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dornase alfa (DNase, IBC Generium / Tigerase)

✓ Approved

Generium Pharmaceutical · DNASE1 · Recombinant Proteins

What is dornase alfa?

dornase alfa is a recombinant proteins developed by Generium Pharmaceutical. It is approved for therapeutic indications via inhaled.

Drug Profile

Brand NamesDNase, IBC Generium, Tigerase
CompanyGenerium Pharmaceutical
Drug ClassRecombinant Proteins
Molecular TargetDNASE1
RouteInhaled
StatusApproved
Sources: ClinicalTrials.gov, Generium Pharmaceutical

Mechanism of Action

Molecular Targets

dornase alfa acts on 1 molecular target:

DNASE1deoxyribonuclease 1 (DNL1, DRNI)
Want deeper analysis?Noah AI can explain complex mechanisms and compare to similar drugs.

Therapeutic Indications

dornase alfa is developed for 1 unique indication across 1 therapeutic area.

Therapeutic AreaConditionPhase
Congenital, familial and genetic disordersCystic fibrosis✓ Approved

Related Research Articles

PubMedJournal of thrombosis and haemostasis : JTH2026-08-28

Successful immune tolerance induction using efanesoctocog alfa in severe hemophilia A: A case series.

Ashraf Aqsa A, Kuhlman Rebekah R, Khan Osman O, Nusrat Sanober S

The development of neutralizing factor VIII (FVIII) inhibitors remains a major therapeutic challenge in severe hemophilia A. We report two patients with complex inhibitor histories who achieved rapid and sustained inhibitor eradication using efanesoctocog alfa. Immune tolerance induction (ITI) was initiated at 100 units (U)/kg 3 times weekly, with subsequent dose reduction after inhibitor clearance. Inhibitor titers declined to undetectable levels (<0.5 Bethesda units [BU]) within 6 weeks in one case and 3 months in the other, with FVIII recovery exceeding 90%. Transition to once-weekly individualized prophylaxis maintained functional FVIII activity and therapeutic FVIII trough levels (4%-6%), allowing discontinuation of emicizumab. Sustained bleed-free outcomes were achieved with individualized prophylactic dosing without inhibitor recurrence. These cases support efanesoctocog alfa-based ITI as a promising strategy for rescue and retreatment patients with complex inhibitor histories.

PMID 42665230
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PubMedKlinicka onkologie : casopis Ceske a Slovenske onkologicke spolecnosti2026-08-28

Treatment of Erdheim-Chester disease from the perspective in 2026.

Adam Zdeněk Z, Štork Martin M, Řehák Zdeněk Z, Boichuk Ivanna I et al.

With the discovery of new drugs, treatment of Erdheim-Chester disease (ECD) is gradually evolving and changing. The text provides an overview of effective drugs available in 2026 for patients with ECD. Treatment options: We consider it appropriate to divide treatment into treatment targeting the inflammatory reaction induced by ECD, and treatment targeting malignant histiocytes. To suppress the inflammatory reaction, high doses of glucocorticoids can be used, but only short-term. For long-term suppression of the inflammatory reaction, anakinra is most commonly used. In rare cases, a similar effect has been reported with canakinumab, as well as with sirolimus and anti-TNF drugs. Anakinra is used both alone and in combination with other drugs. ECD cells, like histiocytosis cells from Langerhans cells, are derived from a monocytic lineage, which similarly to the lymphocytic lineage, is very sensitive to cladribine (2-chloro-2'-deoxyadenosine). This drug is the most effective cytostatic agent for patients with ECD. In several smaller clinical studies, the benefit of treatment with interferon alfa has been described. Thus, interferon alfa, despite its side effects, represents one of the treatment options. In cases of severe ECD and the presence of the BRAFV600E mutation, two drugs are recommended - vemurafenib and dabrafenib. For patients with a severe course without the BRAFV600E mutation, MEK kinase inhibitors trametinib and cobimetinib are recommended. The literature contains conflicting conclusions in studies evaluating the dependence of the effectiveness of these drugs on the detection of mutations in the MAPK signaling pathway. Targeted therapy is associated with numerous side effects, as well as the risk of relapse after its discontinuation. If the administered drugs trigger an inflammatory response, anakinra is again used to suppress it. Treatment of ECD always requires an assessment of the extent of the disease and degree of organ damage in order to choose the appropriate therapy.

