CRISPR Therapeutics(CRSP)

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Citi's 17th Annual BioPharma Conference Company Conference Presentation
2022-09-08 18:04
© 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 (CRSP) Citi 17th Annual BioPharma Conference Transcript
2022-09-07 18:29
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 ...
CRISPR Therapeutics(CRSP) - 2022 Q2 - Quarterly Report
2022-08-07 16:00
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 ...
CRISPR Therapeutics AG (CRSP) Management Presents at Innovation Day Conference (Transcript)
2022-06-22 15:40
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]
CRISPR Therapeutics (CRSP) Presents at the 27th Congress of the European Hematology Association (EHA) - Slideshow
2022-06-19 18:09
Efficacy and Safety of a Single Dose of Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease Franco Locatelli, 1 Haydar Frangoul,2 Selim Corbacioglu,3 Josu de la Fuente,4 Donna Wall,5 Maria Domenica Cappellini,6 Mariane de Montalembert,7 Antonis Kattamis,8 Stephan Lobitz,9 Damiano Rondelli,10 Sujit Sheth,11 Martin Steinberg,12 Mark C. Walters,13 Yael Bobruff,14 Chris Simard,14 Yang Song,14 Lanju Zhang,14 Anjali Sharma,15 Suzan Imren,14 Bill Hobbs,14 Stephan Grupp16 ...
CRISPR Therapeutics AG (CRSP) Management Presents at Goldman Sachs 43rd Annual Global Healthcare Conference (Transcript)
2022-06-15 21:09
Summary of CRISPR Therapeutics AG Conference Call Company Overview - **Company**: CRISPR Therapeutics AG (NASDAQ: CRSP) - **Event**: Goldman Sachs 43rd Annual Global Healthcare Conference - **Date**: June 15, 2022 - **Participants**: Lawrence Klein (COO), Salveen Richter (Goldman Sachs) Key Points Industry Focus - **Core Technology**: CRISPR editing technology - **Main Areas of Focus**: - Ex vivo applications (hemoglobinopathies, rare diseases) - Oncology (allogeneic CAR-T therapies) - Regenerative medicine (beta cell replacement for type 1 diabetes) - In vivo applications [3][4] Clinical Programs and Updates - **Clinical Programs**: - CTX110 (CD19), CTX120 (BCMA), CTX130 (CD70) - Data expected by the end of the month for CTX120 and CTX130 [5][6] - **Durable Responses**: Achieved durable remissions with a single dose of allogeneic cell therapy [7] - **Competitive Dynamics**: Acknowledgment of competitive fields, with ongoing efforts to improve immunovasion and potency [8] Financial Position - **Cash Balance**: Approximately $2.2 billion, providing multiple years of runway [9] - **Capital Allocation**: Majority of capital directed towards clinical programs, with oncology being the largest area of expenditure [10] In Vivo Programs - **Strategic Decision**: Initial focus on ex vivo programs due to lower technical hurdles; in vivo programs are now accelerating [12][13] - **Potential for Approval**: Exa-Cel (formerly CTX001) on track for approval, with significant clinical benefits observed [16] Regulatory and Clinical Strategy - **Regulatory Submission Timeline**: Confidence in submitting for regulatory approval by year-end, with ongoing discussions with the FDA regarding patient follow-up duration [17][18] - **Endpoint Adjustments**: Shifted from transfusion reduction to transfusion independence as primary endpoints based on clinical results [19] Competitive Landscape - **Comparison with Other Therapies**: Observations on the competitive landscape, particularly regarding autologous therapies and the need for off-the-shelf solutions [30][31] - **Differentiation Strategy**: Focus on beta2M edit for immune evasion, aiming to avoid harsher lymphodepletion [30] Regenerative Medicine - **Type 1 Diabetes Program**: Ongoing clinical trials with a focus on safety and immunoagent trials, aiming for unencapsulated, injectable cell therapies [45][46] Intellectual Property (IP) Status - **IP Landscape**: Strong position in ex-U.S. markets; ongoing appeal regarding U.S. patents held by Broad Institute [52][54] Future Outlook - **In Vivo Pipeline**: Anticipation of unveiling significant progress in in vivo programs at the upcoming Innovation Day [56] Additional Insights - **Patient Population**: Approximately 30,000 patients identified for current standard of care conditioning in beta thalassemia and sickle cell disease [26] - **Innovation in Preconditioning**: Ongoing efforts to develop targeted conditioning regimens to enhance patient access [27] - **Market Need**: Recognition of the unmet need in T-cell lymphomas and the potential for new therapies [38] This summary encapsulates the critical insights and developments discussed during the conference call, highlighting the strategic direction and clinical advancements of CRISPR Therapeutics AG.
