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-Initial CTX310™ Phase 1 clinical data demonstrates dose-dependent decreases in triglycerides (TG) and low-density lipoprotein (LDL), with peak reduction of up to 82% in TG and up to 81% in LDL, with a well-tolerated safety profile; presentation anticipated at a medical meeting in the second half of 2025-.

-初步的CTX310™第一阶段临床数据显示，甘油三酯（TG）和低密度脂蛋白（LDL）呈剂量依赖性下降，甘油三酯峰值降低高达82%，低密度脂蛋白峰值降低高达81%，且安全性良好；预计将在2025年下半年的医学会议上展示。-

-CASGEVY

-CASGEVY

®

®

continues to gain momentum; more than 65 authorized treatment centers (ATCs) activated globally for CASGEVY, and more than 90 patients have had cells collected across all regions; new patient initiations expected to grow significantly in 2025-

动力持续增强；全球已启动超过65家授权治疗中心 (ATCs) 用于CASGEVY，所有地区已有超过90名患者的细胞被收集；预计2025年新患者入组将显著增长。

-Clinical trial ongoing for CTX320

-CTX320的临床试验正在进行中

™

™

, targeting the LPA gene; top-line data update on track for the second quarter of 2025-

，针对LPA基因；2025年第二季度的初步数据更新按计划进行中-

-Clinical trials ongoing for next-generation CAR T product candidates, CTX112™ and CTX131™, targeting CD19 and CD70 across multiple indications; broad updates for CTX112 in oncology and autoimmune diseases expected mid-2025 with CTX131 updates also expected in 2025-

- 针对CD19和CD70的下一代CAR T产品候选物CTX112™和CTX131™正在进行临床试验，涵盖多种适应症；预计2025年年中将发布CTX112在肿瘤和自身免疫疾病领域的广泛更新，CTX131的更新也预计将在2025年发布 -

-Strong balance sheet with approximately

-资产负债表强劲，约为

$1.86 billion

18.6亿美元

in cash, cash equivalents, and marketable securities as of

截至……的现金、现金等价物和可出售证券

March 31, 2025

2025年3月31日

-

-

ZUG, Switzerland

楚格，瑞士

and

和

BOSTON

波士顿

,

，

May 06, 2025

2025年5月6日

(GLOBE NEWSWIRE) --

（环球新闻社）——

CRISPR Therapeutics

CRISPR Therapeutics

(Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today reported financial results for the first quarter ended March 31, 2025.

(Nasdaq: CRSP)，一家专注于为严重疾病开发变革性基因药物的生物制药公司，今日公布了截至2025年3月31日的第一季度财务业绩。

“CRISPR Therapeutics remains focused on executing our strategic priorities and advancing our portfolio of innovative therapies. We are highly encouraged by the initial data from our Phase 1 trial for CTX310, which demonstrates the power of our

“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。我们对CTX310一期试验的初步数据感到非常鼓舞，这展示了我们

in vivo

体内

gene editing platform to deliver paradigm changing medicines to patients with serious cardiovascular disease,” said

基因编辑平台，为患有严重心血管疾病的患者提供范式转变的药物，”

Samarth Kulkarni

萨马尔·库尔卡尼

, Ph.D., Chairman and Chief Executive Officer of

，博士，董事长兼首席执行官

CRISPR Therapeutics

CRISPR疗法

. “Additionally, we are pleased with the continued progress of Casgevy and the broader pipeline, and we look forward to sharing further clinical updates in the months ahead.”

“此外，我们对Casgevy及更广泛的管线持续取得的进展感到高兴，并期待在未来几个月分享更多的临床更新。”

Recent Highlights and Outlook

最近的亮点和展望

In Vivo Liver Editing Programs

体内肝脏编辑程序

CTX310™ targets

CTX310™ 目标

ANGPTL3

ANGPTL3

, a gene that encodes for key protein involved in the regulation of low-density lipoprotein (LDL) and triglyceride (TG) levels – both well-established 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 adverse effects on overall health.

，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）的已确定风险因素。ANGPTL3的功能缺失突变与LDL和TG水平显著降低以及ASCVD风险减少相关，且对整体健康无不良影响。

In the .

在...中。

U.S.

美国

alone, more than 40 million patients are affected by elevated LDL, severely elevated TGs or both – representing a large addressable patient population. CTX310 is initially focused on a high-risk subset of this group with the greatest unmet medical need and limited effective treatment options.

