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 ...

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.

® © 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 ...

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 ...

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 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 ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 Title of each classTrading Symbol(s) Name of each exchange on which registered FORM 10-K (Mark One) ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2021 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 ...

Key Points Industry/Company * **Company**: CRISPR Therapeutics AG (NASDAQ:CRSP) * **Industry**: Biotechnology, Gene Editing Therapies Core Views and Arguments * **CRISPR Technology**: CRISPR Therapeutics is a leading developer of CRISPR Cas9 gene editing therapies. The technology allows for precise editing of DNA at the genome level, offering potential cures for various diseases. * **Pipeline**: The company has a robust pipeline with multiple programs targeting rare and common diseases. Key programs include: * **CTX001**: A gene-edited therapy for sickle cell disease and beta thalassemia, demonstrating transformative results in clinical trials. * **Allogeneic CAR-T Programs**: Three programs targeting CD19, BCMA, and CD70, showing promising results in early trials. * **Type 1 Diabetes Therapy**: A partnership with ViaCyte to develop a potentially curative therapy using CRISPR technology. * **In Vivo Gene Editing Programs**: Multiple programs using AAV or lipid nanoparticle delivery methods for various rare diseases. * **Partnerships**: CRISPR Therapeutics has formed strategic partnerships with companies like Vertex Pharmaceuticals and ViaCyte to advance its pipeline and expand its capabilities. Other Important Points * **Financials**: CRISPR Therapeutics has a strong financial position with over $2.5 billion in cash reserves, providing ample funding for its pipeline and operations. * **Regulatory Approval**: The company is actively working on regulatory submissions for CTX001 and other programs, aiming for approval and commercialization. * **Longevity**: CRISPR Therapeutics is focused on extending human longevity through its gene editing technologies, targeting diseases that significantly impact lifespan.

Summary of CRISPR Therapeutics Conference Call Company Overview - **Company**: CRISPR Therapeutics (NASDAQ: CRSP) - **Event**: 4th Annual Evercore ISI HealthCONx Conference - **Date**: December 1, 2021 - **Participants**: Samarth Kulkarni (CEO), Liisa Bayko (Evercore ISI) Key Points Industry and Technology - CRISPR technology is evolving rapidly, with various strategies being employed by different companies in the biotech space [2][3] - CRISPR Therapeutics has chosen to focus on both ex vivo and in vivo approaches, prioritizing ex vivo for initial product development [3][4] Strategic Focus - The company aims to create self-sustaining business franchises, starting with therapies for sickle cell disease and thalassemia, which are expected to be transformative for patients [3][4] - Future franchises include smart cells in oncology, regenerative medicine, and in vivo approaches for both rare and common diseases [4][6] Market Potential - The company has set ambitious goals, aiming to become a $75 billion to $100 billion company within the next five to seven years, contingent on the success of its product development strategies [7] Regulatory and Clinical Development - CRISPR Therapeutics plans to file for regulatory approval by the end of next year, with ongoing discussions with regulators to meet clinical and nonclinical requirements [9][10] - The company is currently in pivotal trials for its second franchise and has activated its third franchise for Type 1 diabetes [7][9] Commercialization Challenges - The commercialization of CRISPR therapies is likened to medical devices rather than traditional pharmaceuticals, requiring a different approach to market entry and patient access [11][12] - Initial patient access may be limited due to the complexity and cost of procedures, but growth is expected as more physicians gain experience [12][14] Competitive Landscape - The company acknowledges the competitive environment but believes that early mover advantages and the unique nature of their therapies will help maintain market share [15][21] - The FDA's RMAT designation for their allogeneic CAR-T therapy indicates regulatory support, which may provide a competitive edge [17][18] Product Development Insights - The company is focused on improving its CAR-T therapies, with ongoing development of GEN2 and GEN3 versions that aim to enhance efficacy and safety [25] - The potential for combining CAR-T and CAR NK therapies is recognized, leveraging the strengths of both modalities [27] Future Outlook - Key value inflection points for 2022 include advancements in the Type 1 diabetes program, pivotal trial data for CD19, and progress in in vivo programs [33][34] - The company is optimistic about its capabilities and the potential for significant breakthroughs in its clinical programs [34] Additional Considerations - The company emphasizes the importance of pharmacoeconomics and the transformative value of its therapies for underserved patient populations [14][15] - The CEO highlighted the need for a shift in investor perception regarding allogeneic therapies, advocating for a more nuanced understanding of their potential benefits [18][19] This summary encapsulates the critical insights and strategic directions discussed during the conference call, providing a comprehensive overview of CRISPR Therapeutics' current status and future aspirations in the biotech industry.

CRISPR THERAPEUTICS ® © 2021 CRISPR Therapeutics 1 Creating transformative gene-based medicines for serious diseases Corporate Overview | Q4 2021 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 ...