Key Points CRISPR Therapeutics and BioNTech have climbed along with many of their peers over the past week. Both are innovative biotechs with catalysts that could drive their stocks higher in the next few years. 10 stocks we like better than CRISPR Therapeutics › On Sept. 30, Pfizer announced a deal with the U.S. government that will allow the pharmaceutical leader to avoid President Donald Trump's tariffs for three years in exchange for slashing the prices of certain drugs in the U.S. and investin ...

Key Points CRISPR Therapeutics has a couple of pipeline candidates with massive opportunities. The company has already shown that it can get approval for its gene-editing medicines. Despte sizable upside potential for the stock, investors should proceed with caution. 10 stocks we like better than CRISPR Therapeutics › Becoming a millionaire through stock investing is possible, but it requires patience, discipline, and the acumen to make informed investment choices. Not every company can generate ...

Investment Rating - The report maintains an "Outperform" rating for the pharmaceutical and biotechnology sector [5][41]. Core Views - The pharmaceutical sector has shown resilience, outperforming the overall market with a 1.40% increase, while the total A-share market declined by 1.17% [1][32]. - ANGPTL3 has gained significant interest from multinational pharmaceutical companies (MNCs) due to its unique mechanism of action, which is non-LDLR dependent, making it effective in patients with LDLR deficiencies [3][30]. - The report highlights the diverse drug forms targeting ANGPTL3, with MNCs like Novartis, Lilly, Amgen, and AstraZeneca actively investing in this area, indicating a potential for multi-target and multi-drug combinations in the future [3][16]. Summary by Sections Market Performance - The overall A-share market fell by 1.17%, with the Shanghai and Shenzhen 300 index down by 0.81%. The biotechnology sector, however, rose by 1.40%, indicating strong performance relative to the broader market [1][32]. - Specific segments within the pharmaceutical sector showed varied performance, with chemical pharmaceuticals up by 3.92% and medical services up by 1.69%, while medical devices and traditional Chinese medicine saw declines [1][32]. ANGPTL3 Developments - A collaboration between Argo Biopharma and Novartis was announced, focusing on RNA drugs for cardiovascular diseases, with a total potential payment of $5.2 billion, including a $160 million upfront payment [2][11]. - ANGPTL3's mechanism allows for significant reductions in LDL-C levels in patients with HoFH, outperforming traditional treatments like PCSK9 inhibitors [3][31]. Company Earnings Forecasts and Ratings - Key companies in the sector, such as Mindray Medical, WuXi AppTec, and Aier Eye Hospital, have been rated as "Outperform" with projected net profits for 2024 ranging from 1.4 billion to 116.7 billion yuan [4][41]. - The report provides detailed earnings forecasts for various companies, indicating a positive outlook for the sector as a whole [4][41]. Valuation Metrics - The TTM price-to-earnings ratio for the pharmaceutical and biotechnology sector stands at 40.75x, significantly higher than the overall A-share market's 19.80x [37][38]. - Sub-sectors such as chemical pharmaceuticals and biological products have even higher valuations, indicating strong investor interest and growth potential [37][38]. Recommended Stocks - The report recommends several companies, including Mindray Medical for its strong market position and international expansion, WuXi AppTec for its comprehensive drug development services, and Aier Eye Hospital for its leading position in the eye care sector [41][42].

Core Insights - CRISPR Therapeutics (CRSP) is the first company to market a CRISPR/Cas9-based therapy, achieving significant success with its gene therapy Casgevy for sickle cell disease and transfusion-dependent beta-thalassemia [1][2] - The company is transitioning focus from ex vivo therapies to in vivo candidates, with ongoing phase I studies for CTX310 and CTX320 [2][3] - CTX310 has shown promising results, with reductions of up to 82% in triglycerides and 86% in low-density lipoprotein levels [3][9] Company Developments - Casgevy was developed in partnership with Vertex Pharmaceuticals, which leads global development and commercialization, sharing costs and profits in a 60:40 ratio [2] - CRISPR Therapeutics is expanding its pipeline with plans to advance CTX340 and CTX450 into clinical studies by the end of 2025 [4][9] - The company’s efforts to diversify its pipeline beyond Casgevy are seen as a positive move in the emerging gene therapy market [5] Competitive Landscape - Competition exists from chronic therapies like Bristol Myers' Reblozyl and Novartis' Adakveo for the same indications as Casgevy [5] - Other companies, such as Beam Therapeutics and Intellia Therapeutics, are also developing CRISPR-based therapies, which may pose competitive threats [6][7] Financial Performance - CRSP shares have increased by 34.7% year-to-date, outperforming the industry average rise of 3.2% [8] - The company is currently trading at a price-to-book (P/B) ratio of 2.68, lower than the industry average of 3.10, indicating a potential valuation opportunity [10] - Estimates for CRISPR Therapeutics' loss per share for 2025 have widened from $5.67 to $6.38, while estimates for 2026 have narrowed from $4.42 to $4.02 [11]

