Group 1: Stock Performance - CRISPR Therapeutics AG shares increased by 9.6% to close at $60.08, with notable trading volume compared to typical sessions [1] - The stock has gained 27.7% over the past four weeks, indicating strong investor interest [1] Group 2: Pipeline Developments - The rise in CRISPR's stock price is attributed to positive sentiment regarding its pipeline, particularly the in vivo candidate CTX310 targeting ANGPTL3 for atherosclerotic heart disease [2] - Updated data from an early-stage study showed a single dose of CTX310 resulted in peak reductions of up to 82% in triglyceride levels and up to 86% in LDL levels, generating excitement for other pipeline candidates [2] Group 3: Financial Expectations - The company is expected to report a quarterly loss of $1.54 per share, reflecting a year-over-year change of -3.4%, while revenues are projected at $5.89 million, a significant increase of 1032.9% from the previous year [3] - The consensus EPS estimate for the quarter has remained unchanged over the last 30 days, suggesting that stock price movements may be influenced by trends in earnings estimate revisions [4] Group 4: Industry Context - CRISPR Therapeutics is part of the Zacks Medical - Biomedical and Genetics industry, where Wave Life Sciences also operates, closing the last trading session 10.8% higher at $7.7 [4] - Wave Life Sciences has seen a consensus EPS estimate change of -6.6% over the past month, indicating a potential decline in performance compared to the previous year [5]

-New Phase 1 clinical data for CTX310™ continues to demonstrate dose-dependent reductions in triglycerides (TG) and low-density lipoprotein (LDL), with peak reduction of up to 82% in TG and up to 86% in LDL, with a well-tolerated safety profile- -Complete Phase 1 data presentation for CTX310 anticipated at a medical meeting in the second half of 2025- -Data update for CTX320™, targeting the LPA gene, now expected in the first half of 2026- -Preclinical in vivo cardiovascular program CTX340™ advancing toward ...

Key Takeaways CRSP is the first to market a CRISPR/Cas9 therapy, with Casgevy approved for SCD and TDT in multiple regions CRSP's pipeline spans ex vivo and in vivo therapies, including early success with CTX310. NTLA focuses on late-stage in vivo programs but faces setbacks, including a liver safety concern with nex-z.CRISPR Therapeutics (CRSP) and Intellia Therapeutics (NTLA) are leading developers of therapies that utilize the Nobel Prize-winning CRISPR/Cas9 gene editing technology. While CRSP is the f ...

Creating transformative gene-based medicines for serious diseases Corporate Overview Q2 2025 CRISPR THERAPEUTICS – CONFIDENTIAL © 2025 CRISPR Therapeutics | 11 Forward-Looking Statements Statements contained in this presentation and other related materials regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ material ...

Core Insights - CRISPR Therapeutics (CRSP) shares have increased by 14% over the past month, primarily due to positive results from its in vivo gene therapy candidates [1][10] Group 1: In Vivo Therapy Developments - CRSP reported promising initial results from its early-stage study on CTX310, a CRISPR-based gene therapy targeting ANGPTL3 for atherosclerotic heart disease, showing peak reductions of up to 82% in triglyceride levels and 81% in low-density lipoprotein levels [2][3] - The success of CTX310 has generated excitement for another in vivo candidate, CTX320, which targets lipoprotein(a) and is expected to release initial data by the end of the month [4] Group 2: Casgevy and Market Position - Casgevy, CRSP's approved ex vivo gene therapy for sickle cell disease and transfusion-dependent beta-thalassemia, has seen a steady uptake post-launch, with $14.2 million in product revenues recorded in Q1, up from $8 million in the previous quarter [6][8] - As of May 1, over 65 authorized treatment centers have been activated globally, with nearly 90 patients undergoing their first cell collection [8] Group 3: Future Pipeline and Competition - CRSP plans to expand its pipeline with two additional in vivo programs, CTX340 and CTX450, by the end of the year, while also advancing two next-generation CAR-T therapy candidates [12][13] - The company faces competition from other firms utilizing CRISPR technology, such as Beam Therapeutics and Intellia Therapeutics, which are developing their own therapies for similar indications [14][15] Group 4: Stock Performance and Valuation - CRSP shares have outperformed the industry and the S&P 500 Index, rising 9% year-to-date compared to a 1% decline in the industry [17] - The stock is currently trading at a price-to-book value (P/B) ratio of 2.03, which is lower than the industry average of 3.14, indicating a discount [20]

Independent drug launches are highly unpredictable, but CRISPR Therapeutics can lean on its collaboration partner, Vertex Pharmaceuticals (VRTX 0.40%). Vertex successfully markets the first and only FDA-approved treatments that address the underlying cause of cystic fibrosis, another rare disease. With product sales that grew past $11 billion last year, it's clear Vertex knows a thing or two about selling rare disease drugs. CRISPR Therapeutics is still losing money, but it also finished March with $1.9 bil ...

Core Insights - CRISPR Therapeutics reported a first-quarter 2025 loss of $1.58 per share, wider than the Zacks Consensus Estimate of a loss of $1.27 per share, and compared to a loss of $1.43 per share in the same period last year [1] - Total revenues for the quarter were $0.86 million, significantly missing the Zacks Consensus Estimate of $5 million, and up from $0.5 million in the year-ago period [1] - The company's shares fell nearly 12% following the weak quarterly performance, with a year-to-date decline of 15.8%, compared to the industry decline of 2.3% [2] Financial Performance - Research and development expenses decreased by approximately 5% year over year to $72.5 million, attributed to reduced employee-related costs [6] - General and administrative expenses increased by 7.2% year over year to $19.3 million [6] - Collaboration expenses rose to $57.5 million in the first quarter from $47.0 million in the previous year, driven by costs related to the Casgevy therapy [6] - As of March 31, 2025, the company had cash, cash equivalents, and marketable securities totaling $1.86 billion, down from $1.90 billion as of December 31, 2024 [7] Product Development and Pipeline - CRISPR Therapeutics and Vertex Pharmaceuticals' Casgevy therapy received approval for treating sickle cell disease and transfusion-dependent beta thalassemia in several countries [4] - Vertex reported Casgevy sales of $14.2 million in the first quarter, with over 65 authorized treatment centers activated and more than 90 patients initiating cell collection [5] - The company is evaluating two in-vivo candidates, CTX310 and CTX320, in separate phase I clinical studies targeting atherosclerotic heart disease [8] - Initial data from the CTX310 study showed dose-dependent decreases in LDL and TG levels, with peak reductions of up to 82% in TG and 81% in LDL [9] - CTX320 is targeting the LPA gene in patients with elevated Lp(a), with updates expected in the second quarter of 2025 [10] - CRISPR is also conducting phase I/II studies on next-generation allogeneic CAR T product candidates, CTX112 and CTX13, with updates anticipated in mid-2025 [10][11]

-Initial CTX310™ Phase 1 clinical data demonstrates dose-dependent decreases in triglycerides (TG) and low- density lipoprotein (LDL), with peak reduction of up to 82% in TG and up to 81% in LDL, with a well-tolerated safety profile; presentation anticipated at a medical meeting in the second half of 2025- -CASGEVY® continues to gain momentum; more than 65 authorized treatment centers (ATCs) activated globally for CASGEVY, and more than 90 patients have had cells collected across all regions; new patient in ...