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The U.S. Food and Drug Administration Completes Review of Cibus' Altered Lignin Alfalfa Developed in Partnership with S&W Seed Company for use in Livestock Feed
Globenewswire· 2025-06-24 11:00
SAN DIEGO, June 24, 2025 (GLOBE NEWSWIRE) -- Cibus, Inc. (Nasdaq: CBUS) (the "Company"), a leading agricultural technology company that develops and licenses plant traits to seed companies, announced today that it has completed the U.S. Food and Drug Administration's (FDA) Plant Biotechnology Consultation Program for its altered lignin alfalfa trait with the FDA notifying Cibus that it has no further questions concerning the use of altered lignin alfalfa in food or feed. This press release contains "forward ...
Jennifer Doudna on the Future of AI and Gene Editing
Bloomberg Originals· 2025-06-23 14:48
Welcome to the Bloomberg Tech Conference. This is our annual event where we bring together a powerhouse lineup of big tech voices. Oh, we're going to be doing a live taping of the circuit today.So, Dylan, our director of photography, is here up from LA. Look at his little shirt. Says the circuit on it.Say hi for the socials. Hi for the socials. 1 2 3 4 5 6 7 8 9 10.So excited. Please welcome to the stage Jennifer Dana, Nobel Laurate in chemistry and founder of the innovative genomics institute for a convers ...
2 Biotech Stocks to Buy Before They Soar 84% and 240%, According to Certain Wall Street Analysts
The Motley Fool· 2025-06-21 22:10
Core Insights - Biotech stocks present significant growth opportunities due to ongoing research and potential breakthroughs in treatments [1] - Wall Street identifies two biotech candidates with projected gains exceeding 80% and 200% in the next 12 months [2] Company Summaries Viking Therapeutics - Viking Therapeutics (VKTX) experienced a surge in stock price following positive phase 2 trial results for its weight loss drug VK2735, but has since returned to pre-announcement levels [4] - The company is advancing VK2735 in both injectable and pill forms, with high demand for weight loss drugs, indicating potential for future growth [5] - Viking is set to begin phase 3 trials for the injectable version in Q2 and expects phase 2 data for the pill version in the second half of the year, with the weight loss drug market projected to reach $100 billion [6] - Wall Street forecasts a 240% increase in Viking's stock price, contingent on trial outcomes [7] CRISPR Therapeutics - CRISPR Therapeutics (CRSP) saw its stock rise leading up to the approval of its first product, Casgevy, but has since declined as investors locked in gains [8] - The company is positioned for potential growth as Casgevy requires a longer rollout process, with expectations for significant increases in new patient initiations this year [10] - Positive phase 1 data for a gene-editing candidate targeting high cholesterol has been reported, with further updates expected on candidates for lipoprotein(a), oncology, and autoimmune diseases [11][12] - Wall Street anticipates an 84% gain for CRISPR Therapeutics, making it a potential buying opportunity for growth investors [12]
Biotech Stock Surging on Billion-Dollar Buyout
Schaeffers Investment Research· 2025-06-17 13:53
Biotech stock Verve Therapeutics Inc (NASDAQ:VERV) is up 75.3% to trade at $10.99 at last check amid news that peer Eli Lilly (LLY) will buy the company for $10.50 per share, or roughly $1.3 billion. The pair have already been partnering on several drugs, with the move allowing Eli Lilly to push further into gene editing. VERV is now trading at its highest level since April 2024, and sports an 11.2% year-to-date lead. The shares have been using the $6 region and 100-day moving average as support in recent w ...