单独来看，超过4000万患者受到高LDL、严重升高的TGs或两者同时升高的影响，这代表着一个庞大的可治疗患者群体。CTX310最初聚焦于这一群体中具有最大未满足医疗需求且有效治疗选择有限的高风险亚群。

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

CTX310 正在进行一项首次人体剂量递增的 I 期临床试验，针对 ANGPTL3，纳入四个患者组，这些患者低密度脂蛋白（LDL）、甘油三酯（TG）或两者均升高，包括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），其甘油三酯（TG）水平 >300 mg/dL 和/或低密度脂蛋白胆固醇（LDL-C）>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。

TG and LDL, both of which are validated as surrogate endpoints for clinical benefit and accepted by regulatory agencies, were assessed at various timepoints..

TG和LDL均被验证为临床获益的替代终点，并被监管机构接受，已在多个时间点进行了评估。

Top-line data reported today are 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 of a data cutoff date of

今天报告的初步数据来自前四个队列的前10名患者（基于瘦体重的剂量DL1 [0.1 mg/kg]、DL2 [0.3 mg/kg]、DL3 [0.6 mg/kg] 和 DL4 [0.8 mg/kg]），截至数据截止日期，每个参与者至少有30天的随访。

April 16, 2025

2025年4月16日

.

。

A single dose of CTX310 demonstrated dose-dependent decreases in ANGPTL3, TGs, and LDL. Based upon ANGPTL3 knockdown, DL1 and DL2 were minimally active doses, whereas treatment at DL3 and DL4 resulted in reductions of up to 75% of baseline levels in ANGPTL3. CTX310 has been well-tolerated, with no treatment-related severe adverse events (SAEs) and no grade ≥3 adverse events (AEs) reported.

单剂量的CTX310显示出ANGPTL3、甘油三酯（TGs）和低密度脂蛋白（LDL）呈剂量依赖性下降。基于ANGPTL3的敲低效果，DL1和DL2为最低活性剂量，而DL3和DL4剂量治疗则使ANGPTL3基线水平降低多达75%。CTX310具有良好的耐受性，未报告与治疗相关的严重不良事件（SAEs），也未出现≥3级不良事件（AEs）。

No clinically significant changes in alanine aminotransferase (ALT), aspartate aminotransferase (AST), bilirubin, or platelets were observed at any dose level. There were no dose-dependent trends in any of these laboratory measurements..

在任何剂量水平下，未观察到丙氨酸氨基转移酶（ALT）、天冬氨酸氨基转移酶（AST）、胆红素或血小板有临床显著变化。这些实验室指标中没有任何剂量依赖性的趋势。

Mean % Change from Baseline at Day 30 post-infusion

输注后第30天从基线的平均百分比变化

(+/- SEM)

（± 标准误）

Dose Level (DL)

剂量水平 (DL)

DL1 + DL2

DL1 + DL2

0.1 + 0.3 mg/kg

0.1 + 0.3 毫克/千克

(n=6)

(n=6)

DL3

DL3

0.6 mg/kg

0.6 毫克/千克

(n=3)

(n=3)

DL4

DL4

0.8 mg/kg

0.8毫克/千克

(n=1)

(n=1)

Patient type

患者类型

HeFH (4), MDL, sHTG

他FH（4），MDL，sHTG

MDL (2), HeFH

MDL（2），HeFH

sHTG

sHTG

Triglycerides

甘油三酯

-10.6% ± 13.1%

-10.6% ± 13.1%

-55.7% ± 8.0%

-55.7% ± 8.0%

-81.9%

-81.9%

LDL

低密度脂蛋白

34.8% ± 27.0%

34.8% ± 27.0%

-28.5% ± 24.4%

-28.5% ± 24.4%

-64.6%

-64.6%

Compelling individual patient responses highlight the therapeutic potential of CTX310: a DL4 patient with sHTG had an 82% reduction in triglycerides from a baseline of 1073 mg/dL at day 30, and a DL3 patient with HeFH had an 81% reduction in LDL-C from a baseline of 256 mg/dL at day 90 – supporting the potential for targeted efficacy in high-risk populations..

引人注目的个体患者反应凸显了CTX310的治疗潜力：一名患有sHTG的DL4患者在第30天时甘油三酯水平较基线1073 mg/dL减少了82%，而一名患有HeFH的DL3患者在第90天时LDL-C水平较基线256 mg/dL减少了81%——这支持了其在高风险人群中的潜在靶向疗效。

These initial results represent a significant milestone in the advancement of CRISPR Therapeutics’ proprietary lipid nanoparticle (LNP) delivery technologies for gene editing in the liver. The Company plans to present the CTX310 Phase 1 data at a medical meeting in the second half of 2025.

