Drug Database
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Factor VIII (ReFacto AF / Xyntha)

✓ Approved

Sobi · F8 · Recombinant Proteins

What is Factor VIII?

Factor VIII is a recombinant proteins developed by Sobi. It is approved for therapeutic indications via injectable (others) or intravenous (iv).

Drug Profile

Brand NamesReFacto AF, Xyntha
CompanySobi
Drug ClassRecombinant Proteins, Cell-based Therapies
Molecular TargetF8
RouteInjectable (Others), Intravenous (IV)
StatusApproved
Sources: ClinicalTrials.gov, Sobi

Mechanism of Action

Molecular Targets

Factor VIII acts on 1 molecular target:

F8coagulation factor VIII (AHF, FVIII)
Want deeper analysis?Noah AI can explain complex mechanisms and compare to similar drugs.

Therapeutic Indications

Factor VIII is developed for 1 unique indication across 1 therapeutic area.

Therapeutic AreaConditionPhase
Congenital, familial and genetic disordersFactor VIII deficiency✓ Approved

Related Research Articles

PubMedNursing & health sciences2026-07-17

Risk Stratification and Nursing Strategy for Recurrent Readmission in Primary Venous Thromboembolism: A Study Based on NT-proBNP and Factor VIII.

Wang Xia X, Li Jia J, Yu Ying Y, Zhang Shujin S et al.

To identify factors influencing recurrent readmission in patients diagnosed with Venous thromboembolism (VTE) who underwent interventional or surgical procedures and propose an evidence-based nursing strategy. This retrospective cohort study included 121 patients with VTE admitted to Shanxi Bethune Hospital (February 2022-February 2025). Patients were divided into recurrence and non-recurrence groups based on readmission. Independent predictors were identified using logistic regression. Model performance was assessed by ROC curves, nomograms, and calibration plots. Recurrent readmission occurred in 24 patients (19.83%). Independent predictors included BMI (OR = 1.625), NT-proBNP (OR = 1.014), factor VIII (OR = 1.032), MMAS-8 (OR = 0.587), and AHSMSRS scores (OR = 0.950). The combined model showed excellent predictive accuracy (AUC = 0.849; 95% CI: 0.767-0.930), with good calibration. Elevated BMI, NT-proBNP, and factor VIII, along with poor medication adherence and self-management, predict VTE recurrence. Early identification and targeted nursing interventions may reduce readmission rates.

PMID 42464685
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PubMedSage open pediatrics2026-07-17

Hemophilia and Type 1 Diabetes in a Pediatric Patient: Case Report of an Extremely Rare Association.

Virú-Loza Manuel André MA

Reports on the association between hemophilia and pediatric diabetes are very scarce in the literature, with no more than four worldwide. This report describes the case of a 14-year-old male with severe hemophilia A and type 1 diabetes who was brought to the emergency department due to fever and bruising on his back and jaw following trauma. The patient responded well to plasma-derived factor VIII, drainage, antibiotic therapy, and management with NPH and Regular insulin. There were no problems with the use of subcutaneous insulins during hospitalization. Previously reported cases in the literature describe potential bleeding and bruising associated with devices used for managing pediatric diabetes. However, severe bleeding and bruising that significantly impede diabetes treatment do not appear to be present, although individualized management is still required.

PMID 42466207
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PubMedbioRxiv : the preprint server for biology2026-07-17

AAV VP1 unique region (VP1u) determines GPR108 dependence for AAV transduction of human airway epithelium and its rescue by Doxorubicin.

