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 ...

CRISPR Therapeutics AG (NASDAQ:CRSP) Barclays Global Healthcare Conference Call March 17, 2022 10:15 AM ET Company Participants Sam Kulkarni - Chief Executive Officer Conference Call Participants Gena Wang - Barclays Disclaimer*: This transcript is designed to be used alongside the freely available audio recording on this page. Timestamps within the transcript are designed to help you navigate the audio should the corresponding text be unclear. The machine-assisted output provided is partly edited and is de ...

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.

CRISPR Therapeutics (NASDAQ:CRSP) 4th Annual Evercore ISI HealthCONx Conference December 1, 2021 12:35 PM ET Company Participants Samarth Kulkarni – Chief Executive Officer Conference Call Participants Liisa Bayko – Evercore ISI Liisa Bayko Hi, good afternoon. This is Liisa Bayko, biotech analyst at Evercore ISI. I’m very pleased to be joined today by one of the leading CRISPR companies, CRISPR. Today, we have CEO, Sam joining me to discuss the company’s suite of products. Sam, I just want start off by a br ...

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 ...