December has been a big month for CRISPR Therapeutics (CRSP -9.01%). On Dec. 8, the Food and Drug Administration (FDA) granted approval for CRISPR's gene-editing therapy, Casgevy, as a treatment for sickle cell disease. It was a monumental decision as it also marked the first approval for a therapy utilizing the CRISPR/Cas9 gene-editing technology.Despite this positive news, shares of CRISPR Therapeutics have stalled in December. In fact, the stock is down since the news of the approval, and month to date i ...

"The thrill of victory and the agony of defeat."That phrase became well-known years ago thanks to sportscaster Jim McKay. And it pretty much sums up the different trajectories for CRISPR Therapeutics (CRSP -9.01%) and Pfizer (PFE -1.88%) in 2023. CRISPR Therapeutics has enjoyed the thrill of victory, while Pfizer has experienced the agony of defeat.But which of these two biopharmaceutical stocks is the better pick for a new bull market? Motley Fool contributors Adria Cimino and Keith Speights make the argum ...

Not applicable. Item 4. Mine Safety Disclosures. Not applicable. Item 5. Other Information. On September 7, 2023, Dr. Phuong Khanh Morrow, our Chief Medical Officer, adopted a trading arrangement intended to satisfy the affirmative defense conditions of Securities Exchange Act Rule 10b5-1(c) (a "Rule 10b5-1 trading arrangement") with respect to the sale of up to an aggregate of 94,699 common shares of the Company pursuant to the terms of such trading plan, inclusive of common shares that will be mandated to ...

Summary of CRISPR Therapeutics AG Conference Call Company Overview - **Company**: CRISPR Therapeutics AG (NASDAQ: CRSP) - **Event**: Citi's 18th Annual BioPharma Conference Call - **Date**: September 6, 2023 - **Participants**: - Sam Kulkarni - CEO - Yigal Nochomovitz - Citigroup Analyst Key Points Industry and Company Mission - CRISPR Therapeutics is on the verge of potentially launching the first CRISPR-based medicine, exa-cel, later in 2023, having dosed over 200 patients across various programs [4][5] - The company aims to expand its reach to treat multiple diseases, aspiring to become a $25 billion company in the next three to four years [5][6] Exa-cel and Market Strategy - Exa-cel is targeted at hemoglobinopathies, with PDUFA dates set for December 1 and March [8] - The company is preparing for an Advisory Committee meeting, anticipating standard questions around safety and efficacy [9][10] - The partnership with Vertex is crucial for commercialization, leveraging Vertex's experience in rare diseases [16][33] Market Dynamics and Patient Demand - There is a strong unmet need for therapies in sickle cell disease, with a significant number of patients willing to undergo transplant processes for transformative therapies like exa-cel [21] - The company expects a strong uptake once the drug is launched, with many treatment centers already familiar with the process [20] Competitive Landscape - CRISPR Therapeutics is optimistic about its market share in sickle cell disease, noting a preference among patients for CRISPR-based therapies over other modalities [25] - The company acknowledges competition but emphasizes the safety and efficacy of its CRISPR approach [25] Future Developments - The company is exploring a gentler conditioning regimen that could expand the market for exa-cel from 25,000 severe sickle cell patients to potentially 50,000 or more [30][31] - CRISPR Therapeutics has a 60-40 profit-sharing agreement with Vertex, with milestones tied to the approval of exa-cel [33] Immuno-Oncology Programs - The company is advancing its immuno-oncology platform, particularly with CTX110 and CTX112, which are designed to improve CAR-T cell therapies [36][37] - CTX112 is expected to be significantly more potent than CTX110, with early data showing promising expansion rates [39][40] Diabetes Regenerative Medicine - CRISPR Therapeutics is collaborating with Vertex on diabetes therapies, focusing on editing induced pluripotent stem cells (iPSCs) to create immune-stealth pancreatic cells [59][60] - The company is optimistic about the potential of its VCTX211 program, which aims to eliminate the need for immunosuppression in Type 1 diabetes patients [60][61] In Vivo Programs - The company plans to enter clinical trials for its ANGPTL3 and Lp(a) programs, leveraging advancements in CRISPR delivery to the liver [74][75] - ANGPTL3 targets a rare disease population, while Lp(a) has a broader market potential, with 11 million patients in the U.S. alone [77] Conclusion - CRISPR Therapeutics is positioned to make significant advancements in gene editing therapies, with a strong focus on commercialization, patient needs, and expanding its treatment capabilities across various diseases [5][21][25]

☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2023 For the transition period from to . Switzerland Not Applicable (State or other jurisdiction of incorporation or organization) (I.R.S. Employer Identification No.) Accelerated filer☐ ☐ If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standard ...