PMID 42663152
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PubMedLife (Basel, Switzerland)2026-08-27

Complete Hematological Response Coupled with Molecular Response in Polycythemia Vera Treatment with Ropeginterferon Alfa-2b.

Vecchio Veronica V, Cacciola Rossella R, Cacciola Emma E

Background: Polycythemia vera (PV) is characterized by mutation JAK2 V617F, elevated blood counts, and risk of thrombosis. Ropeginterferon alfa-2b (ropeg) approved for PV at a low-starting dose and slow-titration schema with efficacy at 12 months with assessment visits every three months. This period might be at thrombotic risk resulting from inadequate control of hematological parameters and JAK2. The study was aimed at assessing its efficacy at less than 12 months with assessment visits every two weeks. Methods: Thirteen patients with Hydroxyurea (HU)/Intolerance (HU/I) high-risk PV and pruritus were treated with ropeg at a low-starting dose and slow-titration schema with assessment visits every two weeks. The primary endpoint was the complete hematological response (CHR) at less than 12 months. The secondary endpoints were the CHR rates, CHR dose, durable CHR, and reduction in JAK2V617F variant allele frequency (VAF). Results: The CHR of patients was 8 weeks in 30.7% (4/13), 16 weeks in 30.7% (4/13), and 24 weeks in 38.4% (5/13), respectively, with a mean time of 4.15 months. Durable CHR was at 20, 28, and 36 weeks, respectively, with a mean time of 6.55 months. JAK2 VAF continuously declined from 43.38% at baseline to 12.96% at durable CHR. Eleven patients (85%) achieved partial molecular response (PMR) and two (15.38%) complete molecular responses (CMRs) and all patients resolved the pruritus. No adverse event (AE) or thrombosis occurred. Conclusions: We report that ropeg is efficacious under the low-starting dose and slow-titration regimen in obtaining CHR and molecular response (MR) at less than 12 months.

PMID 42652908
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PubMedRSC advances2026-08-27

Enhanced CO2 adsorption via one-step thermochemical activation of Alfa fiber.

Akaya Hicham H, Ouzrour Zineb Z, El Aalami Badr B, Babin Alexandre A et al.

In this work, Moroccan Alfa fibers were converted into porous biochars through a one-step thermochemical activation using KOH, ZnCl2, and melamine at impregnation ratios of 0.5, 1.0, and 1.5. The influence of the activating agents on the physicochemical properties and CO2 adsorption behavior of the resulting materials was systematically investigated using structural and surface characterization techniques. Chemical activation substantially enhanced pore development compared with the non-activated biochar, with KOH producing the most developed hierarchical micro-mesoporous structure. Among all samples, Af-K1 exhibited the highest CO2 adsorption capacity, reaching 3.70 mmol g-1 at 30 °C and 1 bar, which increased to 5.26 mmol g-1 at 0 °C. This superior performance was primarily attributed to its larger micropore volume and optimized pore-size distribution. Af-K1 also demonstrated excellent regeneration stability, retaining approximately 99% of its initial adsorption capacity after ten adsorption-desorption cycles. Furthermore, the material exhibited an IAST-predicted CO2/N2 selectivity of 58 for a simulated flue gas mixture (15% CO2), while model extrapolation suggested a selectivity of 24-41 under direct air capture (DAC) conditions (400 ppm CO2). These predicted DAC values should be regarded as indicative and require experimental validation under low-pressure and mixed-gas conditions. Thermodynamic analysis indicated that CO2 adsorption is spontaneous and exothermic, with ΔH° = -13.19 kJ mol-1 and ΔS° = -0.041 kJ mol-1 K-1, while ΔG° remained negative (-2.11 to -0.28 kJ mol-1) over the investigated temperature range, confirming the thermodynamic favorability of the adsorption process. These findings demonstrate that one-step KOH activation provides an efficient and sustainable strategy for producing high-performance biochar adsorbents for CO2 capture and separation.

