Summary of Cibus (CBUS) Conference Call - May 21, 2025 Company Overview - **Company Name**: Cibus (Ticker: CBUS) - **Industry**: Agricultural Biotechnology - **Focus**: Development of gene-edited crop traits in canola, rice, and soy using a rapid trait development system - **Current Rating**: Buy with a price target of $23.50 [1] Core Points and Arguments Gene Editing vs. GMO - **Definition**: Gene editing involves making precise changes to an organism's DNA, while GMOs involve transferring genes from one organism to another [3][4] - **Regulatory Recognition**: Global regulators have acknowledged that gene-edited crops are indistinguishable from natural occurrences, leading to recent approvals in various countries, including Ecuador and the EU [6] Rapid Trait Development System (RTDS) - **Technology**: Utilizes a gene repair oligonucleotide to make precise edits at the single-cell level, allowing for multiple changes within genes and across genomes [7][8] - **Speed Advantage**: Traditional plant breeding can take 10-30 years; Cibus aims to deliver new traits within 12 months, significantly improving time-to-market [9][11] Revenue Model - **Pre-Revenue Status**: Currently, Cibus is a pre-revenue company, planning to generate income through royalties from developed traits [12] - **Market Potential**: Traits developed for major crops like soy, which covers over 250 million acres in North and South America, can lead to substantial royalty income [13] - **Royalty Estimates**: Expected royalties of $10 to $15 per acre for traits related to weed management, with potential for significant revenue as traits are adopted [14][15] Developed and Developing Traits - **Current Traits**: - **Rice**: Two herbicide-tolerant traits for efficient weed management [20][21] - **Canola**: Pod shatter reduction trait to preserve yield during harvest [22] - **Future Traits**: Advanced traits in development include additional herbicide tolerance and disease tolerance for canola and soy [23][24] Farmer Value Proposition - **Cost Reduction**: Traits can reduce input costs for farmers, improving profit margins by decreasing the need for herbicides and other inputs [25][26] - **Market Dynamics**: Farmers are willing to pay a premium for traits that enhance productivity and reduce operational costs [27] Timeline for Revenue Generation - **Projected Start**: Traits expected to be planted and royalties to begin flowing in 2027, with a gradual increase through 2029 [28][29] Near-Term Catalysts - **Customer Announcements**: New customer acquisitions in the rice portfolio and advancements in field trials for advanced traits [30][31] - **Wheat Platform**: Expansion into wheat traits is also being explored [31] Bio Fragrance Business - **Overview**: Cibus has discovered a yeast that produces oil, which can be used to create bio fragrances for consumer packaged goods (CPG) companies [32][33] - **Revenue Potential**: Expected nominal revenues in 2025, with significant growth potential in the tens of millions of dollars annually [34] Key Drivers for Business Growth - **Predictability and Speed**: Ability to deliver traits within 12 months is a significant competitive advantage [36] - **Regulatory Environment**: Harmonization of regulations globally opens up markets, allowing Cibus to operate similarly to conventional breeding programs [36][37] Other Important Content - **Market Size**: Major crops involved cover over 500 million acres, indicating a vast market opportunity for Cibus [36] - **Investor Interest**: Recent press releases have generated significant interest from prospective partners and customers [36] This summary encapsulates the key points discussed during the Cibus conference call, highlighting the company's innovative approach to agricultural biotechnology and its potential for future growth.

