Pirtobrutinib

#痛风临床试验 #IBI3011 #信达生物 #IL—1RAP单抗 #痛风急性发作新药临床招募 简要入排 本临床药物为人源化抗IL-1RAP单克隆抗体；目前招募痛风急性发作患者；既往至少有2次痛风关节炎发作史且该次目标关节的疼痛得到完全缓解；在给药前4天内发生痛风急性发作；痛风急性发作的目标关节VAS疼痛评分（0-100mm）≥50mm；全国多中心。 研究中心 江苏苏州 广东广州、江门 河北廊坊、沧州 江西萍乡 山东临沂 四川成都 浙江湖州 安徽合肥、蚌埠 山西临汾 具体启动情况以后期咨询为准 【研究中心若有更新请拉至文末查看】 试验名称 一项评估IBI3011首次人体给药的安全性、耐受性及药代动力学特征的单次给药剂量递增研究。 试验药物 随着经济发展与生活水平提高，我国痛风患病率逐年持续攀升，2019年患病人数已超300万。关节局部尿酸盐晶体沉积可诱发炎症反应，引起痛风急性发作，可导致关节结构破坏和功能障碍，严重影响患者生活质量。 人白细胞介素1受体辅助蛋白（IL-1RAP）是IL-1家族的共受体，可与IL-1R1、ST2和IL-36R等受体形成二聚体，介导IL-1α/β、IL-33和IL-36α/β/γ等信号通路的激活，这些信号通路在多种自身免疫及炎症性疾病（如急性痛风性关节炎、化脓性汗腺炎等）的发生发展中起关键作用。相较抗IL-1β抗体，靶向IL-1RAP能同时阻断多条IL-1家族炎症通路，有望迅速控制炎症，缓解痛风症状，从而优化降尿酸治疗的时机。 IBI3011是国内首个靶向炎症和自免疾病的IL-1RAP的单克隆抗体。其可通过与IL-1RAP结合，阻断其与相关受体的相互作用，从而同时抑制多条IL-1家族炎症通路，达到控制炎症、缓解症状的目的。 在痛风与关节炎动物模型中，该分子可显著抑制IL-1与IL-36信号通路的激活，延缓疾病进展。在猴子上表现出优异的药代动力学特性与安全性。此外，该分子已开发高浓度制剂，便于皮下给药。 研究药物：IBI3011（I期） 登记号：CTR20254422 试验类型：对照试验（VS 安慰剂） 适应症：痛风急性发作 申办方：信达生物制药（苏州）有限公司 延伸阅读 信达生物成立于2011年，致力于研发、生产和销售肿瘤、自身免疫、代谢、眼科等重大疾病领域的创新药物。公司已有18个产品获得批准上市，它们分别是信迪利单抗注射液，贝伐珠单抗注射液，阿达木单抗注射液，利妥昔单抗注射液，佩米替尼片，奥雷巴替尼片，雷莫西尤单抗注射液，塞普替尼胶囊，伊基奥仑赛注射液，托莱西单抗注射液，氟泽雷塞片，匹妥布替尼片，己二酸他雷替尼胶囊，利厄替尼片，替妥尤单抗N01注射液，玛仕度肽注射液，匹康奇拜单抗注射液和伊匹木单抗N01注射液。 备注：以上信息来源于药企官网 注：上下滑动可查看全部内容 用药周期 IBI3011注射液的规格：300mg（2.0ml）/瓶；用法用量：按试验方案给药；用药时程：仅给药1次。 入选标准 1、理解并签署知情同意书。 2、年龄≥18周岁，男性或女性。 3、A部分的健康受试者体重指数（Body Mass Index, BMI）≥18.0kg/m2且≤26.0kg/m2；B部分的痛风急性发作受试者BMI≥18.0kg/m2且≤40.0kg/m2。 4、体格检查、生命体征检查、实验室检查（血常规、尿常规、血生化等）、12导联心电图、腹部B超、胸部X片等筛选期完成的检查结果正常或异常无临床意义（仅针对A部分的健康受试者）。 5、在试验期间及给药后6个月内无育儿计划且能采取充分的避孕措施。 6、痛风急性发作患者符合美国风湿病学会（American College of Rheumatology, ACR 2015年痛风分类诊断标准）。 7、既往至少有2次痛风关节炎发作史且该次目标关节的疼痛得到完全缓解。 8、在给药前4天内发生痛风急性发作（根据受试者的症状、体征、主诉、病历记录等，由研究者判定）。 9、筛选时，受试者痛风急性发作的目标关节VAS疼痛评分（0-100mm）≥50mm。 排除标准 1、有特定过敏史者（哮喘、荨麻疹、湿疹等），或过敏体质者（如对两种或以上药物、食物和花粉过敏）者；或既往使用生物制剂有超敏反应病史者。 2、有晕血、晕针史者，或不能耐受静脉穿刺者。 3、1个月内参加过其他药物临床试验，或距上一次临床试验时间短于试验药物5个半衰期者（以时间较长者为准）。 4、随机前3个月内有活疫苗或减毒疫苗接种史，或预期在参加研究期间接种疫苗者。 5、筛选前8周内有过献血或失血400mL者。 6、筛选前6个月内有严重感染、严重外伤或外科重大手术者。 7、随机前30天内存在需要使用全身药物治疗的感染，或筛选时仍存在的感染状态。 8、不能进行肌肉注射/静脉输注者（例如正在接受抗凝血药治疗、血小板减少、已知患有出血性疾病如特发性血小板减少性紫癜的患者、慢性牙周病患者。 9、合并恶性肿瘤病史。 10、妊娠期或哺乳期女性。 11、不愿意按照方案要求进行避孕的受试者，在本研究过程中及末次给药后6个月内，有妊娠（或捐献精子）计划，不愿意采取可靠避孕措施的育龄女性（男性）受试者。 12、筛选前3个月内平均每天酒精摄入超过2个单位（1单位酒精≈360mL酒精含量为5%的啤酒或45mL酒精含量为40%的烈酒或150mL酒精含量为 12%的葡萄酒），或在用药前48小时内饮酒，试验期间不能戒酒者，或酒精呼气试验阳性者（仅针对A部分健康受试者）。 13、筛选前12个月内有药物滥用史、药物依赖史或药物筛查结果阳性者。 14、血清病毒学检测人免疫缺陷病毒抗体（HIV-Ab）、梅毒非特异性抗体（RPR）、抗丙型肝炎病毒抗体（HCV-Ab）阳性、乙肝病毒（HBV）表面抗原或e抗原（HBeAg）任一阳性者。 15、有QT间期延长病史，或筛选时QTc男性＞450ms，女性470ms。 16、健康受试者有临床或影像学证据表明受试者存在活动性结核，或有证据表明受试者处于结核潜伏期，如：在筛选时（或筛选前6个月内结核菌素（Purified Protein Derivative, PPD）试验提示硬结直径≥10mm或判定为强阳性（ （+++）者，或 T-SPOT.TB阳性，或与确诊活动性结核患者同时处于密闭空间数天者； 痛风急性发作受试者 诊断为结核病或具有结核病的风险因素，例如但不局限于：a) 需要服用抗潜伏性结核病的抗菌药物，例如异烟肼（不能因要进入试验，在试验前提前终止已开始的抗菌药物治疗）；b) 在过去12个月内与活动性肺结核病人有过密切接触（即在相对封闭的环境中相处达数天或数周）；c) 高度怀疑或确诊活动性结核感染者。 17、有中枢神经系统、心血管系统、消化系统、呼吸系统、泌尿系统、血液系统、代谢障碍等系统疾病病史者（仅针对A部分健康受试者）。 18、存在神经精神病史，或研究者认为不适合参加本临床试验者。 19、接受研究药物给药前指定时间内使用以下任何药物或治疗措施：a) 糖皮质激素药物：i. 在给药前24小时内，系统使用了10mg泼尼松或相当剂量的激素；ii. 长期使用糖皮质激素治疗（定义为泼尼松剂量5mg/d或相当剂量，28天）；iii. 给药前 14天内向目标关节内注射糖皮质激素类药物；iv. 给药前14天内接受过肌肉注射或静脉给药的糖皮质激素类药物；b) 在给药前24小时内使用了麻醉品（阿片类或曲马多）；c) 给药前在该药物的自身一个半衰期内服用过 NSAIDS类及其他止痛药物；d) 给药前12小时内使用过秋水仙碱；e) 冰敷治疗：给药前 4小时内使用过冰袋或其他冰敷治疗；f) 在给药前30天内或5个半衰期内（以较长者为准）使用了任何IL-1阻断剂、TNF抑制剂、其他生物制剂。 