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Origin Agritech Strengthens Gene Editing Platform through Patent License Agreement with Shunfeng BioTech
Prnewswire· 2025-11-10 12:00
Core Viewpoint - Origin Agritech Ltd. has entered into a patent license agreement with Shandong Shunfeng Biotechnology Co., Ltd. for the Cas-SF01 gene editing tool, enhancing its capabilities in gene editing technologies for corn breeding [1][5][6] Group 1: Partnership and Technological Advancements - The collaboration builds on Origin Agritech's long-term relationship with China Agricultural University, focusing on key regulatory genes for corn leaf angle [2] - Using Shunfeng BioTech's Cas-SF01 platform, researchers developed edited corn induction lines, ZmRAVL1-KO1 and ZmDWF4-KO1, which optimize corn leaf angle for better yield potential [3][4] Group 2: Strategic Objectives and Commercialization - The edited induction lines aim to achieve a "smart plant architecture," improving light interception and airflow, thus allowing for higher planting density and greater yield [3][4] - Origin Agritech has initiated the biosafety assessment process for the edited lines, with an estimated timeline of 1-2 years for obtaining final biosafety certification [5] Group 3: Innovation and Future Goals - The patent license agreement is a significant step in Origin Agritech's gene editing breeding strategy, integrating research resources with innovative biotechnological tools [6] - The company is committed to advancing its gene editing innovations to contribute to agricultural technology and national food security in China [6]
CRISPR Therapeutics Announces Positive Phase 1 Clinical Data for CTX310® Demonstrating Deep and Durable ANGPTL3 Editing, Triglyceride and Lipid Lowering
Globenewswire· 2025-11-08 14:56
Core Insights - CRISPR Therapeutics announced positive Phase 1 clinical trial results for CTX310, a CRISPR/Cas9 gene-editing therapy targeting ANGPTL3, demonstrating significant reductions in triglycerides and LDL cholesterol after a single intravenous infusion [1][2][11] Clinical Data Summary - The Phase 1 trial showed a mean reduction in circulating ANGPTL3 of -73% (maximum -89%), triglycerides (TG) by -55% (maximum -84%), and low-density lipoprotein (LDL) by -49% (maximum -87%) at the highest dose [1][12] - Among participants with elevated baseline TG (>150 mg/dL), a mean reduction of 60% in TG was observed at therapeutic doses [1][12] - CTX310 was well tolerated with no serious adverse events related to treatment and no significant changes in liver transaminases [1][7][9] Safety and Tolerability - The trial included 15 participants who received ascending doses of CTX310, with all participants completing at least 28 days of follow-up [7][9] - Adverse events were generally mild to moderate, with one allergic reaction and infusion-related reactions in three participants, all of which resolved [8][9] Efficacy Highlights - The results indicate that CTX310 has the potential to provide durable lipid-lowering effects following a single-course IV administration, which could transform treatment for patients with severe dyslipidemia [11] - The study's findings support the advancement of CTX310 into Phase 1b clinical trials, focusing on severe hypertriglyceridemia and mixed dyslipidemia [11] Company Overview - CRISPR Therapeutics is a leading gene editing company focused on developing transformative medicines for serious diseases, with a diversified pipeline that includes CTX310 and other investigational programs targeting cardiovascular diseases [14]
Cellectis Reports Third Quarter 2025 Financial Results and Provides Business Update
Globenewswire· 2025-11-07 21:30
Core Insights - Cellectis reported promising clinical data for its product candidates lasme-cel (UCART22) and eti-cel (UCART20x22), indicating their potential to improve outcomes in relapsed/refractory B-cell acute lymphoblastic leukemia (r/r B-ALL) and non-Hodgkin lymphoma (r/r NHL) respectively [3][4][11] Financial Results - As of September 30, 2025, Cellectis had $225 million in cash, cash equivalents, and fixed-term deposits, which is projected to fund operations into the second half of 2027 [4][26] - Consolidated revenues and other income for the nine-month period ended September 30, 2025, were $67.4 million, up from $34.1 million in the same period in 2024, primarily driven by activities related to the AstraZeneca collaboration [29] - Consolidated net loss attributable to shareholders for the nine-month period ended September 30, 2025, was $41.3 million, compared to a loss of $42.7 million in the same period in 2024 [34] Clinical Development - The Phase 1 BALLI-01 study of lasme-cel showed an overall response rate (ORR) of 68% with the Cellectis-manufactured product, and 100% in the target Phase 2 population [6][7] - The first interim analysis for the pivotal Phase 2 BALLI-01 trial is expected in Q4 2026, with a Biologics License Application (BLA) anticipated in 2028 [8][9] - For eti-cel, preliminary data indicated an ORR of 86% and a complete response (CR) rate of 57% in the ongoing study [11] Pipeline Highlights - Lasme-cel is positioned as a potentially transformative therapy for r/r B-ALL, with a projected peak gross sales potential of approximately $700 million by 2035, which could increase to $1.3 billion with label expansions [9] - The company plans to present further updates on eti-cel at the ASH 2025 Annual Meeting [10][14] Innovation and Collaboration - Cellectis is advancing its gene editing technology, including the use of circular single-stranded DNA (CssDNA) as a non-viral template for gene therapy, which has shown high gene insertion frequency in hematopoietic stem and progenitor cells [16][22] - A strategic collaboration with AstraZeneca aims to develop up to 10 novel cell and gene therapy products targeting high unmet medical needs [23]