PMID 42656812
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PubMedInternational journal of molecular sciences2026-08-27

The Non-Coding rs5945619 Variant at Xp11.22 and Its Putative Regulatory Effects on Nearby Genes in Ecuadorian Mestizo Women with Breast Cancer: A Case Series and In Silico Cis-eQTL Analysis.

Tamayo-Trujillo Rafael R, Zambrano Ana Karina AK, Guevara-Ramírez Patricia P, Paz-Cruz Elius E et al.

Breast cancer (BC) is a heterogeneous disease influenced by genetic and regulatory mechanisms. Despite this, X-linked non-coding variants remain underexplored, particularly in admixed Latin American populations. Therefore, this study aimed to characterize the non-coding variant rs5945619 at Xp11.22 in Ecuadorian mestizo women with BC and to assess its potential regulatory effects on nearby genes through in silico cis-eQTL analysis. A prospective observational case series was conducted in 21 Ecuadorian mestizo women with histologically confirmed BC. Tumor DNA was extracted and analyzed using the Illumina TruSight Cancer Sequencing Panel. The rs5945619 variant was identified from sequencing data and compared with reference allele frequencies from dbSNP, ALFA, and the 1000 Genomes Project. Regulatory potential was assessed using RegulomeDB v2.2, and tissue-specific cis-eQTL associations were explored using GTEx. All 21 patients carried the genetic variant rs5945619 in a heterozygous state. The mean age at diagnosis was 54.5 ± 12.2 years, and invasive breast carcinoma of no special type was the predominant histological subtype. The T-allele frequency in the study cohort was 0.50, whereas Latin American reference populations showed higher frequencies ranging from 0.69 to 0.83. RegulomeDB assigned rs5945619 a rank of 1f and a functional score of 0.22, supporting its regulatory potential. GTEx analysis identified tissue-specific cis-eQTL signals, including LINC01496 upregulation in testis, NUDT11 and LINC01496 downregulation in prostate, and modest GSPT2 downregulation in mammary tissue. This study provides the first characterization of rs5945619 in Ecuadorian mestizo women with BC and suggests its role as a potential X-linked regulatory variant, requiring further validation. Although universal heterozygosity was observed, the small sample size, tumor-only design, and absence of a healthy control cohort prevent any causal, risk-factor, or tumor-driven selection inference. The in silico evidence supports a tissue-dependent regulatory model involving LINC01496 and NUDT11, mainly in prostate/testis, and a modest GSPT2 signal in mammary tissue. Therefore, breast cancer-specific eQTL analyses, functional validation, and larger ancestry-informed case-control studies are required to determine the biological and clinical relevance of this locus.

PMID 42653091
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PubMedFrontiers in cardiovascular medicine2026-08-26

Fabry cardiomyopathy presenting as hypertrophic phenotype with left ventricular outflow tract obstruction: a case series.

Hosyanto Felycia Fernanda FF, Li Yuanzhu Y, Luo Suxin S, Huang Bi B

Fabry disease is a cardiomyopathy with multisystemic manifestations that is easily to be misdiagnosed as another type of cardiomyopathy. Fabry cardiomyopathy may present with left ventricular outflow tract obstruction (LVOTO), mimicking obstructive hypertrophic cardiomyopathy (oHCM). We present three cases of genetically confirmed Fabry disease that were initially misdiagnosed as oHCM, highlighting the importance of early recognition and tailored management. We performed Fabry disease screening on patients diagnosed with oHCM and ultimately identified three individuals who have received oHCM-specific treatments including mavacamten and alcohol septal ablation. Three patients (2 females, 1 male; aged 48-66 years) who were initially diagnosed with and managed for oHCM based on septal hypertrophy and elevated left ventricular outflow tract gradients (LVOTG), was found to have GLA gene mutations. Enzyme replacement therapy with agalsidase alfa in two patients was associated with stabilization or improvement in wall thickness and LVOTO. Fabry cardiomyopathy can present with a hypertrophic phenotype and significant LVOTO, and is often misdiagnosed as oHCM. This further underscores the importance of genetic testing in the etiological diagnosis of hypertrophic cardiomyopathies. Enzyme replacement therapy may ameliorate obstruction, while mavacamten and septal reduction therapies require caution without definitive genotype-phenotype correlation.

PMID 42643330
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