CRISPR Therapeutics (NasdaqGM:CRSP) FY Conference January 12, 2026 11:15 AM ET Company ParticipantsDiana Ramos - SurgeonCharles Bruce - Medical ExpertSamarth Kulkarni - CEOConference Call ParticipantsNone - AnalystBrian Cheng - Senior Biotech AnalystBrian Chengto another session at the 44th J.P. Morgan Healthcare Conference. I'm Brian Chang. I'm one of the senior biotech analysts here at the firm. On stage, we have Sam from CRISPR Therapeutics. I'll now pass the mic to their CEO, Sam, for a short presentati ...

There are some risks to consider before jumping on the bandwagon.Once again, the technology sector drove much of the market's gains this year. The healthcare industry, on the other hand, has been a bit of a laggard.However, there have been notable standouts among medically focused stocks. The list of strong performers includes CRISPR Therapeutics (CRSP +0.44%), a biotech company, and HCA Healthcare (HCA +0.03%), a leading hospital chain. They have outperformed the S&P 500, (^GSPC +0.32%) rising by 44% and 5 ...

Core Insights - CRISPR Therapeutics reported a narrower loss of $1.17 per share for Q3 2025, compared to the Zacks Consensus Estimate of a loss of $1.32, but wider than the loss of $1.01 per share in the same quarter last year [2][8] - Total revenues for the quarter were $0.9 million, significantly missing the Zacks Consensus Estimate of $6.7 million, and up from $0.6 million in the year-ago period [2][8] Financial Performance - The company experienced a 28% year-over-year decline in R&D expenses to $58.9 million, attributed to reduced manufacturing and employee-related costs [9] - General and administrative expenses fell 3% year-over-year to $16.9 million [9] - Net collaboration expenses increased to $57.1 million from $11.2 million in the previous year, primarily due to reaching a deferral limit on costs related to the Casgevy program [9] - As of September 30, 2025, the company had cash, cash equivalents, and marketable securities totaling $1.94 billion, up from $1.72 billion as of June 30, 2025 [10] Product Development and Pipeline - CRISPR Therapeutics and Vertex Pharmaceuticals' Casgevy therapy has been approved for sickle cell disease and transfusion-dependent beta thalassemia, with Vertex expecting over $100 million in revenues for Casgevy this year [5][6] - The company completed enrollment for pediatric trials of Casgevy and plans to present initial data at the American Society of Hematology annual meeting on December 6, 2025 [11] - CRISPR is advancing two next-generation CAR-T therapy candidates, CTX112 and CTX131, with updates expected by year-end [12] - The company is also studying in-vivo candidates CTX310 and CTX320, with CTX310 showing significant reductions in ANGPTL3, LDL, and triglyceride levels [13] - A collaboration with Sirius Therapeutics is diversifying the pipeline into RNA therapeutics, with an investigational RNA therapy SRSD107 in mid-stage studies [14] Market Performance - Shares of CRISPR Therapeutics have increased by 39% year-to-date, outperforming the industry growth of 11% [3]

Core Insights - CRISPR Therapeutics (CRSP) is set to report Q3 2025 earnings, with sales estimated at $6.71 million and a loss of $1.32 per share, reflecting a slight widening of loss estimates from $6.59 to $6.66 over the past month [1][4][5] Earnings Performance - The company has shown a decent earnings surprise history, beating estimates in three of the last four quarters with an average surprise of 18.41%, including a 12.24% beat in the most recent quarter [2][3] Earnings Prediction - A positive Earnings ESP of +3.53% and a Zacks Rank of 3 suggest a potential earnings beat for the upcoming report [5][4] Revenue Sources - Current revenue includes grants and collaboration income from a partnership with Vertex Pharmaceuticals (VRTX), particularly from the recently approved gene therapy, Casgevy, for blood disorders [6][7] Product Updates - Casgevy, the first marketed product in CRSP's portfolio, has seen rising sales, which are expected to positively impact collaboration expenses in the upcoming quarter [7][8] Pipeline Development - CRISPR is advancing its pipeline with next-generation CAR-T therapies and in-vivo candidates, with updates anticipated during the earnings call [9][10] Stock Performance - CRSP shares have increased nearly 63% this year, outperforming the industry and broader market indices [12][13] Investment Thesis - The approval of Casgevy marks a significant milestone for CRISPR, establishing its first commercial revenue stream, with expectations for steady revenue growth as treatment centers increase [15][16] Valuation - CRSP shares trade at a modest discount to the industry, with a price/book ratio of 3.40 compared to the industry average of 3.51 [18] Future Outlook - Continued investment in innovation and updates on the immuno-oncology pipeline are expected to act as catalysts for the stock [19]