这些初步结果代表了CRISPR Therapeutics公司在肝脏基因编辑领域专有的脂质纳米颗粒（LNP）递送技术的重大里程碑。公司计划在2025年下半年的医学会议上展示CTX310一期临床试验的数据。

CTX320 is in an ongoing Phase 1 clinical trial targeting the

CTX320正处于针对某一目标的持续第一阶段临床试验中。

LPA

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 are prevalent in up to 20% of the global population. Dose escalation is ongoing, with an update expected in the second quarter of 2025..

脂蛋白(a) [Lp(a)] 升高的患者中的基因，这是一种遗传决定的风险因素，与主要不良心血管事件 (MACE) 发生率增加相关。Lp(a) 水平升高在高达 20% 的全球人口中普遍存在。剂量递增正在进行中，预计将在 2025 年第二季度更新。

CRISPR Therapeutics

CRISPR Therapeutics

continues to advance two preclinical programs: CTX340™, targeting angiotensinogen (AGT) for the treatment of refractory hypertension, and CTX450™, targeting 5’ aminolevulinic acid synthase 1 (ALAS1) for the treatment of acute hepatic porphyrias (AHP). Both candidates are currently in IND/CTA-enabling studies..

继续推进两个临床前项目：CTX340™，靶向血管紧张素原 (AGT)，用于治疗难治性高血压；以及CTX450™，靶向5'-氨基乙酰丙酸合成酶1 (ALAS1)，用于治疗急性肝卟啉症 (AHP)。这两个候选药物目前正在进行IND/CTA支持性研究。

Hemoglobinopathies and CASGEVY® (exagamglogene autotemcel [exa-cel])

血红蛋白病和CASGEVY®（exagamglogene autotemcel [exa-cel]）

CASGEVY is approved in the

CASGEVY已获批准在

U.S.

美国

,

，

Great Britain

英国

, the EU, the

，欧盟，

Kingdom of Saudi Arabia

沙特阿拉伯王国

(KSA), the

（KSA），

Kingdom of Bahrain

巴林王国

(

(

Bahrain

巴林

),

),

Canada

加拿大

,

，

Switzerland

瑞士

and the

和

United Arab Emirates

阿拉伯联合酋长国

(

(

UAE

阿联酋

) for the treatment of both SCD and TDT, and launches are ongoing. Building on the foundational launch in 2024, significant progress is being made to bring this transformative therapy to patients worldwide.

）用于治疗SCD和TDT，上市进程正在进行中。在2024年基础性上市的基础上，正在取得重大进展，以将这种变革性的疗法带给全球患者。

As of

截至

May 1

5月1日

, more than 65 authorized treatment centers (ATCs) have been activated globally and approximately 90 patients have had their first cell collection. The number of new patients initiating cell collection is expected to grow significantly throughout 2025.

，全球已有超过65家授权治疗中心（ATC）被激活，大约90名患者完成了他们的首次细胞采集。预计到2025年，新启动细胞采集的患者数量将大幅增长。

Vertex has secured a formal reimbursement agreement with

Vertex已经获得了正式的报销协议

NHS

国家医疗服务体系

England, enabling access to CASGEVY for patients with SCD. This follows an earlier agreement, reaching in

英格兰，使SCD患者能够获得CASGEVY。这是在早前达成的一项协议之后进行的，

August 2024

2024年8月

, providing access for eligible patients with TDT. A similar reimbursement agreement has been established in

，为符合条件的TDT患者提供使用机会。类似的报销协议已经在

Wales

威尔士

for eligible SCD and TDT patients. Following a positive assessment, national reimbursement was finalized in

对于符合条件的SCD和TDT患者。在积极评估后，国家报销最终确定。

Austria

奥地利

. In the

。在

Middle East

中东

, reimbursement was also finalized across the majority of Emirates, following regulatory approval in the

，监管机构批准后，多数酋长国也完成了报销流程的最终确定，

UAE

阿联酋

.

。

A manufacturing license application has been submitted to the

制造许可申请已提交给

U.S. Food and Drug Administration

美国食品药品监督管理局

(FDA), with commercial production in

（FDA），商业生产于

Portsmouth, New Hampshire

朴茨茅斯，新罕布什尔州

expected to begin in the second half of 2025. This submission is part of the planned ramp-up of CASGEVY manufacturing capacity as demand for the therapy increases.

