-New Phase 1 clinical data for CTX310™ continues to demonstrate dose-dependent reductions in triglycerides (TG) and low-density lipoprotein (LDL), with peak reduction of up to 82% in TG and up to 86% in LDL, with a well-tolerated safety profile-

CTX310™ 的新一期临床数据显示，甘油三酯 (TG) 和低密度脂蛋白 (LDL) 呈剂量依赖性降低，TG 最高降低达 82%，LDL 最高降低达 86%，且安全性良好，耐受性佳。

-Complete Phase 1 data presentation for CTX310 anticipated at a medical meeting in the second half of 2025-

预计在2025年下半年的医学会议上展示CTX310的第一阶段完整数据。

-Data update for CTX320™, targeting the

-CTX320™的数据更新，目标是

LPA

LPA

gene, now expected in the first half of 2026-

基因，现在预计在2026年上半年。

-Preclinical

-临床前

in vivo

体内

cardiovascular program CTX340™ advancing toward IND / CTA filings targeting refractory hypertension-

心血管项目 CTX340™ 正在向针对难治性高血压的 IND/CTA 申请推进。

ZUG

楚格

, Switzerland and BOSTON,

瑞士和波士顿，

June 26, 2025

2025年6月26日

(GLOBE NEWSWIRE) -- CRISPR Therapeutics (NASDAQ: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases today announced updates across its

(GLOBE NEWSWIRE) -- CRISPR Therapeutics（纳斯达克股票代码：CRSP），一家专注于为严重疾病开发革命性基因药物的生物制药公司，今天宣布了其在各方面的最新进展。

in vivo

体内

cardiovascular disease programs. These include new data for CTX310™, targeting ANGPTL3, as well as continued progress on CTX320™, targeting the

心血管疾病项目。这些项目包括针对ANGPTL3的CTX310™的新数据，以及针对CTX320™的持续推进，

LPA

LPA

gene, and CTX340™, targeting the

基因，以及针对的CTX340™，

AGT

agt

gene.

基因。

“CRISPR Therapeutics remains focused on executing against our strategic priorities and advancing our portfolio of innovative therapies,” said Samarth Kulkarni, Ph.D., Chairman and Chief Executive Officer of CRISPR Therapeutics. “The additional data from our ongoing Phase 1 clinical trial for CTX310 reinforces the potential of our platform to transform the treatment of serious cardiovascular diseases.

“CRISPR Therapeutics 继续专注于执行我们的战略重点，并推进我们创新疗法的产品组合，”CRISPR Therapeutics董事长兼首席执行官Samarth Kulkarni博士表示，“我们正在进行的CTX310一期临床试验的更多数据进一步证实了我们的平台在治疗严重心血管疾病方面的潜力。”

We are progressing with our dose-finding study and expect to share complete data at a medical meeting in the second half of this year. For CTX320, we are continuing our dose-finding study and anticipate sharing data in the first half of 2026, reflecting a strategic decision to incorporate emerging insights from the evolving Lp(a) treatment landscape.”.

我们正在推进剂量探索研究，并预计在今年下半年的医学会议上分享完整数据。对于CTX320，我们将继续进行剂量探索研究，并预计在2026年上半年分享数据，这反映了我们战略上决定结合不断演变的Lp(a)治疗领域的新兴见解。

CTX310, targeting

CTX310，目标

ANGPTL3

血管生成素样蛋白3

CTX310 targets

CTX310 目标

ANGPTL3

血管生成素样蛋白3

, a gene that encodes for key protein involved in the regulation of low-density lipoprotein (LDL) and triglyceride (TG) levels – both of which are recognized risk factors for atherosclerotic heart disease (ASCVD). Loss-of-function mutations in ANGPTL3 are associated with significantly reduced levels of LDL and TGs, as well as reduced risk of ASCVD, without known adverse health effects.

，该基因编码一种关键蛋白质，参与调节低密度脂蛋白（LDL）和甘油三酯（TG）水平——这两者均为动脉粥样硬化性心脏病（ASCVD）的公认风险因素。ANGPTL3的功能缺失突变与LDL和TG水平显著降低以及ASCVD风险降低相关，且无已知不良健康影响。

More than 40 million patients in the .

超过4000万名患者在。

U.S.

美国

alone are affected by elevated LDL, severely elevated TG or both – representing a significant unmet need and a large addressable population. CTX310 is initially focused on high-risk patients with the greatest unmet medical need and limited effective treatment options.

