RICHMOND, Calif.--(

加利福尼亚州里士满--(

BUSINESS WIRE

商业热线

)--Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced updated data from the Phase 1/2 STAAR study evaluating isaralgagene civaparvovec, or ST-920, a wholly owned gene therapy product candidate for the treatment of Fabry disease. Updated data continue to support the potential of isaralgagene civaparvovec as a one-time, durable treatment option for Fabry disease that can improve patient outcomes..

)--基因组医学公司Sangamo Therapeutics，Inc.（Nasdaq:SGMO）今天宣布了1/2期STAAR研究的最新数据，该研究评估了ISARAGAGENE civaparvovec或ST-920，这是一种用于治疗法布里病的全资基因治疗候选产品。最新数据继续支持isaralgagene civaparvovec作为法布里病的一次性持久治疗选择的潜力，可以改善患者的预后。。

These data will be presented at the 21

这些数据将在21

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Annual WORLD

年度世界

Symposium

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TM公司

in San Diego, CA on February 6, 2025, in an oral presentation in the Clinical Applications session from 9:30-10:30am P.T. and a poster presentation from 3:30-5:30pm P.T. (Poster Ref: 146). These data will also be available on Sangamo’s website on the

2025年2月6日，在加利福尼亚州圣地亚哥，于上午9:30-10:30在临床应用会议上进行口头演示，并于下午3:30-5:30进行海报演示（海报参考号：146）。这些数据也将在Sangamo的网站上提供

Presentations

演示文稿

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These updated data from the Phase 1/2 STAAR study are highly encouraging, particularly the positive mean eGFR slope observed in patients with at least one year of follow-up, indicating improvements in renal function, an important predicter of morbidity and mortality in Fabry disease. Additionally, these data show the strong safety and sustained benefit profiles of ST-920, as well as its ability to improve key quality of life measures,” said Professor Derralynn Hughes, MA Dphil FRCP FRCPath, Royal Free London NHS Foundation Trust and investigator of the Phase 1/2 STAAR study.

来自1/2期STAAR研究的这些最新数据非常令人鼓舞，特别是在至少一年随访的患者中观察到的平均eGFR斜率为阳性，表明肾功能有所改善，这是法布里病发病率和死亡率的重要预测指标。此外，这些数据显示ST-920具有强大的安全性和持续效益，以及改善关键生活质量指标的能力，”Derralynn Hughes教授，MA Dphil FRCP FRCPath，皇家自由伦敦NHS基金会信托基金会和1/2期STAAR研究的研究者说。

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These data support the potential of ST-920 to be a single-dose, durable treatment option for people living with Fabry disease.”

这些数据支持ST-920成为法布里病患者单剂量持久治疗选择的潜力。”

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Following our alignment with the FDA on an Accelerated Approval Pathway for ST-920, we are thrilled with how the data are progressing, particularly the positive one-year mean eGFR slope data that will serve as the primary efficacy endpoint for our regulatory submission,” said Nathalie Dubois-Stringfellow, Ph.

在我们与FDA就ST-920的加速批准途径达成一致后，我们对数据的进展感到兴奋，特别是作为我们监管提交的主要疗效终点的阳性一年平均eGFR斜率数据，”Nathalie Dubois Stringfellow博士说。

D, Chief Development Officer at Sangamo. “.

D、 Sangamo首席开发官。“。

We look forward to building upon the STAAR study’s positive results as we advance our interactions with the FDA ahead of the potential BLA submission in the second half of 2025 and we also continue to engage with the European Medicines Agency.”

我们期待着在STAAR研究的积极成果基础上再接再厉，在2025年下半年提交BLA之前推进与FDA的互动，并继续与欧洲药品管理局合作。”

Updated Phase 1/2 STAAR Study Results (as of the September 12, 2024 cut-off date)

更新的1/2期STAAR研究结果（截至2024年9月12日截止日期）

Safety (all dosed patients):

安全性（所有给药患者）：

Isaralgagene civaparvovec continued to be generally well-tolerated, with the majority of adverse events being grade 1-2 in nature.

Isaralgagene civaparvovec总体上耐受性良好，大多数不良事件本质上是1-2级。

No liver function test (LFT) elevations post-dosing requiring steroids occurred. No adverse events led to study discontinuation and there were no deaths.