SOHM Inc. Releases White Paper on ABBIE, a Cas9-Guided Integrase Enabling Stable Gene Integration and Creation of SKOV3-GYS1 Cell Line for Drug Discovery
Accessnewswire· 2025-11-06 14:30
CHINO HILLS, CALIFORNIA / ACCESS Newswire / November 6, 2025 / SOHM, Inc. (OTCID:SHMN), a pharmaceutical and biotechnology company specializing in generic drugs and gene-editing tools, a leader in Gene Editing and Cell Engineering is pleased to announced the release of a technical white paper describing ABBIE, a Cas9-guided integrase platform that enables donor-DNA integration without double-strand breaks or viral vectors. Using ABBIE, the team established SKOV3-GYS1, an ovarian cancer cell line with stable ...
CRISPR Therapeutics Options Trading: A Deep Dive into Market Sentiment - CRISPR Therapeutics (NASDAQ:CRSP)
Benzinga· 2025-11-05 17:01
Core Insights - Financial giants are showing a bearish sentiment towards CRISPR Therapeutics, with 44% of traders exhibiting bearish tendencies compared to 33% who are bullish [1] - The analysis of options history indicates a significant interest in a price range of $35.0 to $70.0 for CRISPR Therapeutics over the past quarter [2][3] Options Trading Overview - A total of 9 unusual trades were identified, with 4 puts valued at $169,270 and 5 calls valued at $277,530 [1] - The largest options trades observed include a bearish call trade with a strike price of $70.00 and a total trade price of $136.5K, alongside several neutral and bearish put trades [9] Company Performance and Analyst Insights - CRISPR Therapeutics is focused on gene editing using Crispr/Cas9 technology, with its first approved drug targeting sickle-cell disease and transfusion-dependent beta-thalassemia [10][11] - Recent analyst insights suggest an average target price of $87.0, with one analyst maintaining a Buy rating at a target price of $93 and another adjusting their target to $81 [12][13] Current Market Position - The trading volume for CRISPR Therapeutics stands at 772,300, with the stock price at $56.26, reflecting a decrease of -0.27% [15]
SOHM Notes FDA's Evolving Gene-Editing Framework in Context of Ongoing ABBIE Platform Development
Accessnewswire· 2025-11-04 14:30
Core Insights - SOHM, Inc. is a pharmaceutical and biotechnology company focused on generic drugs and gene-editing tools, recognized as a leader in Gene Editing and Cell Engineering [1] - Recent FDA commentary and industry analyses indicate an evolution in regulatory pathways for genome-editing technologies, emphasizing the growing regulatory focus on precision-engineered genetic tools [1] - The developments also highlight advancements in non-viral delivery approaches and scalable manufacturing frameworks within the industry [1]
Precision BioSciences Reports Third Quarter 2025 Financial Results and Provides Business Update
Businesswire· 2025-11-03 12:01
Core Insights - Precision BioSciences, Inc. reported financial results for Q3 2025, highlighting significant advancements in its gene editing pipeline and promising Phase 1 safety and efficacy data for its product PBG [1] Financial Performance - The company announced its financial results for the third quarter ended September 30, 2025, indicating progress in its operations [1] Business Update - Precision BioSciences emphasized strong progress in its gene editing pipeline throughout the third quarter, showcasing the effectiveness of its proprietary ARCUS® platform [1]
Intellia Therapeutics, Inc. (NASDAQ:NTLA) Faces Clinical Trial Challenges but Holds Potential for Growth
Financial Modeling Prep· 2025-10-28 18:22
Core Insights - Intellia Therapeutics, Inc. is a clinical-stage biotechnology company focused on gene editing using CRISPR/Cas9 technology, with a price target set at $29 by JMP Securities, indicating a potential 96% increase from its current trading price of $14.79 [1][5] Company Overview - Intellia specializes in developing treatments for genetic diseases and competes with other biotech firms like CRISPR Therapeutics and Editas Medicine [1] - The company's current stock price is $13.97, reflecting a 5.54% decrease, with a market capitalization of approximately $1.5 billion [4][5] Clinical Trials and Challenges - Intellia is facing challenges with its MAGNITUDE clinical trials for the Nex-Z treatment due to a safety event that led to a protocol pause, impacting the development timeline [2][5] - The pause was initiated after a serious liver issue in a patient, resulting in a temporary halt in dosing and screening for the one-time gene-editing therapy aimed at heart conditions [2] Communication and Consultation - The announcement regarding the trial pause was discussed in a conference call with Intellia's leadership, including CEO John Leonard and CMO David Lebwohl, involving major financial institutions like Jefferies LLC and Goldman Sachs Group, Inc. [3] - This precautionary measure aims to ensure patient safety and includes consultations with experts and regulatory authorities [3]