CRISPR Therapeutics AG (NASDAQ:CRSP) JMP Securities Life Sciences Conference May 15, 2023 11:30 AM ET Company Participants Sam Kulkarni - CEO Conference Call Participants Silvan Türkcan - JMP Securities Silvan Türkcan Thanks. Welcome back. My name is Silvan Türkcan, a senior analyst for JMP Securities. I cover precision medicines. It’s my pleasure to host Sam Kulkarni, CEO of CRISPR Therapeutics. Thanks for joining us today. Sam Kulkarni Thank you, Silvan, for having us. Question-and-Answer Session Q - Silv ...

Financial Data and Key Metrics Changes - The company has a strong balance sheet that supports multiple clinical programs running in parallel, currently having 7 clinical trials with plans to expand to double-digit trials [5] - The addressable market for exa-cel is estimated at $50 billion, based on a potential pricing of $2 million per patient and a patient population of 25,000 in the U.S. [27] Business Line Data and Key Metrics Changes - The company is transitioning from ex vivo to in vivo programs, with expectations to bring new forms of editing to the clinic within the next two years [4] - The company is focusing on developing next-generation CAR-T therapies, with CTX110 and CTX130 in registrational trials and CTX131 targeting solid tumors [47][48] Market Data and Key Metrics Changes - The company sees significant unmet needs in the sickle cell space, especially after the failure of a confirmatory trial for crizanlizumab, positioning exa-cel as a critical option for patients [9] - There is a growing interest in the company's next-gen CAR-T therapies, with increasing inquiries from pharmaceutical companies and investigators [38] Company Strategy and Development Direction - The company aims to become a leader in gene editing, aspiring to be the next Genentech in the biotech space [4] - The strategy includes optimizing manufacturing processes for both autologous and allogeneic therapies to enhance scalability and reduce costs [19][54] Management's Comments on Operating Environment and Future Outlook - Management expresses confidence in regulatory support from agencies like the FDA and EMA for gene-editing therapies, highlighting a deterministic mechanism of action with CRISPR-Cas9 [7][9] - The company is optimistic about the potential of gentler conditioning agents to expand the patient population eligible for therapies, which could significantly increase market penetration [28] Other Important Information - The company is developing in vivo programs targeting ANGPTL3 and LPA, which are expected to have a meaningful impact on cardiovascular health [55] - The partnership with Vertex is crucial for commercial launch strategies, with Vertex taking the lead on pricing and reimbursement discussions [25] Q&A Session Summary Question: What is the current status of the regulatory environment for gene editing? - Management finds the FDA and other regulatory agencies supportive of gene editing, emphasizing the importance of consistent manufacturing processes [7][9] Question: How does CRISPR's manufacturing capacity compare for autologous versus allogeneic therapies? - The company has modular manufacturing facilities that allow for scalability, with autologous therapies producing one patient dose at a time, while allogeneic therapies can generate multiple doses simultaneously [18][19] Question: What are the competitive advantages of CRISPR's therapies compared to others in the market? - The deterministic nature of CRISPR's gene editing provides a significant advantage over viral-based therapies, which are perceived to have more variability and unknowns [8][32] Question: What is the company's approach to next-generation CAR-T therapies? - The company is rapidly advancing next-gen CAR-T therapies, leveraging learnings from first-generation products to improve efficacy and safety profiles [35][40] Question: How does CRISPR plan to address the challenges in solid tumors? - The company is focusing on next-gen edits to combat T-cell exhaustion in solid tumors, with promising data from ongoing studies [51][52]

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, 2023 or UNITED STATES SECURITIES AND EXCHANGE COMMISSION For the transition period from to . (State or other jurisdiction of incorporation or organization) Baarerstrasse 14 6300 Zug, Switzerland Not Applicable (Address of principal executive offices) (Zip Code) Switzerland Not Applicable (I.R.S. Employer Identification No.) +41 (0)41 561 32 77 (R ...

Jon Terrett Session Type: Drug Development Special Track Session Session Title: New Drugs on the Horizon: Part 1 Employee of: CRISPR Therapeutics Stockholder in: CRISPR Therapeutics CRISPR THERAPEUTICS® standard character mark and design logo, CTX001™, CTX110®, CTX112™, CTX121™, CTX130™, CTX131™, CTX310™, CTX320™, CTX330™, COBALT™, VCTX210™, VCTX211™ and VCTX212™ are trademarks and registered trademarks of CRISPR Therapeutics AG. All other trademarks and registered trademarks are the property oftheir respec ...

CRISPR Therapeutics AG (NASDAQ:CRSP) Cowen 43rd Annual Healthcare Conference Call March 6, 2023 10:30 AM ET Company Participants Sam Kulkarni - Chief Executive Officer Conference Call Participants Tyler Van Buren - Cowen Tyler Van Buren Good morning, everyone. Tyler Van Buren here senior biotech analyst at TD Cowen. Thank you very much for joining TD Cowen’s 43rd Annual Healthcare Conference. For next session, it’s a privilege to have CRISPR here and in particular, Sam Kulkarni, the CEO. Sam, thank you very ...