没有发生需要类固醇的给药后肝功能测试（LFT）升高。没有不良事件导致研究中断，也没有死亡。

Efficacy (all dosed patients):

疗效（所有给药患者）：

Elevated expression of α-Gal A activity maintained for up to 47 months for the longest treated patient, and up to 27 months for the longest treated patient receiving the highest dose (2.63x10

对于治疗时间最长的患者，α-半乳糖A活性的表达升高维持了47个月，对于接受最高剂量（2.63x10）治疗时间最长的患者，α-半乳糖A活性的表达升高维持了27个月

13

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vg/kg).

vg/kg）。

All 18 patients who began the study on ERT have been withdrawn from ERT and all remain off ERT as of today. Plasma lyso-Gb3 levels in these patients remained stable following ERT withdrawal for up to 33 months for the longest treated patient.

开始ERT研究的所有18名患者均已退出ERT，截至今日，所有患者均未接受ERT。对于治疗时间最长的患者，ERT停药后长达33个月，这些患者的血浆lyso-Gb3水平保持稳定。

Of the 10 patients who had measurable titers of total antibodies (Ab) or neutralizing antibodies (Nab) against α-Gal A associated with ERT at baseline, total Ab or NAb titers decreased markedly in nine patients and became undetectable in seven following ST-920 treatment.

在基线时与ERT相关的针对α-GalA的总抗体（Ab）或中和抗体（Nab）滴度可测量的10名患者中，9名患者的总Ab或Nab滴度显着降低，7名患者在ST-920治疗后检测不到。

Efficacy (23 dosed patients followed for at least 12 months):

A positive mean annualized eGFR slope of 3.061 mL/min/1.73m

年平均eGFR斜率为3.061 mL/min/1.73m

2

2

/year (95% confidence interval: 0.863, 5.258) was observed, indicating notable improvements in kidney function.

/观察到年份（95%置信区间：0.863，5.258），表明肾功能显着改善。

Improvements in disease severity were reported in the Fabry Outcome Survey adaptation of the Mainz Severity Score Index (FOS-MSSI) age-adjusted score, with 15 patients showing improvements in their total MSSI score and seven patients improving their FOS-MSSI disease category.

美因茨严重程度评分指数（FOS-MSSI）年龄调整评分的法布里结果调查适应症报告了疾病严重程度的改善，其中15名患者的总MSSI评分有所改善，7名患者的FOS-MSSI疾病类别有所改善。

Significant improvements continued to be observed in the short form-36 (SF-36) quality of life (QoL) scores reported, with a mean change in General Health score of 10.6 (p=0.0020). For context, a three- to five-point change on any SF-36 score is considered a minimally clinically important difference..

在报告的简表36（SF-36）生活质量（QoL）评分中继续观察到显着改善，总体健康评分的平均变化为10.6（p=0.0020）。在上下文中，任何SF-36评分的三到五分变化都被认为是临床上最小的重要差异。。

Significant improvements in physical component, bodily pain, physical, vitality, social function, and emotional SF-36 scores were also observed.

还观察到身体成分，身体疼痛，身体，活力，社会功能和情绪SF-36评分的显着改善。

Statistically significant improvements continued to be seen in the gastrointestinal symptom rating scale (GSRS) compared to baseline.

与基线相比，胃肠道症状评定量表（GSRS）继续出现统计学上显着的改善。

Collectively, Sangamo believes these data continue to support the potential for isaralgagene civaparvovec as a one-time, durable treatment for Fabry disease that can improve patient outcomes.

总的来说，Sangamo认为这些数据继续支持isaralgagene civaparvovec作为法布里病一次性持久治疗的潜力，可以改善患者的预后。

Enrollment and dosing are complete in the Phase 1/2 STAAR study. In October 2024, Sangamo announced that the FDA had provided a clear regulatory pathway to Accelerated Approval for isaralgagene civaparvovec using data from ongoing Phase 1/2 STAAR study, avoiding the requirement for an additional registrational study and accelerating estimated time to potential approval by approximately three years.

在1/2期STAAR研究中完成了登记和给药。2024年10月，Sangamo宣布，FDA已经提供了一个明确的监管途径，使用正在进行的1/2期STAAR研究的数据加速批准isaralgagene civaparvovec，避免了额外注册研究的要求，并将潜在批准的估计时间缩短了约三年。

The FDA agreed in a Type B interaction that data from the ongoing Phase 1/2 STAAR study can serve as the primary basis for approval under the Accelerated Approval Program, using eGFR slope at 52 weeks across all patients as an intermediate clinical endpoint..

