Workflow
Gene editing
icon
Search documents
Precision BioSciences Receives U.S. FDA Clearance of Investigational New Drug Application for First-in-Class PBGENE-DMD for Treatment of Duchenne Muscular Dystrophy
Businesswire· 2026-02-11 12:01
DURHAM, N.C.--(BUSINESS WIRE)--Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for high unmet need diseases, today announced that the Company received a Study May Proceed notification from the U.S. Food and Drug Administration (FDA). This allows Precision BioSciences to initiate IRB activities and clinical trial site activation for the FUNCTION-DMD Phase 1/2 clinical trial. ...
Citizens Maintains Outperform Rating On CRISPR Therapeutics (CRSP) Citing Undervalued Pipeline
Yahoo Finance· 2026-02-05 12:19
Core Viewpoint - CRISPR Therapeutics is identified as a stock that has the potential to double by 2030, with analysts maintaining positive ratings despite some adjustments in price targets [1][2]. Group 1: Analyst Ratings and Price Targets - Citizens maintained an Outperform rating on CRISPR Therapeutics while reducing the price target from $86 to $80, citing an undervalued pipeline and potential late-stage development for up to seven opportunities in 2027 [1]. - Bank of America analyst Alec Stranahan reduced the price target for CRISPR Therapeutics from $93 to $90 while maintaining a Buy rating, reflecting adjustments across its US Biopharmaceuticals coverage [2]. Group 2: Market Context and Trends - Various factors are aligning positively for the biotech sector, including rewards for positive data, large-cap biopharma investing in M&A and licensing, an increasing backlog of private companies, better access to capital, and minimal effects from drug price regulation [4]. - The firm suggests that while biotech has returned, the primary uncertainty lies in the longevity of this trend [4]. Group 3: Company Overview - CRISPR Therapeutics is a gene editing company that develops gene-based medicines for serious human diseases using its CRISPR/Cas9 platform [5].
Beam Therapeutics Senior Vice President Sells 6,198 Shares for $216,933
The Motley Fool· 2026-02-01 08:46
Core Insights - Beam Therapeutics is focused on gene-editing medicines and has recently seen a significant insider transaction involving the sale of shares by a senior executive [1][5]. Transaction Summary - On January 22, 2026, Bethany J. Cavanagh, Senior Vice President, sold 6,198 shares for approximately $216,933, which represented 15.11% of her direct holdings at that time [1][2][7]. - Post-transaction, Cavanagh holds 34,813 shares valued at around $1,192,693.38 based on the closing price of $34.26 on the trade date [2][7]. Company Overview - Beam Therapeutics reported a total revenue of $55.70 million and a net income of -$414.64 million for the trailing twelve months (TTM) [3]. - The company has 510 employees and experienced a 6.6% price change over the past year as of January 31, 2026 [3]. Company Snapshot - Beam Therapeutics specializes in the research, development, and commercialization of gene editing therapies, targeting conditions such as leukemia and sickle cell disease [4]. Strategic Priorities - The company announced its strategic priorities for 2026, which include advancing genetic medicines and moving products from clinical trials to distribution [5]. - The FDA has shown alignment with one of its leading medicine candidates, and the company aims to advance more solutions through clinical trials for approval by the end of the year [5]. Financial Outlook - Beam Therapeutics expects to have sufficient cash runway to operate until 2029, supported by existing reserves [6]. - The stock increased approximately 11% in 2025, with minimal price change noted as January 2026 concluded [8].
Eli Lilly partners with Seamless Therapeutics on gene editing treatments for hearing loss
Proactiveinvestors NA· 2026-01-28 16:24
Company Overview - Proactive is a financial news publisher that provides fast, accessible, informative, and actionable business and finance news content to a global investment audience [2] - The company operates with a team of experienced and qualified news journalists, ensuring independent content production [2] Market Focus - Proactive specializes in medium and small-cap markets while also covering blue-chip companies, commodities, and broader investment stories [3] - The news team delivers insights across various sectors, including biotech and pharma, mining and natural resources, battery metals, oil and gas, crypto, and emerging digital and EV technologies [3] Technology Adoption - Proactive is recognized for its forward-looking approach and enthusiastic adoption of technology to enhance workflows [4] - The company utilizes automation and software tools, including generative AI, while ensuring that all content is edited and authored by humans [5]
Cathie Wood Is Trimming Her Palantir Stake Again. How Should You Play PLTR in January 2026?