Hao Siyuan S, Habib Ariful A, Zhang Xiujuan X, Ning Kang K et al.

rAAV2.5T was identified through directed evolution of an AAV capsid library in polarized human airway epithelium (HAE) cultured at an air-liquid interface (ALI). The capsid gene of rAAV2.5T is a chimera of the N-terminal unique region of AAV2 VP1 (VP1u) and the VP2 and VP3 regions of AAV5 with a single A581T substitution at the variable region (VR) VIII of the capsids. GPR108, a G protein-coupled receptor, is known as an essential host factor for the transduction of rAAV2 but not of rAAV5. Both AAV2 and AAV5 VP1u colocalized well with GPR108 and, to a lesser extent, with the trans -Golgi network (TGN). GPR108 knockout (KO) abolished rAAV2.5T transduction in both HeLa cells and HAE-ALI cultures. Remarkably, short-term treatment with doxorubicin (DOX) at 2 µM completely restored transduction, indicating that DOX can compensate for the loss of GPR108 function. DOX enhanced rAAV2.5T transduction by 50-100-fold in wild-type HAE-ALI cultures and by over 300-fold in the GPR108-deficient cultures. Mechanistic studies demonstrated that this enhancement resulted from altered intracellular trafficking that promoted efficient vector nuclear import, rather than increased vector internalization, proteasome inhibition, or activation of the DNA damage response. Importantly, we identified that the N-terminal 15 amino acids of AAV2 VP1u as the primary determinant of rAAV2.5T dependence on GPR108 for transduction. Collectively, these findings demonstrate that productive transduction of rAAV2.5T in polarized HAE cultures depends on GPR108-mediated intracellular trafficking that limits efficient nuclear entry, and that DOX can relieve this constraint by promoting efficient vector import. AAV2.5T is an airway-tropic vector with considerable promise for pulmonary gene therapy. We found that host factor GPR108 is required for rAAV2.5T trafficking from the TGN to the nucleus and that this step constitutes a major bottleneck to productive transduction in polarized HAE. In contrast, KIAA0319L (AAVR) plays a key role in AAV intracellular trafficking from the endosome to the TGN but not in internalization into polarized HAE during apical transduction. Transient treatment with low-dose doxorubicin (DOX, 2 µM) enhanced rAAV2.5T transduction in HAE by 50-100-fold through a significant increase in vector nuclear import. Notably, DOX can overcome the transduction deficit caused by GPR108 deficiency, but not that caused by AAVR deficiency. Mechanistically, the N-terminal 15 amino acids of the VP1u confer GPR108 dependence during rAAV2.5T apical transduction of polarized HAE. DOX bypasses this requirement by promoting efficient nuclear import without affecting vector internalization, inhibiting proteasomes, or inducing DNA damage response.

PMID 42465353
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PubMedJournal of the American Heart Association2026-07-17

Shear Difference: Flow Type Dictates Endothelial Flow-Responsive Gene Programs in a 3-Dimensional-Printed In Vitro Model.

Shah Nasir A NA, Rye Kerry-Anne KA, Endre Zoltan H ZH, Barber Tracie J TJ et al.

Endothelial cells are mechanosensitive and adopt distinct phenotypes in response to hemodynamic forces. These responses are difficult to study in conventional in vitro platforms, which rarely reproduce vessel-scale geometry or clinically relevant flow. Our aim was to determine how flow impacts endothelial morphology and transcriptional activity in a 3-dimensional macrofluidic model. Idealized vessels were 3-dimensional printed using a water-soluble polyvinyl alcohol and cast in polydimethylsiloxane. After core dissolution, human microvascular endothelial cell line-1 cells were grown on the lumen and perfused for 24 hours under static, continuous, or pulsatile flow. The pulsatile waveform was derived from arteriovenous fistula Doppler profiles and scaled to match mean volumetric flow (~100 mL/min) and time-averaged wall shear stress (~1.5 dyn/cm2) to continuous flow. Morphology was assessed using immunofluorescence. Bulk RNA-sequencing and gene set enrichment analysis were performed. Relative to static culture, continuous flow increased cell eccentricity (0.74 versus 0.48; P<0.0001) and reduced orientation variability (Δ=-47.1°, P<0.0001). Differential gene expression was extensive (continuous versus static: 2103 genes; pulsatile versus static: 2643 genes; pulsatile versus continuous: 384 genes). Continuous flow reduced interferon signaling and the Hallmark inflammatory response program relative to static, whereas tumor necrosis factor-α/nuclear factor-κ-light-chain enhancer of activated B cells signaling was increased. Under matched mean shear, pulsatile flow enriched cell cycle/checkpoint programs. Conversely, continuous flow enriched oxidative phosphorylation and p53 pathways. Transforming growth factor-β signaling was enriched in pulsatile flow. Under matched mean shear, pulsatile and continuous flow were associated with distinct endothelial morphologic and transcriptional signatures. This macrofluidic platform provides a validated, waveform-controlled testbed for mechanistic and translational studies.