20、确诊为继发性痛风（如由化疗引起、移植性痛风等）。 21、有证据显示或怀疑为感染/脓毒性关节炎，或者其他急性炎症性关节炎。 22、筛选期的实验室检查指标符合以下任何一条：a) 肝功能检查提示总胆红素（TBIL）高于正常值上限ULN 1.5倍及以上；b) 丙氨酸氨基转移酶（ ALT）或天门冬氨酸氨基转移酶AST）高于正常值上限（ULN 3倍及以上；c) 检查提示血甘油三酯>300mg/dL或>3.42mmol/L；脾脏B超或体格检查提示脾脏中度及以上肿大者（中度肿大定义为脾缘超过肋下2cm，在脐水平线以上；超过脐水平线或前正中线则为高度肿大 /巨脾）；d) 估算的肾小球滤过率（estimand Glomerular Filtration Rate）eGFR 60ml/min/1.73m2。 23、存在重要的疾病，包括但不限于：a) 难治性高血压（同时使用3种不同类别的降压药，血压仍高于140/90mmHg），或筛选期血压高于180/110mmHg；b) 控制不佳的糖尿病（静脉血空腹血糖＞7.0mmol/L，非空腹血糖10mmol/L c) 既往存在充血性心力衰竭病史；d) 筛选前6个月内发生心肌梗死、心绞痛，行经皮冠状动脉介入治疗，冠脉搭桥手术、脑出血、脑梗死等；e) 确诊为慢性肾脏病3期或需要接受肾脏透析治疗者。 24、给药前再次评估目标关节VAS疼痛评分<50mm。 更多项目信息请参考： http://www.chinadrugtrials.org.cn/index.html 温馨提示：文章入排信息来源于国家临床试验官网公示信息，具体情况以实际咨询为准！ 报名方式 参与临床试验，获取国际前沿新药使用机会！更有一线专家团队为你的治疗保驾护航！ 【印塔健康】目前有乙肝、脂肪肝、银屑病、特应性皮炎、类风湿性关节炎、强直、高血压、高血脂、糖尿病、贫血、阿尔茨海默症、溃疡性结肠炎、红斑狼疮、哮喘、青光眼、黄斑变性、慢阻肺、间质性肺病、肺纤维化......等慢病临床试验正在招募患者！ 扫描下方二维码添加医学运营一对一咨询或点击左下角“阅读原文”直接报名！我们将会在24小时内（工作日）与您电话或微信联系，请保持手机畅通！ End 【重要提示】印塔健康旗下公众号【家属说慢病新药新疗法资讯】所有文章信息仅供参考，具体治疗谨遵医嘱！ RECOMMEND 推荐阅读 1 ABP-745招募急性痛风性关节炎 2 URAT1抑制剂ABP-671招募痛风 3 URAT1抑制剂XNW3009招募痛风

-2026 is poised to be a data- and milestone-rich year across the portfolio- -CASGEVY® launch acceleration continues, supporting multi-billion-dollar potential- -Broad pipeline supported by leading gene-editing and siRNA platforms across cardiovascular, autoimmune, oncology and rare diseases- -Starting 2026 with a strong balance sheet with approximately $2 billion in cash, cash equivalents, and marketable securities- ZUG, Switzerland and BOSTON, Jan. 12, 2026 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today highlighted its strategic priorities and anticipated 2026 milestones. "Entering 2026, CRISPR Therapeutics is well positioned, with CASGEVY® gaining momentum, and multiple programs with encouraging data advancing rapidly through clinical trials across a diverse set of therapeutic areas,” said Samarth Kulkarni, Ph.D., Chairman and Chief Executive Officer of CRISPR Therapeutics. “Across the portfolio, clinical data demonstrated the transformative potential of CTX310™ for patients suffering from severe cardiovascular disease, as well as zugo-cel in oncology and autoimmune diseases. We also added a siRNA pillar through our partnership with Sirius and are advancing our lead siRNA asset, CTX611™ targeting Factor XI through the clinic. Beyond these clinical stage programs, we have multiple additional assets in preclinical development that leverage our best-in-class advanced editing platform. Together, our broad portfolio, strong balance sheet and effective operating model reinforce our confidence as we move into the next phase." Anticipated Key Milestones in 2026CRISPR Therapeutics anticipates several key milestones across its portfolio: Continued global commercialization and adoption of CASGEVY, with ongoing quarterly updates.Global regulatory submissions for CASGEVY in patients ages 5 – 11 are expected to be initiated by Vertex in the first half of 2026.Updates from CTX310® are expected in the second half of 2026.Updates from the Lp(a) program are expected in 2026.Top-line Phase 2 clinical data from CTX611 in patients