33 results about "Safety profile" patented technology

A compound or a pharmaceutically acceptable salt thereof for use in a method of treatment of a disease or condition in which signalling through the PI3Kδ pathway is pathologically implicated in patients, for example cancer and inflammatory or autoimmune diseases. The compound is provided at a specified dose and has been found to have a favourable safety profile in humans, in particular with regard to hepatotoxicity, diarrhoea / colitis, respiratory infections, and hematologic toxicities.

This invention discloses the application and method of a STAT1-targeting inhibitor in the treatment of immune thrombocytopenic purpura (ITP). It employs ivermectin, a STAT1 nuclear translocation inhibitor, and fludarabine, a STAT1 activation inhibitor. By injecting either the activation inhibitor or the nuclear translocation inhibitor, the platelet count in a mouse model of ITP is increased. Fludarabine, a fluorinated nucleotide analog of vidarabine, is non-radioactive and a small-molecule phosphorylation inhibitor. Ivermectin is a small-molecule inhibitor of nuclear translocation mediated by α / β1 introgression protein. Both have high bioavailability and are widely used to treat various hematological diseases with good safety profiles. Their application in treating ITP is safe, effective, and shows high compliance.

This invention discloses the application of American ginseng glycoprotein extract in the preparation of drugs for treating alcoholic liver disease, belonging to the field of biomedical technology. This invention verifies that TD can significantly reduce serum ALT, AST, and TG levels in ALD mice, effectively reducing liver damage and lipid accumulation; inhibit NLRP3 protein expression and its downstream targets, effectively alleviating liver inflammation and pyroptosis; and improve intestinal flora imbalance in ALD mice. American ginseng glycoprotein extract has multi-target protective effects against alcoholic liver disease, reducing alcohol-induced liver tissue inflammation and oxidative stress damage. It has a mild composition and high safety profile, and can synergistically exert effects in liver protection, anti-inflammation, and antioxidation, making it suitable for adjunctive intervention and long-term management of alcoholic liver disease, providing a natural, mild, and effective protective pathway for alcoholic liver damage.

The application provides novel N-heterocyclic compounds, such as quinoline or isoquinoline compounds, preferably quinazoline compounds, as calcineurin inhibitors (CNIs,) that provide improved safety profiles, and the pharmaceutical composition and formulation thereof, as well as a method of using the N-heterocyclic compounds for the treatment of, inter alia, an inflammatory-related disease or disorder.

In the field of gene therapy, a major hurdle is the design and identification of constructs and gene therapy vectors providing therapeutic effects while displaying satisfactory safety profiles. In the treatment of Diamond-Blackfan Anemia (DBA), therapies alleviating several crucial anemia symptoms, such as blood or bone marrow cellularity, hemoglobin levels, erythrocytes levels, or platelet levels, while showing satisfactory safety profiles remain a challenge. The present invention provides constructs encoding ribosomal protein genes involved in DBA, such as genes encoding RPS19, RPS17, RPS24, RPS10, RPL35a, RPL11, RPS26, and RPL5, vectors, methods, cells, and medical uses thereof, addressing these challenges and finding particular applications in the field of autologous cell therapy treatment of DBA. Further, the present invention provides a non-genotoxic conditioning protocol for preparing a subject prior to cell therapy treatment for DBA using construct of the present invention.

This disclosure pertains to the field of biomedicine and relates to the use of an anti-HER2 antibody-drug conjugate (ADC) for the treatment of breast cancer. It also relates to a method for treating a subject with breast cancer, the method comprising administering an anti-HER2 antibody-drug conjugate to the subject. Furthermore, it relates to the use of the anti-HER2 antibody-drug conjugate in the preparation of a medicament for neoadjuvant therapy of breast cancer. The method provides benefit to breast cancer subjects and has a good safety profile.

A novel treatment regimen is provided for the treatment of autoimmune disorders. Said novel treatment regimen provides for an efficacious treatment of autoimmune disorders with an advantageous safety profile and / or a high quality of life for the patient. Said novel treatment regimen provides for an advantageous benefit-risk ratio for patients endangered by the risk of infections.

