CRISPR Therapeutics Expands into siRNA Therapies with $95M Sirius Partnership for Thromboembolic Disorders
• CRISPR Therapeutics is diversifying beyond gene editing through a strategic $95 million partnership with Sirius Therapeutics to develop SRSD107, a long-acting siRNA therapy targeting Factor XI for thromboembolic disorders.
• In Phase 1 trials, SRSD107 demonstrated impressive efficacy with over 93% reduction in Factor XI levels and doubled blood clotting time, with effects lasting up to six months from a single dose.
• The collaboration includes a 50-50 cost and profit-sharing structure for SRSD107 development, with CRISPR leading U.S. commercialization and retaining rights to license two additional siRNA targets.
Armata's Bacteriophage Therapy Shows Promising Results in Treating Staphylococcus aureus Bacteremia
• Armata Pharmaceuticals' Phase 1b/2a diSArm trial demonstrated that AP-SA02, a novel intravenous bacteriophage therapy, met all primary endpoints for safety, tolerability, and clinical response in complicated Staphylococcus aureus bacteremia.
• The bacteriophage therapy significantly improved clinical outcomes compared to best available antibiotic therapy alone, with 100% of AP-SA02-treated patients responding at end of study versus 75% in the placebo group (p=0.020).
• AP-SA02 was well-tolerated with no serious adverse events related to the study drug, showing efficacy against both methicillin-sensitive and methicillin-resistant S. aureus infections, marking a breakthrough in phage therapy for systemic bacterial infections.
Pipeline Progress: Over 25 New Therapies in Development for Metastatic Liver Cancer
• DelveInsight's latest report reveals a robust pipeline with over 20 companies developing 25+ therapies for metastatic liver cancer, offering new hope for patients with limited treatment options.
• Several promising clinical trials are underway in 2025, including studies evaluating novel combinations such as AK104 plus lenvatinib, tislelizumab with lenvatinib, and triplet therapy of nivolumab, relatlimab and bevacizumab.
• Key emerging therapies include exoASO-STAT6 targeting tumor-associated macrophages, STP705 dual TGF-ß1/COX-2 inhibitor, and ONCR-177, an oncolytic viral immunotherapy expressing five transgenes to fight tumors through multiple mechanisms.
Rising Obesity in Type 1 Diabetes Patients Signals Need for New Cardiovascular Treatment Approaches
• Type 1 diabetes, traditionally associated with lean individuals, is now frequently complicated by obesity, leading to increased cardiovascular risks and necessitating new treatment approaches.
• Current projections indicate 50% of U.S. adults will be obese by 2030, prompting experts to recommend early cardiovascular screening and preventive strategies for T1D patients.
• GLP-1 receptor agonists show promise in addressing both weight management and cardiovascular risk reduction in T1D patients, potentially transforming traditional insulin-focused treatment paradigms.
Leading Cardiologist Deepak Bhatt Highlights Critical Challenges in Clinical Trial Interpretation
• Dr. Deepak Bhatt, Director of Mount Sinai Heart, addressed key pitfalls in clinical trial interpretation at ESC Congress 2024, drawing from his extensive experience leading landmark cardiovascular trials.
• Research indicates a significant 17-year gap between scientific discoveries and their implementation in real-world clinical practice, highlighting the need for better interpretation and translation of trial results.
• As a senior TIMI investigator with over 275,000 citations, Dr. Bhatt's expertise spans major trials including REDUCE-IT, SCORED, and THEMIS, positioning him as a leading authority on trial methodology.
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