<p>马萨诸塞州剑桥，2025 年 12 月 7 日（GLOBE NEWSWIRE）——Prime Medicine, Inc.（纳斯达克：PRME），一家致力于提供新一类差异化一次性治愈性基因疗法的生物技术公司，今天宣布在《新英格兰医学杂志》（NEJM）上发表了 PM359 的 1/2 期临床数据，该产品是公司针对 p47phox 慢性肉芽肿病（CGD）的研究性自体造血干细胞产品。这些数据还将在 2025 年 12 月 6 日至 9 日在佛罗里达州奥兰多举行的第 67 届美国血液学会（ASH）年会上以海报形式展示。</p>
<p>该出版物标题为 “针对 p47-phox 慢性肉芽肿病的 Prime Editing”，报告了在 PM359 的 1/2 期试验中治疗的两名患者的初步数据，该试验旨在评估成人和儿童研究参与者的安全性、生物活性和初步疗效。两名患者均经历了快速的中性粒细胞和血小板植入，以及持久的 NADPH 氧化酶活性恢复和早期临床获益，且没有任何安全隐患。这些结果共同提供了 Prime Editing 在人体中的安全性和有效性的首次证明，并支持 PM359 作为 CGD 的精准治疗策略的潜力：</p>
<ul>
<li>参与研究的两名患者均有 CGD 相关并发症的病史，包括 CGD 相关性结肠炎（CAC）以及皮肤和软组织感染，并且均接受长期预防治疗。</li>
<li>两名患者均经历了快速的中性粒细胞植入，分别在第 30 天达到 69% 和 83% 的二氢罗丹明阳性（DHR+）中性粒细胞，远远超过 20% 的临床获益最低预期阈值。两名患者的 DHR 活性在一段时间内保持稳定，表明基因修正发生在骨髓的长期再生造血干细胞（HSCs）中。</li>
<li>两名患者在输注后均未出现新的 CGD 相关并发症或显著的合并疾病；此外，患者 1 停止了美沙拉嗪治疗，未出现 CAC 复发，患者 2 的粪便钙保护素水平显著下降，慢性 CAC 症状减轻。</li>
<li>在两名患者中均未发生与 PM359 相关的临床显著不良事件，所有观察到的毒性均与以布斯尔法为基础的预处理一致。</li>
</ul>
<p>“这些首次在人体中的数据的发表突显了 Prime Editing 作为下一代治疗平台的潜力，能够为患者提供有意义的益处，并且可以在临床规模上进行生产和交付，” Prime Medicine 首席医学官 Mohammed Asmal 博士表示。“除了展示早期临床疗效外，这些结果还提供了关于 Prime Editing 安全性特征和相较于其他基因编辑技术潜在优势的重要见解。如在 NEJM 出版物中所述，我们观察到在单次动员周期后可行修正细胞的高恢复率，以及输注后造血系统的快速重建。这些都支持我们认为 Prime Editing 的机制不会引发双链断裂，可能比其他方法更易被 HSCs 和其他细胞类型耐受，因此对患者更安全。”</p>
<p><strong>关于 Prime Medicine</strong></p>
<p>Prime Medicine 是一家领先的生物技术公司，致力于为患者创造和提供下一代基因编辑疗法。该公司正在部署其专有的 Prime Editing 平台，这是一种多功能、精确且高效的基因编辑技术，旨在开发新一类差异化的一次性治愈性基因疗法。Prime Editors 旨在在基因内的正确位置进行正确的编辑，同时最小化不必要的 DNA 修改，具有修复几乎所有类型基因突变的潜力，并在许多不同的组织、器官和细胞类型中发挥作用。综合来看，Prime Editing 的多功能基因编辑能力可能会为数千种潜在适应症打开机会。</p>
<p>Prime Medicine 目前正在推进多样化的研究性治疗项目组合，围绕我们的核心关注领域：肝脏、肺部以及免疫学和肿瘤学。在每个核心领域，Prime Medicine 最初专注于一组高价值项目，每个项目针对具有良好生物学理解和明确临床开发及监管路径的疾病，并预计为扩展到其他机会奠定基础。随着时间的推移，该公司打算最大化 Prime Editing 广泛而多样的治疗潜力，以及 Prime Editing 平台的模块化，以快速高效地扩展到当前管道之外的疾病，可能包括其他遗传疾病、免疫疾病、癌症、传染病，以及针对常见疾病中的遗传风险因素，这些疾病共同影响数百万人。有关更多信息，请访问 www.primemedicine.com。</p>
<p>© 2025 Prime Medicine, Inc. 保留所有权利。PRIME MEDICINE、Prime Medicine 标志和 PASSIGE 是 Prime Medicine, Inc.的商标。所有其他商标均为其各自所有者的财产。</p>
<p><strong>前瞻性声明</strong></p>
<p>本新闻稿包含 1995 年《私人证券诉讼改革法案》修订版所定义的前瞻性声明，包括但不限于关于 Prime Medicine 的信念和期望的隐含和明确声明：关于其 PM359 的 1/2 期试验数据的重要性；PM359 作为一种精准且安全的 CGD 治疗策略的潜力；Prime Editing 的安全性和有效性，包括与其他方法的比较；Prime Editing 纠正致病突变和治愈疾病的潜力；其业务、项目和技术的战略计划；以及 Prime Editing 在数千种潜在适应症中解锁机会的潜力。</p>
<p>本新闻稿中的任何前瞻性声明均基于管理层当前的期望和信念，并受到多种风险、不确定性和重要因素的影响，这些因素可能导致实际事件或结果与本新闻稿中表达或暗示的任何前瞻性声明存在重大差异，包括但不限于与以下内容相关的风险：与 Prime Medicine 的产品候选者进入临床试验相关的不确定性；潜在产品候选者的临床前和 IND 启用研究的授权、启动和实施，以及其他开发要求的不确定性，包括与开设 IND 和获得监管批准相关的不确定性；与新技术的开发和优化相关的风险，临床前研究或临床研究的结果未能预测未来研究的结果；Prime Medicine 能够建立和维护其 Prime Editing 技术的知识产权保护范围；Prime Medicine 识别和签订未来许可协议和合作的能力；Prime Medicine 对其现金流持续时间和未来财务表现的预期；以及一般经济、行业和市场条件。这些风险和不确定性在 Prime Medicine 最近的 10-K 表格年度报告中的 “风险因素” 部分以及随后的任何向证券交易委员会的提交文件中有更详细的描述。此外，任何前瞻性声明仅代表 Prime Medicine 截至今天的观点，不应被视为其在任何后续日期的观点。Prime Medicine 明确声明不承担根据适用法律更新任何前瞻性声明的义务。对于任何此类前瞻性声明的准确性，不作任何明示或暗示的陈述或保证。</p>
<p><strong>投资者和媒体联系人</strong></p>
<p>Gregory Dearborn</p>
<p>Prime Medicine</p>
<p>857-209-0696</p>
<p>gdearborn@primemedicine.com</p>
<p>Hannah Deresiewicz</p>
<p>Precision AQ</p>
<p>212-362-1200</p>