A novel treatment regimen treats autoimmune disorders. Said novel treatment regimen efficaciously treats autoimmune disorders with an advantageous safety profile and / or a high quality of life for the patient. Said novel treatment regimen shows an advantageous benefit-risk ratio for patients endangered by the risk of infections.

The present invention is in the field of viral immunotherapy. The invention provides 1) an efficient recombinant poxvirus expressing a toxic conditional gene product that affects the viability of the recombinant poxvirus itself if left unregulated, or 2) a recombinant poxvirus expressing a conditional gene product with an improved safety profile, a method of producing them, a composition comprising them and therapeutic uses related thereto.

Provided are compositions and methods for treating cancers, in particular those characterized with PSMA expression such as prostate cancer. The compositions include a conditionally-activatable XTENylated Protease-Activated bispecific T Cell Engager targeting PSMA (PSMA-XPAT) that remain inactive until activated by proteases present in tumor microenvironment. The PSMA-XPAT exhibited exceptional safety profiles even at high doses and were efficacious in cancer patients resistant to even multiple lines of prior therapies.

Disclosed are improved Disabled Infectious Single Cycle (DISC) Herpes Simplex Virus (HSV) and their uses in the treatment of cancer or as a vaccine. Also disclosed are improved methods of making DISC HSV.

This invention belongs to the field of pharmaceutical technology, and relates to diphenylmethane derivatives, their preparation methods, and applications. The diphenylmethane derivative is a compound represented by Formula I, its optical isomer, or a pharmaceutically acceptable salt thereof: wherein X is CH2; Y is selected from: -NH-, -CO-, -NHCONH-. The diphenylmethane derivative of this invention is stable, easy to store, and readily prepared, exhibiting good feasibility. It demonstrates outstanding in vitro and in vivo efficacy and good safety profile when used to prepare anticardioprotective drugs (for heart failure and myocardial hypertrophy).

To provide antibodies that selectively and specifically bind to human monocarboxylate transporter 1 (MCT1), have desirable developability and patient-safety profiles, and can be used to treat MCT1 associated disorders, such as autoimmune diseases.SOLUTION: To provide antibodies having specific sequences and inhibiting MCT1 mediated responses (e.g., metabolic product transport, T-cell and B-cell proliferation) and / or driving regulatory T-cell differentiation, as well as compositions comprising such MCT1 antibodies, and methods of using such MCT1 antibodies and compositions.SELECTED DRAWING: None

The present disclosure provides a novel panel of fully humanized bispecific ADCs targeting CDH17 and another clinically validated target such as EGFR and HER2 for gastrointestinal cancers treatment. The bispecific ADCs offer improved target coverage, internalization rate and cytotoxicity over parental antibodies, as well as safety profile for a wider therapeutic window.

This invention relates to a macromolecular anticancer drug and a method for preparing it with controllable molecular weight. The macromolecular copper chelating agent of this invention possesses excellent copper ion binding capacity, significant copper-reducing effect, good safety profile, and good therapeutic and preventative effects against cancer. The method for preparing the controllable molecular weight of this invention is simple, has high yield, uses inexpensive and readily available raw materials, and operates under mild reaction conditions. It enables the large-scale, simple, and efficient preparation of the polymer of this invention, and allows for precise control of its molecular weight, thereby obtaining a macromolecular anticancer drug with a long metabolic time in vivo and good therapeutic effect on cancer metastasis.

The invention provides an antitumor drug with excellent antitumor efficacy and safety profile, exhibiting superior therapeutic effects. An anti-TROP2 antibody is provided, comprising: CDRH1 composed of an amino acid sequence of sequence identification number 23, CDRH2 composed of an amino acid sequence of sequence identification number 24, and CDRH3 composed of an amino acid sequence of sequence identification number 25 in the variable region of its heavy chain; and CDRL1 composed of an amino acid sequence of sequence identification number 26, CDRL2 composed of an amino acid sequence of sequence identification number 27, and CDRL3 composed of an amino acid sequence of sequence identification number 28 in the variable region of its light chain.