undergoing total knee arthroplasty (TKA) are expected in the second half of 2026.Updates across autoimmune disease and immuno-oncology for zugocabtagene geleucel (zugo-cel; formerly CTX112™) are expected in the second half of 2026.The Company expects to initiate a clinical trial for CTX460 in alpha-1 antitrypsin deficiency (AATD) in mid-2026.The Company expects to initiate a clinical trial for CTX340 in refractory hypertension in the first half of 2026. Portfolio Highlights and Outlook Hemoglobinopathies and CASGEVY (exagamglogene autotemcel [exa-cel]) CASGEVY now approved in the U.S., the United Kingdom, the EU, the Kingdom of Saudi Arabia, the Kingdom of Bahrain, Qatar, Canada, Switzerland, the United Arab Emirates, and Kuwait for patients 12 years and older with sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT).CASGEVY exceeded $100M in revenue in 2025, reflecting more than 60 patients receiving CASGEVY infusions.Achieved a nearly three-fold increase in patient initiations and first cell collections in 2025 compared to 2024.Regulatory submissions for CASGEVY in patients aged 5-11 years with SCD and TDT are expected in the first half of 2026.CASGEVY awarded a Commissioner’s National Priority Voucher for pediatric submissions in SCD and TDT, enabling accelerated regulatory review once submissions are complete.Continued development of next-generation conditioning approaches with the potential to expand addressable patient populations for SCD and TDT with CASGEVY.Continued development of a lipid nanoparticle (LNP) based in vivo approach for editing hematopoietic stem cells (HSCs), with the potential to address a broader patient population in SCD and TDT. In Vivo Liver Editing CRISPR Therapeutics continues to advance a diversified portfolio of in vivo gene editing programs leveraging its proprietary LNP delivery platform. In 2025, CRISPR Therapeutics presented data for CTX310, demonstrating deep and durable reductions of triglycerides (TG) and low-density lipoprotein (LDL) following a single-course intravenous infusion, with a well-tolerated safety profile. Based on the positive Phase 1 results, the Company has advanced CTX310, targeting angiopoietin-related protein 3 (ANGPTL3), into Phase 1b clinical trials, prioritizing development in severe hypertriglyceridemia (sHTG) and refractory hypercholesterolemia. The Company expects to provide an update in the second half of 2026.CTX320™, targeting LPA, has demonstrated reductions of up to 73% in the dose escalation phase of the clinical trial. In parallel, the Company is advancing a next-generation LPA program, CTX321™, incorporating an updated guide RNA that demonstrates approximately two-fold greater potency in preclinical testing, while utilizing the same LNP delivery system. CTX321 is currently in IND/CTA-enabling studies, with an Lp(a) program update expected in 2026.In addition to its clinical-stage programs, CRISPR Therapeutics continues to advance several preclinical in vivo gene editing candidates, including: CTX460™, targeting SERPINA1 for the treatment of alpha-1 antitrypsin deficiency (AATD), is the first investigational candidate to emerge from the