预计将在2025年下半年开始。此次提交是计划中的CASGEVY制造能力提升的一部分，因为对该疗法的需求正在增加。

CRISPR Therapeutics

CRISPR Therapeutics

continues to advance its next-generation approaches designed to significantly broaden the addressable patient population for SCD and TDT. The Company’s internally developed targeted conditioning program, an anti-CD117 (c-Kit) antibody-drug conjugate (ADC), remains on track in preclinical development.

继续推进其旨在大幅扩大镰状细胞病（SCD）和地中海贫血（TDT）可治疗患者群体的下一代方法。公司内部开发的靶向调理方案，一种抗CD117（c-Kit）抗体药物偶联物（ADC），在临床前开发阶段仍按计划进行。

In parallel, the Company is making continued progress in its .

同时，公司正在不断取得进展。

in vivo

体内

editing platform aimed at enabling direct editing of hematopoietic stem cells (HSC) without the need for conditioning. By potentially eliminating the need for conditioning, this approach could unlock access to transformative therapies for a significantly larger patient population.

旨在实现无需调理即可直接编辑造血干细胞 (HSC) 的编辑平台。通过可能消除对调理的需求，这种方法可以为更广泛的患者群体提供获得变革性治疗的机会。

Immuno-Oncology and Autoimmune Disease Programs

免疫肿瘤学与自身免疫疾病项目

Clinical trials are ongoing for its next-generation allogeneic CAR T product candidates, CTX112™ and CTX131™, targeting CD19 and CD70, respectively, across multiple indications. Both candidates incorporate novel potency edits which can lead to significantly higher CAR T cell expansion and cytotoxicity, potentially establishing them as best-in-class allogeneic CAR T products for their respective targets.

其下一代同种异体 CAR T 产品候选物 CTX112™ 和 CTX131™ 分别针对 CD19 和 CD70 的多项适应症正在进行临床试验。这两个候选产品都包含新型效力编辑，可以显著提高 CAR T 细胞的扩增和细胞毒性，有望使其成为各自靶点的最佳同种异体 CAR T 产品。

CTX112 is being developed for hematologic malignancies and autoimmune diseases and has the potential to be best-in-class based on preliminary data. .

CTX112 正在被开发用于血液系统恶性肿瘤和自身免疫性疾病，并且根据初步数据有潜力成为同类最佳。

Encouraging clinical data from the ongoing Phase 1/2 clinical trial of CTX112 in relapsed or refractory B-cell malignancies supported the FDA’s decision to grant Regenerative Medicine Advanced Therapy (RMAT) designation for the treatment of relapsed or refractory follicular lymphoma and marginal zone lymphoma. .

来自正在进行的CTX112在复发或难治性B细胞恶性肿瘤中的I/II期临床试验的良好临床数据，支持了FDA授予其再生医学先进疗法（RMAT）资格，用于治疗复发或难治性滤泡性淋巴瘤和边缘区淋巴瘤。

CTX112 is also in an ongoing Phase 1 clinical trial in autoimmune diseases, including indications such as systemic lupus erythematosus (SLE), systemic sclerosis and inflammatory myositis. Preliminary safety, pharmacokinetic, and pharmacodynamic data from oncology trials support its potential in autoimmune indications.

CTX112目前也正在进行一项针对自身免疫性疾病的1期临床试验，包括系统性红斑狼疮（SLE）、系统性硬化症和炎性肌病等适应症。来自肿瘤学试验的初步安全性、药代动力学和药效学数据支持其在自身免疫性疾病中的潜力。

The Company plans to provide an update for both oncology and autoimmune disease in mid-2025..

公司计划在2025年年中提供肿瘤学和自身免疫疾病方面的更新。

Clinical trials for CTX131™ are ongoing in both solid tumors and hematologic malignancies, with updates expected in 2025. In parallel, an Investigational New Drug (IND) application for glypican-3 (GPC3)-targeted gene-edited autologous CAR T program for the treatment of hepatocellular carcinoma has been opened by our partner, .

CTX131™的临床试验正在实体瘤和血液系统恶性肿瘤中进行，预计将于2025年更新数据。与此同时，我们的合作伙伴已经为针对甘露糖蛋白3（GPC3）的基因编辑自体CAR T细胞项目提交了研究性新药（IND）申请，用于治疗肝细胞癌。

Roswell Park Comprehensive Cancer Center

罗斯威尔公园综合癌症中心

.

