CRISPR Therapeutics AG (NASDAQ:CRSP) Citi’s 2023 Virtual Oncology Leadership Summit Conference February 22, 2023 1:00 PM ET Company Participants Sam Kulkarni - CEO Conference Call Participants Yigal Nochomovitz - Citi Yigal Nochomovitz All right. Great. Welcome, everyone. I'm Yigal Nochomovitz, one of the biotech analysts here at Citi. This is the second day of our Virtual Oncology Leadership Summit. So it's my pleasure to introduce the CEO of CRISPR Therapeutics, Sam Kulkarni. Sam, welcome. Thank you so mu ...

Item 11. Executive Compensation. The information required by this item is incorporated by reference to our Proxy Statement for our 2023 Annual General Meeting of Shareholders to be filed with the SEC within 120 days after the end of the fiscal year ended December 31, 2022. Item 12. Security Ownership of Certain Beneficial Owners and Management and Related Stockholder Matters. The information required by this item is incorporated by reference to our Proxy Statement for our 2023 Annual Meeting of Stockholders ...

CRISPR Therapeutics AG (NASDAQ:CRSP) SVB Securities Global Biopharma Conference Call February 15, 2023 1:40 PM ET Company Participants Sam Kulkarni - Chief Executive Officer Conference Call Participants Mani Foroohar - SVB Securities Mani Foroohar Good afternoon and good morning to those still on the West Coast. Welcome to this next session of our Annual Global Biopharma Conference here at SVB Securities. I am Mani Foroohar, senior analyst, exec medicines team. And I am very fortunate to be hosting Sam Kulk ...

Exa-cel Program - Exa-cel 有潜力成为首个获批的 CRISPR 药物，Vertex 和 CRISPR 正在联合准备在 2022 年第四季度提交 BLA/MAA 申请[9] - 在针对输血依赖型地中海贫血 (TDT) 的研究中，42/44 (95%) 的患者停止了红细胞输血，持续时间从 0.8 个月到 36.2 个月不等[10] - 在镰状细胞病 (SCD) 患者中，31/31 (100%) 的患者没有发生血管闭塞危象 (VOC)，持续时间从 2.0 个月到 32.3 个月不等[10] - Exa-cel 有巨大的潜在市场，如果获得批准，可以覆盖美国和欧盟超过 3 万名患有严重 SCD 和 β-地中海贫血的患者[9] Immuno-Oncology Pipeline - CRISPR Therapeutics 在 4 项试验中对超过 100 名患者使用了 CRISPR 编辑的 CAR-T 细胞，实现了同种异体 CAR-T 的概念验证[5] - CTX110 的潜在注册试验正在进行中[13] - CTX130 在 T 细胞淋巴瘤中显示出积极数据，并在实体瘤中首次显示出有意义的活性[13] - 在 DL3 及以上剂量水平下，CTX130 的总缓解率 (ORR) 为 70%，完全缓解率 (CR) 为 30%[43] In Vivo Platform - 公司已建立用于体内基因敲除的即插即用 LNP/mRNA 平台，从肝脏开始[69] - 在非人灵长类动物 (NHP) 中，剂量依赖性肝脏编辑高达 70%[74] - CTX310 项目在非人灵长类动物中使血清 ANGPTL3 蛋白降低约 90%[77] - CTX320 项目在非人灵长类动物中使血清 Lp(a) 降低超过 90%[81] Financial Status - 公司拥有约 20 亿美元的现金储备[89]

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2022 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to . Commission File Number: 001-37923 CRISPR THERAPEUTICS AG (Exact name of registrant as specified in its charter) Switzerland Not Applicable (State or other juris ...

CRISPR Therapeutics AG (NASDAQ:CRSP) Morgan Stanley 20th Annual Global Healthcare Conference Call September 12, 2022 11:40 AM ET Company Participants Samarth Kulkarni - Chief Executive Officer Conference Call Participants Terence Flynn - Morgan Stanley Terence Flynn Okay, great. Thanks for joining us, everybody. I’m Terence Flynn, Morgan Stanley, pharma and biotech analyst, and we’re very pleased to be hosting CRISPR Therapeutics this morning. First, for important disclosures, please see the Morgan Stanley ...