Company’s SyNTase™ editing platform. The Company expects to initiate a clinical trial for CTX460 in mid-2026.CTX340™, targeting angiotensinogen (AGT) for refractory hypertension, is currently in IND/CTA-enabling studies. The Company expects to initiate a clinical trial for CTX340 in the first half of 2026. siRNA-based Programs CRISPR Therapeutics’ small interfering RNA (siRNA)-based portfolio includes clinical-stage programs in cardiovascular and thromboembolic diseases, developed in collaboration with Sirius Therapeutics. CTX611 (SRSD107), a long-acting siRNA targeting Factor XI (FXI), is in an ongoing Phase 2 clinical trial in patients undergoing total knee arthroplasty (TKA). The Company expects to provide an update in the second half of 2026.CRISPR Therapeutics will lead global Phase 3 development of CTX611, excluding Greater China. The program targets a range of thromboembolic and clotting-related indications and represents a multi-billion-dollar market opportunity, including arterial fibrillation (AF), venous thromboembolism (VTE), ischemic stroke, cancer-associated thrombosis (CAT), chronic kidney disease (CKD), peripheral vascular disease (PVD), chronic coronary artery disease (CAD). Autoimmune Disease and Immuno-OncologyZugocabtagene geleucel (zugo-cel; formerly CTX112™) continues to advance in both autoimmune disease and hematologic malignancies. In autoimmune disease, Phase 1 clinical trials are ongoing across multiple indications, including systemic lupus erythematosus (SLE), systemic sclerosis (SSc), and inflammatory myositis and a second Phase 1 trial in immune thrombocytopenia purpura (ITP) and warm autoimmune hemolytic anemia (wAIHA). The first patient with SLE, refractory to 9 prior therapies with a baseline Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2K) score of 8, has maintained drug-free DORIS clinical remission through month 9 following CAR-T therapy. The second SLE patient with a baseline SLEDAI-2K score of 8, has sustained B cell depletion with SLEDAI-2K score of 0 through month 2 following CAR-T therapy. The Company expects to provide updates in the second half of 2026.The Phase 1/2 clinical trial of zugo-cel in B-cell malignancies is ongoing. The Company expects to provide updates in the second half of 2026. CRISPR Therapeutics has also established a collaboration and clinical supply agreement with Lilly to evaluate zugo-cel together with pirtobrutinib in aggressive B-cell lymphomas, further expanding the program’s development in oncology. Regenerative MedicineCRISPR Therapeutics continues to advance its regenerative medicine portfolio, including its efforts in diabetes. Clinical data generated from CTX211™ were promising, demonstrating detectable C-peptide levels 12 months after implantation. These data have informed the Company’s approach to hypoimmune cell engineering, supporting a transition to a next-generation candidate, CTX213™. CTX213 has demonstrated compelling preclinical efficacy and is progressing towards the clinic. The Company expects to provide additional updates as development progresses. About CASGEVY® (exagamglogene autotemcel [exa-cel]) CASGEVY® is a non-viral, ex