RNA interference (RNAi)

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Why Alnylam Pharmaceuticals (ALNY) is a Top Momentum Stock for the Long-Term
ZACKS· 2025-09-24 14:51
Core Insights - Zacks Premium provides various tools for investors to enhance their stock market strategies and confidence [1] - The Zacks Style Scores are designed to complement the Zacks Rank, helping investors identify stocks likely to outperform the market in the short term [2] Zacks Style Scores Overview - Stocks are rated from A to F based on value, growth, and momentum characteristics, with A being the highest score [3] - The Style Scores are categorized into four types: Value Score, Growth Score, Momentum Score, and VGM Score [3][4][5][6] Value Score - Focuses on identifying undervalued stocks using financial ratios such as P/E, PEG, and Price/Sales [3] Growth Score - Concentrates on a company's financial health and future growth potential, analyzing projected and historical earnings, sales, and cash flow [4] Momentum Score - Targets stocks with upward or downward price trends, utilizing recent price changes and earnings estimate revisions to identify buying opportunities [5] VGM Score - Combines Value, Growth, and Momentum Scores to provide a comprehensive assessment of stocks, aiding in the selection of the most attractive investment opportunities [6] Zacks Rank Integration - The Zacks Rank uses earnings estimate revisions to identify stocks with the highest potential returns, with 1 (Strong Buy) stocks historically achieving an average annual return of +23.64% since 1988 [7][8] - Investors are encouraged to focus on stocks with a Zacks Rank of 1 or 2 and Style Scores of A or B for optimal investment success [9] Company Spotlight: Alnylam Pharmaceuticals - Alnylam Pharmaceuticals is a biopharmaceutical company specializing in RNA interference therapeutics, with a pipeline targeting genetic, cardio-metabolic, and hepatic infectious diseases [11] - The company has received multiple FDA approvals for its products, including Onpattro, Givlaari, Oxlumo, and Amvuttra, addressing various medical conditions [11] - Currently, Alnylam has a Zacks Rank of 3 (Hold) and a VGM Score of B, with a strong Momentum Style Score of A, indicating potential for upward movement [12][13]
Press Release: Sanofi’s SAR446268 earns US fast track designation for the treatment of non-congenital myotonic dystrophy type 1
Globenewswire· 2025-09-23 05:00
Core Insights - The FDA has granted fast track designation to Sanofi's SAR446268, a one-time AAV gene therapy for treating non-congenital myotonic dystrophy type 1 (DM1), aimed at expediting its development and review process [1][7] Group 1: Product Development - SAR446268 utilizes a vectorized RNA interference (RNAi) approach to silence DMPK expression, potentially addressing key symptoms of DM1 such as muscle weakness and myotonia [2][3] - The therapy is currently undergoing a first-in-human, phase 1-2 study to assess its safety, tolerability, and efficacy, with the first patient expected to be enrolled in late 2025 [3] Group 2: Disease Overview - Myotonic dystrophy type 1 is a rare genetic disorder affecting approximately 1 in 2,300 people globally, characterized by progressive muscle weakness and various systemic effects [4] - There are currently no approved treatments for DM1, highlighting the unmet medical need that SAR446268 aims to address [4][7] Group 3: Company Profile - Sanofi is an R&D driven biopharma company focused on improving lives through innovative medicines and vaccines, with a commitment to addressing urgent healthcare challenges [5]
Silexion Therapeutics Announces Pricing of $6.0 Million Public Offering
Globenewswire· 2025-09-11 13:17
Core Viewpoint - Silexion Therapeutics Corp. has announced a public offering of 1,500,000 ordinary shares and accompanying warrants at a price of $4.00 per share, aiming to raise approximately $6.0 million to advance its pre-clinical studies and for general corporate purposes [1][2]. Group 1: Offering Details - The public offering includes 1,500,000 ordinary shares, series A warrants, and series B warrants, all priced at $4.00 per share [1]. - Series A warrants will have an exercise price of $4.00, exercisable immediately, and will expire five years from issuance [1]. - Series B warrants will also have an exercise price of $4.00, exercisable immediately, but will expire twelve months from issuance [1]. - The offering is expected to close on or about September 12, 2025, pending customary closing conditions [1]. Group 2: Financial Aspects - The gross proceeds from the offering are expected to be $6.0 million before deducting fees and expenses [2]. - The net proceeds will be utilized to advance pre-clinical studies and for general corporate purposes [2]. Group 3: Company Background - Silexion Therapeutics is focused on developing RNA interference therapies for KRAS-driven cancers, particularly targeting solid tumors with mutated KRAS oncogenes [5]. - The company has conducted a Phase 2a clinical trial for its first-generation product, showing a positive trend compared to chemotherapy alone [5]. - Silexion is committed to advancing therapeutic options in oncology, specifically for locally advanced pancreatic cancer [5].