单独受到LDL升高、TG严重升高或两者同时影响的患者——代表着一个重要的未满足需求和庞大的可治疗人群。CTX310最初专注于那些具有最大未满足医疗需求和有限有效治疗选择的高风险患者。

CTX310 is in an ongoing Phase 1 first-in-human clinical trial targeting ANGPTL3 in four patient groups: homozygous familial hypercholesterolemia (HoFH), severe hypertriglyceridemia (sHTG), heterozygous familial hypercholesterolemia (HeFH), or mixed dyslipidemias (MDL). Eligible participants have levels of TG >300 mg/dL and/or LDL-C >100 mg/dL (or >70 mg/dL for subjects with ASCVD).

CTX310 正在进行一项首次用于人体的 1 期临床试验，针对 ANGPTL3，涵盖四个患者群体：纯合子家族性高胆固醇血症 (HoFH)、严重高甘油三酯血症 (sHTG)、杂合子家族性高胆固醇血症 (HeFH) 或混合型血脂异常 (MDL)。符合条件的参与者甘油三酯 (TG) 水平 >300 mg/dL 和/或低密度脂蛋白胆固醇 (LDL-C) 水平 >100 mg/dL（对于患有动脉粥样硬化性心血管疾病 (ASCVD) 的受试者，LDL-C >70 mg/dL）。

Both LDL and TG are validated surrogate endpoints accepted by regulatory agencies..

LDL 和 TG 均为监管机构认可的经验证的替代终点。

These new results build upon previously disclosed results from the first 10 patients across the first four cohorts (lean body weight-based doses of DL1 [0.1 mg/kg], DL2 [0.3 mg/kg], DL3 [0.6 mg/kg] and DL4 [0.8 mg/kg]) with at least 30 days of follow-up for each participant. As dose-range finding continues, data to date demonstrate peak reductions of up to 82% in TG and LDL reductions of up to 86% at DL4 without any clinically significant changes in liver enzymes and a safety and tolerability profile consistent with previous findings..

这些新的结果基于之前公布的前四个队列中前10名患者的结果（按瘦体重计算的剂量DL1 [0.1 mg/kg]、DL2 [0.3 mg/kg]、DL3 [0.6 mg/kg] 和 DL4 [0.8 mg/kg]），每位参与者至少进行了30天的随访。随着剂量范围探索的继续，迄今为止的数据表明，在DL4剂量下，甘油三酯（TG）水平最高降低了82%，低密度脂蛋白（LDL）水平最高降低了86%，且肝酶无任何临床显著变化，安全性和耐受性与之前的发现一致。

The Company anticipates presenting the complete Phase 1 data for CTX310 at a medical meeting in the second half of 2025.

公司预计将在2025年下半年的医学会议上展示CTX310的完整一期临床数据。

CTX320™, targeting

CTX320™，目标定位

LPA

LPA

CTX320 is in an ongoing Phase 1 clinical trial targeting the LPA gene in patients with elevated lipoprotein(a) [Lp(a)], a genetically determined risk factor associated with increased incidence of major adverse cardiovascular events (MACE). Elevated Lp(a) levels affect up to 20% of the global population and remains unaddressed by current therapies..

CTX320 正在进行一项一期临床试验，针对脂蛋白(a) [Lp(a)]基因，这是一种与主要不良心血管事件 (MACE) 发生率增加相关的遗传风险因素。高Lp(a)水平影响全球高达20%的人口，且目前的治疗方法尚未能解决这一问题。

The Phase 1 trial is enrolling patients and dose-finding is ongoing. An update is now expected in the first half of 2026, reflecting a strategic decision to incorporate emerging insights from the evolving Lp(a) landscape.

第一阶段试验正在招募患者，剂量探索正在进行中。更新预计将于2026年上半年发布，反映了一个战略决策，即结合不断演变的Lp(a)领域的新兴见解。

CTX340, targeting angiotensinogen (AGT)

CTX340，靶向血管紧张素原 (AGT)

CRISPR Therapeutics

CRISPR治疗公司

is also advancing its preclinical

也在推进其临床前

in vivo

体内

cardiovascular program CTX340, targeting angiotensinogen (AGT) for the treatment of refractory hypertension. CTX340 is currently progressing through IND/CTA-enabling studies.