Lilly to acquire Verve Therapeutics to advance one-time treatments for people with high cardiovascular risk
Prnewswire· 2025-06-17 10:45
Core Viewpoint - Eli Lilly and Company has announced a definitive agreement to acquire Verve Therapeutics, a clinical-stage company focused on genetic medicines for cardiovascular disease, with the aim of transforming treatment from chronic care to a single-dose solution [1][2][3] Company Overview - Verve Therapeutics is developing gene editing medicines targeting atherosclerotic cardiovascular disease (ASCVD), with its lead program VERVE-102 focusing on the PCSK9 gene, which is linked to cholesterol levels [2][7] - VERVE-102 is currently in a Phase 1b clinical trial and has received Fast Track designation from the U.S. FDA, indicating its potential significance in treating conditions like heterozygous familial hypercholesterolemia [2][3] Acquisition Details - Under the acquisition agreement, Lilly will offer $10.50 per share in cash for Verve's outstanding shares, totaling approximately $1.0 billion, with potential additional contingent value rights (CVR) bringing the total to up to $13.50 per share, or approximately $1.3 billion [3][4] - The purchase price represents a premium of about 113% over Verve's 30-day volume-weighted average trading price as of June 16, 2025 [4] - The transaction is expected to close in the third quarter of 2025, subject to customary closing conditions [3][4] Strategic Rationale - Lilly's acquisition of Verve is seen as a strategic move to enhance its capabilities in cardiometabolic disease and genetic medicines, aligning with Verve's vision to provide lifelong cardiovascular risk reduction through innovative treatments [1][3] - The collaboration is anticipated to accelerate the development of Verve's gene editing programs, addressing significant unmet medical needs in cardiovascular care [3][7] Shareholder Support - Verve's board of directors unanimously recommends that shareholders tender their shares in the tender offer, and key stakeholders representing approximately 17.8% of Verve's outstanding common stock have signed support agreements [5][4]
Intellia Therapeutics Announces Positive Three-Year Data from Phase 1 Trial of Lonvoguran Ziclumeran (lonvo-z) in Patients with Hereditary Angioedema (HAE) at the European Academy of Allergy and Clinical Immunology Congress
Globenewswire· 2025-06-15 14:45
Core Insights - Intellia Therapeutics announced promising three-year follow-up data from the Phase 1 portion of its ongoing Phase 1/2 study for lonvoguran ziclumeran (lonvo-z) in patients with hereditary angioedema (HAE) [1][2] - All 10 patients in the Phase 1 study were attack-free and treatment-free for a median of nearly two years, demonstrating the potential of lonvo-z as a one-time therapy [2][6] - The global Phase 3 HAELO trial has completed screening ahead of schedule, with over half of the patients screened in the United States [4][6] Clinical Results - A single dose of lonvo-z led to a mean reduction in monthly HAE attack rate of 98% compared to pre-treatment baseline [2][6] - Patients showed deep, dose-dependent, and durable reductions in plasma kallikrein protein levels throughout the study [2][6] - The treatment was well tolerated, with the most frequent adverse events being infusion-related reactions, which were mostly Grade 1 and resolved without complications [3][6] Development Plans - The ongoing Phase 3 HAELO trial is randomized, double-blind, and placebo-controlled, assessing the safety and efficacy of lonvo-z at the 50 mg dosage [4][5] - Intellia plans to submit a biologics license application (BLA) in 2026, aiming for a U.S. launch in 2027 [4][8] - New data from the Phase 2 portion of the ongoing Phase 1/2 study is expected to be presented in the second half of 2025 [4][10] About Lonvo-z - Lonvo-z is based on CRISPR/Cas9 technology and aims to prevent HAE attacks by inactivating the kallikrein B1 (KLKB1) gene [8] - The therapy has received multiple regulatory designations, including Orphan Drug and RMAT Designation from the FDA [8][9] - Intellia is focused on leveraging gene editing technology to develop novel therapies that address unmet medical needs [9]
ElevateBio CEO Ger Brophy on AWS partnership for AI technology
CNBC Television· 2025-06-12 17:00
So what most people realize is, you know, there's amazing cell and gene therapies coming through in the market at the moment and they're done and actuated in different ways. Uh crisper gene editing is a way in which you can make very precise edits to a patient's DNA. And what that allows you to do is identify new targets, but also to correct some diseases, more importantly expand the number of treatable diseases.And we're working with our partners. We've built the world's most powerful crisper gene editing ...
CNBC Disruptor 50: ElevateBio CEO Ger Brophy on AI and CRISPR for rare disease treatments
CNBC Television· 2025-06-12 11:47
Crispr Technology & Applications - Crispr 基因编辑技术能够对患者的 DNA 进行精确编辑,从而识别新的治疗靶点并纠正某些疾病,扩大可治疗疾病的范围 [2] - Crispr 技术与 AI 结合,加速识别靶点和疗法,从而加快新疗法上市的速度 [3] - 癌症,尤其是无法以其他方式治疗的儿童疾病和罕见癌症,可能是 Crispr 技术首先产生影响的领域 [5] Regulatory Landscape - FDA 对新的治疗方法和技术平台持支持态度,并倾向于快速批准 [5] - 美国卫生与公众服务部 (HHS) 部长承诺支持细胞和基因治疗领域,这对行业来说是一个巨大的推动力 [7] - 众议院拨款委员会也表达了对细胞和基因治疗领域的支持,释放了积极信号 [7] Manufacturing & Supply Chain - 该公司主要在美国本土进行生产,对进口尤其是来自中国的依赖程度不高 [9] - 公司在马萨诸塞州进行生产,并在匹兹堡进行扩张 [9] - 匹兹堡的团队致力于支持当地学校的 STEM (科学、技术、工程和数学) 项目 [9]
Verve Therapeutics (VERV) FY Conference Transcript
2025-06-09 16:20