Group 1: CRISPR Therapeutics - CRISPR Therapeutics has seen its shares rise approximately 85% this year due to promising advancements in its pipeline programs [3] - The company's CTX310 aims to lower LDL cholesterol and triglycerides, addressing a significant health need for around 40 million people in the U.S. with elevated levels [3] - Other notable programs include CTX112 for blood cancers and CTX320 for lowering lipoprotein(a), with the potential for better outcomes than existing therapies [5] - The company has an approved drug, Casgevy, which could contribute to revenue in the future [5] - CRISPR Therapeutics is positioned for potential new medicine launches, supported by successful clinical trials, despite inherent clinical risks [6] Group 2: Spotify Technologies - Spotify Technologies is the leading music streaming platform, holding a significant market share despite competition from major corporations like Apple, Amazon, and Alphabet [7] - The company's shares have increased by about 50% in 2025, reflecting market recognition of its strong brand and network effects [8] - Spotify's strategic move into podcasting has contributed to its growth, although it has impacted profit margins [9] - As of the second quarter, Spotify had 696 million monthly active users, an 11% year-over-year increase, with a goal to reach 1 billion MAUs by 2030 [11] - The company is enhancing its platform through AI initiatives, such as Spotify DJ, which is expected to drive user engagement and increase ad sales [12]

Group 1: Pfizer and the Healthcare Sector - Pfizer announced a deal with the U.S. government to avoid tariffs for three years by reducing drug prices and investing in local manufacturing [1] - This deal positively impacted Pfizer and the broader healthcare sector, with expectations of similar agreements in the future [1] Group 2: CRISPR Therapeutics - CRISPR Therapeutics is a gene-editing company with one approved drug, Casgevy, which targets two rare blood diseases and is priced at $2.2 million in the U.S. [4][5] - The company estimates around 60,000 potential patients for Casgevy in the launched countries, indicating future sales potential [5] - Significant clinical progress is anticipated with leading candidates CTX310 and CTX320, which target cardiovascular risk factors and could address up to 40 million patients in the U.S. [6][7] Group 3: BioNTech - BioNTech, alongside CRISPR Therapeutics, has seen stock price increases and possesses significant upside potential due to innovative developments [2][8] - Both companies are positioned well within the biotech sector, with catalysts that could drive stock prices higher in the coming years [8]

Core Insights - Achieving millionaire status through stock investing is feasible but demands patience, discipline, and informed decision-making [1] - Investing in ETFs that track major indexes is a low-risk strategy, but selecting individual stocks with high upside potential may yield better returns [2] Company Overview: CRISPR Therapeutics - CRISPR Therapeutics is a mid-cap biotech company whose shares have declined over 60% from their all-time highs in early 2021 [3] - The company relies on strong clinical and regulatory advancements to thrive, with potential for significant stock appreciation over the next five years if it meets market expectations [4] Pipeline Progress - CRISPR's leading pipeline candidate, CTX310, aims to lower LDL cholesterol and triglycerides by inactivating the ANGPTL3 gene, addressing a significant unmet medical need [5] - CTX310 has shown promising results in clinical trials, leading to substantial reductions in LDL and triglyceride levels, with a potential market of 40 million high-risk patients in the U.S. [6] - Another candidate, CTX320, targets lipoprotein(a) levels, which are associated with heart attack and stroke risks, indicating CRISPR's focus on high unmet needs in large patient populations [7] Investment Considerations - CRISPR Therapeutics has demonstrated the ability to gain approval for its gene-editing therapies, presenting substantial upside potential for investors [8]

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 Insights - CRISPR Therapeutics reported a second-quarter 2025 loss of $2.40 per share, wider than the previous year's loss of $1.49, primarily due to a $96.3 million expense related to a collaboration with Sirius Therapeutics [1][6] - Adjusted loss, excluding special items, was $1.29 per share, better than the Zacks Consensus Estimate of a loss of $1.47 [2] - Total revenues for the quarter were $0.89 million, significantly below the Zacks Consensus Estimate of $6.6 million, compared to $0.5 million in the same period last year [2] Financial Performance - CRISPR Therapeutics' stock fell over 8% in after-market trading following the wider-than-expected loss, continuing the downward trend in pre-market trading [3] - The stock has increased by 51% year-to-date, outperforming the industry growth of 2% [3] - Research and development expenses decreased by 13% year-over-year to $69.9 million, while general and administrative expenses fell by 3% to $18.9 million [8] Product Development and Sales - Casgevy, a CRISPR/Cas9 gene-edited therapy developed in partnership with Vertex Pharmaceuticals, saw sales of $30.4 million in Q2, up from $14.2 million in the previous quarter [5][6] - Over 75 treatment centers have been activated globally for Casgevy, with approximately 115 patients completing their first cell collection since its launch [6][7] - The company is advancing its CAR-T and in-vivo therapy pipelines, with updates expected later this year [10][11] Pipeline Expansion - CRISPR Therapeutics is developing two next-generation CAR-T therapy candidates, CTX112 and CTX131, currently in phase I/II studies [10] - The company is also studying in-vivo candidates CTX310 and CTX320, with promising early data showing significant reductions in LDL and triglyceride levels [11] - A collaboration with Sirius Therapeutics has diversified the pipeline into RNA therapeutics, with a focus on the investigational siRNA candidate SRSD107 [12][13] Financial Position - As of June 30, 2025, CRISPR Therapeutics had cash and marketable securities totaling $1.72 billion, down from $1.86 billion at the end of March 2025 [9]