：。

Generally well-tolerated with no serious adverse events (SAEs) related to TSHA-102 or dose-limiting toxicities (DLTs) as of 52-week assessment post-treatment for patient one and 36-week assessment post-treatment for patient two

一般耐受性良好，截至患者1治疗后52周评估和患者2治疗后36周评估，没有与TSHA-102相关的严重不良事件（SAE）或剂量限制性毒性（DLT）

Sustained and new improvements observed across multiple clinical domains relative to baseline, as of 52-weeks post-treatment for patient one, based on clinical observations reported by the Principal Investigator (PI), including:

根据首席研究者（PI）报告的临床观察，截至患者1治疗后52周，在多个临床领域相对于基线观察到持续和新的改善，包括：

Motor skills: improved hand function and gained ability to sit unassisted for first time in over a decade and move legs (patient one), and improved hand stereotypies for the first time since regression at age three and improved posture and stability (patient two)

运动技能：十年多来首次改善了手部功能，获得了独立坐着和移动腿的能力（患者一），自三岁回归以来首次改善了手部刻板印象，并改善了姿势和稳定性（患者二）

Communication/Socialization: improved social interest, vocalization and ability to use eye-gaze driven communication device (patient one), and improved social interest with increased response to spoken words and eye contact (patient two)

沟通/社会化：提高社会兴趣、发声和使用眼神驱动的沟通设备的能力（患者一），提高社会兴趣，增加对口语和眼神交流的反应（患者二）

Autonomic function: improved breathing patterns, normalized sleep quality/duration for first time in 20 years and improved circulation (patient one), and improved breathing patterns and circulation (patient two)

自主神经功能：改善呼吸模式，20年来首次恢复正常的睡眠质量/持续时间，改善循环（患者一），改善呼吸模式和循环（患者二）

Seizures: stable seizure events (patient one), and significantly reduced seizure events (patient two)

癫痫发作：癫痫发作事件稳定（患者1），癫痫发作事件明显减少（患者2）

Seizure Diary and caregiver reports:

癫痫发作日记和护理人员报告：

Patient one at week 52 post-treatment: stable seizure events at lower levels of anti-seizure medication relative to baseline

治疗后第52周的患者一：相对于基线水平，抗癫痫药物水平较低时癫痫发作稳定

Patient two at week 36 post-treatment: significantly reduced seizure events at 25% lower levels of anti-seizure medication relative to baseline (2-4 seizures per week), with 8.5 months reported seizure-free

治疗后第36周的患者2：相对于基线水平（每周2-4次癫痫发作），抗癫痫药物水平降低了25%，癫痫发作事件显着减少，据报道无癫痫发作8.5个月

Clinical improvements seen across multiple efficacy measurements relative to baseline include:

相对于基线，多项疗效测量的临床改善包括：

Patient one at week 52 post-treatment: Sustained improvement in Clinical Global Impression–Improvement (CGI-I), Clinical Global Impression–Severity (CGI-S) and Seizure Diaries, with new improvement in Revised Motor Behavior Assessment (R-MBA), Parental Global Impressions–Improvement (PGI-I) and Rett Syndrome Behavior Questionnaire (RSBQ) following completion of steroid and sirolimus taper.

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Patient two at week 25 post-treatment: Sustained improvement in CGI-I and PGI-I, with new improvement in R-MBA and Seizure Diaries following completion of steroid taper

患者2在治疗后第25周：CGI-I和PGI-I持续改善，在完成类固醇减量后，R-MBA和癫痫发作日记有了新的改善

Colleen Buhrfiend, M.D., Assistant Professor of Pediatrics at RUSH University Medical Center said, “Following treatment with TSHA-102, both pediatric patients with different genotypes and disease severity had challenging side effects related to immunosuppressant treatment but showed a well-tolerated safety profile with no SAEs or DLTs related to TSHA-102 as of week 22 and week 11 post-treatment for the first and second pediatric patient, respectively, as well as some initial improvements across multiple clinical domains and early evidence of new developmental gains.

拉什大学医学中心儿科助理教授科琳·布尔菲德（ColleenBuhrfiend）医学博士说：“在接受TSHA-102治疗后，不同基因型和疾病严重程度的儿科患者都有与免疫抑制剂治疗相关的具有挑战性的副作用，但在治疗后第22周和第11周，第一名和第二名儿科患者的安全性良好，没有与TSHA-102相关的SAE或DLT，以及多个临床领域的一些初步改善和新的发展进展的早期证据。

Specifically, at week 12 post-treatment, the first patient’s truncal stability and balance improved, which enabled her to sit unassisted for a longer duration and move her leg on her own to better take a step with assistance. Her hand function improved, and she was able to hold an object for three minutes following treatment compared to up to 12 seconds pre-treatment.

