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dornase alfa (DNase, IBC Generium / Tigerase)

✓ Approved

Generium Pharmaceutical · DNASE1 · 重组蛋白

什么是 dornase alfa？

dornase alfa 是一种重组蛋白，由Generium Pharmaceutical研发。该药已获批，用于治疗相关适应症，给药途径：Inhaled。

药物档案

商品名DNase, IBC Generium, Tigerase
公司Generium Pharmaceutical
药物类别重组蛋白
分子靶点DNASE1
给药途径Inhaled
状态Approved
来源： ClinicalTrials.gov, Generium Pharmaceutical

作用机制

分子靶点

dornase alfa 作用于 1 个分子靶点：

DNASE1deoxyribonuclease 1 (DNL1, DRNI)
需要更深入的分析？Noah AI 可解释复杂机制并与同类药物比较。

治疗适应症

dornase alfa 针对 1 个适应症，涉及 1 个治疗领域。

治疗领域疾病/病症分期
Congenital, familial and genetic disordersCystic fibrosis✓ Approved

相关研究文献

PubMedJournal of clinical medicine2026-06-12

Growth Patterns in MPS IVA and MPS IIIA: A Longitudinal Single-Center Study.

Carmon Lior L, Nassar Majd M, Idan Daphna D, Leifman Dar D et al.

Background/Objectives: Mucopolysaccharidoses (MPS) are lysosomal storage disorders characterized by impaired glycosaminoglycan degradation, leading to multisystem involvement and progressive growth impairment. Longitudinal growth data in MPS IVA and MPS IIIA, including the association of ERT with growth outcomes, remain limited. This study aimed to characterize growth trajectories in MPS IVA and MPS IIIA and to assess the association of ERT with Elosulfase alfa on growth outcomes in MPS IVA patients. Methods: We retrospectively analyzed growth data from 39 patients with MPS subtypes IIIA and IVA followed at a single center between 2004 and 2024. Height and weight standard deviation scores (SDS) were calculated relative to CDC growth references and modeled using linear mixed-effects models (LMM). In the MPS IVA subgroup, the effect of ERT with Elosulfase alfa was assessed using LMM and paired SDS comparisons. Results: Growth impairment was evident across both subtypes with distinct trajectories. MPS IIIA patients showed significant height decline after age six with progressive weight loss in later childhood. MPS IVA patients exhibited persistently severe short stature and a tendency toward overweight with advancing age. Among the 16 MPS IVA patients treated with Elosulfase alfa who were included in the analysis, height SDS declined significantly during treatment (-0.127 SDS/year [95% CI: -0.194, -0.061], p < 0.001), and the rate of decline was not significantly affected by age at ERT initiation (interaction p = 0.53). Conclusions: ERT with Elosulfase alfa did not prevent progressive height loss relative to population norms. The rate of height SDS decline was not significantly influenced by the timing of ERT initiation (interaction p = 0.53), and causal conclusions cannot be drawn from this observational data.

PMID 42279040
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PubMedMolecules (Basel, Switzerland)2026-06-12

Establishment of an N-Glycan Profiling Method for Three ERT Enzymes Used in Gaucher Disease Therapy.

Chen Jinliang J, Hu Xinyue X, Wang Lyuyin L, Xu Kaixin K et al.

N-glycosylation, particularly terminal mannose exposure, is a critical quality attribute affecting macrophage targeting and the clinical efficacy of enzyme replacement therapy for Gaucher disease. This study developed a universal, sensitive, and quantitative method to compare the N-glycan profiles of three recombinant human glucocerebrosidase products from different expression systems: imiglucerase, velaglucerase alfa, and velaglucerase beta. Using 2-aminobenzamide labeling combined with HILIC-UPLC-FLD and high-resolution mass spectrometry, an N-glycan profiling platform was established. A multidimensional calibration system integrating retention time, glucose unit values, and mass-to-charge ratios was constructed, and collision-induced dissociation tandem MS was used to identify isomers and phosphorylated glycans. The method showed good specificity, linearity, precision, and accuracy. Glycan profiling revealed clear product-dependent differences: imiglucerase was enriched in core-fucosylated Man3 structures, velaglucerase alfa was dominated by Man9 and contained more phosphorylated and sialylated glycans, whereas velaglucerase beta showed a highly homogeneous Man5 profile. These findings demonstrate how distinct manufacturing strategies shape glycosylation patterns and provide a basis for biosimilar development and comparability assessment.

PMID 42280207
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PubMedResearch square2026-06-12

UTag, a cysteine-free thermostable tagging system for tracking single mRNA translation live.

Zhao Ning N, Aguilera Luis L, Chen Szuhsuan S, Sears Rhiannon R et al.

Spatiotemporal regulation of mRNA translation is central to gene expression. Over the past decade, translation has become directly observable in live cells at single-mRNA resolution by tagging nascent chains with tandem arrays of short epitope tags recognized by genetically encodable fluorescent intracellular antibodies (intrabodies). While this technology has revolutionized our understanding of translation regulation, the current toolbox of tagging systems remains limited. Here, we developed a novel and tight-binding intrabody against a short (11-amino acid) HIV protease epitope (named UTag). To ensure robust intracellular folding of the anti-UTag intrabody, we further engineered a cysteine-free variant that folds and functions independently of disulfide-bond formation, as validated by X-ray crystallography. The cysteine-free anti-UTag intrabody retains high binding affinity comparable to the parental intrabody while exhibiting significantly improved thermostability (~80 °C). Importantly, the cysteine-free UTag system enables real-time tracking of single-mRNA translation in live cells with performance on par with the parental UTag system as well as the established SunTag and ALFA-tag. Collectively, these results demonstrate that the newly developed UTag system expands the toolbox for live-cell translation tracking and provides complementary tools for multiplexed applications.

