An investment in these players today could score a big win down the road. Why do investors love biotech stocks? Because these players often work on game-changing technology that could lead to tomorrow's game-changing treatment -- and potentially blockbuster revenue. As an investor, you often have to be patient, as it takes years for a company to bring a candidate through development and reach the billion-dollar revenue stage. And you have to be comfortable with a bit of risk too, because candidate failure a ...

Core Viewpoint - CRISPR Therapeutics has experienced a significant decline in stock price despite the potential of its gene-editing therapy, Casgevy, which could generate substantial revenue in the future [1][2][3] Group 1: Financial Performance - Year-to-date, CRISPR's shares are down 24%, far from its 52-week high of over $91 [1] - The company incurred a net loss of $243 million in the first half of the year, indicating ongoing financial challenges [4] - CRISPR has $2 billion in cash and cash equivalents, providing a solid financial foundation to sustain operations without immediate cash raises [4] Group 2: Product Pipeline and Market Potential - Casgevy, a treatment for sickle cell disease and beta thalassemia, is expected to generate billions in revenue, but analysts predict it won't exceed $1 billion until 2027 [2][3] - CRISPR is also developing other treatments, including CTX211 for type 1 diabetes and multiple cancer therapies, with preliminary data expected later this year [5][6] - The gene therapy market is currently valued at around $6 billion but is projected to grow to over $18 billion by the end of the decade [6] Group 3: Investment Outlook - CRISPR is viewed as a long-term investment opportunity, with a market capitalization of just over $4 billion, suggesting significant upside potential if additional treatments gain approval [7] - The company is not a typical high-risk biotech startup, as it has an approved treatment and ample liquid resources to support its operations [8] - Despite current market focus on artificial intelligence and tech stocks, CRISPR may present a compelling buy for patient investors, with the potential for substantial long-term returns [9]

A month has gone by since the last earnings report for CRISPR Therapeutics AG (CRSP) . Shares have lost about 7.7% in that time frame, underperforming the S&P 500. Will the recent negative trend continue leading up to its next earnings release, or is CRISPR Therapeutics due for a breakout? Before we dive into how investors and analysts have reacted as of late, let's take a quick look at its most recent earnings report in order to get a better handle on the important catalysts. Q2 Loss Wider-Than-Expected, S ...

These companies are nearly in the sweet spot of risk versus reward in biotech. Many investors rightfully identify biotech stocks as being riskier than average, and those stocks certainly have a habit of earning their reputation. Still, there are a few up-and-coming biotechs that have revenue and a clear path to generating even more in the near term, which makes them notably steadier than their earlier-stage peers that don't yet have the certainty of selling anything at all. With that in mind, let's examine ...

The companies are chasing major innovations. Exciting trends are sweeping through the biotech industry. One of them is the race to develop breakthrough weight loss medicines, an area that is projected to grow by leaps and bounds through the end of the decade. Gene editing, a set of techniques that are allowing researchers to unlock therapies for diseases that have been untouchable, is another one. Companies, particularly relatively small ones that make breakthroughs in these fields, could deliver marketcrus ...

Core Viewpoint - CRISPR Therapeutics (CRSP) has underperformed in the market despite receiving regulatory approval for its gene therapy Casgevy, which is the first CRISPR-based gene-editing therapy approved globally [1][3][11] Company Developments - CRSP's stock has declined 23.6% year-to-date, while the industry has only fallen by 1.7% [1][3] - The company received approval for Casgevy, a one-shot gene therapy for sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT), in the U.S. and Europe [3][4] - Casgevy is expected to provide a new treatment option for approximately 35,000 patients in the U.S. and Europe, with revenue generation anticipated to start in the second half of 2024 [4][5] Partnership Insights - CRISPR has a solid partnership with Vertex Pharmaceuticals, which began in 2015, focusing on gene editing therapies for various conditions [6][7] - The collaboration has expanded to include therapies for cystic fibrosis, Duchenne Muscular Dystrophy (DMD), and Myotonic Dystrophy Type 1 (DM1) [6] - CRISPR is eligible for over $1.3 billion in milestone payments and royalties from therapies developed under this partnership [7] Financial Metrics - CRISPR's shares are trading at a price-to-book (P/B) ratio of 2.05, which is lower than the industry average of 4.58 [8] - Loss estimates for 2024 have slightly improved from $5.65 to $5.61, while 2025 estimates have widened from $4.44 to $4.92 due to increased R&D costs [8][9] Market Position - The approval of Casgevy gives CRISPR an advantage over competitors like Beam Therapeutics and Editas Medicine, which are still in earlier stages of developing their therapies for SCD and TDT [11] - Management is also focusing on in vivo candidates, with two new candidates currently in phase I clinical studies [12]

Bevan Goldswain/E+ via Getty Images Introduction Shares of CRISPR Therapeutics (NASDAQ:CRSP) have been on a rollercoaster ride as of late. I've been following the company closely for over a year now. Remember that CRISPR secured FDA approval last December for its gene therapy targeting sickle cell disease, Casgevy. On the same day, bluebird bio (BLUE), too, received the green light for its sickle cell gene therapy, Lyfgenia. My attitude toward CRISPR has always been cautious, even after Casgevy's groundbrea ...