Summary of Verve Therapeutics (VERV) 2025 Conference Call Company Overview - **Company**: Verve Therapeutics - **Industry**: Biotechnology, specifically focusing on gene editing therapies for cholesterol reduction Key Points and Arguments Progress and Pipeline - Verve is developing one-time therapies aimed at lifelong cholesterol reduction through IV infusion targeting the PCSK9 gene [3][4] - Initial Phase 1 data shows well-tolerated safety and significant efficacy with LDL reductions of 50-60%, with one patient achieving a 69% reduction [4] - The company is also working on additional programs targeting ANGPTL3 and LPA, with updates expected in the second half of the year [5][6] Regulatory Environment - The new leadership at the FDA, particularly Dr. Prasad, is seen as an opportunity for Verve, aligning with their focus on meaningful clinical endpoints like LDL levels [10][11] - Verve's PCSK9 therapy is positioned to provide substantial value, potentially reducing coronary disease risk by 80-90% [12] Clinical Development - Transition from first-generation (Vrb 101) to second-generation (Vrb 102) therapies has improved safety profiles due to advancements in lipid nanoparticle delivery systems [15][16] - Ongoing dose escalation studies are expected to yield further efficacy data, with hopes of achieving higher LDL reduction percentages [19] Partnership with Eli Lilly - Verve has a partnership with Eli Lilly, which includes an opt-in decision expected in the second half of the year, where Lilly would cover a third of worldwide development costs and 50% of US commercialization expenses [21][22] - The partnership is focused on a multimodal pipeline for cardiovascular disease targets, including LPA and ANGPTL3 [23] Market Landscape and Competition - Despite the presence of oral medications for cholesterol reduction, Verve's one-time therapy addresses a significant unmet need, as many patients do not achieve treatment goals with existing options [29][30] - Patient and physician preferences indicate a strong demand for one-time therapies over daily pills [30][31] ANGPTL3 Target - The ANGPTL3 target is supported by strong genetic evidence and pharmacologic validation, with the potential to lower both LDL cholesterol and triglycerides [34] - Previous safety concerns with other ANGPTL3 therapies are attributed to specific drug-related issues rather than the target itself [36] Future Directions - Verve is optimistic about the upcoming data updates for both PCSK9 and ANGPTL3 programs, with plans to leverage learnings from previous studies to enhance study designs [40] - The company is also exploring monetization opportunities for its proprietary GalNAc LNP delivery technology, which has shown promising safety profiles [49][51] Competitive Landscape - The emergence of competitors like CRISPR in the gene editing space is acknowledged, with Verve focusing on differentiating its approach through established safety and efficacy data [38][53] Additional Important Insights - The ongoing trials and partnerships are critical for Verve's strategy to establish itself in the competitive landscape of cholesterol-lowering therapies [47][48] - The company remains confident in the potential of its therapies to address significant cardiovascular health challenges, particularly in light of the limitations of current treatment options [45][46]

Precision BioSciences (DTIL) Conference May 21, 2025 03:15 PM ET Speaker0 Welcome everyone to this session of our May twenty twenty five MicroCap conference. I'm Alex Hanman, and I serve as an equity research analyst here at Sidoti and Company. Today, we're pleased to be in conversation with CFO Alex Kelly and VP of Investor Relations, Naresh Thanna, of Precision Biosciences, ticker d t I l. During the presentation, please feel welcome to submit questions using the Zoom Q and A interface at the bottom of yo ...

智通财经5月21日电，美国麻省理工学院与哈佛大学博德研究所刘如谦团队联合哥伦比亚大学科学家， 成功研发出新型基因编辑工具evoCAST。这款工具能像编程般精准地将完整基因或多个基因嵌入人类基 因组特定位置，为遗传疾病治疗带来突破性进展。相关成果发表于最新一期《科学》杂志。 新型基因编辑工具精准嵌入完整基因 ...

Group 1 - Cibus, Inc. is a leading agricultural biotechnology company focused on gene editing technologies to develop specific traits in seeds [1][3] - The company will present its advancements in gene-editing technology, regulatory developments, and commercialization strategy at the AGP Annual Virtual Healthcare Company Showcase [2][3] - Cibus addresses significant productivity and sustainability challenges for farmers, with an estimated global economic cost of $300 billion annually due to diseases and pests [3] Group 2 - Cibus operates as a technology company rather than a seed company, licensing traits to seed companies in exchange for royalties [3] - The company has a pipeline of five productivity traits, focusing on weed management in rice, pod shatter reduction, and disease resistance [3] - Cibus aims to develop and commercialize plant traits more efficiently and cost-effectively than conventional breeding methods [3]

Core Viewpoint - CRISPR Therapeutics has faced financial struggles despite clinical advancements, with shares down 24% over the past three years, while the S&P 500 gained 41%. However, potential catalysts could double its stock price by 2030, indicating a compound annual growth rate of about 14.9% [1] Group 1: Clinical Progress and Products - CRISPR Therapeutics has developed Casgevy, a one-time treatment for sickle cell disease and transfusion-dependent beta-thalassemia, showcasing the potential of its gene-editing technology [2] - The complexity of ex vivo gene-editing treatments, which involve collecting, editing, and reinserting patient cells, presents manufacturing and administration challenges [3] - Although Casgevy was approved in late 2023, it has not yet significantly impacted financial results, but it is expected to exceed $1 billion in annual sales at its peak due to limited competition [5][6] Group 2: Market Potential - The addressable market for Casgevy is estimated at 58,000 patients in targeted regions, with a treatment cost of $2.2 million in the U.S., indicating substantial revenue potential [6] - The competitive landscape for CRISPR and Vertex Pharmaceuticals appears favorable, particularly in the Middle East where they have received product approvals [5] Group 3: Future Catalysts - CRISPR Therapeutics has several promising candidates in its pipeline, including CTX112 and CTX131 for cancer, with CTX112 receiving Regenerative Medicine Advanced Therapy designation from the FDA [10] - Data from an ongoing clinical trial for CTX320, aimed at lowering lipoprotein(a) levels, is also anticipated, along with other investigational treatments like a functional cure for type 1 diabetes [11] - A 50% success rate in bringing products to market could significantly benefit the company, and clinical wins may positively impact stock performance [12]

