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Cibus, Inc. Announces Pricing of $27.5 Million Public Offering
Globenewswire· 2025-06-05 12:30
SAN DIEGO, June 05, 2025 (GLOBE NEWSWIRE) -- Cibus, Inc. (Nasdaq: CBUS) (the "Company" or "Cibus"), a leading agricultural biotechnology company that uses proprietary gene editing technologies to develop plant traits (or specific genetic characteristics) in seeds, today announced the pricing of its previously announced public offering of 15,714,285 shares of its Class A Common Stock, at a purchase price of $1.75 per share, including to institutional and strategic investors, as well as the Chairman of Cibus' ...
BEAM Up on FDA's Orphan Drug Status for Gene-Editing Therapy
ZACKS· 2025-06-04 16:01
Core Insights - Beam Therapeutics (BEAM) has received orphan drug designation from the FDA for its investigational genome-editing candidate, BEAM-101, aimed at treating sickle cell disease (SCD) [1][7] - The orphan drug designation provides Beam with seven years of market exclusivity post-approval, along with exemptions from FDA application fees and tax credits for qualifying clinical studies [2][7] - Following the announcement, Beam's shares increased by 4.3%, although the stock has seen a 32% decline year-to-date compared to a 2.9% decline in the industry [2][3] Development Activities - BEAM-101 is the lead candidate in Beam's hematology franchise, currently undergoing evaluation in the phase I/II BEACON study for adult SCD patients [4] - Preliminary data from the BEACON study indicated that BEAM-101 treatment resulted in a significant and lasting increase in fetal hemoglobin and a decrease in sickle hemoglobin [5][7] - Updated data from the BEACON study is anticipated to be presented at an upcoming European Hematology Association conference [8] Competitive Landscape - Other companies utilizing CRISPR/Cas9 technology include CRISPR Therapeutics and Intellia Therapeutics, both of which are developing therapies for blood disorders [9][10] - CRISPR Therapeutics, in collaboration with Vertex Pharmaceuticals, has received approvals for its CRISPR/Cas9 gene-edited therapy, Casgevy, for SCD and transfusion-dependent beta thalassemia [10] - Intellia Therapeutics is advancing its investigational therapies, including NTLA-2001 for ATTR amyloidosis and NTLA-2002 for hereditary angioedema, both in late-stage development [11]
Verve Therapeutics (VERV) 2025 Conference Transcript
2025-06-04 14:22
Verve Therapeutics (VERV) 2025 Conference June 04, 2025 09:20 AM ET Speaker0 Okay. Welcome everyone to twenty twenty five Jefferies Global Healthcare Conference. It it is my my name is Roger Song, one of the senior analysts covers MidCap biotech in US. And then it's my pleasure to have the next five star chat with our company, Zach, from the so excuse me. So from the Verve Therapeutics. So it it is a it is it is a very exciting timing for Verve because they just reported a very exciting data from the one tw ...
CRISPR Therapeutics (CRSP) FY Conference Transcript
2025-06-03 17:20
Summary of CRISPR Therapeutics (CRSP) FY Conference Call - June 03, 2025 Company Overview - CRISPR Therapeutics is focused on creating transformative gene-based medicines for serious diseases, leveraging its Nobel Prize-winning CRISPR technology [3][4][32]. Key Products and Pipeline - **KASJEVY**: The flagship product for treating sickle cell disease, with a global addressable market of approximately 60,000 patients and a price point of $2 million, creating a multibillion-dollar market opportunity [10][11]. - **Clinical Trials**: Several ongoing clinical trials with readouts expected in the next 6 to 12 months, which will shape the company's strategic direction [4][8]. - **Franchises**: The company has four key franchises: 1. **Hematology**: KASJEVY as the anchor product. 2. **CAR T Platform**: Targeting oncology and autoimmune diseases using CRISPR technology [5][14]. 3. **In Vivo Platform**: Developing therapies for cardiovascular diseases, including a one-time infusion to reduce triglycerides and LDL cholesterol [6][17]. 4. **Type 1 Diabetes**: Aiming to provide insulin-producing pancreatic islet cells [6][28]. Financial Highlights - Strong balance sheet with $1.86 billion in cash, allowing for purposeful spending and investment in growth opportunities [8][31][50]. - Anticipation of significant revenue generation from KASJEVY and other pipeline products, with a focus on achieving profitability in the future [49][50]. Recent Developments - **Partnership with Vertex**: Vertex is leading the commercialization of KASJEVY, with CRISPR receiving 40% of the net income from the program [31][45]. - **Business Development Deal**: Acquisition of siRNA technology from Sirius Therapeutics targeting Factor XI, which presents a multibillion-dollar opportunity in the anticoagulation space [8][31][33]. Clinical Data and Efficacy - **CTX-310**: Early data shows a 56% average reduction in triglycerides and a 28% reduction in LDL cholesterol, with some patients experiencing reductions of up to 82% and 65%, respectively [19][20][21]. - **CTX-112**: In oncology trials, a 67% overall response rate and a 50% complete response rate were reported, indicating promising efficacy [15][16]. Market Opportunities - Expansion into untapped markets, particularly in the Middle East, with significant patient populations for KASJEVY [12][48]. - Potential for addressing underserved populations in Africa and other regions with innovative therapies [13][45]. Strategic Vision - CRISPR aims to become a sector-leading biotech company with a sustainable business model, focusing on innovation and the development of a diverse pipeline [8][32][40]. - The company is committed to maintaining a high hurdle rate for new assets and partnerships, ensuring that any new initiatives align with its long-term goals [39][40]. Conclusion - 2025 is viewed as a pivotal year for CRISPR Therapeutics, with multiple clinical data readouts and the ongoing launch of KASJEVY expected to drive growth and establish the company as a leader in the biotech sector [31][32].
Verve Therapeutics to Participate in Upcoming Investor Conferences
Globenewswire· 2025-05-28 11:00
Investor Contact Jen Robinson Verve Therapeutics, Inc. jrobinson@vervetx.com Media Contact Ashlea Kosikowski 1AB ashlea@1abmedia.com Jefferies Global Healthcare Conference on Wednesday, June 4, 2025, at 9:20 a.m. ET in New York, NY Goldman Sachs 46 Annual Global Healthcare Conference on Monday, June 9, 2025, at 11:20 a.m. ET in Miami, FL Live webcasts will be available in the investor section of the company's website at www.vervetx.com. The webcasts will be archived for 30 days following the fireside chats. ...
Cibus Provides Webcast of BofA Securities AgTech Virtual Conference Fireside Chat
Globenewswire· 2025-05-27 11:00
SAN DIEGO, May 27, 2025 (GLOBE NEWSWIRE) -- Cibus, Inc. (Nasdaq: CBUS) (the "Company"), a leading agricultural biotechnology company that uses proprietary gene editing technologies to develop plant traits (or specific genetic characteristics) in seeds, today announced that Peter Beetham, Co-Founder, President and Interim Chief Executive Officer, participated in the BofA Securities 2025 AgTech Virtual Conference this week. During the fireside chat, hosted by BofA research analyst Stephen Byrne, management di ...
Cibus (CBUS) Conference Transcript
2025-05-21 21:40
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.
Verve Therapeutics (VERV) 2025 Conference Transcript
2025-05-21 20:35
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 Transcript
2025-05-21 20:15
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 ...
新型基因编辑工具精准嵌入完整基因
news flash· 2025-05-20 22:07
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 ...