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Prime Medicine to Present Preclinical Data Demonstrating Broad Potential of Prime Editing Technology at Upcoming Scientific Meetings

Prime Medicine to Present Preclinical Data Demonstrating Broad Potential of Prime Editing Technology at Upcoming Scientific Meetings

Prime Medicine将在即将举行的科学会议上发布临床前数据,证明Prime编辑技术的广泛潜力
Prime Medicine ·  04/23 00:00

-- Presentations include new preclinical data for PM359, further demonstrating ability of Prime Editors to efficiently, reproducibly and durably correct causative mutation of CGD; on track to enter clinical development in 2024, with initial data expected in 2025 --

— 演讲包括 PM359 的新临床前数据,进一步证明了 Prime Editors 有效、可重复和持久地纠正 CGD 致病突变的能力;有望在 2024 年进入临床开发,预计在 2025 年提供初步数据 —

-- Prime Medicine Co-Founder, David Liu, Ph.D., Awarded ASGCT Outstanding Achievement Award --

—— Prime Medicine 联合创始人刘大卫博士荣获 ASGCT 杰出成就奖——

CAMBRIDGE, Mass., April 23, 2024 (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 that the Company will present preclinical data highlighting the broad potential of its Prime Editing technology and supporting the advancement of its programs at the 3rd Annual LNP Formulation and Process Development Summit (Boston, April 29-May 2, 2024); the American Society of Cell & Gene Therapy 27th Annual Meeting (Baltimore, May 7-11, 2024); and TIDES USA Oligonucleotide & Peptide Therapeutics Conference (Boston, May 14-17, 2024).

马萨诸塞州剑桥,2024年4月23日(GLOBE NEWSWIRE)——致力于提供新一类差异化的一次性治疗遗传疗法的生物技术公司Prime Medicine, Inc.(纳斯达克股票代码:PRME)今天宣布,该公司将提供临床前数据,重点介绍其Prime Editing技术的广泛潜力,并支持其在三大领域的项目进展第三方 年度 LNP 配方和工艺开发峰会(波士顿,2024 年 4 月 29 日至 5 月 2 日);美国细胞与基因疗法学会 27第四 年会(巴尔的摩,2024年5月7日至11日);以及TIDES美国寡核苷酸和肽治疗会议(波士顿,2024年5月14日至17日)。

"In the short time since Prime Editing was first discovered, we have made incredible strides advancing this technology, developing and optimizing Prime Editors for programs across our core areas of focus, and creating new delivery systems that we believe will permit our Prime Editors to safely and effectively access target tissue," said Keith Gottesdiener, M.D., President and Chief Executive Officer of Prime Medicine. "This progress is underscored by our upcoming presentations, which will feature new findings from across our platform and initial pipeline, including new preclinical data for our first program expected to enter clinical development – PM359 for chronic granulomatous disease (CGD), a serious, life-threatening rare disease. In addition to our presentations, we congratulate our Co-Founder, David Liu, on receiving the ASGCT Outstanding Achievement Award, highlighting his work in the gene editing field, including his contributions to the discovery and advancement of Prime Editing. We look forward to coming together with the scientific research community and highlighting our leading position in the field of next-generation gene editing."

Prime Medicine总裁兼首席执行官基思·戈特斯迪纳医学博士表示:“自Prime Editing首次被发现以来的短时间内,我们在推进这项技术、为核心重点领域的项目开发和优化Prime Editors以及创建新的交付系统方面取得了令人难以置信的进展。”“我们即将发表的演讲突显了这一进展,这些演讲将介绍来自我们整个平台和初始产品线的新发现,包括我们第一个有望进入临床开发的项目——用于慢性肉芽肿病(CGD)的 PM359 的新临床前数据,这是一种严重的、危及生命的罕见疾病。除了我们的演讲,我们还祝贺我们的联合创始人刘大卫获得ASGCT杰出成就奖,该奖项旨在表彰他在基因编辑领域的工作,包括他对Prime Editing的发现和进步所做的贡献。我们期待与科研界齐聚一堂,突出我们在下一代基因编辑领域的领先地位。”

Details of the presentations are as follows.

演讲的详情如下。

3rd Annual LNP Formulation and Process Development Summit (Boston)

3第三方 年度LNP配方和工艺开发峰会(波士顿)

  • Presentation Name: Advancing LNP Screening to Incorporate Automated, High-Throughput Readouts
    • Date & Time: Wednesday, May 1, 2024, 2:00 p.m. ET
    • Presenter: Marine Hatit, Ph.D.
  • 演示文稿名称:推进 LNP 筛查以纳入自动化、高通量读数
    • 日期和时间:美国东部时间 2024 年 5 月 1 日星期三下午 2:00
    • 主持人:马琳·哈蒂特博士

American Society of Cell & Gene Therapy (ASGCT) 27th Annual Meeting (Baltimore)

美国细胞与基因疗法学会 (ASGCT) 27第四 年会(巴尔的摩)