FDA在B型相互作用中同意，正在进行的1/2期STAAR研究的数据可以作为加速批准计划下批准的主要基础，使用所有患者52周的eGFR斜率作为中间临床终点。。

The 52-week eGFR slope data from all enrolled patients in the Phase 1/2 STAAR study will be available in the first half of 2025. A potential BLA submission is anticipated in the second half of 2025. Sangamo continues to advance business development discussions regarding a potential ST-920 collaboration agreement..

2025年上半年将提供1/2期STAAR研究中所有入选患者的52周eGFR斜率数据。预计2025年下半年可能会提交BLA。Sangamo继续推进有关潜在ST-920合作协议的业务发展讨论。。

A Current Report on Form 8-K summarizing the updated preliminary results from the Phase 1/2 STAAR study in more detail will be filed by Sangamo, and this press release is subject to the further detail provided in that Form 8-K.

Sangamo将提交一份表格8-K的最新报告，其中更详细地总结了1/2期STAAR研究的最新初步结果，本新闻稿以表格8-K中提供的进一步细节为准。

About the STAAR Study

关于STAAR研究

The Phase 1/2 STAAR study is a global open-label, single-dose, dose-ranging, multicenter clinical study designed to evaluate isaralgagene civaparvovec, or ST-920, a gene therapy product candidate in patients with Fabry disease. Isaralgagene civaparvovec requires a one-time infusion without preconditioning.

1/2期STAAR研究是一项全球开放标签，单剂量，剂量范围的多中心临床研究，旨在评估isaralgagene civaparvovec或ST-920，这是法布里病患者的基因治疗候选产品。Isaralgagene civaparvovec需要一次性输注而无需预处理。

The STAAR study enrolled patients who are on ERT, are ERT pseudo-naïve (defined as having been off ERT for six or more months), or who are ERT-naïve. The FDA has granted Orphan Drug, Fast Track and RMAT designations to isaralgagene civaparvovec, which has also received Orphan Medicinal Product designation and PRIME eligibility from the European Medicines Agency and Innovative Licensing and Access Pathway from U.K.

STAAR研究招募了接受ERT治疗的患者，这些患者是ERT伪天真的（定义为停止ERT治疗六个月或更长时间）或未接受ERT治疗的患者。美国食品和药物管理局（FDA）已授予isaralgagene civaparvovec孤儿药、快速通道和RMAT名称，该公司还获得了欧洲药品管理局（European Medicines Agency）的孤儿药名称和主要资格，以及英国的创新许可和准入途径。

Medicines and Healthcare products Regulatory Agency..

药品和保健品管理局。。

About Fabry Disease

关于法布里病

Fabry disease is a lysosomal storage disorder caused by mutations in the galactosidase alpha gene (GLA), which leads to deficient alpha-galactosidase A (α-Gal A) enzyme activity, which is necessary for metabolizing globotriaosylceramide (Gb3). The buildup of Gb3 in the cells can cause serious damage to vital organs, including the kidney, heart, nerves, eyes, gut and skin.

法布里病是由半乳糖苷酶α基因（GLA）突变引起的溶酶体贮积病，导致α-半乳糖苷酶a（α-GalA）酶活性不足，这是代谢球三糖神经酰胺（Gb3）所必需的。Gb3在细胞中的积累会对重要器官造成严重损害，包括肾脏、心脏、神经、眼睛、肠道和皮肤。

Symptoms of Fabry disease can include decreased or absent sweat production, heat intolerance, angiokeratoma (skin blemishes), vision problems, kidney disease, heart failure, gastrointestinal disturbance, mood disorders, neuropathic pain and tingling in the extremities..

法布里病的症状可能包括出汗减少或缺乏，热不耐受，血管角化瘤（皮肤瑕疵），视力问题，肾脏疾病，心力衰竭，胃肠道紊乱，情绪障碍，神经性疼痛和四肢刺痛。。

About Sangamo Therapeutics

关于Sangamo Therapeutics

Sangamo Therapeutics is a genomic medicine company dedicated to translating ground-breaking science into medicines that transform the lives of patients and families afflicted with serious neurological diseases who do not have adequate or any treatment options. Sangamo believes that its zinc finger epigenetic regulators are ideally suited to potentially address devastating neurological disorders and that its capsid discovery platform can expand delivery beyond currently available intrathecal delivery capsids, including the central nervous system.

Sangamo Therapeutics是一家基因组医学公司，致力于将开创性的科学转化为药物，以改变患有严重神经系统疾病但没有足够或任何治疗选择的患者和家庭的生活。Sangamo认为，其锌指表观遗传调节剂非常适合解决潜在的破坏性神经系统疾病，其衣壳发现平台可以将递送范围扩大到目前可用的鞘内递送衣壳（包括中枢神经系统）之外。

Sangamo’s pipeline also includes multiple partnered programs and programs with opportunities for partnership and investment. To learn more, visit .