Core Insights - Intellia Therapeutics announced positive two-year follow-up data from the Phase 1 trial of investigational nexiguran ziclumeran (nex-z) for hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN) [1][2] - The data presented at the 2025 Peripheral Nerve Society Annual Meeting indicates that a single dose of nex-z leads to significant reductions in serum TTR levels and improvements in neuropathic impairment measures [2][5] ATTRv-PN Results - In the dose-escalation portion (N=15), the mean Neuropathy Impairment Score (NIS) improved by -2.0 at Month 12 and -4.5 at Month 24 [4] - In the dose expansion portion (N=21), the mean NIS improved by -2.1 at Month 12 and -5.2 at Month 24 [4] - The overall mean change in modified NIS +7 (mNIS+7) was -0.6 at Month 12 and -8.5 at Month 24 [4] - Among patients previously on patisiran, the mean mNIS+7 change was -6.3 at Month 12 and -6.5 at Month 24 [4] - The Norfolk Quality of Life-Diabetic Neuropathy (QoL-DN) score improved by -3.5 at Month 12 and -8.5 at Month 24 [4] - Neurofilament light chain (NfL) showed a reduction of -8.6% at Month 12 [4] Safety and Tolerability - Nex-z demonstrated generally favorable safety and tolerability with no new drug-related adverse events reported during the follow-up period [5][7] - The most common treatment-related adverse events were mild to moderate infusion-related reactions, which did not lead to discontinuations [7] Clinical Program and Future Prospects - The ongoing Phase 1 trial is a multi-center study evaluating nex-z in adults with hereditary ATTR amyloidosis [8] - The Phase 3 MAGNITUDE-2 trial is designed to measure clinical outcomes and evaluate the efficacy of a single dose of nex-z, with a potential biologics license application submission by 2028 [5][9] - Nex-z is based on CRISPR technology and aims to be the first one-time treatment for ATTR amyloidosis by inactivating the TTR gene [10]

Core Viewpoint - CRISPR Therapeutics AG is at the forefront of gene editing, having received approval for its first gene editing treatment, CASGEVY, which targets sickle cell disease and beta-thalassemia [2][4]. Company Overview - CRISPR Therapeutics' stock is currently trading at approximately $36.31, showing little change since 2018, and investors from 2020 and 2021 may be facing losses [3][11]. - The company has a significant cash reserve of approximately $1.86 billion as of March 31, 2025, which is expected to support future growth [8]. Product and Market Potential - CASGEVY has been approved in eight jurisdictions and has 65 authorized treatment centers globally, with expectations for significant patient initiation growth by 2025 [6]. - The total addressable market for CASGEVY is estimated to be around 60,000 patients, although this is small compared to larger markets for diseases like cancer and diabetes [6][7]. Future Outlook - The company anticipates that 2026 will mark a turning point, generating enough revenue from CASGEVY to support sustainable growth for ongoing and future clinical trials [8]. - Analysts have a 12-month price target for CRISPR Therapeutics stock at $71.75, indicating a potential upside of 97.60% from the current price [10][12]. Investment Sentiment - Despite the current challenges, over 69% of CRSP stock is owned by institutional investors, and buying has outpaced selling for the last nine quarters [11][12]. - The stock is rated as a Moderate Buy by analysts, although it is not currently considered a top investment compared to other stocks [12][14].

Precision BioSciences (DTIL) Update / Briefing May 15, 2025 08:00 AM ET Speaker0 Thank you for standing by. Hello, and welcome to the Precision Biosciences Business Update Conference Call. I would now like to turn the call over to Naresh Tana, Head of Investor Relations. Please go ahead, sir. Speaker1 Thank you, Dustin. Welcome to Precision Biosciences Muscle Program Update. Thank you all for joining today. I'm joined today by Michael Amoroso, President and Chief Executive Officer of Precision Biosciences. ...

Editas Medicine, Inc. (NASDAQ:EDIT) BofA Securities 2025 Healthcare Conference May 14, 2025 8:15 PM ET Company Participants Amy Parison - CFO Christi Bernett - SVP of IR Conference Call Participants Alec Stranahan - BofA Securities Alec Stranahan Welcome to the last fireside for day 1 of the 2025 Bank of America Healthcare Conference. Thanks for joining this session with Editas. My name is Alec Stranahan. I'm senior biotech analyst at BofA, covering Editas. And I'm pleased to be joined by Amy Parison, Chie ...