心血管项目CTX340，靶向血管紧张素原（AGT）用于治疗难治性高血压。CTX340目前正在进行IND/CTA支持性研究。

About

关于

In Vivo

体内

Programs

程序

CRISPR Therapeutics has established a proprietary lipid nanoparticle (LNP) platform for the delivery of CRISPR/Cas9 to the liver. The Company’s

CRISPR Therapeutics 已建立了一个专有的脂质纳米颗粒（LNP）平台，用于将CRISPR/Cas9递送到肝脏。公司

in vivo

体内

portfolio includes its lead investigational programs, CTX310 (directed towards angiopoietin-related protein 3 (

投资组合包括其主要研究项目CTX310（针对血管生成素相关蛋白3（

ANGPTL3

血管生成素样蛋白3

)) and CTX320 (directed towards

)) 和 CTX320（针对

LPA

LPA

, the gene encoding apolipoprotein(a) (apo(a)), a major component of lipoprotein(a) [Lp(a)]). Both are validated therapeutic targets for cardiovascular disease. CTX310 and CTX320 are in ongoing clinical trials in patients with heterozygous familial hypercholesterolemia, homozygous familial hypercholesterolemia, mixed dyslipidemias, or severe hypertriglyceridemia, and in patients with elevated lipoprotein(a), respectively.

，编码载脂蛋白(a)（apo(a)）的基因，是脂蛋白(a) [Lp(a)]的主要成分）。两者均为心血管疾病的有效治疗靶点。CTX310和CTX320正在杂合子家族性高胆固醇血症、纯合子家族性高胆固醇血症、混合型血脂异常或严重高甘油三酯血症患者以及脂蛋白(a)升高的患者中进行持续的临床试验。

In addition, the Company’s research and preclinical development candidates include CTX340 and CTX450™, targeting angiotensinogen (.

此外，该公司研发和临床前开发的候选药物包括CTX340和CTX450™，靶向血管紧张素原（。

AGT

代理人

) for refractory hypertension and 5’-aminolevulinate synthase 1 (

) 用于难治性高血压和 5'-氨基乙酰丙酸合成酶 1 (

ALAS1

ALAS1

) for acute hepatic porphyria (AHP), respectively.

) 分别用于急性肝卟啉病 (AHP)。

About CRISPR Therapeutics

关于CRISPR Therapeutics

Since its inception over a decade ago, CRISPR Therapeutics has evolved from a research-stage company advancing gene editing programs into a leader that celebrated the historic approval of the first-ever CRISPR-based therapy. The Company has a diverse portfolio of product candidates across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine, cardiovascular, autoimmune, and rare diseases.

十多年来，CRISPR Therapeutics已从一家致力于推进基因编辑项目的研发阶段公司，发展成为庆祝首个基于CRISPR疗法历史性获批的领导者。该公司在包括血红蛋白病、肿瘤学、再生医学、心血管、自身免疫和罕见疾病在内的广泛疾病领域拥有多种候选产品组合。

In 2018, CRISPR Therapeutics advanced the first-ever CRISPR/Cas9 gene-edited therapy into the clinic to investigate the treatment of sickle cell disease and transfusion-dependent beta thalassemia. Beginning in late 2023, CASGEVY.

2018年，CRISPR Therapeutics公司将首个CRISPR/Cas9基因编辑疗法推进到临床，以研究镰状细胞病和依赖输血的β地中海贫血的治疗。从2023年底开始，CASGEVY。

®

®

(exagamglogene autotemcel [exa-cel]) was approved in several countries to treat eligible patients with either of these conditions. The Nobel Prize-winning CRISPR technology has revolutionized biomedical research and represents a powerful, clinically validated approach with the potential to create a new class of potentially transformative medicines.

(exagamglogene autotemcel [exa-cel]) 已在多个国家获批用于治疗符合这些条件的患者。荣获诺贝尔奖的CRISPR技术已经彻底改变了生物医学研究，并代表了一种强大且经过临床验证的方法，有潜力创造一类可能具有变革性的新药物。

To accelerate and expand its efforts, CRISPR Therapeutics has formed strategic partnerships with leading companies including Vertex Pharmaceuticals. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Boston, Massachusetts and San Francisco, California.

为了加速和扩大其努力，CRISPR Therapeutics 已与包括 Vertex Pharmaceuticals 在内的领先公司建立了战略合作伙伴关系。CRISPR Therapeutics AG 总部位于瑞士楚格，其全资美国子公司 CRISPR Therapeutics, Inc. 以及研发业务基地分别位于马萨诸塞州波士顿和加利福尼亚州旧金山。

To learn more, visit .

要了解更多信息，请访问。

www.crisprtx.com

www.crisprtx.com

.

。

CRISPR THERAPEUTICS

CRISPR治疗学

®

®

standard character mark and design logo CTX310™, CTX320™, CTX340™ and CTX450™ are trademarks and registered trademarks of CRISPR Therapeutics AG. CASGEVY

标准字符标记和设计标志 CTX310™、CTX320™、CTX340™ 和 CTX450™ 是 CRISPR Therapeutics AG 的商标及注册商标。CASGEVY

®

®

and the CASGEVY logo are registered trademarks of Vertex Pharmaceuticals Incorporated. All other trademarks and registered trademarks are the property of their respective owners.