Sangamo的渠道还包括多个合作项目和有合作和投资机会的项目。要了解更多信息，请访问。

www.sangamo.com

www.sangamo.com

and connect with us on

并与我们联系

LinkedIn

LinkedIn

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Forward-Looking Statements

前瞻性声明

This press release contains forward-looking statements regarding Sangamo’s current expectations. These forward-looking statements include, without limitation, statements relating to: the safety and efficacy and therapeutic potential of isaralgagene civaparvovec, including the potential for it to be a one-time, durable treatment option for Fabry disease that can improve patient outcomes; the presentation of clinical data from the Phase 1/2 STAAR study; the potential for isaralgagene civaparvovec to qualify for the FDA’s Accelerated Approval program, including the adequacy of data generated in the Phase 1/2 STAAR study to support any such approval; expectations concerning the availability of additional data to support a potential BLA submission for isaralgagene civaparvovec, and the timing of such submission; the potential to accelerate the expected timeline to approval of isaralgagene civaparvovec; Sangamo’s plans to advance discussions with the FDA and the European Medicines Agency; Sangamo’s plans to seek a potential collaboration partner for ST-920; and other statements that are not historical fact.

本新闻稿包含有关Sangamo当前期望的前瞻性声明。；1/2期STAAR研究的临床数据介绍；isaralgagene civaparvovec有资格获得FDA加速批准计划的潜力，包括1/2期STAAR研究中生成的数据是否足以支持任何此类批准；关于提供额外数据以支持isaralgagene civaparvovec的潜在BLA提交的预期，以及此类提交的时间；加快批准isaralgagene civaparvovec的预期时间表的潜力；桑加莫计划推进与FDA和欧洲药品管理局的讨论；Sangamo计划为ST-920寻找潜在的合作伙伴；以及其他非历史事实的陈述。

These statements are not guarantees of future performance and are subject to certain risks and uncertainties that are difficult to predict. Factors that could cause actual results to differ include, but are not limited to, risks and uncertainties related to Sangamo’s lack of capital resources to obtain regulatory approval for and commercialize its product candidates in a timely manner or at all, including the ability to secure a collaboration partner for ST-920; the uncertain timing and unpredictable nature of clinical trial results, including the risk that the therapeutic effects observed in the latest preliminary clinical data from the Phase 1/2 STAAR study will not be d.

这些声明并不能保证未来的业绩，并且会受到某些难以预测的风险和不确定性的影响。可能导致实际结果不同的因素包括但不限于与Sangamo缺乏资本资源相关的风险和不确定性，无法及时或根本获得监管部门对其候选产品的批准并将其商业化，包括确保ST-920合作伙伴的能力；临床试验结果的不确定时间和不可预测性，包括在1/2期STAAR研究的最新初步临床数据中观察到的治疗效果不会被d的风险。

There can be no assurance that Sangamo and its current or potential future partners will be able to develop commercially viable products. Actual results may differ materially from those projected in these forward-looking statements due to the risks and uncertainties described above and other risks and uncertainties that exist in the operations and business environments of Sangamo and its collaborators.

无法保证Sangamo及其当前或潜在的未来合作伙伴能够开发出商业上可行的产品。由于上述风险和不确定性以及Sangamo及其合作者的运营和业务环境中存在的其他风险和不确定性，实际结果可能与这些前瞻性声明中预测的结果存在重大差异。

These risks and uncertainties are described more fully in Sangamo’s Securities and Exchange Commission, or SEC, filings and reports, including in Sangamo’s Annual Report on Form 10-K for the year ended December 31, 2023, as supplemented by its Quarterly Report on Form 10-Q for the quarter ended September 30, 2024, each filed with the SEC, and future filings and reports that Sangamo makes from time to time with the SEC.

这些风险和不确定性在Sangamo的证券交易委员会（SEC）的文件和报告中有更全面的描述，包括截至2023年12月31日的Sangamo 10-K表格年度报告，以及截至2024年9月30日的10-Q表格季度报告（每个季度都提交给SEC），以及Sangamo不时向SEC提交的未来文件和报告。

Forward-looking statements contained in this announcement are made as of this date, and Sangamo undertakes no duty to update such information except as required under applicable law..

本公告中包含的前瞻性声明自即日起发布，Sangamo不承担更新此类信息的义务，除非适用法律要求。。