具体来说，在治疗后第12周，第一位患者的躯干稳定性和平衡性得到改善，这使她能够在无人帮助的情况下坐更长的时间，并自行移动腿部，以便在有帮助的情况下更好地迈出一步。她的手功能得到了改善，治疗后她能够拿着物体三分钟，而治疗前长达12秒。

Additionally, she communicated new words using an eye-gaze driven communication device and gained the ability to identify object functions for the first time. At week eight post-treatment, the second pediatric patient’s gait, speed and stability improved, resulting in the ability to walk longer distances.

此外，她使用眼睛注视驱动的交流设备交流新词，并首次获得了识别物体功能的能力。在治疗后第八周，第二名儿科患者的步态，速度和稳定性得到改善，从而能够走更长的距离。

Her hand function showed initial improvement, and she gained some new skills that were previously lost, including the ability to stand up from a chair and walk up a stair. The initial improvements observed across multiple areas of disease in both pediatric patients are encouraging early signs of possible benefit.”.

她的手功能显示出初步的改善，她获得了一些以前失去的新技能，包括从椅子上站起来和走上楼梯的能力。在两名儿科患者的多个疾病领域观察到的初步改善正在鼓励可能获益的早期迹象。”。

REVEAL Phase 1/2 Pediatric Trial (U.S. and U.K.): an open-label, randomized, dose-escalation and dose-expansion study evaluating the safety and preliminary efficacy of TSHA-102 in pediatric females with Rett syndrome due to MECP2 loss-of-function mutation. TSHA-102 is administered as a single lumbar intrathecal injection.

REVEAL 1/2期儿科试验（美国和英国）：一项开放标签，随机，剂量递增和剂量扩展研究，评估TSHA-102在因MECP2功能丧失突变而患有Rett综合征的儿科女性中的安全性和初步疗效。TSHA-102作为单次腰椎鞘内注射给药。

Part A of the study will focus on determining MAD and MTD in patients aged 5 to 8 years old. Part B is the dose expansion phase and will evaluate TSHA-102 at the MAD or MTD in two age cohorts (5 to 8 years and 3 to 5 years)..

该研究的A部分将重点确定5至8岁患者的MAD和MTD。B部分是剂量扩展阶段，将在两个年龄组（5至8岁和3至5岁）的MAD或MTD中评估TSHA-102。。

Completed dosing in cohort one (low dose, n=2) of 5.7x1014 total vg

队列一（低剂量，n=2）完成剂量为5.7x1014总vg

Received IDMC approval of Company’s request to advance early to cohort two (high dose, n=3) evaluating 1x1015 total vg, with dosing to occur following IDMC review of the 42-day safety data from the first high dose patient in the adolescent and adult trial

获得IDMC批准公司要求提前进行第二组（高剂量，n=3）评估1x1015总vg，剂量将在IDMC审查青少年和成人试验中第一名高剂量患者的42天安全性数据后进行

Dosing of first pediatric patient in cohort two expected in the third quarter of 2024

预计2024年第三季度第二组中第一名儿科患者的剂量

Initial available safety and efficacy data from cohort two expected in the second half of 2024

预计2024年下半年第二组的初步可用安全性和有效性数据

Initial results from the first pediatric patient (6 years old; MECP2 deletion; associated with moderate phenotype) and second pediatric patient (7 years old; missense MECP2 mutation; associated with milder phenotype) with pseudo stationary symptoms, stage three Rett syndrome dosed with TSHA-102 in the low dose cohort:.

第一名患有假性静止症状的儿科患者（6岁；MECP2缺失；与中度表型相关）和第二名患有假性静止症状的儿科患者（7岁；错义MECP2突变；与轻度表型相关）的初步结果，在低剂量队列中服用TSHA-102的第三阶段Rett综合征：。

Generally well-tolerated with no SAEs related to TSHA-102 or DLTs as of 22-week assessment post-treatment for patient one and 11-week assessment post-treatment for patient two; there were two SAEs reported in the second pediatric patient that were not deemed treatment-related (both were related to underlying disease and one was also attributed to immunosuppression) and have resolved .