。

CRISPR Therapeutics’ immuno-oncology and autoimmune disease efforts are supported by a wholly-owned,

CRISPR Therapeutics的免疫肿瘤学和自身免疫疾病研究由其全资支持，

U.S.

美国

manufacturing facility located in

制造工厂位于

Framingham, MA.

马萨诸塞州弗雷明汉。

This investment enables the production of clinical and commercial-stage good manufacturing practice (GMP) materials across the Company’s allogeneic cell therapy programs.

这项投资使得公司能够在其同种异体细胞治疗项目中生产临床和商业阶段的良好生产规范（GMP）材料。

Regenerative Medicine Programs

再生医学项目

CRISPR Therapeutics

CRISPR治疗学

continues to advance its regenerative medicine efforts in Type 1 diabetes (T1D). In addition to CTX211, the Company continues to advance next-generation programs focusing on induced pluripotent stem cell (iPSC) derived, allogeneic, gene-edited, beta islet cell precursors. These approaches aim to achieve insulin independence in T1D patients without the need for chronic immunosuppression.

继续推进其在1型糖尿病（T1D）领域的再生医学工作。除了CTX211，公司还在持续推进下一代项目，重点放在诱导多能干细胞（iPSC）衍生的、同种异体的、经过基因编辑的胰岛β细胞前体。这些方法旨在帮助T1D患者实现胰岛素独立，而无需长期使用免疫抑制剂。

The Company expects to provide an update in 2025..

公司预计将在2025年提供更新。

Upcoming Events

即将举行的活动

The Company will participate in the following events in May:

公司将在五月份参加以下活动：

3rd Annual

第三届年度

H.C. Wainwright BioConnect Investor Conference

H.C. Wainwright BioConnect 投资者会议

,

，

May 20

5月20日

2025 RBC Capital Markets Global Healthcare Conference,

2025年RBC资本市场全球医疗保健会议，

May 20

5月20日

First Quarter 2025 Financial Results

2025年第一季度财务业绩

Cash Position:

现金头寸：

Cash, cash equivalents, and marketable securities were

现金、现金等价物和可出售证券

$1,855.3 million

1855.3百万美元

as of

截至

March 31, 2025

2025年3月31日

, compared to

，与...相比

$1,903.8 million

1903.8百万美元

as of

截至

December 31, 2024

2024年12月31日

. The decrease in cash was primarily driven by operating expenses, offset by proceeds from interest income and employee option exercises.

现金的减少主要是由运营费用驱动的，部分被利息收入和员工期权行权带来的收益所抵消。

R&D Expenses:

研发费用：

R&D expenses were

研发费用为

$72.5 million

7250万美元

for the first quarter of 2025, compared to

2025年第一季度，与

$76.2 million

7620万美元

for the first quarter of 2024. The decrease in R&D expense was primarily driven by a decrease in employee-related expenses, including stock-based compensation expenses.

2024年第一季度。研发费用的减少主要是由于员工相关费用的减少，包括基于股票的薪酬费用。

G&A Expenses:

管理费用：

General and administrative expenses were

一般和行政费用为

$19.3 million

1930万美元

for the first quarter of 2025, compared to

2025年第一季度，与

$18.0 million

1800万美元

for the first quarter of 2024.

适用于2024年第一季度。

Collaboration Expense:

协作费用：

Collaboration expense, net, was

净合作费用为

$57.5 million

5750万美元

for the first quarter of 2025, compared to

2025年第一季度，与……相比

$47.0 million

4700万美元

for the first quarter of 2024. The increase in collaboration expense, net, was primarily attributable to costs related to CASGEVY and collaboration expenses related to

2024年第一季度。协作费用净额的增加主要归因于与CASGEVY相关的成本以及与协作费用相关的

in vivo

体内

HSC editing, offset by CASGEVY product sales.

HSC编辑，由CASGEVY产品销售抵消。

Net Loss:

净亏损：

Net loss was

净亏损为

$136.0 million

1.36亿美元

for the first quarter of 2025, compared to a net loss of

2025年第一季度，相比净亏损

$116.6 million

1.166亿美元

for the first quarter of 2024.