Core Viewpoint - Crispr Therapeutics AG, in partnership with Vertex, has received approvals for its CRISPR/Cas9 gene editing therapy, Casgevy, targeting Sickle Cell Disease and transfusion-dependent beta thalassemia, but has yet to report revenue figures from the drug due to the complex treatment process [2][3][7]. Treatment and Revenue Potential - Casgevy is priced at approximately $2.2 million per treatment, with around 20 patients having cells collected as of mid-July, suggesting potential revenue of at least $44 million for the first half of 2024 [7]. - The number of authorized treatment centers (ATCs) has increased from 9 at launch to 35, with a target of 75, indicating a growing capacity to treat patients [7][9]. - If each ATC can treat 30 patients annually, potential annual revenues for Crispr Therapeutics could reach $1.98 billion if all 75 centers are operational [7][9]. Efficacy and Market Position - Over 90% of patients treated with Casgevy in pivotal studies remain transfusion-independent and free from vaso-occlusive crises, highlighting the therapy's effectiveness [8]. - The competitive landscape includes other therapies, but Casgevy offers a permanent cure, differentiating it from alternatives like Pfizer's Oxbryta [8][9]. Future Developments - Crispr Therapeutics is exploring ways to reduce the preconditioning burden for patients, potentially expanding the addressable patient population significantly [9][10]. - The company is also working on in vivo therapies that could eliminate the need for complex treatment processes, which may enhance its market position [9][10]. Pipeline and Financial Health - Crispr Therapeutics has a robust pipeline, including allogeneic therapies and in vivo programs, which could significantly increase its market cap if successful [12]. - Despite a net loss of $126.4 million in Q2, the company maintains a strong cash position of approximately $1.7 billion, ensuring a secure funding runway for ongoing research and development [12]. Investment Outlook - The current market cap of Crispr Therapeutics is $4.2 billion, with expectations that revenues from Casgevy could exceed $1 billion annually by 2025 or 2026, potentially increasing the market cap to over $10 billion [14].

CRISPR Therapeutics (CRSP) incurred a loss of $1.49 per share in the second quarter of 2024, which was wider than the Zacks Consensus Estimate of a loss of $1.37. In the year-ago period, the company had incurred a loss of 98 cents per share. The company's total revenues, though negligible, were entirely from grant revenues, which were $0.5 million in the second quarter. The reported figure significantly missed the Zacks Consensus Estimate of $8 million. In the year-ago quarter, the company generated collabo ...