一般耐受性良好，截至患者1治疗后22周评估和患者2治疗后11周评估，没有与TSHA-102或DLTs相关的SAE；在第二位儿科患者中报告了两种SAE，它们不被认为与治疗有关（两者都与潜在疾病有关，一种也归因于免疫抑制），并且已经解决。。

Significant challenges with AEs dues to immunosuppressive regimen

Initial improvements observed across multiple clinical domains relative to baseline as of 12-weeks post-treatment for patient one and 8-weeks post-treatment for patient two, based on clinical observations reported by the PI:

根据PI报告的临床观察，截至患者1治疗后12周和患者2治疗后8周，相对于基线，在多个临床领域观察到的初步改善：

Motor skills: improved hand function with the ability to hold an object for three minutes vs up to 12 seconds at baseline, improved truncal stability and balance with the gained ability to move her leg on her own to better take a step with assistance and sit unassisted for a longer duration, and improved swallowing and oral intake relative to gastrostomy tube feeding (patient one), and improved hand function and gait, speed and stability when walking with some new skills gained, including standing up from a chair and walking up a stair (patient two).

运动技能：改善手部功能，能够握住物体三分钟，而基线时最多可达12秒，改善躯干稳定性和平衡性，能够独立移动腿部，以便在帮助下更好地迈出一步，并在更长的时间内独自坐着，相对于胃造口管喂养，吞咽和口腔摄入得到改善（患者一），步行时手部功能和步态得到改善，速度和稳定性得到改善，并获得了一些新技能，包括从椅子上站立和爬楼梯（患者二）。

Communication/Socialization: improved communication and ability to use eye-gaze driven communication device and gained visual reception and receptive language skills (patient one), and improved social interest and eye contact (patient two)

沟通/社会化：提高沟通能力和使用眼神驱动的沟通设备的能力，获得视觉接收和接受语言技能（患者一），提高社会兴趣和眼神交流（患者二）

Autonomic function: reduced breath holding (patient one), and improved breathing patterns (patient two)

自主神经功能：减少屏气（患者一），改善呼吸模式（患者二）

Seizures: stable seizure events (patient one), and increase in days reported seizure-free since dosing (patient two)

癫痫发作：癫痫发作事件稳定（患者1），自给药后报告无癫痫发作的天数增加（患者2）

Seizure Diary and caregiver reports:

癫痫发作日记和护理人员报告：

Patient one at 22-weeks post-treatment: stable seizure events relative to baseline

患者一在治疗后22周：相对于基线稳定的癫痫发作事件

Patient two at 11-weeks post-treatment: increase in days reported seizure-free since dosing relative to baseline (2-4 seizures daily), although a new anti-seizure medication was added to patient two’s regimen at week four, which she has maintained through week 11 post-treatment

患者2在治疗后11周：相对于基线（每天2-4次癫痫发作），自给药以来报告无癫痫发作的天数增加，尽管患者2在第4周的治疗方案中添加了新的抗癫痫药物，但她一直维持到治疗后11周

Clinical improvements seen across multiple efficacy measurements relative to baseline include:

相对于基线，多项疗效测量的临床改善包括：

Patient one at 12-weeks post-treatment: CGI-I, PGI-I, R-MBA, Adapted Mullen Scales of Early Learning (MSEL-A) and Seizure Diaries

治疗后12周的患者一：CGI-I，PGI-I，R-MBA，适应的马伦早期学习量表（MSEL-A）和癫痫发作日记

Patient two at 8-weeks post-treatment: CGI-I, PGI-I, RSBQ, R-MBA and Seizure Diaries

治疗后8周的患者2：CGI-I，PGI-I，RSBQ，R-MBA和癫痫发作日记

Presentation with additional details and accompanying figures are available through Taysha’s website here.

Taysha的网站上提供了更多详细信息和附图。

Conference Call and Webcast Information

电话会议和网络广播信息

Taysha management will hold a conference call and webcast today at 8:00 a.m. ET to discuss these clinical data for TSHA-102 in Rett syndrome. Participants can register for the live webcast here. The live webcast and replay will be available through Taysha’s website here.

