在进行生物分布进行表征时，研究团队还观察到 AAV.CPP.16 对 肺部 的转导作用比 AAV9 更强。因此，研究团队决定进一步探索 AAV.CPP.16 作为鼻内基因递 送载体，在基因治疗和基因编辑方面的应用，以期用于潜在的呼吸系统疾病和空气传播病毒感染的治疗。 2025 年 5 月 22 日，哈佛医学院 Fengfeng Bei 教授、昆明医科大学第二附属医院 蒲军 教授及上海交通大学医学附属第九人民医院 范先群 院士团队合作 （ 阳 志 、 Yizheng Yao 为共同第一作者） ，在 Cell 子刊 Cell Reports Medicine 上发表了题为 ： Cross-species tropism of AAV.CPP.16 in the respiratory tract and its gene therapies against pulmonary fibrosis and viral infection 的研究论文 【2】 。 撰文丨王聪 编辑丨王多鱼 排版丨水成文 呼吸系统 的基因治疗和基因编辑的进展，因缺乏有效的递送载体而受阻。 腺相关病毒 （AAV） 是最常用的体内基因传递病毒 ...

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 ...

Core Viewpoint - A new gene editing tool named evoCAST has been developed by a team from MIT and Harvard's Broad Institute in collaboration with scientists from Columbia University, enabling precise insertion of complete genes into specific locations in the human genome, marking a significant advancement in the treatment of genetic diseases [1] Group 1 - The evoCAST tool allows for the precise embedding of complete genes or multiple genes into the human genome [1] - This development represents a breakthrough in the treatment of hereditary diseases [1] - The research findings have been published in the latest issue of the journal Science [1]

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]

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. ...

Corteva (CTVA) Conference Call Summary Company Overview - Corteva is a leading seed and crop protection company [1] - The conference featured CFO David Johnson and CTO Sam Eathington discussing the company's performance and outlook [2] Financial Performance - Corteva reported a 15% year-over-year increase in EBITDA for Q1, totaling $1.2 billion [3] - Pricing for seeds was in line with expectations, while crop protection prices saw a slight decline [3] - New products and biologicals in crop protection experienced double-digit growth [4] - The company anticipates $400 million in cost improvements for the year, with $200 million already realized in Q1 [5] - Foreign exchange (FX) impacts were significant, with a $90 million headwind in Q1 and an expected total negative impact of $275 million for the year [6][11] Market Outlook - Corteva's guidance for the year includes a 10% increase in EBITDA, projecting $3.7 billion [9] - Crop protection pricing is expected to be low single-digit negative in the second half of the year, contrasting with earlier expectations of flat pricing [10] - The company is optimistic about volume growth, particularly in biologicals and new products [11] Industry Dynamics - Corteva's performance in Q1 was notably better than competitors like FMC and Bayer, attributed to increased planted acres and successful new product launches [14][15] - The company is monitoring grower sentiment, which remains positive, with corn acres being planted as expected [24][25] - In South America, there is potential for growth, particularly in Brazil, with expectations of a low to mid single-digit price mix lift [30] Strategic Initiatives - Corteva is focused on mitigating tariff impacts, estimating a potential $50 million effect, but believes it can manage this through supply chain options and pricing adjustments [17][21] - The company is investing in gene editing and other technologies to enhance crop yields and disease resistance, with significant potential for future growth [49][52] - Corteva aims to achieve royalty neutrality by 2028, transitioning from paying royalties to licensing its own traits [80][82] Research and Development - R&D spending is stable at about 8% of sales, with a focus on efficiency and effectiveness [76][79] - The integration of recent acquisitions, such as Stoller and Symborg, is progressing well, with positive yield results in Brazil [72][73] Long-term Growth Drivers - Key future products include the fungicide Aviza and hybrid wheat, both expected to contribute significantly to revenue [58][60] - Gene editing is anticipated to double yield gains, creating substantial value in the agricultural sector [62] Conclusion - Corteva is positioned as a technology-driven company with multiple growth opportunities, focusing on innovation in seed and crop protection [93][96] - The management emphasizes the importance of long-term strategies and the potential for significant market impact through advancements in agricultural technology [96]

Core Insights - Editas Medicine has achieved therapeutically relevant gene editing levels in the HBG1/2 promoter and a favorable biodistribution profile in non-human primates, supporting the development of treatments for sickle cell disease and beta thalassemia [1][2]. Group 1: Study Findings - New data from preclinical studies in non-human primates will be presented at the European Hematology Association (EHA) 2025 Congress, highlighting the potential of Editas' proprietary HSC-tLNP for gene editing [1][2]. - The study demonstrated editing levels exceeding the predicted threshold of ≥25% required for therapeutic benefit with a single dose [5]. Group 2: Presentation Details - The poster presentation titled "Targeted Lipid Nanoparticle Delivery Enables In Vivo HBG1/2 Genome Editing In Non-Human Primates" is scheduled for June 14, 2025, at the Allianz MiCo in Milan, Italy [4]. - The presentation will be accessible on the Editas Medicine website during the conference [3]. Group 3: Company Overview - Editas Medicine focuses on translating CRISPR/Cas12a and CRISPR/Cas9 genome editing systems into in vivo medicines for serious diseases [4]. - The company is the exclusive licensee of Broad Institute's Cas12a and Cas9 patent estates for human medicines [4].