Top 20 biopharma market caps fall 5.7% amid industry challenges
Yahoo Finance· 2025-09-10 16:16
Industry Overview - The biopharmaceutical industry is experiencing challenges due to ongoing tariffs and drug pricing pressures from the Trump administration, leading to a 5.7% decline in the combined market capitalisation of the top 20 global biopharmaceutical companies, from $3.92 trillion on March 31, 2025, to $3.70 trillion on June 30, 2025 [1] Company Performances - Alnylam achieved the largest market capitalisation growth of 21.8%, reaching $42.5 billion, driven by strong sales of its RNA interference therapeutic Amvuttra, which saw a 59% year-on-year increase in global sales to $310 million in Q1 2025 [2] - Novartis reported a 7.7% increase in market capitalisation in Q2 2025, bolstered by strong sales of blockbuster drugs such as Entresto, Cosentyx, Kesimpta, and Kisqali, along with the acquisition of Regulus Therapeutics for $1.7 billion, enhancing its renal disease portfolio [3] - Bristol Myers Squibb experienced the largest decline in market capitalisation at 22.9% due to mixed Q1 2025 financial results and negative trial outcomes, particularly the failure of its Phase III ARISE trial for Cobenfy [4] - Regeneron and Sanofi saw market capitalisation declines of 18.7% and 13.8%, respectively, following the failure of their partnered drug itepekimab in the Phase III AERIFY-2 trial, which aimed to reduce exacerbations of chronic obstructive pulmonary disease [5]
Alnylam Pharmaceuticals, Inc. (NASDAQ: ALNY) Targets New Heights in Biopharmaceuticals
Financial Modeling Prep· 2025-09-05 20:11
Company Overview - Alnylam Pharmaceuticals, Inc. is a leading player in the biopharmaceutical industry, focusing on RNA interference (RNAi) therapeutics aimed at silencing specific genes associated with various diseases [1] - The company competes with other biotech firms such as Moderna and BioNTech, positioning itself among the leaders in the sector [1] Recent Developments - On September 5, 2025, Kostas Biliouris from BMO Capital set a price target of $470 for ALNY, indicating a potential upside of about 3.8% from its trading price of $452.81 at that time [2] - Alnylam participated in Citi's Biopharma Back to School Conference on September 3, 2025, where key executives discussed the company's strategic initiatives and future plans [3] Stock Performance - Currently, ALNY's stock is priced at $452.13, reflecting a slight decrease of 0.31% or $1.41, with a daily trading range between $444.66 and $455.05 [4] - Over the past year, ALNY has experienced significant volatility, with a high of $469.81 and a low of $205.87 [4] Market Position - Alnylam's market capitalization is approximately $59.26 billion, indicating a substantial presence in the market [5] - The stock has a trading volume of 264,290 shares on the NASDAQ exchange, highlighting active trading and investor interest in Alnylam's growth potential [5]
Silexion Therapeutics to Present at the Upcoming 27th Annual H.C. Wainwright Global Investment Conference
Globenewswire· 2025-09-02 12:45
Core Insights - Silexion Therapeutics Corp. is a clinical-stage biotechnology company focused on RNA interference therapies for KRAS-driven cancers [1][3] - The company will present at the 27th Annual H.C. Wainwright Global Investment Conference on September 10, 2025 [1][2] - Silexion's lead product candidate targets locally advanced pancreatic cancer and has shown positive trends in a Phase 2a clinical trial compared to chemotherapy alone [3] Company Presentation Details - The presentation is scheduled for September 10, 2025, at 2:00 PM ET [2] - An updated company presentation is available on Silexion's investor website [2] - Management will be available for one-on-one investor meetings during the conference [2] Company Background - Silexion Therapeutics is dedicated to developing innovative treatments for solid tumor cancers with mutated KRAS oncogenes, which are prevalent in human cancers [3] - The company aims to advance therapeutic options in oncology, particularly for patients with unsatisfactorily treated cancers [3]
Silexion Therapeutics Announces Positive Results in Preclinical Study Demonstrating Significant SIL204 Efficacy in Human Lung Cancer Cell Lines
Globenewswire· 2025-07-09 12:30
Core Insights - Silexion Therapeutics Corp. announced positive preclinical data for SIL204, demonstrating significant efficacy in human lung cancer cell lines, particularly those with KRAS G12D mutations [1][4][6] - The company is conducting a new study on an untested KRAS mutation, with results expected soon, which could further validate SIL204 as a potential pan-KRAS treatment [2][3] - Silexion's dual-route administration strategy is progressing, with plans to initiate a Phase 2/3 clinical trial in Q2 2026 for KRAS-driven solid tumors [3] Study Findings - The study showed dose-dependent inhibition in lung cancer cells with KRAS G12D mutations, highlighting SIL204's potential as a versatile therapeutic [4][7] - The lipid-conjugated delivery system enhances SIL204's entry into tumor cells, addressing a significant barrier for siRNA technology [5][6] Market Opportunity - KRAS mutations are prevalent in various cancers, with approximately 90% in pancreatic cancers, 45% in colorectal cancers, and 30% in lung cancers, representing a significant market opportunity [8] - The global treatment market for these cancers exceeds US $30 billion annually, with many KRAS variants challenging to target with conventional therapies, underscoring the potential of Silexion's RNAi-based strategy [8][9] Company Overview - Silexion Therapeutics is focused on developing RNA interference therapies for solid tumors driven by KRAS mutations, with SIL204 as its next-generation candidate showing promise in preclinical studies [10]