Yahoo Finance· 2026-01-15 21:16
Core Insights - Ark Invest, led by Cathie Wood, is focusing on innovation themes such as autonomy, gene editing, and advanced air mobility, adding companies like Archer Aviation, Joby Aviation, and Deere and Company to its portfolio [1] - Wood sold approximately 58,000 shares of Palantir Technologies, which represents less than 15% of her total stake, indicating profit-taking and rebalancing rather than a negative outlook on the company [2] - Analysts, including Dan Ives from Wedbush and Tyler Radke from Citigroup, have positive projections for Palantir, with Ives suggesting it could become a trillion-dollar company and Radke upgrading the stock to "Buy" with a price target increase from $210 to $235 [2][3] Company Performance - Palantir Technologies has diversified its revenue sources, with nearly half coming from the commercial segment, reducing its reliance on government contracts [4] - The stock has experienced a significant gain of about 159% over the past year, although it had a remarkable 340% gain in 2024, making it the best performer in the S&P 500 [5] - Currently, Palantir's stock is trading at a forward price-to-earnings (P/E) ratio of 225, which is considerably higher than its five-year average, suggesting that much of the positive outlook is already priced in [6]
CRISPR Therapeutics AG (CRSP) Presents at 44th Annual J.P. Morgan Healthcare Conference Transcript
Seeking Alpha· 2026-01-12 20:36
Core Insights - CRISPR Therapeutics is focused on transforming medicine through gene editing, with significant progress across four franchises [2] - The company has an approved product, CASGEVY, in collaboration with Vertex, which has multibillion-dollar revenue potential and is expanding its addressable population [2] - Ongoing efforts in in vivo gene editing aim to improve access for patients with sickle cell disease [2] Franchise Developments - The company has made advancements in its in vivo platform, particularly in gene editing of the liver [3] - Transformative data for CTX310 related to hypercholesterolemia was published in November of the previous year [3] - A partnership has been established to enhance the company's RNA platform, indicating a strategic expansion of capabilities [3]
Precision BioSciences Sets Strategic Priorities for 2026 Focused on Continued Advancement of Clinical-Stage Programs PBGENE-HBV for Chronic Hepatitis B and PBGENE-DMD for Duchenne Muscular Dystrophy
Businesswire· 2026-01-12 12:01
Core Viewpoint - Precision BioSciences, Inc. is advancing its gene editing therapies through its proprietary ARCUS® platform, focusing on unmet medical needs and outlining strategic priorities for 2026, with significant progress in its lead programs and a strong financial position to support upcoming milestones [1][2]. Clinical Programs - The PBGENE-HBV program is in a Phase 1/2a ELIMINATE-B trial aimed at curing chronic Hepatitis B by eliminating cccDNA, with positive dose-dependent effects and antiviral activity reported [3][4]. - Safety data from the PBGENE-HBV trial indicated no dose-limiting toxicities at doses up to 0.8 mg/kg, with manageable adverse events observed [4][5]. - Additional dosing cohorts are being explored to optimize the therapeutic index, with 12 participants having completed at least one dose administration [5][6]. - The PBGENE-DMD program is set to begin its Phase 1/2 FUNCTION-DMD trial in late Q1 or early Q2 2026, targeting DMD patients with specific mutations, with initial data expected by the end of 2026 [8][10]. Partnered Programs - ECUR-506, developed by iECURE, is being evaluated for neonatal onset OTC deficiency and has received FDA RMAT designation, with data expected in the first half of 2026 [11]. - The azer-cel CAR T treatment is being developed by Imugene for diffuse large B-cell lymphoma, with Precision receiving an $8 million milestone payment due to progress [12]. - Additional milestone payments are anticipated from TG Therapeutics for azer-cel's ongoing Phase 1 trial in progressive multiple sclerosis [13]. Financial Position - Precision BioSciences expects to report approximately $137 million in cash and equivalents as of December 31, 2025, with a cash runway projected through 2028 to support ongoing clinical programs [14].
Prime Medicine to Present at 44th Annual J.P. Morgan Healthcare Conference
Globenewswire· 2026-01-07 13:00
Core Insights - Prime Medicine, Inc. is set to present a corporate overview at the 44th Annual J.P. Morgan Healthcare Conference on January 14, 2026, highlighting its commitment to innovative genetic therapies [1] Company Overview - Prime Medicine is a biotechnology company focused on developing a new class of one-time curative genetic therapies using its proprietary Prime Editing platform, which allows for precise and efficient gene editing [3] - The Prime Editing technology aims to make accurate edits within genes while minimizing unwanted DNA modifications, potentially addressing a wide range of genetic mutations across various tissues and organs [3] Therapeutic Programs - The company is advancing a diversified portfolio of investigational therapeutic programs centered on liver, lung, immunology, and oncology [4] - Prime Medicine is initially targeting high-value programs with well-understood biology and clear clinical development paths, with plans to expand into additional opportunities over time [4] - The company aims to leverage the broad therapeutic potential of Prime Editing to address genetic diseases, immunological diseases, cancers, infectious diseases, and genetic risk factors in common diseases, impacting millions of people [4]
Intellia Therapeutics to Present at the 44th Annual J.P. Morgan Healthcare Conference
Globenewswire· 2026-01-07 12:30
Core Viewpoint - Intellia Therapeutics, Inc. is set to present at the 44th Annual J.P. Morgan Healthcare Conference, highlighting its focus on CRISPR-based therapies in gene editing [1] Company Overview - Intellia Therapeutics, Inc. is a leading clinical-stage gene editing company dedicated to revolutionizing medicine through CRISPR-based therapies [3] - The company aims to develop novel, first-in-class medicines that address significant unmet medical needs and advance treatment paradigms for patients [3] - Intellia is expanding its CRISPR-based platform with innovative editing and delivery technologies to maximize the potential of gene editing [3] Event Details - The presentation will take place on January 14, 2026, at 9:00 a.m. PT during the J.P. Morgan Healthcare Conference in San Francisco [1] - A live webcast of the presentation will be available on Intellia's website, with a replay accessible for approximately 30 days [2]
Intellia Therapeutics to Present at the 44th Annual J.P. Morgan Healthcare Conference
Globenewswire· 2026-01-07 12:30
Core Insights - Intellia Therapeutics, Inc. is set to present at the 44th Annual J.P. Morgan Healthcare Conference on January 14, 2026, at 9:00 a.m. PT, highlighting its focus on CRISPR-based therapies [1] - The company is recognized as a leading clinical-stage gene editing firm, aiming to revolutionize medicine through innovative gene editing technologies [3] Company Overview - Intellia Therapeutics specializes in developing first-in-class medicines that address significant unmet medical needs, leveraging its expertise in gene editing [3] - The company is committed to advancing the treatment paradigm for patients by expanding its CRISPR-based platform with novel editing and delivery technologies [3] Investor Information - A live webcast of the presentation will be available on Intellia's website, with a replay accessible for approximately 30 days [2]