-Positive Phase 1 data for CTX310® presented in a late-breaking presentation at the American Heart Association (AHA) Scientific Sessions and simultaneously published in The New England Journal of Medicine- -CASGEVY® momentum accelerating; nearly 300 patients have been referred to Authorized Treatment Centers (ATCs), approximately 165 patients have completed their first cell collection and 39 have received infusions across all regions; Vertex expects clear line of sight to over $100 million in total CASGEVY ...

Accessibility StatementSkip Navigation Commercialization Roadmap: Advancing Biosafety Evaluation Origin Agritech has initiated the biosafety assessment process for the edited lines. Additionally, this license agreement provides a substantial advantage by facilitating biosafety approval for Origin's current and future gene editing transformants utilizing Hi-3 one-step gene-editing method, for some of which the Company has already applied. The Company estimates obtaining final biosafety certification will req ...

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]

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]

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 ...

Financial giants have made a conspicuous bearish move on CRISPR Therapeutics. Our analysis of options history for CRISPR Therapeutics (NASDAQ:CRSP) revealed 9 unusual trades.Delving into the details, we found 33% of traders were bullish, while 44% showed bearish tendencies. Out of all the trades we spotted, 4 were puts, with a value of $169,270, and 5 were calls, valued at $277,530.What's The Price Target?Analyzing the Volume and Open Interest in these contracts, it seems that the big players have been eyei ...

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]

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]

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]

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].