<p>hannah.deresiewicz@precisionaq.com</p>
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PRME

<p>Prime Medicine 宣布在《新英格兰医学杂志》上发表了 PM359 的 1/2 期临床数据，这是一种用于慢性肉芽肿病的基因疗法。数据显示了安全性和有效性，并将在 ASH 年会上进行展示。试验中的两名患者均显示出显著的临床益处且没有安全隐患，突显了 Prime Editing 作为治疗平台的潜力。Prime Medicine 旨在将其基因编辑疗法扩展到多种疾病</p>

<p>CAMBRIDGE, Mass., Dec. 07, 2025 (GLOBE NEWSWIRE) -- Prime Medicine, Inc. (Nasdaq: PRME), a biotechnology company committed to delivering a new class of differentiated one-time curative genetic therapies, today announced the publication of Phase 1/2 clinical data with PM359, the Company’s investigational autologous hematopoietic stem cell product for p47phox chronic granulomatous disease (CGD) in the <em>New England Journal of Medicine (NEJM).</em> The data will also be presented in a poster session at the 67th American Society of Hematology (ASH) Annual Meeting, December 6-9, 2025 in Orlando, Florida.</p>
<div class="lb-trans"><p>马萨诸塞州剑桥，2025 年 12 月 7 日（GLOBE NEWSWIRE）——Prime Medicine, Inc.（纳斯达克：PRME），一家致力于提供新一类差异化一次性治愈性基因疗法的生物技术公司，今天宣布在《新英格兰医学杂志》（NEJM）上发表了 PM359 的 1/2 期临床数据，该产品是公司针对 p47phox 慢性肉芽肿病（CGD）的研究性自体造血干细胞产品。这些数据还将在 2025 年 12 月 6 日至 9 日在佛罗里达州奥兰多举行的第 67 届美国血液学会（ASH）年会上以海报形式展示。</p>
</div><p>The publication, titled “Prime Editing for p47-phox Chronic Granulomatous Disease,” reports initial data for two patients treated in the Phase 1/2 trial of PM359, which was designed to assess safety, biological activity and preliminary efficacy in adult and pediatric study participants. Both patients experienced rapid neutrophil and platelet engraftment, as well as durable restoration of NADPH oxidase activity and early clinical benefit, without any safety concerns. Together, these results provide the first-in-human demonstration of the safety and efficacy of Prime Editing, and support the potential for PM359 as a precise therapeutic strategy for CGD:</p>
<div class="lb-trans"><p>该出版物标题为 “针对 p47-phox 慢性肉芽肿病的 Prime Editing”，报告了在 PM359 的 1/2 期试验中治疗的两名患者的初步数据，该试验旨在评估成人和儿童研究参与者的安全性、生物活性和初步疗效。两名患者均经历了快速的中性粒细胞和血小板植入，以及持久的 NADPH 氧化酶活性恢复和早期临床获益，且没有任何安全隐患。这些结果共同提供了 Prime Editing 在人体中的安全性和有效性的首次证明，并支持 PM359 作为 CGD 的精准治疗策略的潜力：</p>
</div><ul>
<li>Both patients enrolled in the study had a history of prior CGD-defining complications, including CGD-associated colitis (CAC), and skin and soft tissue infections, and both were maintained on long-term prophylactic therapy.</li>
<li>Both patients experienced rapid neutrophil engraftment, achieving 69% and 83% dihydrorhodamine-positive (DHR+) neutrophils by Day 30, respectively, far in excess of the 20% projected minimum threshold for clinical benefit. DHR activity remained stable over time in both patients, suggesting that gene correction occurred in the long-term repopulating hematopoietic stem cells (HSCs) of the bone marrow.</li>
<li>Both patients remain free of new CGD-related complications or significant intercurrent illnesses post-infusion; additionally, Patient 1 stopped his mesalamine treatment and has not experienced a flare of CAC, and Patient 2’s levels of fecal calprotectin have decreased substantially, and his chronic CAC symptoms have abated.</li>