© 2022 CRISPR Therapeutics 1 Creating transformative gene-based medicines for serious diseases Corporate Overview | Q3 2022 Forward-Looking Statements The presentation and other related materials may contain a number of "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding CRISPR Therapeutics' expectations about any or all of the following: (i) its plans and expectations for its preclinical studies, clinical trials ...

CRISPR Therapeutics AG (NASDAQ:CRSP) Citi 17th Annual BioPharma Conference September 7, 2022 8:50 AM ET Company Participants Sam Kulkarni – Chief Executive Officer Conference Call Participants Yigal Nochomovitz – Citi Yigal Nochomovitz All right, well, welcome everyone. I’m Yigal Nochomovitz, one of the biotech analysts here at Citi. It's my great pleasure and privilege to have with me, the CEO of CRISPR Therapeutics, Sam Kulkarni. Welcome. Sam Kulkarni Thank you, Yigal for having us. Yigal Nochomovitz Grea ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2022 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to . Commission File Number: 001-37923 CRISPR THERAPEUTICS AG (Exact name of registrant as specified in its charter) Switzerland Not Applicable (State or other jurisdicti ...

Summary of CRISPR Therapeutics AG Innovation Day Conference Call Company Overview - **Company**: CRISPR Therapeutics AG (NASDAQ: CRSP) - **Date**: June 21, 2022 - **Key Participants**: - Samarth Kulkarni (CEO) - Phuong Khanh Morrow (CMO) - Sumanta Kumar Pal (Co-Director, Kidney Cancer Program) - Swaminathan Iyer (Professor, Lymphoma/Myeloma) - Alireza Rezania (SVP, Head of Regenerative Medicine) Core Industry Focus - **Industry**: Gene Editing and Biotechnology - **Key Areas**: Hemoglobinopathies, Immuno-oncology, Regenerative Medicine, In Vivo Therapies Key Points and Arguments Company Progress and Financials - CRISPR Therapeutics has made significant advancements in gene editing, with over 500 employees and a state-of-the-art GMP facility [6] - The company has over $2 billion in its balance sheet, positioning it as a leader in the CRISPR-based revolution [6] - The company is on the verge of filing a Biologics License Application (BLA) for CTX001 (exa-cel) for hemoglobinopathies, which could be the first CRISPR-edited product approved globally [5][6] Hemoglobinopathies - **Exa-cel**: A gene therapy targeting sickle cell disease and beta-thalassemia, showing promising results: - 42 out of 44 patients with beta-thalassemia became transfusion independent [10] - All patients with sickle cell disease were free of vaso-occlusive crises [10] - The therapy could potentially address around 30,000 patients with these conditions, with plans to expand its reach through targeted conditioning and in vivo delivery [12] - Development of a cKit antibody drug conjugate (ADC) is underway to enhance gene editing delivery [13] Immuno-oncology - **CTX110**: An allogeneic CAR-T therapy targeting CD19, with ongoing patient dosing and optimization [18] - **CTX120**: A BCMA-targeting CAR-T therapy, pivoting to a next-generation program due to competitive landscape and efficacy concerns [19] - **CTX130**: A novel allogeneic CAR-T therapy targeting CD70, with unique edits to enhance efficacy and reduce toxicity: - High CD70 expression in T-cell lymphomas presents a significant unmet need [22][24] - Early data shows a 70% overall response rate and a 30% complete response rate in T-cell lymphoma patients [31][35] Safety and Efficacy Data - CTX130 demonstrated a favorable safety profile with no severe adverse events related to the therapy [29][40] - The efficacy data from the COBALT lymphoma trial showed promising responses across various disease sites, with a 90% disease control rate [31][32] Future Directions - Plans to develop next-generation CAR-T therapies (CTX131) with additional potency edits [35][53] - Ongoing collaborations with academic centers for novel targets beyond CD70, including CD83 and Glypican-3 [56] - The company aims to leverage its CRISPR platform for further innovations in gene editing and therapy development [57] Market Potential - The company believes that the introduction of therapies like Axi-Cel will expand the patient population and improve access to treatments for conditions like sickle cell disease [60] - The predictability and reliability of CRISPR manufacturing provide a competitive advantage over traditional viral methods [64] Additional Important Content - The call emphasized the importance of continuous innovation in gene editing and the potential for CRISPR technology to transform medicine [6][11] - The company is committed to addressing significant unmet medical needs in oncology and hematology through its innovative therapies [24][37]