Core Insights - Metagenomi, Inc. has made significant advancements in gene editing technologies, particularly for treating neurological disorders through extrahepatic in vivo gene editing [1][2] - The company presented three abstracts at the ASGCT 28 Annual Meeting, showcasing its proprietary gene editing toolbox, including compact nucleases and CRISPR-associated transposases (CAST) [1][2] Gene Editing Technologies - The metagenomics discovery platform utilizes natural microbial evolution and AI-guided protein optimization to identify and enhance novel gene editing systems [2] - Compact Type II and Type V nucleases have been developed for efficient in vivo editing, with a focus on delivering large therapeutic genes [2][9] - The CAST system has shown promise in addressing challenges related to targeted and precise large gene integration [2] Presentation Highlights - A compact type II CRISPR system (MG21-1) demonstrated up to 69% target protein reduction in the CNS with localized AAV9 delivery [6] - An ultra-compact type V nuclease (MG119-28) achieved over 50% restoration of dystrophin protein levels in DMD model myocytes and 64% knockdown of target protein in the motor cortex [6] - The type V-K CAST system has improved integration efficiency by over 50 times compared to natural systems, indicating its potential for therapeutic applications [9] Company Overview - Metagenomi leverages AI and machine learning to develop next-generation genome editing therapeutics, analyzing over 7.4 billion proteins to target various genetic mutations [7] - The company has a lead program in Hemophilia A, aiming to provide lifelong protection from bleeding events and joint damage [7]

Summary of Intellia Therapeutics Conference Call Company Overview - **Company**: Intellia Therapeutics - **Industry**: Gene Editing and Biotechnology - **Focus**: In vivo applications of gene editing with a focus on late-stage programs and clinical studies [4][5] Key Assets and Programs - **Phase III Studies**: Two assets with three Phase III studies in progress, targeting different indications [4][5] - **ATTR Program**: Focused on TTR (transthyretin) amyloidosis with two Phase III studies, one for Polyneuropathy (PN) and one for Centimeters [6][8] - **HAE Program**: A pivotal Phase III program for Hereditary Angioedema (HAE) with aggressive enrollment and a timeline for updates [33][36] Market Dynamics and Competitive Landscape - **ATTR Market**: Increasing diagnosis rates and a high level of unmet need despite existing therapies. The company believes its approach to significantly reduce TTR levels will provide a competitive edge [6][7][10] - **Centimeters Opportunity**: The company is optimistic about the trajectory of the Centimeters opportunity, with strong enrollment momentum and investigator enthusiasm [8][10] - **HAE Market**: Aiming for a paradigm shift from chronic therapy to a one-time treatment, targeting zero attacks and no ongoing therapy for patients [37][38] Clinical Data and Efficacy - **TTR Knockdown**: The company has demonstrated consistent TTR knockdown in Phase I studies, with a nadir reached in 28 days [16][22] - **Durability of Response**: Emphasis on the importance of durability in treatment response, with ongoing data collection to support long-term efficacy [11][13][48] - **Functional Endpoints**: The studies are tracking various functional endpoints, including mortality, hospitalization, and quality of life metrics [18][19] Financial and Operational Strategy - **Cash Position**: The company has a cash balance exceeding $700 million, which is expected to support the execution of Phase III programs and commercial infrastructure development [52][54] - **Restructuring**: A recent restructuring aimed at focusing on key research areas while ensuring financial stability and operational efficiency [53][55] - **Market Strategy**: Plans to build the company through successful launches in smaller markets before tackling larger opportunities [25][27] Regulatory and Market Engagement - **FDA Interactions**: The company maintains a positive relationship with the FDA, running gold standard randomized placebo-controlled studies, which are expected to align with regulatory expectations [58][59] - **Market Research**: Ongoing market research to understand patient and physician perspectives, which will inform commercial strategies [35][36] Future Outlook - **Upcoming Milestones**: Anticipated data releases and updates on clinical trials over the next 12 to 18 months, with a focus on building a robust safety database and demonstrating treatment efficacy [30][56] - **Long-term Vision**: The company aims to leverage its gene editing technology to provide significant benefits to patients and the healthcare system, with a focus on sustainable growth and value creation [11][31][39]