Taysha管理层将于美国东部时间今天上午8:00举行电话会议和网络广播，讨论Rett综合征中TSHA-102的这些临床数据。参与者可以在此处注册直播。现场直播和重播将通过Taysha的网站提供。

2024 IRSF Rett Syndrome Scientific Meeting Presentation Details

2024年IRSF Rett综合征科学会议介绍详情

These data will also be presented by Elsa Rossignol, M.D., FRCP, FAAP, Principal Investigator of the REVEAL adolescent and adult trial at CHU Sainte-Justine and Colleen Buhrfiend, M.D., of RUSH University Medical Center at the 2024 International Rett Syndrome Foundation (IRSF) Rett Syndrome Scientific Meeting during a poster presentation on Tuesday, June 18 at 5:15 p.m.

这些数据也将由Elsa Rossignol，M.D.，FRCP，FAAP在2024年国际Rett综合征基金会（IRSF）Rett综合征科学会议上于6月18日星期二下午5:15在拉什大学医学中心的CHU Sainte Justine和Colleen Buhrfiend进行的REVEAL青少年和成人试验的主要研究者进行展示。

MT and during an oral presentation on Wednesday, June 19 at 11:00 a.m. MT..

MT，并于6月19日星期三上午11:00进行口头陈述。。

About TSHA-102

关于TSHA-102

TSHA-102 is a self-complementary intrathecally delivered AAV9 investigational gene transfer therapy in clinical evaluation for Rett syndrome. Designed as a one-time lumbar intrathecal treatment, TSHA-102 aims to address the genetic root cause of the disease by delivering a functional form of MECP2 to cells in the CNS.

TSHA-102是一种自我补充的鞘内注射AAV9研究性基因转移疗法，用于Rett综合征的临床评估。TSHA-102被设计为一次性腰椎鞘内治疗，旨在通过向中枢神经系统的细胞递送功能形式的MECP2来解决该疾病的遗传根源。

TSHA-102 utilizes a novel miRNA-Responsive Auto-Regulatory Element (miRARE) technology designed to mediate levels of MECP2 in the CNS on a cell-by-cell basis without risk of overexpression. TSHA-102 has received Regenerative Medicine Advanced Therapy, Fast Track and Orphan Drug and Rare Pediatric Disease designations from the FDA, Orphan Drug designation from the European Commission and Innovative Licensing and Access Pathway designation from the Medicines and Healthcare products Regulatory Agency..

TSHA-102利用一种新型的miRNA响应性自动调节元件（miRARE）技术，旨在逐个细胞介导中枢神经系统中MECP2的水平，而不存在过表达的风险。TSHA-102已获得美国食品和药物管理局（FDA）的再生医学高级疗法、快速通道和孤儿药以及罕见儿科疾病指定、欧盟委员会的孤儿药指定以及美国药品和保健品管理局（Medicines and Healthcare products Regulatory Agency）的创新许可和获取途径指定。。

About Rett Syndrome

关于Rett综合征

Rett syndrome is a rare neurodevelopmental disorder caused by mutations in the X-linked MECP2 gene encoding methyl CpG-binding protein 2 (MeCP2), which is essential for regulating neuronal and synaptic function in the brain. The disorder is characterized by loss of communication and hand function, slowing and/or regression of development, motor and respiratory impairment, seizures, intellectual disabilities and shortened life expectancy.

Rett综合征是一种罕见的神经发育障碍，由编码甲基CpG结合蛋白2（MECP2）的X连锁MECP2基因突变引起，这对于调节大脑中的神经元和突触功能至关重要。该疾病的特征是失去沟通和手部功能，发育减慢和/或消退，运动和呼吸障碍，癫痫发作，智力残疾和预期寿命缩短。

Rett syndrome progression is divided into four key stages, beginning with early onset stagnation at 6 to 18 months of age followed by rapid regression, plateau and late motor deterioration. Rett syndrome primarily occurs in females and is one of the most common genetic causes of severe intellectual disability.

Rett综合征的进展分为四个关键阶段，从6至18个月大的早期发作停滞开始，然后是快速消退，高原和晚期运动恶化。Rett综合征主要发生在女性中，是严重智力残疾的最常见遗传原因之一。

Currently, there are no approved disease-modifying therapies that treat the genetic root cause of the disease. Rett syndrome caused by a pathogenic/likely pathogenic MECP2 mutation is estimated to affect between 15,000 and 20,000 patients in the U.S., EU, and U.K..