vivo 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 BCL11A 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 VOCs for patients with SCD and transfusion requirements for patients with TDT. CASGEVY is approved for eligible SCD and TDT patients 12 years and older by multiple regulatory bodies around the world. About the CRISPR Therapeutics - Vertex Collaboration for CASGEVY CRISPR Therapeutics and Vertex established a strategic research collaboration in 2015 to discover and develop therapies using CRISPR/Cas9 technology to address the underlying genetic causes of human disease. CASGEVY is the first approved therapy to emerge from this collaboration. Under an amended collaboration agreement, Vertex leads global development, manufacturing, and commercialization of CASGEVY and shares program costs and profits worldwide 60/40 with CRISPR Therapeutics. Vertex is the manufacturer and exclusive license holder of CASGEVY. About In Vivo Programs CRISPR Therapeutics has established a proprietary lipid nanoparticle (LNP) delivery platform to enable gene editing in the liver using both CRISPR/Cas9 and its novel, proprietary SyNTase™ editing technologies. The Company’s in vivo portfolio includes its lead investigational programs, CTX310 (directed towards angiopoietin-related protein 3 (ANGPTL3)) and CTX320 (directed towards 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 being developed for patients with heterozygous familial hypercholesterolemia, homozygous familial hypercholesterolemia, mixed dyslipidemias, or severe hypertriglyceridemia, and in patients with elevated lipoprotein(a), respectively. In addition, the Company’s research and preclinical development candidates include: CTX460™, targeting SERPINA1 for the treatment of alpha-1 antitrypsin deficiency (AATD), CTX340™, targeting AGT for the treatment of refractory hypertension and CTX321, targeting LPA for the treatment of patients with elevated lipoprotein(a). About CTX611 (SRSD107) CTX611 is a novel double-stranded siRNA, designed to target the human coagulation FXI messenger RNA and inhibit FXI protein expression. Through modulation of the intrinsic coagulation pathway, CTX611 is intended to provide anticoagulant and antithrombotic effects. Supported by clinical experience to date, CTX611 is being developed as a long-acting FXI inhibitor with the potential to support infrequent, including semi-annual, subcutaneous administration. About Zugocabtagene Geleucel (zugo-cel; formerly CTX112) Zugocabtagene geleucel (zugo-cel) is a wholly-owned, allogeneic chimeric antigen receptor (CAR) T cell therapy product candidate targeting Cluster of Differentiation 19 (CD19), in development for both autoimmune and immuno-oncology indications. Zugo-cel is an off-the-shelf allogeneic CAR-T that utilizes CRISPR Cas9 for targeted gene knockout and CAR insertion for immune evasion and enhanced T effector cell potency. Zugo-cel is given following a standard lymphodepletion regimen without the need for HLA matching. Zugo-cel is being investigated in ongoing clinical trials in adult patients with systemic lupus erythematosus, systemic sclerosis, and inflammatory myositis