CASGEVY标志是Vertex Pharmaceuticals公司的注册商标。所有其他商标和注册商标均为其各自所有者的财产。

CRISPR Special Note Regarding Forward-Looking Statements

CRISPR关于前瞻性声明的特别说明

Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements.

本新闻稿中包含的关于非历史事实的陈述是1995年《私人证券诉讼改革法案》所指的“前瞻性声明”。由于此类声明受到风险和不确定性的约束，实际结果可能与这些前瞻性声明所表达或暗示的结果有重大差异。

Such statements include, but are not limited to, statements made by Dr. Kulkarni in this press release, as well as regarding any or all of the following: (i) CRISPR Therapeutics preclinical studies, clinical trials and pipeline products and programs, including, without limitation, status of such studies and trials (including guidance) and expectations regarding data, safety and efficacy generally; (ii) data included in this press release, as well as the ability to use data from ongoing and planned clinical trials for the design and initiation of further clinical trials; (iii) regulatory submissions and authorizations, including related timelines; and (iv) the therapeutic value, development, and commercial potential of gene editing and delivery technologies and therapies, including CRISPR/Cas9.

此类声明包括但不限于库尔卡尼博士在本新闻稿中的声明，以及关于以下任何或所有内容的声明：(i) CRISPR Therapeutics 的临床前研究、临床试验和产品及项目管线，包括但不限于这些研究和试验的状态（包括指导）以及对数据、安全性和有效性的总体预期；(ii) 本新闻稿中包含的数据，以及利用正在进行和计划中的临床试验数据设计和启动更多临床试验的能力；(iii) 监管提交和授权，包括相关时间表；以及 (iv) 基因编辑和递送技术及疗法（包括 CRISPR/Cas9）的治疗价值、开发和商业潜力。

Risks that contribute to the uncertain nature of the forward-looking statements include, without limitation, the risks and uncertainties discussed under the heading “Risk Factors” in its most recent annual report on Form 10-K and in any other subsequent filings made by CRISPR Therapeutics with the U.S.

导致前瞻性声明具有不确定性的风险包括但不限于其最近的年度报告中“风险因素”标题下讨论的风险和不确定性，以及CRISPR Therapeutics向美国证券交易委员会提交的任何其他后续文件中提及的风险。

Securities and Exchange Commission. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. The Company disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this pres.

证券交易委员会。现有和潜在投资者被警告不要过分依赖这些前瞻性陈述，这些陈述仅在作出时有效。公司否认有任何义务或承诺更新或修改本新闻稿中包含的任何前瞻性陈述。

This press release also contains information regarding our industry, our business and the markets for certain of our product candidates, including data regarding the estimated size of those markets, and the incidence and prevalence of certain medical conditions. Unless otherwise expressly stated, we obtained this industry, business, market and other data from market research firms and other third parties, including medical publications, government data and similar sources.

本新闻稿还包含有关我们行业、业务以及某些产品候选市场的信息，包括这些市场的估计规模，以及某些医疗状况的发病率和患病率的数据。除非另有明确说明，我们从市场研究公司和其他第三方获得了这些行业、业务、市场及其他数据，包括医学出版物、政府数据和类似来源。

Information that is based on estimates, forecasts, projections, market research or similar methodologies is inherently subject to uncertainties and actual events or circumstances may differ materially from events and circumstances reflected in this information..

基于估计、预测、推测、市场研究或类似方法的信息本质上存在不确定性，实际事件或情况可能与本信息中反映的事件和情况有重大差异。

This press release discusses CRISPR/Cas9 gene editing investigational therapies and is not intended to convey conclusions about efficacy or safety as to those investigational therapies or uses of such investigational therapies. There is no guarantee that any investigational therapy will successfully complete clinical development or gain approval from applicable regulatory authorities..

本新闻稿讨论了CRISPR/Cas9基因编辑的在研疗法，并不旨在传达有关这些在研疗法或其使用的有效性和安全性的结论。无法保证任何在研疗法将成功完成临床开发或获得相关监管机构的批准。

Investor Contact:

投资者联系人：

+1-617-307-7503

+1-617-307-7503

ir@crisprtx.com

ir@crisprtx.com

Media Contact:

媒体联系人：

+1-617-315-4493

+1-617-315-4493

media@crisprtx.com

media@crisprtx.com

Source: CRISPR Therapeutics AG

来源：CRISPR Therapeutics AG