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Factor IX (AlphaNine SD / AlphaNine)

✓ Approved

Mitsubishi Tanabe Pharma Corporation · F9 · Cell-based Therapies

What is Factor IX?

Factor IX is a cell-based therapies developed by Mitsubishi Tanabe Pharma Corporation. It is approved for therapeutic indications via injectable (others) or intravenous (iv).

Drug Profile

Brand NamesAlphaNine SD, AlphaNine
CompanyMitsubishi Tanabe Pharma Corporation
Drug ClassCell-based Therapies
Molecular TargetF9
RouteInjectable (Others), Intravenous (IV)
StatusApproved

Mechanism of Action

Molecular Targets

Factor IX acts on 1 molecular target:

F9coagulation factor IX (P19, F9 p22)
Want deeper analysis?Noah AI can explain complex mechanisms and compare to similar drugs.

Therapeutic Indications

Factor IX is developed for 3 unique indications across 2 therapeutic areas.

Therapeutic AreaConditionPhase
Vascular disordersExtravasation blood✓ Approved
Congenital, familial and genetic disordersFactor IX deficiency✓ Approved
Vascular disordersHaemorrhage✓ Approved

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To evaluate the reliability and construct validity of the Turkish version of the Oral Health Impact Profile-17 (OHIP-17-TR) in patients receiving endodontic treatment. This methodological, cross-sectional validation study included 229 adult patients undergoing or having recently completed endodontic treatment. Participants completed the OHIP-17-TR questionnaire. Internal consistency was assessed using Cronbach alpha and corrected item-total correlations. Construct validity was evaluated through exploratory factor analysis (EFA). Sampling adequacy was examined using the Kaiser-Meyer-Olkin (KMO) measure and Bartlett test of sphericity. Test-retest reliability was not assessed. The OHIP-17-TR showed high internal consistency, with a Cronbach alpha of 0.894. Corrected item-total correlations ranged from 0.345 to 0.675. The KMO value was 0.882, and the Bartlett test was significant (P 0.001), indicating suitability for factor analysis. Exploratory factor analysis identified a four-factor structure explaining 51.8% of the total variance, with all items demonstrating salient factor loadings. Subscale Cronbach alpha values ranged from 0.689 to 0.862. The OHIP-17-TR demonstrated good reliability and acceptable construct validity in this sample of endodontic patients. These findings support the use of OHIP-17-TR as a brief and practical patient-reported outcome measure for assessing oral health-related quality of life in endodontic research and clinical practice.

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The small intestine microbiome: composition, host interactions and therapeutic potential.

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The human microbiome, the collection of microorganisms that inhabit the human body, has been extensively studied over the past two decades and has been shown to play a critical part in health and disease. These research efforts renewed interest in developing microbial therapeutics to promote human health and treat disease. However, the vast majority of human gut microbiome research has been focused on fecal microbial strains and communities, leaving most of the gastrointestinal tract understudied. The dynamic environment of the small intestine provides distinct opportunities for microorganisms to interact with the host and diet. In this Review, we discuss the current state of research on the microbiome of the small intestine, how interactions between the small intestine and its microbiome influence host physiology, and we posit that small intestinal microorganisms are a unique source of novel therapeutics for treating human disease. Next-generation microbial therapeutics that emerge from the small intestinal microbiome are anticipated to be relevant to improving small intestinal diseases as well as systemic human health.

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Pulmonary delivery of nucleic acid therapeutics represents a transformative strategy for treating a broad spectrum of respiratory diseases, including lung cancer, pulmonary fibrosis, chronic obstructive pulmonary disease, and acute lung injury. Despite their promise, effective clinical translation is hampered by a cascade of physiological barriers spanning the airway mucus layer, mucociliary clearance, alveolar macrophage uptake, cellular internalization, and endosomal sequestration. In this review, we adopt a barrier-oriented framework to systematically dissect these extracellular, cellular, and intracellular obstacles, highlighting how disease-specific microenvironments further modulate delivery efficiency. We then critically examine emerging engineering strategies-including nanocarrier surface modifications, biomimetic vesicles, aerosol optimization, and stimuli-responsive formulations that are designed to overcome these barriers. Integrating mechanistic insights with disease-specific considerations, we analyze how distinct pulmonary pathologies necessitate tailored delivery solutions, emphasizing that successful translation requires coupling nanoengineering with the unique cellular and molecular landscapes of target tissues. Finally, we discuss translational challenges, including manufacturing scalability, long-term safety, and regulatory constraints, and outline emerging solutions that may enable next-generation inhaled nucleic acid therapies. By providing a synthesis of barrier-oriented design principles and disease-driven targeting strategies, this review offers a comprehensive roadmap for accelerating the clinical adoption of inhaled nucleic acid therapeutics.

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Frey Linus S LS, Verstraeten William W, Tran Mai P MP, Venkert Dana D et al.

RNA aptamers are short, single-stranded RNA molecules that fold into specific three-dimensional structures and selectively bind molecular targets. Despite applications in therapeutics, diagnostics, and biosensing, tools for aptamer validation remain scarce compared to well-established pipelines for other biomolecular binders such as antibodies. Here, we introduce SecAntiGami─a secondary-antibody-inspired RNA origami─as a modular tool for signal enhancement and aptamer characterization. SecAntiGami binds aptamers of interest through programmable kissing-loop interactions and carries a fluorescent aptamer for visualization. It amplifies aptamer-dependent size increases in dynamic light scattering assays and enables fluorescence-based aptamer localization. We rationally design three different variants of a biotin-binding aptamer and use SecAntiGami to demonstrate that all of them retain their functionality, enabling efficient integration of aptamers into RNA origami structures. SecAntiGami serves as a versatile tool for qualitative aptamer validation for synthetic biology, structural RNA nanotechnology, RNA therapeutics, and diagnostics.

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End-of-Life Care Practices of Korean Intensive Care Nurses: A Cross-Cultural Adaptation and Validation Study.

Kang Sook Jung SJ, Lee Ji Seon JS

End-of-life care is increasingly provided in Korean intensive care units (ICUs), yet no validated ICU-specific instrument assesses nurses' end-of-life care practices. This study cross-culturally adapted an Australian instrument measuring critical care nurses' end-of-life care practices and evaluated its validity and reliability. Following translation and content validity review (51 to 43 items), construct validity was examined using confirmatory factor analysis in a cross-sectional survey, alongside known-groups validity and reliability. Among 189 ICU nurses, the instrument was refined to a six-factor, 22-item version demonstrating good model fit (comparative fit index = 0.96; root mean square error of approximation = 0.047). Nurses with prior end-of-life care education scored significantly higher (U = 3548.5, p = 0.029; rank-biserial r = 0.19, 95% confidence interval [0.02, 0.35]). Internal consistency (Cronbach's alpha = 0.91) and test-retest stability (intraclass correlation coefficient = 0.93) were satisfactory. The Korean version is valid and reliable for assessing these practices.

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