CRISPR Therapeutics (CRSP) Investor Presentation - Slideshow
2022-05-10 15:35
® © 2021 CRISPR Therapeutics 1 CRISPR Creating transformative gene-based medicines for serious diseases Corporate Overview | Q2 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) the safety, efficacy and clinical progress of our various clin ...
CRISPR Therapeutics(CRSP) - 2022 Q1 - Quarterly Report
2022-05-08 16:00
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 March 31, 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 jurisdict ...
CRISPR Therapeutics AG's (CRSP) CEO Sam Kulkarni Presents At Barclays Global Healthcare Conference (Transcript)
2022-03-17 16:17
Summary of CRISPR Therapeutics AG Conference Call Company Overview - **Company**: CRISPR Therapeutics AG (NASDAQ:CRSP) - **CEO**: Sam Kulkarni - **Event**: Barclays Global Healthcare Conference Call on March 17, 2022 - **Key Focus**: Development of CRISPR-based therapies, particularly in hemoglobinopathies, immuno-oncology, regenerative medicine, and in vivo gene editing [3][4][5] Core Points and Arguments Company Progress and Strategy - CRISPR Therapeutics has a diversified portfolio with five clinical programs, aiming to be the first to file a Biologics License Application (BLA) for a CRISPR-edited product, specifically CTX001 for sickle cell disease and beta thalassemia [3][4] - The company has dosed over 150 patients, gaining significant clinical experience [4] - Strong financial position with over $2.3 billion in cash, allowing aggressive investment in capabilities during a biotech market downturn [6][7] CTX001 Program - CTX001 involves editing bone marrow stem cells to elevate fetal hemoglobin, addressing deficiencies in beta-globin [9][10] - Initial data from 15 thalassemia and 7 sickle cell patients showed remarkable outcomes, with patients experiencing significant improvements in their conditions [10][11] - The company is in alignment with regulators regarding the filing package and aims to submit the BLA by the end of the year [13][19] Pricing and Market Considerations - Pricing negotiations for therapies in Europe are complex, with significant differences between proposed prices and what health systems are willing to pay [22][23] - The pharmacoeconomic argument supports a high price point due to the substantial cost of care for patients with sickle cell disease [24][25] Allogeneic CAR-T Programs - CRISPR is developing allogeneic CAR-T therapies, with a focus on CD19 non-Hodgkin lymphoma, aiming for a pivotal trial that could lead to market approval [26][30] - The company believes that allogeneic CAR-Ts have greater improvement potential compared to autologous therapies and bispecifics [28][29] - CTX110 is positioned to fulfill an unmet need in the market, with a strong competitive position bolstered by recent developments in the competitive landscape [30][31] Future Developments - The company plans to provide updates on clinical datasets and timelines for filing as more data becomes available [18][19] - Focus on in vivo gene editing and regenerative medicine, with plans for an R&D day to disclose targets and pipeline breadth [41][42] Other Important Content - The company emphasizes the importance of collaboration with regulators to bring transformative therapies to patients [16][19] - There is a strong belief in the long-term value creation potential of their therapies, with expectations of sustained growth over several years [21][30] - The competitive landscape is evolving, and CRISPR Therapeutics is positioning itself to capitalize on these changes [30][31]
CRISPR Therapeutics' (CRSP) CEO Sam Kulkarni on Cowen 42nd Annual Health Care Conference (Transcript)
2022-03-07 20:27
CRISPR Therapeutics AG (NASDAQ:CRSP) Cowen 42nd Annual Health Care Conference March 7, 2022 10:30 AM ET Company Participants Sam Kulkarni – Chief Executive Officer Conference Call Participants Tyler Van Buren – Cowen Tyler Van Buren Good morning, everyone. And welcome to Cowen’s 42nd Annual Health Care Conference. My name is Tyler Van Buren. I’m a Senior Biotech Analyst here at Cowen. For this next session we have a fireside chat with CRISPR Therapeutics. It’s my pleasure to introduce Sam Kulkarni, the Chie ...