Crispr And Intellia:Advances In Vivo Gene Editing As 2025 Results Strengthen Outlook(CRSP)
Seeking Alpha· 2025-10-24 12:15
Core Insights - The article discusses the performance and outlook of Crispr Therapeutics (NASDAQ: CRSP) and Intellia Therapeutics (NASDAQ: NTLA) in the context of the gene therapy sector, which has been facing regulatory uncertainties and pricing challenges [1]. Company Analysis - Crispr Therapeutics and Intellia Therapeutics are highlighted as key players in the gene therapy market, with a focus on their potential to address various clinical needs through novel treatments [1]. - The author has a beneficial long position in Crispr Therapeutics, indicating confidence in the company's future performance [2]. Industry Context - The gene therapy sector is currently experiencing challenges due to regulatory uncertainties, which may impact stock performance and investor sentiment [1].
Precision BioSciences (NasdaqCM:DTIL) FY Conference Transcript
2025-10-21 14:30
Summary of Precision BioSciences FY Conference Call Company Overview - **Company**: Precision BioSciences (NasdaqCM:DTIL) - **Technology**: Proprietary gene editing technology called Arcus, which is a nuclease-based system distinct from CRISPR Cas technologies [4][5] Key Points Discussed Industry Context - **Gene Editing Technologies**: The session focused on advancements in genome editing technologies, particularly the applications of gene insertion and targeting viral genomes [1][2] - **Participants**: Included executives from Precision BioSciences, eCure, Harbor Site, and Seamless Therapeutics, discussing their respective technologies and programs [2] Precision BioSciences' Arcus Technology - **Unique Features**: Arcus nucleases create staggered overhang cuts, allowing for high-efficiency gene insertion in both dividing and non-dividing cells [30] - **Clinical Applications**: The technology is being utilized in partnership with eCure for clinical gene insertion, specifically targeting conditions like ornithine transcarbamylase (OTC) deficiency [5][28] Clinical Data and Outcomes - **OTC Deficiency Treatment**: A patient treated at six and a half months of age showed significant improvement, with a 50% reduction in ammonium levels and no hyperammonemic events post-treatment [36][37] - **Regulatory Approval**: The program has received clearance to operate in multiple regions, including the US, UK, Spain, and Australia [32] Comparison with Traditional Gene Therapy - **Gene Insertion vs. Gene Addition**: Traditional gene addition approaches can lead to dilution of therapeutic effects in actively dividing tissues, whereas gene insertion ensures that the therapeutic gene is integrated into the genome, providing sustained expression [18][19] - **Targeting Multiple Mutations**: The ability to insert large DNA fragments addresses diseases with multiple mutations, which traditional gene editing systems cannot effectively target [12][15] Market Potential and Competitive Landscape - **Hepatitis B Program**: Precision BioSciences is pursuing a hepatitis B program aimed at eliminating cccDNA, which is crucial for curing chronic infections [54][56] - **Commercial Viability**: The potential market for hepatitis B treatments is significant, with a large patient population and a clear unmet need for effective therapies [62] Regulatory Outlook - **Positive Regulatory Interactions**: The company has had favorable interactions with regulatory agencies, indicating a supportive environment for gene editing technologies [81][83] - **Flexibility in Development**: Regulators are showing a willingness to adapt traditional trial designs to accommodate the unique challenges of rare diseases [86] Future Directions - **Expansion Beyond Rare Diseases**: There is a focus on applying gene editing technologies to larger patient populations and various indications beyond rare genetic disorders [94][95] - **Technological Advancements**: Continuous improvements in gene editing technologies and cost efficiencies are expected to enhance the commercial viability of these therapies [95] Additional Insights - **Investor Sentiment**: Investors are increasingly interested in technologies that address unmet medical needs and have clear commercial potential [76][78] - **Long-term Impact**: The ability to treat young patients with severe genetic disorders could have significant societal and economic benefits [90] This summary encapsulates the key discussions and insights from the Precision BioSciences FY Conference Call, highlighting the company's innovative approach to gene editing and its implications for future therapies.