目前，还没有批准的疾病缓解疗法来治疗疾病的遗传根源。据估计，由致病性/可能致病性MECP2突变引起的Rett综合征会影响美国的15000至20000名患者。S、 ，欧盟和美国。K、 。。

About Taysha Gene Therapies

关于Taysha基因疗法

Taysha Gene Therapies (Nasdaq: TSHA) 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. Its lead clinical program TSHA-102 is in development for Rett syndrome, a rare neurodevelopmental disorder with no approved disease-modifying therapies that address the genetic root cause of the disease.

Taysha Gene Therapes（纳斯达克：TSHA）是一家临床阶段生物技术公司，专注于推进基于腺相关病毒（AAV）的基因疗法，用于治疗中枢神经系统的严重单基因疾病。它的主要临床项目TSHA-102正在针对Rett综合征进行开发，Rett综合征是一种罕见的神经发育障碍，没有经过批准的疾病缓解疗法来解决该疾病的遗传根源。

With a singular focus on developing transformative medicines, Taysha aims to address severe unmet medical needs and dramatically improve the lives of patients and their caregivers. The Company’s management team has proven experience in gene therapy development and commercialization. Taysha leverages this experience, its manufacturing process and a clinically and commercially proven AAV9 capsid in an effort to rapidly translate treatments from bench to bedside.

Taysha专注于开发变革性药物，旨在解决严重未满足的医疗需求，并显着改善患者及其护理人员的生活。该公司的管理团队在基因治疗开发和商业化方面拥有丰富的经验。Taysha利用这一经验，其制造过程以及临床和商业证明的AAV9衣壳，努力将治疗从台式快速转化为床边。

For more information, please visit www.tayshagtx.com..

有关更多信息，请访问www.tayshagtx.com。。

Forward-Looking Statements

前瞻性声明

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as “anticipates,” “believes,” “expects,” “intends,” “projects,” “plans,” and “future” or similar expressions are intended to identify forward-looking statements.

本新闻稿包含1995年《私人证券诉讼改革法案》所指的前瞻性声明。诸如“预期”、“相信”、“期望”、“打算”、“项目”、“计划”和“未来”等词语或类似表达旨在识别前瞻性陈述。

Forward-looking statements include, but are not limited to, statements concerning the potential of TSHA-102 and Taysha’s other product candidates, to positively impact quality of life and alter the course of disease in the patients Taysha seeks to treat, its research, development and regulatory plans for its product candidates, including the anticipated timelines for reporting data for the TSHA-102 REVEAL trials and the trial design of the TSHA-102 REVEAL trials, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed and the potential market opportunity for Taysha’s product candidates.

前瞻性声明包括但不限于关于TSHA-102和Taysha其他候选产品对Taysha寻求治疗的患者的生活质量和疾病进程产生积极影响的潜力的声明，其候选产品的研究，开发和监管计划，包括报告TSHA-102 REVEAL试验数据的预期时间表和TSHA-102 REVEAL试验的试验设计，这些候选产品获得FDA或同等外国监管机构监管批准的潜力，以及这些候选产品是否会成功分销和上市，以及Taysha候选产品的潜在市场机会。

Forward-looking statements are based on management’s current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements.

前瞻性陈述基于管理层当前的预期，并受到各种风险和不确定性的影响，这些风险和不确定性可能导致实际结果与此类前瞻性陈述所表达或暗示的结果产生重大不利影响。因此，这些前瞻性声明不构成对未来业绩的保证，请注意不要过度依赖这些前瞻性声明。

Risks regarding Taysha’s business are described in detail in its SEC filings, including in Taysha’s Annual Report on Form 10-K for the full-year ended December 31, 2023, which is available on the SEC’s website at www.sec.gov. Additional information will be made available in other filin.

Taysha的业务风险在其向美国证券交易委员会提交的文件中有详细描述，包括Taysha截至2023年12月31日的10-K表年度报告，该报告可在美国证券交易委员会的网站www.SEC.gov上查阅。其他文件将提供更多信息。

Company Contact:

公司联系人：

Hayleigh Collins

Hayleigh Collins

Director, Head of Corporate Communications, and Investor Relations

董事、企业传播和投资者关系主管

Taysha Gene Therapies, Inc.

Taysha基因治疗公司。

hcollins@tayshagtx.com

hcollins@tayshagtx.com

Media Contact:

媒体联系人：

Carolyn Hawley

Carolyn Hawley

Inizio Evoke

启动Evoke

Carolyn.hawley@inizioevoke.com

Carolyn.hawley@inizioevoke.com