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1.WO/2020/103777INTERLEUKIN 21 PROTEIN (IL21) MUTANT AND USE THEREOF
WO 28.05.2020
Int.Class C07K 14/54
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
435from animals; from humans
52Cytokines; Lymphokines; Interferons
54Interleukins (IL)
Appl.No PCT/CN2019/119088 Applicant HYQUO MOLECULE (BEIJING) TECHNOLOGY CO., LTD Inventor ZHANG, Xin
Provided in the present invention are an interleukin 21 protein (IL21) mutant and a use thereof. In the mutant, both ILE at position 16 and SER at position 70 of wild-type IL21 are mutated into CYS, and disulfide bonds are formed between the two mutated CYSs. Also provided is a fusion protein containing the IL21 protein mutant. The IL21 protein mutant or fusion protein can be used for regulating or activating immunity, resisting tumors and promoting the differentiation and proliferation of B cells, T cells or NK cells.
2.WO/2020/104467RECOMBINANT VECTORS SUITABLE FOR THE TREATMENT OF IPEX SYNDROME
WO 28.05.2020
Int.Class C12N 15/86
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
79Vectors or expression systems specially adapted for eukaryotic hosts
85for animal cells
86Viral vectors
Appl.No PCT/EP2019/081820 Applicant INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) Inventor ANDRE, Isabelle
IPEX (Immune dysregulation Polyendocrinopathy X linked) syndrome is a primary immunodeficiency caused by mutations in the gene encoding the transcription factor forkhead box P3 (FOXP3), which leads to the loss of function of thymus-derived CD4+CD25+ regulatory T (tTreg) cells. Preclinical and clinical studies suggest that T cell gene therapy approaches designed to selectively restore the repertoire of Treg cells by transfer of wild type FOXP3 gene is a promising potential cure for IPEX. However, there is still a need for a vector that can be used efficiently for the preparation of said Treg cells. The inventors thus compared 6 different lentiviral constructs according to 4 criteria (vector titers, level of transduction of human CD4+ T cells, level of expression of FOXP3 and ΔLNGFR genes, degree of correlation between both expression) and selected one construct comprising a bidirectional PGK-EF1a promoter that showed remarkable efficiency.
3.WO/2020/106997METHODS AND MATERIALS FOR REDUCING AGE-RELATED STRIATED MUSCLE AND COGNITIVE DECLINE
WO 28.05.2020
Int.Class C07K 14/71
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
435from animals; from humans
705Receptors; Cell surface antigens; Cell surface determinants
71for growth factors; for growth regulators
Appl.No PCT/US2019/062682 Applicant UNIVERSITY OF PITTSBURGH - OF THE COMMONWEALTH SYSTEM OF HIGHER EDUCATION Inventor AMBROSIO, Fabrisia
This document provides methods and materials for treating aging. For example, a mammal having, or at risk for developing, an age-related impairment (e.g., age-related cognitive decline) can be treated by increasing the level of one or more myokine polypeptides (e.g., one or more Klotho polypeptides) within cells within the mammal. This document also provides methods and materials for increasing the ability of muscle progenitor cells to regenerate muscle cells by increasing the level(s) of one or more myokine polypeptides (e.g., an α-Klotho polypeptide) within a muscle progenitor cell.
4.WO/2020/107037COMPOUNDS AND METHODS FOR TREATING NEURODEGENERATIVE DISEASES
WO 28.05.2020
Int.Class C07K 16/44
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
16Immunoglobulins, e.g. monoclonal or polyclonal antibodies
44against material not provided for elsewhere
Appl.No PCT/US2019/063113 Applicant PRESIDENT AND FELLOWS OF HARVARD COLLEGE Inventor MORDES, Daniel, A.
Disclosed are compounds and methods for treating diseases and conditions related to toxic dipeptide repeats including Amyotrophic Lateral Sclerosis (ALS) and frontotemporal dementia (FTD). Also disclosed are method of screening for agents that treat or prevent diseases and conditions related to toxic dipeptide repeats.