<li>No clinically significant adverse events attributable to PM359 occurred in either patient, and all observed toxicities were consistent with busulfan-based conditioning.</li>
</ul>
<div class="lb-trans"><ul>
<li>参与研究的两名患者均有 CGD 相关并发症的病史，包括 CGD 相关性结肠炎（CAC）以及皮肤和软组织感染，并且均接受长期预防治疗。</li>
<li>两名患者均经历了快速的中性粒细胞植入，分别在第 30 天达到 69% 和 83% 的二氢罗丹明阳性（DHR+）中性粒细胞，远远超过 20% 的临床获益最低预期阈值。两名患者的 DHR 活性在一段时间内保持稳定，表明基因修正发生在骨髓的长期再生造血干细胞（HSCs）中。</li>
<li>两名患者在输注后均未出现新的 CGD 相关并发症或显著的合并疾病；此外，患者 1 停止了美沙拉嗪治疗，未出现 CAC 复发，患者 2 的粪便钙保护素水平显著下降，慢性 CAC 症状减轻。</li>
<li>在两名患者中均未发生与 PM359 相关的临床显著不良事件，所有观察到的毒性均与以布斯尔法为基础的预处理一致。</li>
</ul>
</div><p>“Publication of these first-in-human data highlights Prime Editing’s promise as a next-generation therapeutic platform, which is capable of delivering meaningful benefits to patients and which can be manufactured and delivered at clinical scale,” said Mohammed Asmal, M.D., Ph.D., Chief Medical Officer of Prime Medicine. “Beyond demonstrating early clinical efficacy, these results offer important insights into Prime Editing’s safety profile and potential advantages over other gene editing technologies. As described in the <em>NEJM</em> publication, we observed high recovery rates of viable corrected cells after a single mobilization cycle, as well as the rapid reconstitution of the hematopoietic system after infusion. Both support our belief that the mechanism of Prime Editing, which does not induce double-strand breaks, may be better tolerated by HSCs and other cell types – and therefore safer for patients – than other approaches.”</p>
<div class="lb-trans"><p>“这些首次在人体中的数据的发表突显了 Prime Editing 作为下一代治疗平台的潜力，能够为患者提供有意义的益处，并且可以在临床规模上进行生产和交付，” Prime Medicine 首席医学官 Mohammed Asmal 博士表示。“除了展示早期临床疗效外，这些结果还提供了关于 Prime Editing 安全性特征和相较于其他基因编辑技术潜在优势的重要见解。如在 NEJM 出版物中所述，我们观察到在单次动员周期后可行修正细胞的高恢复率，以及输注后造血系统的快速重建。这些都支持我们认为 Prime Editing 的机制不会引发双链断裂，可能比其他方法更易被 HSCs 和其他细胞类型耐受，因此对患者更安全。”</p>
</div><p><strong>About Prime Medicine</strong></p>
<div class="lb-trans"><p><strong>关于 Prime Medicine</strong></p>
</div><p>Prime Medicine is a leading biotechnology company dedicated to creating and delivering the next generation of gene editing therapies to patients. The Company is deploying its proprietary Prime Editing platform, a versatile, precise and efficient gene editing technology, to develop a new class of differentiated one-time curative genetic therapies. Designed to make only the right edit at the right position within a gene while minimizing unwanted DNA modifications, Prime Editors have the potential to repair almost all types of genetic mutations and work in many different tissues, organs and cell types. Taken together, Prime Editing’s versatile gene editing capabilities could unlock opportunities across thousands of potential indications.</p>
<div class="lb-trans"><p>Prime Medicine 是一家领先的生物技术公司，致力于为患者创造和提供下一代基因编辑疗法。该公司正在部署其专有的 Prime Editing 平台，这是一种多功能、精确且高效的基因编辑技术，旨在开发新一类差异化的一次性治愈性基因疗法。Prime Editors 旨在在基因内的正确位置进行正确的编辑，同时最小化不必要的 DNA 修改，具有修复几乎所有类型基因突变的潜力，并在许多不同的组织、器官和细胞类型中发挥作用。综合来看，Prime Editing 的多功能基因编辑能力可能会为数千种潜在适应症打开机会。</p>