CASGEVY Approval and Efficacy - CASGEVY, the first-ever approved CRISPR-based gene-editing therapy, has been approved in the United States, European Union, Great Britain, Kingdom of Saudi Arabia, and Kingdom of Bahrain for treating severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT) in patients aged 12 and older[51] - CASGEVY has shown efficacy as a potential one-time functional cure for severe SCD and TDT, with safety data consistent with autologous stem cell transplant and myeloablative conditioning[51] Next-Generation CAR T Cell Therapies - The company is advancing next-generation CAR T cell therapies, including CTX112 (targeting CD19) and CTX131 (targeting CD70), which show significantly higher CAR T cell expansion and functional persistence compared to first-generation candidates[53] - CTX112 is being investigated in clinical trials for relapsed or refractory CD19-positive B-cell malignancies and systemic lupus erythematosus, with early studies showing long-lasting remissions in autoimmune indications[54] - CTX131 is being developed for solid tumors and hematologic malignancies, including T cell lymphomas, with ongoing clinical trials assessing its safety and efficacy[55] In Vivo Gene Editing Programs - The company's in vivo gene editing programs, CTX310 and CTX320, target cardiovascular disease by disrupting ANGPTL3 and LPA genes, respectively, with up to 20% of the global population having elevated Lp(a) levels[57] Type 1 Diabetes Therapy Development - CTX211, an allogeneic, gene-edited, hypoimmune stem cell-derived product candidate for type 1 diabetes, is in a Phase 1/2 clinical trial, with the company receiving $170 million in upfront and milestone payments from Vertex in 2023[59] Next-Generation Editing Modalities - The company's CRISPR-X research team is developing next-generation editing modalities, including whole gene correction and insertion without requiring homology-directed repair or viral delivery of DNA[60] Strategic Partnerships and Collaborations - Strategic partnerships with Vertex, ViaCyte, and Bayer are accelerating the development of gene-editing therapies, with Vertex co-developing and co-commercializing CASGEVY and licensing CRISPR/Cas9 technology for diabetes[61] - Additional collaborations with companies like Nkarta, Capsida Biotherapeutics, and MaxCyte support the company's hematopoietic stem cell, immuno-oncology, and in vivo programs[62] Financial Performance and Expenses - The company has not generated any revenue from product sales and does not expect to do so in the near future. Revenue for the three and six months ended June 30, 2024 was not material, while revenue for the same periods in 2023 was $70.0 million and $170.0 million, respectively, primarily from an upfront payment and a research milestone achieved with Vertex[64][71][76] - Research and development expenses for the three months ended June 30, 2024 were $80.2 million, a decrease of $21.4 million compared to $101.6 million in the same period in 2023, primarily due to reduced external research and manufacturing costs[72] - General and administrative expenses for the three months ended June 30, 2024 were $19.5 million, slightly higher than the $19.0 million in the same period in 2023[73] - Collaboration expense, net, for the three months ended June 30, 2024 was $52.1 million, an increase of $7.5 million compared to $44.6 million in the same period in 2023, primarily due to commercial and manufacturing costs related to the CASGEVY program with Vertex[74] - Net loss for the three months ended June 30, 2024 was $126.4 million, compared to a net loss of $77.7 million in the same period in 2023, driven by higher collaboration expenses and lower revenue[70] - For the six months ended June 30, 2024, the company reported a net loss of $243.0 million, compared to a net loss of $130.8 million in the same period in 2023, primarily due to a significant decrease in collaboration revenue and increased collaboration expenses[75] - Research and development expenses decreased by $45.2 million to $156.3 million for the six months ended June 30, 2024, primarily due to reduced external R&D costs ($34.3 million), facility-related expenses ($8.2 million), and employee-related expenses ($4.0 million)[77] - General and administrative expenses decreased by $4.0 million to $37.4 million for the six months ended June 30, 2024, driven by lower employee-related and consulting expenses[78] - Collaboration expense, net, increased by $12.3 million to $99.1 million for the six months ended June 30, 2024, primarily due to commercial and manufacturing costs related to the CASGEVY program[79] - Other income increased by $19.8 million to $50.9 million for the six months ended June 30, 2024, mainly due to higher interest income from cash, cash equivalents, and marketable securities[80] Cash and Financial Position - The company had $2,012.8 million in cash, cash equivalents, and marketable securities as of June 30, 2024, with $154.1 million held outside the U.S. and an accumulated deficit of $1,242.7 million[81] - Net cash provided by operating activities increased by $138.6 million to $14.2 million for the six months ended June 30, 2024, driven by a $270.0 million increase in accounts receivable[86] - Net cash used in investing activities was $224.7 million for the six months ended June 30, 2024, compared to $334.8 million provided in the same period in 2023, due to increased purchases of marketable securities[87] - Net cash provided by financing activities was $305.5 million for the six months ended June 30, 2024, primarily from the sale of $280.0 million in common shares to institutional investors[88] - The company expects its existing cash, cash equivalents, and marketable securities to fund operations for at least the next 24 months, based on current R&D plans and timing expectations[84] - The company's cash, cash equivalents, and marketable securities of $2,012.8 million are primarily invested in U.S. treasury securities, government agency securities, corporate bonds, and commercial paper, with minimal exposure to interest rate risk[91] Legal Proceedings - No material adverse legal proceedings currently pending against the company[96] - No significant developments in previously disclosed legal proceedings[96] Future Outlook and Expectations - The company expects to continue incurring significant research and development expenses as it progresses its current development programs, adds new programs, and prepares regulatory filings[66] - The company has a history of recurring losses and expects to continue incurring losses for the foreseeable future, with expenses likely to increase as it advances its research and development activities[63] - The company has the option to defer specified costs on the CASGEVY program in excess of $110.3 million for the years ended December 31, 2022, 2023, and 2024, with deferred amounts payable as an offset against future profitability of the program[68] - The company's revenue recognition for the three and six months ended June 30, 2023 was primarily related to an upfront payment from Vertex and a research milestone achieved in the second quarter of 2023[64][71][76]