Patent classifications
A61K38/46
Materials and Methods for Treatment of Myotonic Dystrophy Type 1 (DM) and Other Related Disorders
The present application provides materials and methods for treating a patient with one or more conditions associated with DMPK whether ex vivo or in vivo. In addition, the present application provides materials and methods for editing and/or modulating the expression of DMPK gene in a cell by genome editing.
COMPOSITIONS AND METHODS FOR TREATING INFECTIONS AND NETOPATHY
The invention provides a pharmaceutical composition and methods for the treatment of a respiratory disease that results from a viral infection. The pharmaceutical composition can include a peptide that interferes with the Src family kinase-Androgen receptor interaction (i.e., an “SA inhibitor”). The composition can also include Niacin or a Niacin derivative and/or a DNase I or fragment or derivative. The pharmaceutical composition can prevent the development of Acute Respiratory Distress Syndrome (ARDS) associated with Corona vims infection and allow the host adaptive immune response to overcome the infection.
ANTI-TUMOR FUSION PROTEIN, PREPARATION METHOD THEREFOR AND APPLICATION THEREOF
Provided are an anti-tumor fusion protein, a preparation method therefor and an application thereof. Specifically, the fusion protein contacts a CPP element, an optional linking element, and a SH2 domain of SHP2 or SHP1 or an active fragment thereof. The obtained fusion protein has an extremely excellent anti-tumor effect.
Lysosomal storage disease enzymes
The present invention provides compositions of recombinant human lysosomal acid lipase having particular glycosylation patterns for internalization into target cells, a vector containing the nucleic acid encoding human lysosomal acid lipase, a host cell transformed with the vector, pharmaceutical compositions comprising the recombinant human lysosomal acid lipase and method of treating conditions associated with lysosomal acid lipase deficiency.
Epitope focusing by variable effective antigen surface concentration
The present disclosure provides compositions and methods for the generation of an antibody or immunogenic composition, such as a vaccine, through epitope focusing by variable effective antigen surface concentration. Generally, the composition and methods of the disclosure comprise three steps: a “design process” comprising one or more in silico bioinformatics steps to select and generate a library of potential antigens for use in the immunogenic composition; a “formulation process”, comprising in vitro testing of potential antigens, using various biochemical assays, and further combining two or more antigens to generate one or more immunogenic compositions; and an “administering” step, whereby the immunogenic composition is administered to a host animal, immune cell, subject or patient. Further steps may also be included, such as the isolation and production of antibodies raised by host immune response to the immunogenic composition.
Epitope focusing by variable effective antigen surface concentration
The present disclosure provides compositions and methods for the generation of an antibody or immunogenic composition, such as a vaccine, through epitope focusing by variable effective antigen surface concentration. Generally, the composition and methods of the disclosure comprise three steps: a “design process” comprising one or more in silico bioinformatics steps to select and generate a library of potential antigens for use in the immunogenic composition; a “formulation process”, comprising in vitro testing of potential antigens, using various biochemical assays, and further combining two or more antigens to generate one or more immunogenic compositions; and an “administering” step, whereby the immunogenic composition is administered to a host animal, immune cell, subject or patient. Further steps may also be included, such as the isolation and production of antibodies raised by host immune response to the immunogenic composition.
COMPOSITIONS AND METHODS FOR TREATING AND/OR PREVENTING COAGULOPATHY AND/OR SEPSIS IN PATIENTS SUFFERING FROM BACTERIAL AND/OR VIRAL INFECTIONS
The present disclosure includes compositions and methods for treating, ameliorating, and/or preventing immune mediated pathology associated with a bacterial and/or viral infection.
COMPOSITIONS AND METHODS FOR TREATING BACTERIAL INFECTIONS
Disclosed herein is a composition comprising a biofilm degrading protease, a disulphide bond breaking agent and an antibiotic, as well as a method for debriding biofilm (e.g. on an implant) in a patient's body. The method comprises contacting the biofilm with a combination of a biofilm degrading protease and disulphide bond breaking agent.
Extracellular DNA as a therapeutic target in neurodegeneration
The invention relates to the use of deoxyribonuclease (DNase) enzyme for inhibiting progression and for prevention and treatment of neurodegeneration.
CRISPR/CAS-MEDIATED GENE CONVERSION
CRISPR/CAS-related compositions and methods for altering a cell or treating a disease, for example, by gene conversion, are disclosed.