</div><p>Prime Medicine is currently progressing a diversified portfolio of investigational therapeutic programs organized around our core areas of focus: liver, lung, and immunology and oncology. Across each core area, Prime Medicine is focused initially on a set of high value programs, each targeting a disease with well-understood biology and a clearly defined clinical development and regulatory path, and each expected to provide the foundation for expansion into additional opportunities. Over time, the Company intends to maximize Prime Editing’s broad and versatile therapeutic potential, as well as the modularity of the Prime Editing platform, to rapidly and efficiently expand beyond the diseases in its current pipeline, potentially including additional genetic diseases, immunological diseases, cancers, infectious diseases, and targeting genetic risk factors in common diseases, which collectively impact millions of people. For more information, please visit www.primemedicine.com.</p>
<div class="lb-trans"><p>Prime Medicine 目前正在推进多样化的研究性治疗项目组合，围绕我们的核心关注领域：肝脏、肺部以及免疫学和肿瘤学。在每个核心领域，Prime Medicine 最初专注于一组高价值项目，每个项目针对具有良好生物学理解和明确临床开发及监管路径的疾病，并预计为扩展到其他机会奠定基础。随着时间的推移，该公司打算最大化 Prime Editing 广泛而多样的治疗潜力，以及 Prime Editing 平台的模块化，以快速高效地扩展到当前管道之外的疾病，可能包括其他遗传疾病、免疫疾病、癌症、传染病，以及针对常见疾病中的遗传风险因素，这些疾病共同影响数百万人。有关更多信息，请访问 www.primemedicine.com。</p>
</div><p>© 2025 Prime Medicine, Inc. All rights reserved. PRIME MEDICINE, the Prime Medicine logos, and PASSIGE are trademarks of Prime Medicine, Inc. All other trademarks referred to herein are the property of their respective owners.</p>
<div class="lb-trans"><p>© 2025 Prime Medicine, Inc. 保留所有权利。PRIME MEDICINE、Prime Medicine 标志和 PASSIGE 是 Prime Medicine, Inc.的商标。所有其他商标均为其各自所有者的财产。</p>
</div><p><strong>Forward Looking Statements</strong></p>
<div class="lb-trans"><p><strong>前瞻性声明</strong></p>
</div><p>This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, implied and express statements about Prime Medicine’s beliefs and expectations regarding: the significance of data from its Phase 1/2 trial of PM359; the potential for PM359 to be a precise and safe therapeutic strategy for CGD;; the safety and efficacy of Prime Editing, including in comparison to other approaches; the potential of Prime Editing to correct the causative mutations of, and to cure, diseases; its strategic plans for its business, programs, and technology; and the potential of Prime Editing to unlock opportunities across thousands of potential indications.</p>
<div class="lb-trans"><p>本新闻稿包含 1995 年《私人证券诉讼改革法案》修订版所定义的前瞻性声明，包括但不限于关于 Prime Medicine 的信念和期望的隐含和明确声明：关于其 PM359 的 1/2 期试验数据的重要性；PM359 作为一种精准且安全的 CGD 治疗策略的潜力；Prime Editing 的安全性和有效性，包括与其他方法的比较；Prime Editing 纠正致病突变和治愈疾病的潜力；其业务、项目和技术的战略计划；以及 Prime Editing 在数千种潜在适应症中解锁机会的潜力。</p>