2024年第一季度。

About CASGEVY

关于CASGEVY

®

®

(exagamglogene autotemcel [exa-cel])

(exagamglogene autotemcel [exa-cel])

CASGEVY is a non-viral,

CASGEVY是一种非病毒性的，

ex vivo

离体

CRISPR/Cas9 gene-edited cell therapy for eligible patients with SCD or TDT, in which a patient’s own hematopoietic stem and progenitor cells are edited at the erythroid specific enhancer region of the

镰状细胞病或β-地中海贫血患者符合条件接受CRISPR/Cas9基因编辑细胞治疗，这种治疗会编辑患者自身造血干细胞和祖细胞在红系特异性增强子区域的基因。

BCL11A

BCL11A基因

gene. This edit results in the production of high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is the form of the oxygen-carrying hemoglobin that is naturally present during fetal development, which then switches to the adult form of hemoglobin after birth. CASGEVY has been shown to reduce or eliminate recurrent vaso-occlusive crises (VOCs) for patients with SCD and transfusion requirements for patients with TDT.

基因。这种编辑导致红细胞中产生高水平的胎儿血红蛋白（HbF；血红蛋白F）。HbF是携带氧气的血红蛋白的一种形式，在胎儿发育期间自然存在，出生后则转变为成人形式的血红蛋白。CASGEVY已被证明可以减少或消除SCD患者的反复血管阻塞性危象（VOCs），并降低TDT患者的输血需求。

CASGEVY is approved for certain indications in multiple jurisdictions for eligible patients..

CASGEVY在多个司法管辖区被批准用于某些适应症的符合条件的患者。

About the CRISPR Collaboration and Vertex

关于CRISPR合作与Vertex公司

CRISPR Therapeutics and Vertex entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. CASGEVY represents the first potential treatment to emerge from the joint research program.

CRISPR Therapeutics 和 Vertex 于 2015 年建立了战略研究合作，专注于使用 CRISPR/Cas9 发现和开发针对人类疾病潜在遗传原因的新疗法。CASGEVY 代表了该联合研究计划中出现的首个潜在治疗方法。

Under an amended collaboration agreement, Vertex now leads global development, manufacturing, and commercialization of CASGEVY and splits program costs and profits worldwide 60/40 with CRISPR Therapeutics. Vertex is the manufacturer and exclusive license holder of CASGEVY..

根据修订后的合作协议，Vertex现在领导CASGEVY的全球开发、制造和商业化，并与CRISPR Therapeutics按60/40的比例分担全球项目成本和利润。Vertex是CASGEVY的制造商和独家许可证持有者。

About CTX112

关于CTX112

CTX112 is being developed for both oncology and autoimmune indications. CTX112 is a next-generation, wholly-owned, allogeneic CAR T product candidate targeting Cluster of Differentiation 19, or CD19, which incorporates edits designed to evade the immune system, enhance CAR T potency, and reduce CAR T exhaustion.

CTX112 正在为肿瘤学和自身免疫适应症开发。CTX112 是一种下一代的、全资拥有的、同种异体的 CAR T 候选产品，靶向分化簇 19（即 CD19），它结合了旨在逃避免疫系统、增强 CAR T 效力并减少 CAR T 衰竭的设计编辑。

CTX112 is being investigated in an ongoing clinical trial designed to assess safety and efficacy of the product candidate in adult patients with relapsed or refractory B-cell malignancies who have received at least two prior lines of therapy. In addition, CTX112 is being investigated in an ongoing clinical trial designed to assess the safety and efficacy of the product candidate in adult patients with systemic lupus erythematosus, systemic sclerosis, and inflammatory myositis..

CTX112 正在一项正在进行的临床试验中进行研究，该试验旨在评估该候选产品在复发或难治性 B 细胞恶性肿瘤成年患者中的安全性和有效性，这些患者至少接受过两线既往治疗。此外，CTX112 还在一项正在进行的临床试验中进行研究，该试验旨在评估该候选产品在系统性红斑狼疮、系统性硬化症和炎性肌病成年患者中的安全性和有效性。

About CTX131

关于CTX131

CTX131 is being developed for both solid tumors and hematologic malignancies, including T cell lymphomas (TCL). CTX131 is a next-generation, wholly-owned, allogeneic CAR T product candidate targeting Cluster of Differentiation 70, or CD70, an antigen expressed on various solid tumors and hematologic malignancies.

CTX131 正在被开发用于实体瘤和血液系统恶性肿瘤，包括T细胞淋巴瘤（TCL）。CTX131 是一种下一代的、全资拥有的、同种异体的CAR T候选产品，靶向分化簇70（CD70），这是一种在多种实体瘤和血液系统恶性肿瘤上表达的抗原。

CTX131 incorporates edits designed to evade the immune system, prevent fratricide, enhance CAR T potency, and reduce CAR T exhaustion. CTX131 is being investigated in ongoing clinical trials designed to assess the safety and efficacy of the product candidate in adult patients with relapsed or refractory solid tumors and hematologic malignancies, including TCL..