This invention relates to HeartTAC, an antibody protein that targets and degrades calprotectin, and its applications, belonging to the field of biomedical technology. This invention provides HeartTAC, an antibody protein that targets and degrades calprotectin, the amino acid sequence of which is shown in SEQ ID NO. 6 or SEQ ID NO. 4. This invention employs an extracellular targeted protein degradation scheme to construct HeartTAC, an antibody protein that targets and degrades calprotectin. This protein exhibits good targeting specificity to the heart, can degrade calprotectin in the heart, can significantly improve cardiac function, alleviate cardiac fibrosis, and has good safety profile. It opens up an important new direction for the prevention and control of cardiovascular diseases and has promising application prospects.

The present invention relates to an L-tryptophan-producing engineered strain of Clostridium butyricum and use thereof in the preparation of a drug for treating tumors, and pertains to the technical field of biomedical engineering. The tryptophan operon gene trpEDCBA is overexpressed in Clostridium butyricum by means of a thl promoter and a pMTL82151 shuttle vector to construct an engineered strain of Clostridium butyricum with a high L-tryptophan yield. The strain can specifically target and colonize tumors in mice without additional treatment. The strain secretes butanoic acid and tryptophan metabolites to inhibit IDO enzyme expression, regulate CD8+ T cell metabolism, and change the immune microenvironment of tumors, thereby achieving the purpose of inhibiting tumor growth. In addition, the strain will not affect the physiological structures of the major organs in the body, showing a favorable safety profile.

The present invention provides a compound for preventing or treating ocular diseases, a pharmaceutical composition, and an application thereof. The compound provided by the present invention is effective in controlling ametropia and axial length elongation, has a good safety profile, with no duration of use restrictions, and is capable of meeting the usage needs of adolescents with myopia.

The present disclosure relates to monoclonal antibodies that specifically bind to thymic stromal lymphopoietin (TSLP) and methods for treating atopic dermatitis. The antibodies block TSLP interaction with its receptor complex, preventing downstream inflammatory signaling. Administration involves weekly loading doses followed by biweekly maintenance doses, achieving superior clinical efficacy with >90% reduction in disease severity. The antibodies demonstrate favorable pharmacokinetics with a 25-day half-life, low immunogenicity, and a well-tolerated safety profile. This disclosure also covers methods for evaluating treatment efficacy and safety in moderate to severe atopic dermatitis patients.

This invention discloses a class of androgen receptor modulators, their preparation methods, and applications. The androgen receptor modulators are selected from compounds having the structural formula shown in Formula I. Experimental verification has shown that these compounds are all androgen receptor modulators, capable of significantly reducing the expression levels of androgen receptors in various cells and effectively inhibiting the proliferation of prostate cancer cells. They exhibit good safety profiles and have potential for further development.

The application provides novel N-heterocyclic compounds, such as quinoline or isoquinoline compounds, preferably quinazoline compounds, as calcineurin inhibitors (CNIs,) that provide improved safety profiles, and the pharmaceutical composition and formulation thereof, as well as a method of using the N-heterocyclic compounds for the treatment of, inter alia, an inflammatory-related disease or disorder.

This invention discloses a ternary nanoparticle, its preparation method, and its applications, relating to the field of biomedical technology. The invention constructs a carrier-free, fully active ternary nanoparticle (diPCL NPs). This nanoplatform connects PTX dimer (diPTX), celecoxib (CXB) dimer (diCXB), and lonidamine (LND) dimer (diLND) via disulfide bonds for chemoimmunotherapy of drug-resistant tumors. diPCL NPs accumulate at tumor sites through enhanced permeability and retention (EPR) effects and circulate continuously in the bloodstream. In the high GSH tumor microenvironment, disulfide bond cleavage releases the active drug, exerting chemotherapeutic, immune-activating, sensitizing, and metabolic-regulating effects. diPCL NPs exhibit high antitumor efficacy and good safety profile within the therapeutic window in both breast cancer and paclitaxel-resistant models.

The present application relates to the field of biomedicine, and pertains to an antibody conjugate and an application thereof. The antibody conjugate comprises a heterodimeric antibody, a linker, and an active agent. Also provided is a method for preparing the antibody conjugate. The provided antibody conjugate exhibits excellent anti-tumor activity and / or a relatively good safety profile. The provided antibody conjugate can be used in treatment of tumors or autoimmune diseases.