</div><p>Any forward-looking statements in this press release are based on management’s current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, risks associated with: uncertainties related to Prime Medicine’s product candidates entering clinical trials; the authorization, initiation, and conduct of preclinical and IND-enabling studies and other development requirements for potential product candidates, including uncertainties related to opening INDs and obtaining regulatory approvals; risks related to the development and optimization of new technologies, the results of preclinical studies, or clinical studies not being predictive of future results in connection with future studies; the scope of protection Prime Medicine is able to establish and maintain for intellectual property rights covering its Prime Editing technology; Prime Medicine’s ability to identify and enter into future license agreements and collaborations; Prime Medicine’s expectations regarding the anticipated timeline of its cash runway and future financial performance; and general economic, industry and market conditions. These and other risks and uncertainties are described in greater detail in the section entitled “Risk Factors” in Prime Medicine’s most recent Annual Report on Form 10-K, as well as any subsequent filings with the Securities and Exchange Commission. In addition, any forward-looking statements represent Prime Medicine’s views only as of today and should not be relied upon as representing its views as of any subsequent date. Prime Medicine explicitly disclaims any obligation to update any forward-looking statements subject to any obligations under applicable law. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements.</p>
<div class="lb-trans"><p>本新闻稿中的任何前瞻性声明均基于管理层当前的期望和信念，并受到多种风险、不确定性和重要因素的影响，这些因素可能导致实际事件或结果与本新闻稿中表达或暗示的任何前瞻性声明存在重大差异，包括但不限于与以下内容相关的风险：与 Prime Medicine 的产品候选者进入临床试验相关的不确定性；潜在产品候选者的临床前和 IND 启用研究的授权、启动和实施，以及其他开发要求的不确定性，包括与开设 IND 和获得监管批准相关的不确定性；与新技术的开发和优化相关的风险，临床前研究或临床研究的结果未能预测未来研究的结果；Prime Medicine 能够建立和维护其 Prime Editing 技术的知识产权保护范围；Prime Medicine 识别和签订未来许可协议和合作的能力；Prime Medicine 对其现金流持续时间和未来财务表现的预期；以及一般经济、行业和市场条件。这些风险和不确定性在 Prime Medicine 最近的 10-K 表格年度报告中的 “风险因素” 部分以及随后的任何向证券交易委员会的提交文件中有更详细的描述。此外，任何前瞻性声明仅代表 Prime Medicine 截至今天的观点，不应被视为其在任何后续日期的观点。Prime Medicine 明确声明不承担根据适用法律更新任何前瞻性声明的义务。对于任何此类前瞻性声明的准确性，不作任何明示或暗示的陈述或保证。</p>
</div><p><strong>Investor and Media Contacts</strong></p>
<div class="lb-trans"><p><strong>投资者和媒体联系人</strong></p>
</div><p>Gregory Dearborn</p>
<div class="lb-trans"><p>Gregory Dearborn</p>
</div><p>Prime Medicine</p>
<div class="lb-trans"><p>Prime Medicine</p>
</div><p>857-209-0696</p>
<div class="lb-trans"><p>857-209-0696</p>
</div><p>gdearborn@primemedicine.com</p>
<div class="lb-trans"><p>gdearborn@primemedicine.com</p>
</div><p>Hannah Deresiewicz</p>
<div class="lb-trans"><p>Hannah Deresiewicz</p>
</div><p>Precision AQ</p>
<div class="lb-trans"><p>Precision AQ</p>
</div><p>212-362-1200</p>
<div class="lb-trans"><p>212-362-1200</p>
</div><p>hannah.deresiewicz@precisionaq.com</p>
<div class="lb-trans"><p>hannah.deresiewicz@precisionaq.com</p>
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Prime Medicine Announces The New England Journal of Medicine Publication of PM359  Clinical Data for the Treatment of Chronic Granulomatous Disease | PRME Stock News

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