Core Insights - CRISPR Therapeutics (CRSP) shares have increased by 41.4% over the past three months, driven by positive data from the CTX310 study and strong sales of Casgevy in Q2 2025 [1][9]. In Vivo Pipeline Programs - CRISPR Therapeutics is advancing its in-vivo candidates, CTX310 and CTX320, in separate phase I clinical studies targeting ANGPTL3 and lipoprotein(a) respectively [2]. - Updated data from the CTX310 study showed significant reductions in low-density lipoprotein (LDL) and triglyceride (TG) levels, with peak reductions of up to 86% in LDL and 82% in TG [2][9]. - Data for CTX320 is anticipated in the first half of 2026, raising hopes for its potential in treating heart disease [3]. Casgevy Sales Performance - Casgevy, a one-shot gene therapy developed in partnership with Vertex Pharmaceuticals, was approved for sickle cell disease and transfusion-dependent beta-thalassemia [4]. - After a slow start in 2024 with only $10 million in revenue, Casgevy's sales surged to $30.4 million in Q2 2025, marking a 114.1% sequential increase [5][6]. - Over 75 treatment centers have been activated for Casgevy, with expectations for significant growth in new patient starts throughout 2025 [6]. Other Pipeline Candidates and Collaborations - CRISPR Therapeutics is developing next-generation CAR-T therapy candidates, CTX112 and CTX131, in separate phase I/II studies, with data expected later in 2025 [7]. - The company plans to expand its in-vivo pipeline with two additional programs, CTX340 and CTX450, by the end of the year [8]. - A recent collaboration with Sirius Therapeutics aims to diversify CRSP's pipeline into RNA therapeutics, focusing on the development of SRSD107 for thromboembolic disorders [10][11]. Competitive Landscape - CRISPR Therapeutics faces competition from other companies in the gene-editing space, including Beam Therapeutics and Intellia Therapeutics, which are advancing their own therapies [12]. - Casgevy also competes with chronic therapies like Bristol Myers' Reblozyl and Novartis' Adakveo [13]. Stock Performance and Valuation - CRSP shares have risen 34.8% year-to-date, outperforming the industry and the S&P 500 [14]. - The stock is trading at a price-to-book (P/B) ratio of 2.68, lower than the industry average of 2.95, indicating a potential valuation opportunity [15]. Estimate Movements - Estimates for CRISPR's 2025 loss per share have widened from $5.58 to $6.26, while estimates for 2026 have narrowed from $4.30 to $3.98 [18]. Financial Position - CRISPR Therapeutics has a strong cash balance of approximately $1.7 billion as of June-end, which supports ongoing operations and potential late-stage studies [20].

Core Viewpoint - CRISPR Therapeutics, despite a 24% decline in share price since mid-2022, has potential for significant returns due to its innovative gene-editing therapies, particularly with its first approved product, Casgevy [1][2]. Group 1: Product Development and Market Potential - CRISPR Therapeutics' first approved product, Casgevy, treats sickle cell disease and transfusion-dependent beta-thalassemia, marking a milestone as the first CRISPR-based gene-editing medicine approved [3]. - The treatment faces challenges, including a complex manufacturing process and a high cost of $2.2 million in the U.S., making reimbursement from third-party payers a significant hurdle [4]. - The company has activated 75 authorized treatment centers and secured reimbursement for eligible patients in 10 countries, targeting approximately 60,000 eligible patients [6]. Group 2: Financial Projections - If CRISPR Therapeutics can secure reimbursement for 70% of the target population and treat 30% over the next decade, Casgevy could generate over $27.7 billion, with CRISPR's share estimated at $11.1 billion [7]. - While Casgevy could contribute significantly to the company's revenue, it may primarily serve as proof of concept for the effectiveness of the biotech's approach [8]. Group 3: Future Pipeline and Growth Potential - CRISPR Therapeutics has six candidates in clinical trials, including CTX310, which shows promise in reducing LDL cholesterol and is easier to handle than ex vivo therapies [10]. - The company's future success relies on achieving consistent clinical and regulatory wins for CTX310 and other candidates, which could lead to a substantial increase in share price [11]. - A successful launch of new products in the next five to seven years could make gene-editing medicines more mainstream, encouraging third-party payers and healthcare institutions to support the treatments [12].

Key Takeaways CRISPR Therapeutics (CRSP) reported a second-quarter 2025 loss of $2.40 per share, which was wider than the year-ago period's loss of $1.49. The increased loss was attributable to the payment of $96.3 million (recorded as acquired in-process R&D expenses) made to Sirius Therapeutics as part of a strategic collaboration agreement signed in May. Excluding this special item, the adjusted loss stood at $1.29 per share, narrower than the Zacks Consensus Estimate of a loss of $1.47. No such expense ...