This invention relates to the field of pharmaceutical preparation technology, and more particularly to a herbal spray for treating acute pharyngitis, its preparation method, and its application. This invention provides a herbal spray with the effects of clearing heat and detoxifying, reducing swelling and relieving pain. It can effectively alleviate symptoms such as sore throat and throat discomfort caused by acute pharyngitis. This invention optimizes the preparation method of the spray, resulting in a herbal spray with good properties and stability. When applied topically to the throat, it has a rapid onset of action, is suitable for all age groups, especially children, and has a high safety profile. It is convenient and simple to use, absorbs quickly and completely, and has high patient compliance.

The present disclosure relates to a use in the preparation of a drug for treating hypertrophic cardiomyopathy. Specifically, the present disclosure relates to a use of a compound as represented by formula I, or a pharmaceutically acceptable salt thereof, in the preparation of a drug for preventing and / or treating hypertrophic cardiomyopathy. Compared with Mavacamten and Aficamten, the use of the present disclosure exhibits a shorter steady-state attainment time, a higher safety profile, and no complex restrictions on concomitant drug use.

This invention discloses the application of a natural isoflavone compound in the preparation of drugs for the prevention or treatment of depression. The natural isoflavone compound is hematoxylin A, a derivative of hematoxylin A, or a pharmaceutically acceptable salt of hematoxylin A. Hematoxylin A can effectively reduce oxidative damage to HT22 neurons in the hippocampus induced by glutamate and corticosterone, and can significantly improve the behavioral performance of mice in a chronic restraint stress depression model, exhibiting clear antidepressant activity. Hematoxylin A has a well-defined structure and good safety profile, showing promising application prospects in the development of antidepressant drugs.

The invention discloses a linear long-chain compound containing valine thiazole as well as a preparation method and application of the linear long-chain compound, and belongs to the technical field of medicinal chemistry. The structure of the linear long-chain compound containing valine thiazole is shown in the specification. The valinethiazole-containing linear long-chain compound has the beneficial effects that the valinethiazole-containing linear long-chain compound provided by the invention is novel in structure, the compound 6, the compound 9, the compound 11 and the compound 13 are good in safety, particularly, the MDR reverse activity of the compound 9 on a human oral epidermoid cancer cell line KBV is higher than that of a positive control drug verapamil, the effect is improved by 8 times compared with that of verapamil, and the compound 6, the compound 9, the compound 11 and the compound 13 can be used for preparing the valinethiazole-containing linear long-chain compound. Compared with Dysoxylactam A, the reverse multiple of the Dysoxylactam A is obviously improved from 174.3 times to 288.5 times, and the Dysoxylactam A has the application prospect of becoming a novel tumor drug resistance reversal agent.

Provided are compositions and methods for treating cancers, in particular those characterized with HER2 expression. The compositions include a conditionally-activatable XTENylated Protease-Activated bispecific T Cell Engager targeting HER2 (HER2-XPAT) that remain inactive until activated by proteases present in tumor microenvironment. The HER2-XPAT exhibited exceptional safety profiles even at high doses and were efficacious in cancer patients resistant to even multiple lines of prior therapies.

This invention relates to the application of tanshinone A ester derivatives with carbon chain lengths of C1-C2, as shown in Formula I, in the preparation of drugs for treating idiopathic pulmonary fibrosis. This invention systematically investigated the oral absorption behavior of tanshinone A ester derivatives of different carbon chain lengths in rats, and confirmed through in vitro HFL1 cell models and in vivo bleomycin-induced pulmonary fibrosis mouse models that these short-chain ester derivatives (methyl and ethyl esters) can significantly improve oral bioavailability and inhibit TGF-β1-induced α-SMA expression and extracellular collagen deposition, demonstrating significantly superior characteristics compared to the original tanshinone A and its long-chain ester derivatives. Most importantly, cytotoxicity experiments showed a significantly improved safety profile, demonstrating an excellent therapeutic window. This invention overcomes the shortcomings of low bioavailability and well-defined toxicity of tanshinone A in existing technologies, providing a more efficient and safer new drug option for the treatment of idiopathic pulmonary fibrosis.