CTX131包含旨在逃避免疫系统、防止自相残杀、增强CAR T效力并减少CAR T耗竭的编辑。CTX131正在持续进行的临床试验中进行研究，以评估该候选产品在成年复发或难治性实体瘤和血液恶性肿瘤（包括TCL）患者中的安全性和有效性。

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 (

产品组合包括其主要的在研项目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）。两者都是经过验证的心血管疾病治疗靶点。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中进行临床试验，并分别在脂蛋白(a)升高的患者中进行试验。

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

此外，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

agt

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

) 用于治疗顽固性高血压和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 CTX211

关于CTX211

CTX211 is an allogeneic, gene-edited, stem cell-derived investigational therapy for the treatment of type 1 diabetes (T1D), which incorporates gene edits that aim to make cells hypoimmune and enhance cell fitness. This immune-evasive cell replacement therapy is designed to enable patients to produce their own insulin in response to glucose.

CTX211 是一种同种异体、经过基因编辑的干细胞衍生研究性疗法，用于治疗 1 型糖尿病 (T1D)，其结合了旨在使细胞具有低免疫原性并增强细胞适应性的基因编辑。这种免疫规避细胞替代疗法旨在使患者能够响应葡萄糖自行产生胰岛素。

A Phase 1 clinical trial for CTX211 for the treatment of T1D is ongoing..

CTX211治疗1型糖尿病的I期临床试验正在进行中。

About CRISPR Therapeutics

关于CRISPR治疗学

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.

自从十多年前成立以来，CRISPR Therapeutics已从一家推进基因编辑项目的研究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。该公司拥有涵盖广泛疾病领域的产品候选组合，包括血红蛋白病、肿瘤学、再生医学、心血管、自身免疫和罕见病。

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，并代表了一种强大的、经过临床验证的方法，有望创造出一类潜在的变革性药物。

To accelerate and expand its efforts, CRISPR Therapeutics has formed strategic partnerships with leading companies including .

为了加速和扩大其努力，CRISPR Therapeutics 已与包括在内的领先公司建立了战略合作伙伴关系。

Vertex Pharmaceuticals

顶点制药公司

. CRISPR Therapeutics AG is headquartered in Zug,

CRISPR Therapeutics AG 总部位于楚格，

Switzerland

瑞士

, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Boston, Massachusetts and San Francisco,

，其全资美国子公司 CRISPR Therapeutics, Inc.，以及位于马萨诸塞州波士顿和旧金山的研发业务，

California

加利福尼亚州

. To learn more, visit

. 欲了解更多信息，请访问

www.crisprtx.com

www.crisprtx.com

.

。

CRISPR THERAPEUTICS

CRISPR治疗学

®

®

standard character mark and design logo, CTX112™, CTX131™, CTX211™, 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，CTX112™、CTX131™、CTX211™、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，是根据1995年《私人证券诉讼改革法案》定义的“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中明示或暗示的结果存在重大差异。

Such statements include, but are not limited to, statements made by .

这些声明包括但不限于由...作出的声明。

Dr. Kulkarni

库尔卡尼博士

in this press release, as well as regarding any or all of the following: (i)

在本新闻稿中，以及关于以下任何或全部内容：(i)

CRISPR Therapeutics

CRISPR Therapeutics

preclinical studies, clinical trials and pipeline products and programs, including, without limitation, manufacturing capabilities, status of such studies and trials, potential expansion into new indications 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) .

临床前研究、临床试验和在研产品及项目，包括但不限于生产能力、这些研究和试验的状态、潜在的新适应症扩展以及关于数据、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进一步临床试验的能力；(iii) 。

CRISPR Therapeutics

CRISPR疗法

strategy, goals, anticipated financial performance and the sufficiency of its cash resources; (iv) plans and expectations for the commercialization of, and anticipated benefits of, CASGEVY, including anticipated patient access to CASGEVY; (v) regulatory submissions and authorizations, including timelines for and expectations regarding additional regulatory agency decisions; (vi) the expected benefits of its collaborations; and (vii) the therapeutic value, development, and commercial potential of gene editing and delivery technologies and therapies, including CRISPR/Cas9.

