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20260109 CRISPR Therapeutics - JPM 2026.pdf

上传人: 明**** 编号:1069657 2026-01-17 35页 4.23MB

1、CRISPR Corporate Update44thAnnual J.P.Morgan Healthcare ConferenceJanuary 12,20261 2026 CRISPR TherapeuticsForward-Looking Statements2This presentation and related materials may contain statements regarding matters that are not historical facts and are“forward-looking statements”within the meaning o

2、f 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 regarding any or all

3、 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 gener

4、ally;(ii)data included in this presentation,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)reg

5、ulatory submissions and authorizations,including timelines for and expectations regarding regulatory agency decisions;(v)plans and expectations for the commercialization of,and anticipated benefits of,CASGEVY;(vi)the expected benefits of CRISPR Therapeutics collaborations;and(vii)the therapeutic val

6、ue,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 th

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1. **核心业务与愿景**:CRISPR Therapeutics致力于通过基因编辑技术开发治愈严重疾病的疗法,拥有四大治疗领域(血液病、CAR-T、自身免疫、心血管及罕见病)的多元化管线。 2. **商业化进展**:基因编辑疗法CASGEVY(CRISPR占40%分成)2025年营收超1亿美元,全球10国获批,75个治疗中心激活,患者治疗数显著增长(2026年预计超12倍)。 3. **关键临床数据**: - **CTX310**(心血管):I期显示ANGPTL3靶向治疗降低LDL达48.9%、甘油三酯55.2%,安全性良好,美国潜在患者超100万。 - **Zugo-cel**(CAR-T):在DLBCL中客观缓解率90%,完全缓解率70%;自身免疫病(如SLE)患者实现药物缓解。 - **CTX611**(抗凝):FXI活性降低超93%,潜在每6个月给药一次。 4. **技术平台**:推进体内造血干细胞编辑(HSC)、非病毒体内CAR-T及SyNTase编辑技术,2026年多个项目进入临床(如CTX340高血压、CTX460α-1抗胰蛋白酶缺乏症)。
**CASGEVY前景如何?** **基因编辑新突破?** **CAR-T疗法潜力大?**
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