5.WO/2020/103836OX40 ANTIBODY, PREPARATION METHOD THEREOF AND USE THEREOF
WO 28.05.2020
Int.Class C07K 16/28
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
16Immunoglobulins, e.g. monoclonal or polyclonal antibodies
18against material from animals or humans
28against receptors, cell surface antigens or cell surface determinants
Appl.No PCT/CN2019/119515 Applicant SHANGHAI PHARMAEXPLORER CO., LTD. Inventor CHAI, Xiaojuan
Provided are a murine or human-mouse chimeric antibody targeting OX40 and a preparation method therefor, wherein the antibody binds to the OX40 antigen and has an anti-tumor activity and other activities.
6.WO/2020/106566REGULATABLE FUSOGENIC ONCOLYTIC HERPES SIMPLEX VIRUS TYPE 1 VIRUS AND METHODS OF USE
WO 28.05.2020
Int.Class C12N 15/86
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
79Vectors or expression systems specially adapted for eukaryotic hosts
85for animal cells
86Viral vectors
Appl.No PCT/US2019/061662 Applicant THE BRIGHAM AND WOMEN'S HOSPITAL, INC. Inventor YAO, Feng
Malignant tumors that are resistant to conventional therapies represent significant therapeutic challenges. An embodiment of the present invention provides a regulatable fusogenic oncolytic herpes simplex virus-1 that is more effective at selective killing target cells, such as tumor cells. In various embodiments presented herein, the oncolytic virus described herein is suitable for treatment of solid tumors, as well as other cancers.
7.WO/2020/106776METHODS AND COMPOSITIONS FOR PREPARING NUCLEIC ACIDS THAT PRESERVE SPATIAL-PROXIMAL CONTIGUITY INFORMATION
WO 28.05.2020
Int.Class C12Q 1/6806
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
1Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
68involving nucleic acids
6806Preparing nucleic acids for analysis, e.g. for polymerase chain reaction assay
Appl.No PCT/US2019/062298 Applicant ARIMA GENOMICS, INC. Inventor SCHMITT, Anthony
Provided herein are methods and compositions for preparing nucleic acids in samples that preserve spatial-proximal contiguity information. Samples include, but are not limited to, formalin-fixed paraffin-embedded (FFPE) samples, deeply formalin-fixed samples and samples that comprise protein: cfDNA complexes.
8.WO/2020/106876COMPOSITIONS AND METHODS FOR INCREASING FETAL HEMOGLOBIN AND TREATING SICKLE CELL DISEASE
WO 28.05.2020
Int.Class A61K 38/46
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
38Medicinal preparations containing peptides
16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
43Enzymes; Proenzymes; Derivatives thereof
46Hydrolases (3)
Appl.No PCT/US2019/062461 Applicant FULCRUM THERAPEUTICS, INC. Inventor RAHL, Peter
The present invention relates to compositions and methods of increasing levels of fetal hemoglobin (HbF) in cells. The present invention further relates to methods for treating patients suffering from blood cell diseases, including those associated with reduced amounts of functional adult hemoglobin (HbA), such as sickle cell disease and β-thalassemias.
9.WO/2020/106906METHODS FOR TARGETED NUCLEIC ACID LIBRARY FORMATION
WO 28.05.2020
Int.Class C12N 15/10
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
10Processes for the isolation, preparation or purification of DNA or RNA
Appl.No PCT/US2019/062507 Applicant AVIDA BIOMED, INC. Inventor WANG, Heng
The present disclosure provides targeted hybridization and/or proximity ligation of a probe for amplification and analysis of target sequences. The hybridization of the probe to the target sequences can be direct or through indirect association.
10.WO/2020/106916RECOMBINANT VIRAL VECTORS AND NUCLEIC ACIDS FOR PRODUCING THE SAME
WO 28.05.2020
Int.Class C07K 14/47
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
435from animals; from humans
46from vertebrates
47from mammals
Appl.No PCT/US2019/062531 Applicant STRIDEBIO, INC. Inventor THOMAS, Darby
Described herein are nucleic acids, AAV transfer cassettes and plasmids used in the production of recombinant adeno-associated viral (rAAV) vectors. The disclosed nucleic acids, cassettes and plasmids comprise sequences that express one or more transgenes having therapeutic efficacy in the amelioration, treatment and/or prevention of one or more diseases or disorders.