战略、目标、预期财务表现及其现金资源的充足性；(iv) 对CASGEVY商业化的计划与预期，包括预期患者获取CASGEVY的途径；(v) 监管提交与授权，包括时间表及对其他监管机构决策的预期；(vi) 其合作的预期收益；以及(vii) 基因编辑和递送技术及疗法（包括CRISPR/Cas9）的治疗价值、开发和商业化潜力。

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 .

可能导致前瞻性陈述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，以及任何其他后续文件中提及的风险。

CRISPR Therapeutics

CRISPR疗法

with the

随着

U.S. Securities and Exchange Commission

美国证券交易委员会

. 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. We disclaim any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law..

现有和潜在投资者被警告不要过度依赖这些前瞻性陈述，这些陈述仅在作出时有效。我们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，除非法律要求的范围内。

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、我们的业务以及我们某些候选产品市场的信息，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取了此行业、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此类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，并非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。不能保证任何在研疗法将成功完成临床开发或获得适用监管机构的批准。

Investor Contact:

投资者联系方式：

+1-617-307-7503

+1-617-307-7503

ir@crisprtx.com

投资者关系@CRISPR治疗公司.com

Media Contact:

媒体联系人：

+1-617-315-4493

+1-617-315-4493

media@crisprtx.com

媒体@克里普尔克斯.com

CRISPR Therapeutics AG

CRISPR治疗学股份公司

Condensed Consolidated Statements of Operations

合并简明损益表

(Unaudited, In thousands except share data and per share data)

（未经审计，除股份数据和每股数据外，以千为单位）

Three Months Ended

三个月结束

March 31

3月31日

,

，

2025

2025

2024

2024

Revenue:

收入：

Collaboration revenue

合作收入

$

$

—

—

$

$

—

—

Grant revenue

补助收入

865

865

504

504

Total revenue

总收入

$

$

865

865

504

504

Operating expenses:

营业费用：

Research and development

研究与开发

72,484

72,484

76,172

76,172

General and administrative

一般管理费用

19,296

19,296

17,953

17,953

Collaboration expense, net

协作费用，净值

57,509

57,509

46,966

46,966

Total operating expenses

总运营费用

149,289

149,289

141,091

141,091

Loss from operations

营业亏损

(148,424

(148,424

)

)

(140,587

(140,587

)

)

Total other income, net

总其他收入，净额

13,537

13,537

24,720

24,720

Net loss before income taxes

税前净亏损

(134,887

(134,887

)

)

(115,867

(115,867

)

)

Provision for income taxes

所得税准备金

(1,109

(1,109

)

)

(724

(724

)

)

Net loss

净亏损

(135,996

(135,996

)

)

(116,591

(116,591

)

)

Foreign currency translation adjustment

外币折算调整

41

41

(11

(11

)

)

Unrealized gain (loss) on marketable securities

未实现的可交易证券收益（损失）

2,254

2,254

(3,454

(3,454

)

)

Comprehensive loss

综合损失

$

$

(133,701

(133,701

)

)

$

$

(120,056

（120,056

)

)

Net loss per common share — basic

每股普通股的基本净亏损

$

$

(1.58

(1.58

)

)

$

$

(1.43

(1.43

)

)

Basic weighted-average common shares outstanding

基本加权平均流通股

85,938,720

85,938,720

81,794,630

81,794,630

Net loss per common share — diluted

每股普通股的净亏损——摊薄后

$

$

(1.58

(1.58

)

)

$

$

(1.43

(1.43

)

)

Diluted weighted-average common shares outstanding

稀释加权平均流通股数

85,938,720

85,938,720

81,794,630

81,794,630

CRISPR Therapeutics AG

CRISPR治疗学股份公司

Condensed Consolidated Balance Sheets Data

简明合并资产负债表数据

(Unaudited, in thousands)

（未经审计，单位：千）

As of

截至

March 31, 2025

2025年3月31日

December 31, 2024

2024年12月31日

Cash and cash equivalents

现金及现金等价物

$

$

235,184

235,184

$

$

298,257

298,257

Marketable securities

可销售证券

1,620,101

1,620,101

1,605,569

1,605,569

Working capital

营运资本

1,748,164

1,748,164

1,849,350

1,849,350

Total assets

总资产

2,166,102

2,166,102

2,242,034

2,242,034

Total shareholders' equity

股东权益总额

1,829,160

1,829,160

1,932,080

1,932,080

Source: CRISPR Therapeutics AG

来源：CRISPR Therapeutics AG