Instil Bio Names John Maraganore, Ph.D., to Board Role Supporting the Advancement of PD-L1xVEGF Bispecific Antibody Program
Globenewswire· 2025-06-11 11:00
Core Insights - Instil Bio, Inc. has appointed John Maraganore, Ph.D., to the Board of Directors of its subsidiary Axion Bio, Inc., which is focused on developing AXN-2510, a PD-L1xVEGF bispecific antibody targeting solid tumors, particularly non-small cell lung cancer (NSCLC) [1][2] - The appointment is aimed at enhancing leadership to maximize the value of the AXN-2510 program for both patients and shareholders, with expectations of redefining the standard of care for solid tumors [2] - AXN-2510 is currently in a Phase 2 trial in combination with chemotherapy for first-line NSCLC, with early signs of safety and efficacy being reported [2] Company Overview - Instil Bio is a clinical-stage biopharmaceutical company dedicated to developing novel therapies, with AXN-2510 as its lead asset [3] - Axion Bio, a wholly-owned subsidiary of Instil Bio, is specifically focused on the development of AXN-2510 for multiple solid tumors [4] Leadership Background - Dr. John Maraganore brings over 30 years of biotechnology experience, having previously served as the founding CEO of Alnylam Pharmaceuticals, where he led the company to a market capitalization exceeding $40 billion [2] - His expertise spans drug discovery, clinical development, and strategic partnerships, making him a valuable asset for guiding the advancement of next-generation cancer therapies [2]
Silexion Therapeutics Announces Groundbreaking Preclinical Results: SIL204 Shows Strong Efficacy in Pancreatic, Colorectal, and Lung Cancers
Globenewswire· 2025-05-29 13:05
Core Insights - Silexion Therapeutics has demonstrated the efficacy of its RNAi therapeutic candidate, SIL204, against KRAS-driven cancers, showing a significant inhibition rate of approximately 90% in GP2D human colorectal cancer cells [1][5] - The company plans to conduct further preclinical studies focusing on lung cancer cell lines to expand the understanding of SIL204's therapeutic potential [2][4] Company Overview - Silexion Therapeutics is a clinical-stage biotechnology company specializing in RNA interference therapies targeting KRAS mutations, which are prevalent in various cancers [6] - The company’s first-generation product, LODER™, has shown promising results in Phase 2 trials for non-resectable pancreatic cancer, while SIL204 aims to target a broader range of KRAS mutations [6] Therapeutic Potential - SIL204 has shown significant inhibition of cancer cell proliferation and metabolic activity across multiple cancer types, including pancreatic, colorectal, and lung cancers, indicating its potential as a pan-KRAS therapy [4][5] - KRAS mutations are found in approximately 90% of pancreatic cancers, 45% of colorectal cancers, and 35% of non-squamous non-small-cell lung cancers, representing a substantial market opportunity exceeding US $30 billion annually [4][6]
TransCode Therapeutics Appoints Dr. Phillip D. Zamore to Science Advisory Board
Prnewswire· 2025-05-28 12:00
Company Overview - TransCode Therapeutics, Inc. is a clinical-stage oncology company focused on developing RNA-targeted therapeutics for cancer treatment [1][4] - The company aims to combat metastatic disease through the intelligent design and effective delivery of RNA therapeutics using its proprietary TTX nanoparticle platform [4] Key Appointment - Dr. Phillip D. Zamore has been appointed to the Scientific Advisory Board of TransCode Therapeutics [1] - Dr. Zamore is recognized for his pioneering work in RNA interference (RNAi) and co-founded Alnylam Pharmaceuticals, which developed the first FDA-approved RNAi drug [1][6] Research Contributions - Dr. Zamore's research has advanced the understanding of non-coding RNA and its implications in health and disease, particularly in the processing of microRNAs [2] - His contributions have earned him recognition from prestigious institutions, including the National Academy of Sciences and the National Academy of Medicine [2] Strategic Vision - The company emphasizes the importance of Dr. Zamore's expertise in RNA biology to enhance its pipeline of RNA-targeted cancer therapies [3] - Dr. Zamore expressed enthusiasm for contributing to the development of effective RNA-based treatments for cancer patients [3] Product Focus - TransCode's lead therapeutic candidate, TTX-MC138, targets metastatic tumors that overexpress microRNA-10b, a biomarker associated with metastasis [4] - The company has a portfolio of first-in-class RNA therapeutic candidates aimed at overcoming RNA delivery challenges to access novel genetic targets for various cancers [4]