Patent classifications
C12Y304/21075
COMPLEMENT FACTOR I-RELATED COMPOSITIONS AND METHODS
Provided herein are Complement Factor I (CFI) variants that exhibit at least one improved characteristic relative to a wild type CFI. CFI variants of the disclosure can exhibit tunable specificity and activity. Also included are CFI-containing fusion constructs comprising at least one domain of CFI, for example, wild type full length CFI fused to human serum albumin. Also included are methods of making and using such CFI variants and fusion constructs. The CFI variants and fusion constructs provided herein may be useful for treating a disease or condition associated with dysregulation of the complement system or a deficiency of CFI.
OLIGONUCLEOTIDES FOR SARS-CoV-2 MODULATION
- Anastasia Khvorova ,
- Jonathan Watts ,
- Zachary Kennedy ,
- Annabelle Biscans ,
- Bruno Miguel da Cruz Godinho ,
- Chantal Ferguson ,
- Dimas Echeverria Moreno ,
- Ken Yamada ,
- Daniel O'Reilly ,
- Kathryn Monopoli ,
- Vignesh Narayan Hariharan ,
- Qi Tang ,
- Sarah Davis ,
- Samuel Hildebrand ,
- Socheata Ly ,
- Minwook Shin ,
- Pranathi Meda Krishnamurthy ,
- Nicholas McHugh ,
- Jacquelyn Sousa ,
- Jillian Caiazzi ,
- Yann Thillier ,
- Gitali Devi
This disclosure relates to novel SARS-CoV-2 targeting sequences. Novel SARS-CoV-2 targeting oligonucleotides for the treatment of SARS-CoV-2 infection are also provided.
METHODS OF PRODUCING RECOMBINANT COMPLEMENT PROTEINS, VECTORS AND THERAPEUTIC USES THEREOF
Aspects of the present invention relate to the recombinant production of a mature complement system protein. Certain embodiments of the present invention relate to recombinant production of fully mature human complement Factor I protein (CFI). Included herein are details of an expression vector with which to recombinantly express fully mature human CFI from mammalian cells. Further disclosed are chromatography steps with which to purify recombinantly expressed CFI. Certain aspects of the present invention relate to the use of an expression system in gene therapy and the like. Certain embodiments of the present invention relate to use of said vector as a medicament, for example for use in the treatment of complement-mediated disorders.
Humanized monoclonal antibodies against the extracellular domain of human death receptor 5
The present invention provides a humanized monoclonal antibody against extracellular domain of human death receptor 5, comprising a light chain variable region, whose amino acid sequence has at least 90% identity with the amino acid sequence shown as SEQ ID NO: 1, a heavy chain variable region, whose amino acid sequence has at least 90% identity with the amino acid sequence shown as SEQ ID NO: 2, and constant region derived from human antibody. The present invention also provides nucleotide sequence encoding said humanized monoclonal antibody, a recombinant eukaryotic expression vector, a process for preparing the humanized monoclonal antibody, and the composition and use therefore. Said humanized monoclonal antibody of the present invention shows specific apoptosis-inducing activity against various cancer cells both in vivo and in vitro, and thus it can be used alone or in combination with natural ligand of DR5, apoptosis-inducing ligand associated with tumor nerosis factor or other medicaments for the treatment of a variety of cancers as well as other diseases associated with high DR5 expression.
COMPLEX FOR INTRACELLULAR DELIVERY OF MOLECULES
A fusion protein and a complex containing same, capable of being used for the intracellular delivery of cargo molecules. The fusion protein and complex can implement the efficient release of cargo molecules from endocytic vesicles, thereby significantly improving the cytoplasmic delivery efficiency of the cargo molecules. One cargo molecules can be obtained in cytoplasms, they can exert any function related thereto. The fusion protein and complex provide effective means for affecting biological mechanisms and pathways of cells, and can be used in various fields such as research, treatment, and diagnosis.
BINDING AGENTS TO PRE-FUSION STATE SARS-COV-2 SPIKE PROTEIN
Disclosed is producing recombinant SARS-CoV-2 spike protein in a pre-fusion state, using furin knock out or knockdown mammalian cells (such as HEK293, CHO or other mammalian cells) and using them to generate antibodies and related binding agents. The antibodies/binding agents can be used in SARS-CoV-2 detection assays or in diagnosis of active or prior infection with SARS-CoV-2; in prophylaxis or as a therapeutic; or for prophylactic or therapeutic use against coronaviruses related to SARS-CoV-2.
METHODS AND COMPOSITIONS FOR IMMUNOMODULATION
The methods and uses described herein relate to the modulation of the immune system by modulation of Sema3F levels and/or activity, e.g. suppressing allograft rejection or inflammation by administering a Sema3F agonist or increasing an immune response by administering a Sema3F inhibitor.
Combinatorial gene construct and non-viral delivery for anti-obesity
The invention provides a plasmid comprising two or more anti-obesity genes. Also provided by the invention are compositions and host cells comprising the plasmid and methods of increasing the metabolic activity in a mammal. The invention provides a plasmid comprising two or more of (a) a nucleic acid sequence encoding islet amyloid polypeptide (IAPP), (b) a nucleic acid sequence encoding leptin (LEP), and (c) a nucleic acid sequence encoding fibronectin type III domain containing 5 (FNDC5).
Binding agents to pre-fusion state SARS-CoV-2 spike protein
Disclosed is producing recombinant SARS-CoV-2 spike protein in a pre-fusion state, using furin knock out or knockdown mammalian cells (such as HEK293, CHO or other mammalian cells) and using them to generate antibodies and related binding agents. The antibodies/binding agents can be used in SARS-CoV-2 detection assays or in diagnosis of active or prior infection with SARS-CoV-2; in prophylaxis or as a therapeutic; or for prophylactic or therapeutic use against coronaviruses related to SARS-CoV-2.
TARGETED DELIVERY OF THE ABRIN-A A-CHAIN TO CANCER CELLS
A targeted delivery of recombinant therapeutic protein to inhibit the growth of cancer cells is disclosed. The recombinant protein derived from Abrin-a A-chain able to inhibit the growth of cancer cell and conjugated to stxB to form Abrin-f-stxB. The abrin toxin A chain and the Shiga toxin B chain are linked with the furin linker to form a recombinant therapeutic protein as chemotherapeutic agent to inhibit the growth of cancer cells. A conjugate for the delivery of a compound into cancer cells, comprising a first module mediates cell targeting and facilitates cellular uptake, a second module facilitates transport to the endoplasmic reticulum (ER), a third module mediates translocation from the ER to the cytosol and a compound that is desired to be delivered to the cytosol. The recombinant protein Abrin-f-stxB exhibits high binding affinity of stxB as a drug delivery system to Gb3 receptor in targeted cancer diagnosis and treatment.