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]

Core Viewpoint - CRISPR Therapeutics (CRSP) and Intellia Therapeutics (NTLA) are leading companies in the CRISPR/Cas9 gene editing space, with CRSP being the first to market a CRISPR-based therapy, while NTLA focuses on late-stage in vivo therapies [1][10]. Group 1: CRISPR Therapeutics (CRSP) - CRSP achieved a significant milestone by securing approval for Casgevy (exa-cel) for treating sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT) in late 2023/early 2024, marking the first-ever approval for a CRISPR/Cas9 therapy globally [2][3]. - The commercial uptake of Casgevy has been slow due to the complexity of the treatment process, although VRTX has activated over 65 authorized treatment centers globally, with nearly 90 patients having completed cell collection as of May 1 [4]. - CRSP has multiple other CRISPR candidates in its pipeline, including two CAR-T therapy candidates, CTX112 and CTX131, which are in early-stage studies, with updates expected this year [5]. - Encouraging initial data from an early-stage study on CTX310, an in vivo CRISPR-based gene therapy targeting ANGPTL3 for atherosclerotic heart disease, has raised excitement for CRSP's in vivo programs [6][7]. - Despite the progress, CRSP's pipeline is still in early-stage development, and Casgevy faces competition from chronic therapies like Bristol Myers' Reblozyl and Novartis' Adakveo [8]. Group 2: Intellia Therapeutics (NTLA) - NTLA has shown potential in the biotech space by focusing on in vivo therapies, with two late-stage candidates: lonvo-z for hereditary angioedema (HAE) and nex-z for transthyretin (ATTR) amyloidosis [9][11]. - The pivotal phase III HAELO study for lonvo-z is underway, with enrollment expected to complete by Q3 2025, and regulatory filing planned for the second half of 2026 [12]. - Nex-z is being developed in collaboration with Regeneron Pharmaceuticals and is evaluated in two late-stage studies for ATTR amyloidosis [13]. - NTLA initiated a strategic reorganization to prioritize late-stage candidates, resulting in the cessation of some research programs and a planned workforce reduction of nearly 27% [14]. - A recent setback in the nex-z development due to liver safety concerns has raised questions about the therapy's long-term safety [15]. Group 3: Financial Estimates and Performance - The Zacks Consensus Estimate for CRSP's 2025 sales implies a 7% year-over-year increase, while loss estimates per share are expected to widen by about 28% [16]. - NTLA's 2025 loss per share is expected to improve nearly 20%, with loss estimates for 2025 and 2026 having narrowed over the past 60 days [17]. - Year-to-date, CRSP shares have risen by 19%, while NTLA shares have declined by 18%, compared to a 4% decline in the industry [19]. - CRSP's shares trade at a price/book (P/B) ratio of 2.21, higher than NTLA's 1.27, indicating that CRSP appears more expensive from a valuation standpoint [20]. Group 4: Investment Considerations - Both companies hold a Zacks Rank 3 (Hold), making it challenging to choose one over the other [24]. - CRSP is viewed as a safer investment due to its marketed product and a strong cash balance of $1.9 billion as of March 2025, while NTLA's cash balance was $707 million, raising concerns due to the lack of a stable revenue stream [25]. - Despite setbacks, CRSP's broader pipeline across in vivo and ex vivo therapies offers greater diversification, suggesting potential growth driven by solid fundamentals and positive stock price movement [26].

CASGEVY & Hemoglobinopathies - CASGEVY, a CRISPR-Cas9 therapy for severe sickle cell disease and beta thalassemia, is approved in multiple jurisdictions, with >65 authorized treatment centers activated globally and ~90 patients having initiated cell collection as of May 1st, 2025[9, 23] - The addressable market for CASGEVY is approximately 60,000 severe patients in approved territories[19] - A new CMMI model aims to improve access and health outcomes for sickle cell disease, potentially reducing U S expenditures by $3 billion annually[21] CAR T Programs - CTX112, an allogeneic CAR T therapy, demonstrated an overall response rate (ORR) of 67% and a complete response rate (CR) of 50% in a Phase I/II trial (N=12) for relapsed or refractory B-cell malignancies[40] - Updated CTX112 data (N=25) showed cell expansion comparable to autologous CAR T therapies, with dose-dependent increases in AUC and Cmax[43, 46] - In a subset of patients receiving CTX112 post-T cell engager (TCE) therapy, the overall response rate was 100% (6 patients)[48] In Vivo Programs - Initial results from the CTX310 Phase 1 dose escalation trial (N=10) showed promising efficacy, with one patient at the 0 8 mg/kg dose level experiencing an 82% reduction in triglycerides from a baseline of 1073 mg/dL at day 30[62, 66] - Preclinical data for CTX320 in NHPs demonstrated ~70% editing of LPA and ~95% reduction in plasma Lp(a) sustained at 2 years at 2 mg/kg dose[73] - CTX320 has the potential to benefit >60 million U S patients with elevated Lp(a)[81] Financials & Outlook - The company has a strong balance sheet with approximately $1 86 billion[15]