PMID 42466497
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PubMedXi bao yu fen zi mian yi xue za zhi = Chinese journal of cellular and molecular immunology2026-07-17

[Research progress on the trans-organ regulation of renal fibrosis by type 3 innate lymphoid cells].

Wang Qianyu Q, Guo Zhaoan Z

Type 3 innate lymphoid cells (ILC3s) are a subset of innate immune cells regulated by the transcription factor retinoic acid receptor-related orphan receptor γt (RORγt). They are primarily distributed in the intestinal mucosa and lymphoid tissues, where they secrete cytokines such as interleukin 17(IL-17), IL-22, and granulocyte-macrophage colony-stimulating factor (GM-CSF), playing a crucial role in maintaining intestinal mucosal homeostasis, defending against pathogen invasion, and modulating immune responses. Renal fibrosis (RF) is a pathological process triggered by pathogenic factors such as trauma, infection, inflammation, or metabolic abnormalities. It is characterized by damage to renal parenchymal cells, abnormal deposition of extracellular matrix (ECM), and progressive fibrosis of renal tissue, ultimately leading to irreversible loss of kidney function. Recent studies have revealed that ILC3s not only contribute to intestinal homeostasis but are also involved in the progression of RF. Therefore, this review summarizes the research advances in the mechanisms of ILC3-mediated cross-organ regulation in RF, aiming to provide new perspectives for the prevention and treatment of RF.

PMID 42463315
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PubMedMolecular biology reports2026-07-17

Celastrol at the obesity-cancer interface: critical appraisal of molecular mechanisms, preclinical evidence, and translational barriers.

Omer Asma B AB, Afzal Muhammad M, Saleem Shakir S, Khan Mohd Masih Uzzaman MMU et al.

Obesity is a modifiable risk factor for various malignancies and is mechanistically linked to hyperinsulinemia, insulin-like growth factor 1 (IGF-1) activation, adipokine imbalance, mitochondrial oxidative stress, and chronic low-grade inflammation. Tripterygium wilfordii-derived celastrol is a quinone-methide triterpenoid that possesses multiple activities targeting metabolic, inflammatory, and oncogenic signaling networks. The molecular evidence of celastrol at the obesity-cancer interface is critically discussed, particularly focusing on metabolic reprogramming, adipokine signalling, inflammatory pathways, and the validity of the experimental models used. Celastrol increases leptin sensitivity in cell-based and animal studies, inhibits phosphoinositide 3-kinase/protein kinase B/mechanistic target of rapamycin (PI3K/AKT/mTOR), signal transducer and activator of transcription 3 (STAT3), and nuclear factor kappa B (NF-κB) signaling, disrupts heat shock protein 90-cell division cycle 37 (Hsp90-Cdc37) client-protein stabilization, and induces adenosine monophosphate-activated protein kinase (AMPK) activation, lipophagy, apoptosis, and autophagy. However, most of the antitumor effects observed are obtained from classic cancer cell lines or non-obese xenografts, and there is still limited direct evidence from diet-induced obesity models, adipocyte-tumor co-cultures, and obesity-associated models. The advantages of nanoformulations for solubility and tumor delivery in preclinical systems are significantly qualified by the lack of oral bioavailability, reactive metabolite hepatotoxicity, unclear dose-exposure relationships, narrow therapeutic window, and lack of human oncology trials. As such, at the present time, celastrol should be viewed as a preclinical molecular lead, but not a therapeutic candidate per se.

PMID 42467316
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