Summary of Verve Therapeutics (VERV) FY Conference Call - June 09, 2025 Company Overview - **Company**: Verve Therapeutics - **Focus**: Gene editing for cardiovascular disease, specifically targeting lifelong cholesterol lowering to treat coronary heart disease Key Points and Arguments 1. **HEART-two Data Announcement**: The company recently disclosed data from the HEART-two trial, which showed promising results for their gene editing therapy aimed at lowering LDL cholesterol levels [3][4] 2. **Unmet Need**: Current cholesterol-lowering therapies have a high dropout rate, with up to 50% of patients discontinuing treatment within a year. Verve aims to provide a one-time therapy that offers lifelong efficacy [5] 3. **Efficacy Results**: In the HEART-two trial, the therapy demonstrated an LDL reduction of up to 60%, with a mean reduction of 59% in the highest dose group. All patients in this group experienced over 50% lowering [8][21] 4. **Safety Profile**: The therapy was well tolerated, with only one infusion-related reaction reported among 14 patients. No significant laboratory abnormalities were observed [8][9] 5. **Durability of Effect**: The company reported that the LDL reduction was sustained at 60% two years post-infusion, supporting the vision of a one-time therapy for atherosclerotic cardiovascular disease [10][11] 6. **Comparison of Products**: The difference between the first-generation product (VRRB101) and the second-generation product (VRRB102) lies in the delivery system, which has been improved to enhance safety and efficacy [12][14] 7. **Regulatory Environment**: Recent discussions with the FDA indicate a supportive stance towards gene editing technologies, particularly those addressing chronic diseases like atherosclerosis [45][48] 8. **Market Research Findings**: Surveys indicate a significant openness among patients and physicians towards a one-time gene editing therapy, with about one-third of patients preferring this option over traditional therapies [50][53] 9. **Cost of Manufacturing**: The estimated cost to manufacture the therapy is around $3 per dose, suggesting potential for flexible pricing strategies compared to traditional gene therapies [55][56] 10. **Partnership with Eli Lilly**: The company is preparing to present data to Eli Lilly for potential opt-in for further development, which could extend Verve's cash runway [62][69] Additional Important Insights - **Dosing Strategy**: The company is shifting towards fixed dosing rather than weight-based dosing, as the therapy primarily targets the liver [24][26] - **Long-term Safety Considerations**: The company believes that the low risk of off-target effects, combined with the significant risk of untreated cardiovascular disease, will favor the adoption of their therapy [40][41] - **Future Developments**: Verve is also working on additional products targeting ANGPTL3 and Lp(a), leveraging the same delivery system as VRRB102 [64][66] This summary encapsulates the critical insights from the conference call, highlighting the company's innovative approach to gene editing in cardiovascular disease and its potential impact on patient care and market dynamics.
Prime Medicine (PRME) 2025 Conference Transcript
2025-06-05 15:30
Summary of Prime Medicine Conference Call Company Overview - **Company**: Prime Medicine (PRME) - **Industry**: Biotechnology, specifically gene editing technology Core Points and Arguments 1. **Introduction of Prime Medicine**: The company focuses on prime editing technology, which is described as a next-generation gene editing platform that is versatile and safe for editing genomes, with applications in genetic diseases, oncology, and immunology [4][5][6] 2. **Recent Developments**: Prime Medicine has shown data for its first patient in humans, indicating the potential of prime editing as a cure for chronic granulomatous disease (CGD) [6][24][26] 3. **Pipeline Focus**: The company is concentrating on three major commercial opportunities: Wilson's disease, alpha-1 antitrypsin disease (AATD), and cystic fibrosis, alongside a partnership with Bristol-Myers Squibb for CAR T cell therapies [7] 4. **Comparison with Other Technologies**: Prime editing is positioned as superior to CRISPR and base editing due to its ability to make precise edits without double-stranded breaks, thus minimizing off-target effects [9][17][18] 5. **LNP Delivery System**: Prime Medicine has developed a robust lipid nanoparticle (LNP) platform that has shown high editing efficiency in the liver and is being evaluated for lung delivery in cystic fibrosis [20][21] 6. **Chronic Granulomatous Disease (CGD)**: The company reported rapid engraftment in the first patient treated for CGD, with DHR levels indicating functional neutrophils at 58% and 66% at 15 and 30 days post-treatment, respectively [24][25][26] 7. **Wilson's Disease Program**: Prime Medicine plans to file an IND or CTA in the first half of 2026, targeting approximately 20,000 patients in the US and EU, with preclinical data showing high editing efficiency [32][33][34] 8. **AATD Program**: The company aims to differentiate its AATD program from competitors by focusing on achieving wild-type protein levels, addressing the limitations of existing therapies [53][56][61] Important but Overlooked Content 1. **Cash Position**: As of March, the company reported a cash position of $158 million, which is expected to last through the first half of the following year, following a restructuring that reduced operational costs by 50% [72][73] 2. **Business Development Opportunities**: There is ongoing interest in Prime Medicine's technology, with potential for future deals, although no specific agreements have been promised [75] 3. **Translatability of Preclinical Data**: The company acknowledges the challenges in translating preclinical data to human outcomes but expresses confidence based on observed efficiencies and safety profiles [35][36][37] This summary encapsulates the key points discussed during the conference call, highlighting Prime Medicine's strategic focus, technological advantages, and future plans in the biotechnology sector.