PMID 42282040
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PubMedPLoS biology2026-06-12

IntAct-U-ExM enables super-resolution imaging of isoform-specific actin networks across species.

Dhar Anubhav A, Dey Sucheta S, Mullick Sanjana S, Suman Nishant Kumar NK et al.

Expansion microscopy (ExM) has revolutionized super-resolution imaging in cell biology due to its simple and inexpensive workflow. The use of ExM has revealed several novel insights into the nanoscale architectures of cellular protein complexes, especially the microtubule cytoskeleton in model and non-model systems. Despite tremendous progress in expansion microscopy protocols that preserve cellular ultrastructure (U-ExM), compatible probes for imaging actin isoforms with U-ExM are still lacking and have hindered the study of diverse actin isoforms and networks across model systems. Here, we use IntAct, an internally tagged actin that incorporates into cellular actin networks, to develop and optimize U-ExM for diverse actin structures in yeast, mammalian cells, and primary neurons. Using ALFA-tagged IntAct variants, we achieve robust visualization of actin patches, cables, and rings in yeast, as well as diverse actin architectures including the cortex, stress fibers, filopodia, and lamellipodia in mammalian cells at improved resolution. In primary hippocampal neurons, IntAct efficiently labels actin throughout the soma and neuronal projections, revealing strong enrichment at dendritic spines and synaptic boutons. Notably, we observe a periodic organization of F-actin along axons consistent with the membrane-associated periodic cytoskeleton, thereby resolving the periodic, sub-diffraction actin ring organization. We also detect transient nuclear actin filaments using IntAct-U-ExM underscoring the advantages offered by our approach to image understudied actin structures. Overall, we demonstrate the effectiveness of IntAct-U-ExM for performing super-resolution imaging of various actin structures in an isoform-specific manner and highlight the potential of IntAct to study the nanoscale organization of diverse actin cytoskeletal networks across species.

PMID 42284311
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PubMed3 Biotech2026-06-11

A comparative study of PiggyBac-mediated gene insertion and ubiquitous chromatin opening element incorporation to enhance Darbepoetin alfa expression.

Khoshnood Zeynab Z, Lohrasbi Reyhane R, Hasheminejad Fateme F, Halfinezhad Zahra Z et al.

Stable and robust production of recombinant therapeutic proteins in mammalian cells remains challenging due to position effects and transcriptional silencing of the transgene following random integration. We investigated whether semi-targeted integration and UCOE-incorporation could enhance Darbepoetin alfa (DPO) transgene expression. A PiggyBac-mediated Darbepoetin alfa-secreting cell pool (PB-DPO) was established by semi-targeted insertion of a DPO-containing expression cassette using pB513B-1, a PiggyBac transposon-derived vector, into CHO DG44 host cells. A UCOE-incorporated Darbepoetin alfa-secreting cell pool (UCOE-DPO) was also used to compare DPO production rates between the two groups and the control (pOptiVEC-DPO) cell pool. DPO expression at both transcript and protein levels was compared across DPO-producing cell pools using real-time quantitative PCR, Western blotting, and ELISA. The PB-DPO showed ~ 1.96- and ~ 1.79-fold increases in mRNA and protein levels, respectively, whereas the UCOE-DPO exhibited even stronger enhancement, with ~ 2.94- and ~ 4.53-fold increases compared with the control. In conclusion, PiggyBac-mediated transgene transposition generated a cell population with increased transgene expression, likely through multiple insertions and preferential integration near transcriptionally active regions. Although both modified pools demonstrated increased DPO transcript and protein yields, UCOE-DPO was more effective at overcoming integration-associated silencing. Compared with the semi-targeted transposition method, UCOE incorporation further increased Darbepoetin alfa production by ~ 2.5-fold. The online version contains supplementary material available at 10.1007/s13205-026-04828-6.

PMID 42273071
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PubMedThe New England journal of medicine2026-06-11

Antidotes for Anticoagulation Reversal.

Rocca Bianca B, Ten Cate Hugo H

The global rise in anticoagulant use has increased the number of major bleeding events that warrant timely and effective pharmacologic reversal. Reversal strategies should be informed by the pharmacodynamic and pharmacokinetic features of the anticoagulant and antidote, regulatory indications, quality of evidence, patient-specific factors, and availability of treatment options. Protamine sulfate neutralizes unfractionated heparin, whereas no specific antidotes exist for low-molecular-weight heparins or fondaparinux. Four-factor prothrombin complex concentrates effectively reverse vitamin K antagonists. Idarucizumab specifically reverses dabigatran, although delayed dabigatran rebound can occur. Andexanet alfa targets direct oral factor Xa inhibitors, but uncertainties regarding the efficacy-safety balance, monitoring, rebound, perioperative use, and cost have prompted off-label use of four-factor prothrombin complex concentrates, for which stronger evidence is needed. Key challenges remain, including determination of appropriate dosing, standardization and validation of laboratory monitoring, mitigation of thrombotic risk, and development of guidelines for perioperative treatment. Emerging agents aim to broaden targets and improve safety. Building high-quality evidence remains essential to advancing global, patient-centered anticoagulant and hemostatic care.

PMID 42269153
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