Abstract: The present invention is directed to a monoclonal anti-human BCMA antibody, or a single-chain variable fragment (scFv), comprising VH having the amino acid sequence of SEQ ID NO: 6 and VL having the amino acid sequence of SEQ ID NO: 7. The present invention is also directed to a BCMA chimeric antigen receptor (CAR) comprising from N-terminus to C-terminus: (i) a single-chain variable fragment (scFv) of the present invention, (ii) a transmembrane domain, (iii) at least one co-stimulatory domains, and (iv) an activating domain. The monoclonal antibody of the present invention exhibits selective and high-affinity binding to BCMA. BCMA CAR-T cells based on BCMA scFv of the present invention significantly decreases multiple myeloma tumor growth in an animal model.

Abstract: A system for genetic editing of a transthyretin (TTR) gene, comprising (i) a Cas12i2 polypeptide or a first nucleic acid encoding the Cas12i2 polypeptide, and (ii) an RNA guide or a second nucleic acid encoding the RNA guide, wherein the RNA guide comprises a spacer sequence specific to a target sequence within an TTR gene. Also provided herein are methods for editing a TTR gene using the gene editing system disclosed herein and/or for treating diseases associated with the TTR gene.

Abstract: A method of treating viral infection in an animal is provided. Oleandrin or digoxin are administered to treat viral infection is caused by any of the following virus families: Arterviridae, Astroviridae, Bomaviridae, Circoviridae, Coronaviridae, Chordopoxvirinae, Flaviviridae, Herpesviridae, Orthomyxoviridae, Papillomaviridae, Papovaviridae, Paramyxoviridae, Parvoviridae, Picornaviridae, Poxviridae, Reoviridae, Retroviridae, Rhabdoviridae, and Togaviridae. An antiviral composition may be administered to prevent the disease state of the viral infection. Domesticated, companion, and livestock animals can be treated.

Abstract: The present invention relates to a composition, a preparation method therefor, and a use thereof. The composition comprises a PEDF-derived short peptide (PDSP), a stabilizer, and a buffer agent. The pH of the composition ranges from 4 to 9. The stabilizer is a copolymer of vinyl pyrrolidone and vinyl acetate. The buffer agent is selected from a citric acid buffer system, a phosphoric acid buffer system, a boric acid buffer system, a histidine buffer system, or a combination thereof. The composition has improved stability, small irritation to eyes, and/or high bioavailability.

Abstract: The present disclosure provides one or more engineered nucleases and systems, compositions, and methods thereof, wherein the one or more engineered nucleases can be used to effect binding, cleaving, and/or editing a target polynucleotide sequence. The one or more engineered nucleases can be engineered variants of a small CRISPR/Cas protein.

Abstract: The disclosure describes novel systems, methods, and compositions for the manipulation of nucleic acids in a targeted fashion. The disclosure describes non-naturally occurring, engineered CRISPR systems, components, and methods for targeted modification of DNA, RNA, and protein substrates. Each system includes one or more protein components and one or more nucleic acid components that together target DNA, RNA, or protein substrates.

Abstract: Provided herein are lipid particles, such as lentiviral particles, that incorporate or are pseudotyped with a truncated Baboon Endogenous Retrovirus (BaEV) envelope glycoprotein that contains a cytoplasmic tail with a partial inhibitory R peptide that is less than the full length wildtype BaEV inhibitory R peptide. Also provided herein are polynucleotides encoding the truncated BaEV envelope glycoproteins and producer cells for preparation of the lipid particles, such as lentiviral particles, containing the truncated BaEV envelope glycoproteins, as well as methods for preparing and using the lipid particles, such as lentiviral particles.

Abstract: Provided herein are methods and systems for high-throughput cell line development.

Abstract: Provided herein are gene editing systems and/or compositions comprising RNA guides targeting HAO1 for use in genetic editing of the HAO1 gene. Also provide herein are methods of using the gene editing system for introducing edits to the HAO1 gene and/or for treatment of primary hyperoxaluria (PH), and processes for characterizing the gene editing system.

Abstract: The present disclosure provides a method for extracting Gymnadenia conopsea(L.)R.Br., which includes the following steps: (1) the root tuber of Gymnadenia conopsea(L.)R.Br. is soaked in water so that it can be fully infiltrated until having been taken as a sample, no white core is observed; (2) the root tuber of Gymnadenia conopsea(L.)R.Br. is ultrafinely comminuted by wet method to obtain a dispersion slurry; (3) additional water is supplemented into the dispersion slurry to obtain diluted dispersion followed by heating and adding neutral protease, and then extraction is carried out through circulation and homogenization by the homogenization pump to obtain extracted material fluid; (4) heat preservation and enzyme inactivation; (5) coarse filtration to obtain a coarse filtrate; and (6) fine filtration to obtain a fine filtrate. The disclosure also provides a related extract of Gymnadenia conopsea(L)R.Br.