and in adult patients with relapsed or refractory B-cell malignancies. About the CRISPR Therapeutics and Sirius Therapeutics Collaboration CRISPR Therapeutics and Sirius Therapeutics entered into a strategic collaboration in 2025 to develop and commercialize novel small interfering RNA (siRNA) therapies for thromboembolic disorders and other serious diseases. The lead program, CTX611, is a long-acting siRNA targeting FXI. Under the agreement, the companies will co-develop CTX611 and share costs and profits equally. CRISPR Therapeutics will lead commercialization in the U.S., while Sirius will lead in Greater China. The collaboration also provides CRISPR Therapeutics with the option to license up to two additional siRNA programs. About CRISPR Therapeutics Founded over a decade ago, CRISPR Therapeutics is a leading gene editing company focused on developing transformative medicines for serious diseases. The Company has evolved from a pioneering research-stage organization into an industry leader, marking a historic milestone with the approval of CASGEVY® (exagamglogene autotemcel [exa-cel]), the world’s first CRISPR-based therapy, approved for eligible patients with sickle cell disease and transfusion-dependent beta thalassemia. CRISPR Therapeutics is advancing a broad and diversified pipeline across hemoglobinopathies, oncology, regenerative medicine, cardiovascular and autoimmune, and rare diseases. The Company continues to expand its leadership in gene editing through the development of SyNTase™ editing, a novel and proprietary gene-editing platform designed to enable precise, efficient, and scalable gene correction. To accelerate and expand its impact, CRISPR Therapeutics has established strategic collaborations with leading biopharmaceutical partners, 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. To learn more, visit www.crisprtx.com.CRISPR THERAPEUTICS® standard character mark and design logo, SyNTase™, CTX112™, CTX211™, CTX213™, CTX310®, CTX320™, CTX321™, CTX340™, CTX460™ and CTX611™ are trademarks and registered trademarks of CRISPR Therapeutics AG. 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. CRISPR Special Note Regarding Forward-Looking Statements 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. 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, 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) CRISPR Therapeutics 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 regulatory agency decisions; (vi) the expected benefits of its collaborations; and (vii) the therapeutic value, development, and commercial potential of gene editing technologies and therapies, including CRISPR/Cas9 and SyNTase, as well as other technologies. 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. 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. 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. 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. 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. This press release discusses 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. Investor Contact: +1-617-307-7503 ir@crisprtx.com Media Contact: +1-617-315-4493 media@crisprtx.com