RICHMOND, Calif.--(BUSINESS WIRE)--Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced the outcome of a recent successful interaction with the U.S. FDA, providing a clear regulatory pathway to Accelerated Approval for isaralgagene civaparvovec, or ST-920, its wholly owned gene therapy product candidate for the treatment of Fabry disease..

加利福尼亚州里士满（商业新闻短讯）--基因组医学公司Sangamo Therapeutics，Inc.（纳斯达克：SGMO）今天宣布了最近与美国FDA成功互动的结果，为加速批准isaralgagene civaparvovec（ST-920）提供了明确的监管途径，ST-920是其全资拥有的用于治疗法布里病的候选基因治疗产品。。

The FDA has 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. The complete dataset to support an Accelerated Approval pathway will be available in the first half of 2025.

。支持加速审批途径的完整数据集将于2025年上半年提供。

This approach unlocks a potential BLA submission in the second half of 2025, three years ahead of previous estimates, and avoids the requirement for an additional registrational study to establish clinical efficacy..

这种方法在2025年下半年解锁了潜在的BLA提交，比以前的估计提前了三年，并且避免了需要额外的注册研究来确定临床疗效。。

Sangamo engaged with the FDA on alternative pathways to potential approval following analysis of clinical data from the Phase 1/2 STAAR study showing encouraging safety and efficacy data, including promising preliminary evidence of improved kidney function. Renal manifestations, such as proteinuria or a decreased glomerular filtration rate, occur early in life in almost all male, and in many female, patients with Fabry disease, and can lead to end-stage renal disease and early death.

Sangamo在对1/2期STAAR研究的临床数据进行分析后，与FDA就潜在批准的替代途径进行了接触，这些数据显示了令人鼓舞的安全性和有效性数据，包括有希望的肾功能改善的初步证据。几乎所有男性和许多女性法布里病患者在生命早期都会出现肾脏表现，例如蛋白尿或肾小球滤过率降低，并可能导致终末期肾脏疾病和早期死亡。

In the 18 male and female patients treated with isaralgagene civaparvovec with more than one year of follow-up data, statistically significant improvements were observed in both mean and median eGFR levels, resulting in a positive annualized eGFR slope. Based on these latest data, the FDA agreed that eGFR slope at 52 weeks can serve as an intermediate clinical endpoint to support a potential Accelerated Approval.

在接受isaralgagene civaparvovec治疗的18名男性和女性患者中，随访数据超过一年，平均和中位eGFR水平均观察到统计学上显着的改善，导致eGFR年化斜率为正。根据这些最新数据，FDA同意52周时的eGFR斜率可以作为中间临床终点，以支持潜在的加速批准。

The FDA also advised that eGFR slope at 104 weeks may be assessed to verify clinical benefit..

FDA还建议可以评估104周时的eGFR斜率，以验证临床益处。。

“Fabry is a debilitating disease, for which there is a serious unmet medical need,” said Sandy Macrae, Chief Executive Officer of Sangamo. “I strongly believe in the potential for ST-920 to alleviate many manifestations of Fabry disease and am delighted to have a clear regulatory pathway that could bring this treatment to patients significantly sooner than originally anticipated”..

Sangamo首席执行官桑迪·麦克雷（SandyMacrae）表示：“法布里（Fabry）是一种使人衰弱的疾病，严重的医疗需求尚未得到满足。”。“我坚信ST-920有可能缓解法布里病的许多表现，并且很高兴有一个明确的监管途径，可以比最初预期的更快地为患者带来这种治疗”。。

Dosing was completed in the Phase 1/2 STAAR study in April 2024, with a total of 33 patients dosed. The longest treated patient recently achieved four years of follow-up. The 18th and final patient who started the study on Enzyme Replacement Therapy (ERT) was successfully withdrawn from ERT in September 2024, and all 18 patients remain off ERT as of today.

2024年4月，在1/2期STAAR研究中完成了给药，共有33名患者给药。。2024年9月，开始进行酶替代疗法（ERT）研究的第18位也是最后一位患者成功退出了ERT，截至今日，所有18位患者均未接受ERT治疗。

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..

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 has begun to execute BLA readiness activities for isaralgagene civaparvovec, while continuing to advance ongoing business development discussions with potential collaboration partners.

Sangamo已开始为isaralgagene civaparvovec执行BLA准备活动，同时继续推进与潜在合作伙伴正在进行的业务发展讨论。

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 the safety and tolerability of 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，多中心临床研究，旨在评估isaralgagene civaparvovec或ST-920（法布里病患者的基因治疗候选产品）的安全性和耐受性。Isaralgagene civaparvovec需要一次性输注而无需预处理。

The STAAR study enrolled male and female 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 EMA and Innovative Licensing and Access Pathway from U.K.

STAAR研究招募了接受ERT治疗的男性和女性患者，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。FDA已授予isaralgagene civaparvovec孤儿药、快速通道和RMAT名称，该公司还获得了EMA的孤儿药产品名称和主要资格，以及英国的创新许可和获取途径。

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不出汗、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 in 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。

Sangamo’s pipeline also includes multiple partnered programs and programs with opportunities for partnership and investment. To learn more, visit www.sangamo.com and connect with us on LinkedIn and X..

。要了解更多信息，请访问www.sangamo.com，并通过LinkedIn和X与我们联系。。

Forward-Looking Statements

前瞻性声明

This press release contains forward-looking statements regarding our current expectations. These forward-looking statements include, without limitation, statements relating to: the safety and efficacy and therapeutic potential of isaralgagene civaparvovec; 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 and bring isaralgagene civaparvovec to patients sooner than previously expected; the anticipated advancement of isaralgagene civaparvovec to registration, including Sangamo’s plans to seek a potential collaboration partner; and other statements that are not historical fact.

。这些前瞻性陈述包括但不限于以下方面的陈述：isaralgagene civaparvovec的安全性，有效性和治疗潜力；isaralgagene civaparvovec有资格获得FDA加速批准计划的潜力，包括1/2期STAAR研究中产生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；有可能加速预期的批准时间表，并比以前预期的更快地将isaralgagene civaparvovec带给患者；isaralgagene civaparvovec注册的预期进展，包括Sangamo寻求潜在合作伙伴的计划；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 our lack of capital resources to obtain regulatory approval for and commercialize our product candidates in a timely manner or at all, including our ability to secure a partnership; 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 durable in patients and that final clinical trial data from the study will not validate the safety and efficacy of isaralgagene civaparvovec, including that the 52-week data from the Phase 1.

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。可能导致实际结果产生差异的因素包括但不限于与我们缺乏资本资源相关的风险和不确定性，以及时或根本无法获得监管部门的批准并将我们的候选产品商业化，包括我们建立合作关系的能力；临床试验结果的不确定性和不可预测性，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在患者中不持久的风险，以及该研究的最终临床试验数据将无法验证isaralgagene civaparvovec的安全性和有效性，包括第一阶段的52周数据。

There can be no assurance that we and our current or potential future collaborators 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 our collaborators.

无法保证我们和我们目前或潜在的未来合作者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和我们的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 our Securities and Exchange Commission, or SEC, filings and reports, including in our Annual Report on Form 10-K for the year ended December 31, 2023, as supplemented by our Quarterly Report on Form 10-Q for the quarter ended June 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美国证券交易委员会（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10-K表格年度报告，以及截至2024年6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 we undertake 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，除适用法律要求外，我们不承担更新此类信息的义务。。