Abstract: Methods and systems for quantification of an abundance of one or more payloads (e.g. proteins) in a mixture (e.g. a complex mixture (e.g. in vivo)) using barcodes (e.g. peptide barcodes), binders (e.g. polypeptide binders), and binding agents (e.g. phage) are provided herein.

Abstract: Compositions and methods for genetically modifying felines or feline cells are described. The compositions and methods are useful for knocking out all or a portion of a Fel d 1 gene from a feline genome. Feline cells and organisms in which all or a portion of the Fel d 1 gene is knocked out are also described. The compositions and methods may include reagents and procedures for CRISPR-Cas9-mediated genomic editing of Fel d 1.

Abstract: The present disclosure provides adeno-associated virus (AAV) virions with altered capsid protein that binds heparan sulfate proteoglycans, where the AAV virions exhibit greater infectivity of retinal cells, altered tropism and/or the ability to bind and cross the inner limiting membrane following intravitreal injection. The present disclosure further provides methods of delivering a gene product to a retinal cell in an individual, and methods of treating ocular disease.

Abstract: The present invention is directed to a chimeric antigen receptor (CAR) fusion protein comprising from N-terminus to C-terminus: (i) a single-chain variable fragment (scFv) comprising VH and VL, wherein scFv binds to human PLAP (placental alkaline phosphatase), (ii) a transmembrane domain, (iii) a co-stimulatory domain of CD28, OX-40, GITR, or 4-1BB, and (iv) CD3 an activating domain. The present invention is also directed to T cells, natural killer (NK) cells, or macrophages, modified to express the CAR of the present invention. The present invention is further directed to a method for treating PLAP-positive cancer cells by administering PLAP-CAR-T cells, PLAP-CAR-NK cells, or PLAP-CAR-macrophages to the patients.

Abstract: An immunoconjugate includes an anti-ENO-1 antibody, or a binding fragment thereof, and a therapeutic agent or a label, having the formula: Ab-(L-D)m, wherein Ab is the anti-ENO-1 antibody or the binding fragment thereof, L is a linker or a direct bond, D is the therapeutic agent or the label, and m is an integer from 1 to 12. The antibody may be a monoclonal antibody, which may be a humanized antibody or fully human antibody. A method for treating an inflammatory disease, immune disorder, or cancer includes administering to a subject in need of such treatment a pharmaceutically effective amount of an immunoconjugate containing an antibody against ENO-1, or a binding fragment thereof, and a therapeutic agent covalently conjugated with the antibody.

Abstract: The present disclosure relates to novel SOS1 inhibitors, pharmaceutical compositions containing such compounds, and their use in prevention and treatment of cancer and related diseases and conditions.

Abstract: The present disclosure generally relates to systems and methods for treating vitiligo. In one set of embodiments, the present disclosure comprises a composition comprising ethyl pyruvate. The composition may be formulated for application to the skin of a subject, for instance, as a gel, lotion, cream, ointment, soap, or stick. In some cases, the composition may comprise a lecithin, such as phosphatidylcholine. In certain embodiments. the lecithin is present as a liquid crystal, and/or in liposomes, micelles, or other vesicles. Other aspects of the present disclosure are generally directed to methods of making or using such compositions, methods of promoting such compositions, kits including such compositions, or the like.

Abstract: The invention involves generating a T cell response to subdominant antigens and using the cells to therapeutically change the cellular homeostasis and nature of the immune response. In a preferred embodiment, the cells are generated outside of the patient avoiding the influence of the patient's immunologic milieu. By stimulating and growing the T cells from a patient in a tissue culture to one or more subdominant antigens and the transplanting them into the patient, if enough cells are expanded and transplanted, the transplanted cells overwhelm the endogenous dominant T cells in the response to either break or induce immune tolerance or otherwise modify the immune response to the cells or organism expressing that antigen. When the memory cells are established they are then reflective of this new immunodominance hierarchy so that the desired therapeutic effect is long lasting.

Abstract: The present disclosure relates to novel quinolinone-8-carbonitrile based compounds, pharmaceutical compositions containing such compounds, and their use in prevention and treatment of cancer and related diseases and conditions. The compounds disclosed herein exhibit androgen receptor degradation activity.

Abstract: The invention discloses a disposable reaction device, a tracer synthesizer and a method for producing a tracer. The disposable reaction device comprises: (1) a disposable reagent bottle, which is used for loading the substances required for reaction, including radioisotopes, solvents, reagents, and reactants; the load can be filled directly or through a disposable liquid pipeline into the disposable reactor; (2) disposable reactors, which are used to contain radioisotopes and at least one reagent for reaction, and can be sealed with pierceable materials; (3) disposable liquid pipelines, including evaporation elements, filling elements, retraction elements, and mass transfer pipelines. The beneficial advantages brought by the present invention are: First, there is no need to wait for 10 half-lives of radioactive decay before proceeding to the next tracer production. Secondly, the disposable reaction device enables the synthesis of the same or different tracers to be repeated in a short time.