  • Presentation Name: Development and Delivery of Prime Editors
    • Date & Time: Tuesday, May 7, 2024, 9:45 a.m. ET
    • Presenter: Jonathan Levy, Ph.D.
  • Poster Name: Exon Reframing by Prime Editors Restores Dystrophin
    • Date & Time: Wednesday, May 8, 2024, 12:00 p.m. ET (poster session)
    • Presenter: Michelle O'Connor, Ph.D.
  • Presentation Name: Development of a Prime Edited CD34+ cell Drug Product for the Treatment of P47phox Chronic Granulomatous Disease
    • Date & Time: Wednesday, May 8, 2024, 5:15 p.m. ET
    • Presenter: Jennifer Gori, Ph.D.
  • Presentation Name: Off-Target Analysis Shows Favorable Safety Profile of Prime Editing
    • Date & Time: Friday, May 10, 2024, 2:45 p.m. ET
    • Presenter: Maria Collier, Ph.D.
  • Presentation Name: Prime Editors Precisely Correct Pathogenic RHO Mutations and Preserve Photoreceptors In Vivo
    • Date & Time: Wednesday, May 8, 2024, 3:45 p.m. ET
    • Presenter: Deepak Reyon, Ph.D.
  • 演讲名称:主要编辑器的开发和交付
    • 日期和时间:美国东部时间 2024 年 5 月 7 日星期二上午 9:45
    • 主持人:乔纳森·利维博士
  • 海报名称:Prime Editors 的 Exon Reframing 可恢复肌萎缩蛋白
    • 日期和时间:美国东部时间 2024 年 5 月 8 日星期三下午 12:00(海报发布会)
    • 主持人:米歇尔·奥康纳博士
  • 演讲名称:开发用于治疗p47phox慢性肉芽肿病的主要编辑CD34+细胞药物产品
    • 日期和时间:美国东部时间 2024 年 5 月 8 日星期三下午 5:15
    • 主持人:詹妮弗·戈里博士
  • 演示文稿名称:偏离目标的分析显示 Prime Editing 具有良好的安全性
    • 日期和时间:美国东部时间 2024 年 5 月 10 日星期五下午 2:45
    • 主持人:玛丽亚·科利尔博士
  • 演讲名称:主要编辑精确校正致病性 RHO 突变并将光感受器保留在体内
    • 日期和时间:美国东部时间 2024 年 5 月 8 日星期三下午 3:45
    • 主持人:迪帕克·雷恩博士

TIDES USA Oligonucleotide & Peptide Therapeutics Conference (Boston)

TIDES 美国寡核苷酸和肽疗法会议(波士顿)

  • Workshop: Introduction to Genome Editing
    • Date & Time: Tuesday, May 14, 2024, 9:00 a.m. ET
    • Presenter: Jeffrey Hussman, Ph.D.
  • Presentation Name: Gene Editing, Regulatory and Delivery Challenges and Perspectives
    • Date & Time: Tuesday, May 14, 2024, 4:15 p.m. ET
    • Presenter: Luis Santos, Ph.D.
  • Presentation Name: RNA-Based Approach to Delivering Prime Editing
    • Date & Time: Thursday, May 16, 2024, 10:45 a.m. ET
    • Presenter: Seth Alexander, Ph.D.
  • Presentation Name: Analytical Development for Prime Editing Guide (peg)RNAs
    • Date & Time: Friday, May 17, 2024, 11:45 a.m. ET
    • Presenter: Xiangkun Yang, Ph.D.
  • 研讨会:基因组编辑简介
    • 日期和时间:美国东部时间 2024 年 5 月 14 日星期二上午 9:00
    • 主持人:杰弗里·胡斯曼博士
  • 演讲名称:基因编辑、监管和交付挑战与展望
    • 日期和时间:美国东部时间 2024 年 5 月 14 日星期二下午 4:15
    • 主持人:路易斯·桑托斯博士
  • 演示文稿名称:基于 RNA 的优质编辑方法
    • 日期和时间:美国东部时间 2024 年 5 月 16 日星期四上午 10:45
    • 主持人:塞思·亚历山大博士
  • 演示文稿名称:主要编辑指南(peg)RNA 的分析开发
    • 日期和时间:美国东部时间 2024 年 5 月 17 日星期五上午 11:45
    • 演讲者:杨向昆博士

About Prime Medicine

关于普瑞米医学

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.

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、精确和高效的基因编辑技术,以开发一类新的差异化一次性治疗遗传疗法。Prime Editors旨在仅在基因的正确位置进行正确的编辑,同时最大限度地减少不必要的DNA修改,它有可能修复几乎所有类型的基因突变,并适用于许多不同的组织、器官和细胞类型。总而言之,Prime Editing的多功能基因编辑能力可以为数千种潜在适应症开启机会。

Prime Medicine is currently progressing a diversified portfolio of investigational therapeutic programs organized around core areas of focus: hematology and immunology, liver, lung, ocular and neuromuscular. Across each core area, Prime Medicine's initial focus is on genetic diseases with a fast, direct path to treating patients, and those with high unmet need not currently addressable using other gene editing approaches. Over time, the Company intends to maximize Prime Editing's broad and versatile therapeutic potential to expand beyond the genetic diseases in its initial pipeline, potentially including 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.

Prime Medicine目前正在推进围绕核心重点领域组织的多元化研究性治疗项目组合:血液学和免疫学、肝脏、肺部、眼部和神经肌肉学。在每个核心领域,Prime Medicine最初的重点是遗传性疾病,为患者提供快速、直接的治疗途径,而那些未得到满足的患者目前无法使用其他基因编辑方法来解决的需求。随着时间的推移,该公司打算最大限度地发挥Prime Editing广泛而多功能的治疗潜力,将业务扩展到其最初研发的遗传疾病之外,可能包括免疫疾病、癌症、传染病,并针对共同影响数百万人的常见疾病中的遗传风险因素。欲了解更多信息,请访问 www.primemedicine.com。

2024 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.

2024 Prime Medicine, Inc. 版权所有。PRIME MEDICINE、Prime Medicine 徽标和 PASSIGE 是 Prime Medicine, Inc. 的商标。此处提及的所有其他商标均为其各自所有者的财产。

Forward Looking Statements

前瞻性陈述

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 potential of PM359 to correct the causative mutation of CGD; its expectations regarding the breadth and potential of Prime Editing technology; the expansion of Prime Editing's therapeutic potential and the creation of value through strategic business development to extend the reach and impact of Prime Editing to areas beyond Prime Medicine's current core areas of focus; the anticipated maturation into a clinical-stage company by bringing PM359 into clinical development in 2024 with initial data expected in 2025; and the potential for Prime Editors to repair genetic mutations and offer curative genetic therapies for a wide spectrum of diseases. The words "may," "might," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "expect," "estimate," "seek," "predict," "future," "project," "potential," "continue," "target" and similar words or expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words.

本新闻稿包含经修订的 1995 年《私人证券诉讼改革法》所指的前瞻性陈述,包括但不限于对 Prime Medicine 在以下方面的信念和期望的暗示和明确陈述:PM359 纠正 CGD 致病突变的可能性;其对 Prime Editing 技术广度和潜力的预期;扩大 Prime Editing 的治疗潜力,以及通过战略业务发展创造价值以扩大 Prime 的覆盖范围和影响力编辑 Prime Medicine 当前核心领域以外的领域;通过在 2024 年将 PM359 纳入临床开发,预计将成熟为一家临床阶段的公司,初步数据预计将在 2025 年发布;Prime Editors 有可能修复基因突变并为各种疾病提供治疗性基因疗法。“可能”、“可能”、“将”、“可能”、“应该”、“计划”、“预测”、“打算”、“相信”、“期望”、“估计”、“寻找”、“预测”、“未来”、“项目”、“潜在”、“继续”、“目标” 等词语和类似的词语或表述旨在识别前瞻性陈述,尽管不是所有前瞻性陈述都包含这些识别词。

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 an IND or CTA filing and 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; and general economic, industry and market conditions, including rising interest rates, inflation, and adverse developments affecting the financial services industry. 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.

本新闻稿中的任何前瞻性陈述均基于管理层当前的预期和信念,并受许多风险、不确定性和重要因素的影响,这些风险和不确定性可能导致实际事件或结果与本新闻稿中包含的任何前瞻性陈述所表达或暗示的存在重大差异,包括但不限于与IND或CTA申请和进入临床试验相关的不确定性;临床前和IND支持研究的授权、启动和进行以及其他相关风险潜在候选产品的开发要求,包括与开放IND和获得监管部门批准相关的不确定性;与新技术的开发和优化相关的风险、临床前研究结果或无法预测未来研究结果的临床研究;Prime Medicine能够确立和维护涵盖其Prime Editing技术的知识产权的保护范围;Prime Medicine识别和签订未来许可协议的能力,以及合作;以及总体经济、行业和市场状况,包括利率上升、通货膨胀和影响金融服务行业的不利发展。Prime Medicine最新的10-K表年度报告以及随后向美国证券交易委员会提交的任何文件中题为 “风险因素” 的部分对这些风险和不确定性进行了更详细的描述。此外,任何前瞻性陈述仅代表Prime Medicine截至今天的观点,不应以此为依据来代表其以后的观点。Prime Medicine明确声明不承担任何更新任何前瞻性陈述的义务,但须遵守适用法律规定的任何义务。对于任何此类前瞻性陈述的准确性,不作任何陈述或保证(明示或暗示)。

Investor Contact
Hannah Deresiewicz
Stern Investor Relations, Inc.
212-362-1200
hannah.deresiewicz@sternir.com

投资者联系人
汉娜·德雷西维奇
斯特恩投资者关系有限公司
212-362-1200
hannah.deresiewicz@sternir.com

Media Contact
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dan@1ABmedia.com

媒体联系人
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