Abstract: Recombinant SARS-CoV2 vaccine compositions and methods are presented that have unexpected cross-reactivity against a variety of other coronaviruses, and particularly against SARS-CoV1, MERS-CoV, OC43-CoV, and HKU1-CoV in addition to significant reactivity against SARS-CoV2A. Moreover, the vaccine compositions presented herein also produced cross-reactive memory B cells as well as cross-reactive memory T cells with cross-reactivity spanning a relatively wide range of different coronaviruses.

Abstract: Disclosed is a method of treating or preventing a CMV infection in a recipient of a hematopoietic cell transplant (HCT). The method entails administering an effective amount of a CMV Triplex vaccine composition to a donor and/or recipient of the hematopoietic cells.

Abstract: 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.

Abstract: The present application relates to an immunostimulatory composition including a liposome-nucleic acid complex wherein said complex contains (1) a liposome which, interms of its lipids, includes or consists of a first lipid and a second lipid, wherein the first lipid is a zwitterionic lipid and the second lipid is a cationic lipid, and (2) one or more immunostimulatory oligonucleotides and/or one or more polynucleotides. The application further relates to certain uses of the immunostimulatory composition and to methods for preparing the same. In particular, the application concerns the use of a zwitterionic lipid in an immunostimulatory composition including a liposome-nucleic acid complex to induce type I interferon immune responses and/or to reduce or even bypass TLR-mediated immune responses.

Abstract: The present invention provides a composition for raising an immune response against a tumor. The composition comprises at least one tumor antigen, a saponin-based adjuvant, a TLR ligand and a Flt3 ligand.

Abstract: The present disclosure relates to a novel lipid compound and a lipid nanoparticle composition including the same and, more specifically, the lipid nanoparticle composition includes ionized lipid, helper lipid, PEG-lipid, and additives, and may mitigate changes and side effects in the delivery mechanism and enhance the protein expression efficiency by including a biofriendly vitamin-based novel lipid compound and helper lipids including neutral lipids.

Abstract: A transplant material used for kidney xenotransplantation to primates is provided, the transplant material comprising a kidney anlage with bladder derived from a pig fetus devoid of blood vessels.

Abstract: Compositions and methods for treating rheumatoid arthritis and/or accelerated atherosclerosis are provided, including an inhibitor of oxidized or malondialdehyde-modified low density lipoprotein (LDL) for administration to a subject. Exemplary inhibitors of oxidized LDL include an anti-oxidized LDL antibody, which results in a reduction in the secretion of pro-inflammatory cytokine from primary monocyte in vitro and the plasma cytokine level of inflammatory cytokine in vivo.

Abstract: The novel synergistic combination of immune checkpoint blockade and hematopoietic stem cell transplantation and/or hematopoictic stem cell mobilization yield synergistic effects in disease therapy.

Abstract: Disclosed herein is a composition comprising a recombinant nucleic acid sequence that encodes an antibody to a Zika viral antigen, and functional fragments thereof. The invention also relates to a composition comprising the combination of a first composition that elicits an immune response in a mammal against zika virus and a second composition comprising a recombinant nucleic acid sequence encoding an antibody, a fragment thereof, a variant thereof, or a combination thereof. In some instances, the nucleic acid molecule comprises a nucleotide sequence encoding an anti-ZIKV-Envelope (anti-ZIKV E) Protein antibody.

Abstract: Disclosed herein are methods and compositions related to identification, isolation, and targeting of first responder dendritic cells (FRs). Aspects of the disclosure are directed to FR-targeting agents and methods for use of such agents, including methods for directing a diagnostic, imaging, or therapeutic molecule (e.g., an antigen) to FRs. The present disclosure includes pharmaceutical compositions comprising an FR-targeting agent and an antigen or polynucleotide encoding an antigen. Also disclosed are methods for stimulating an immune response comprising targeting an antigen to FRs.

Abstract: Provided are methods and compositions for obtaining functionally enhanced derivative effector cells obtained from the differentiation of genomically engineered iPSCs. The derivative cells provided herein have stable and functional genome editing that delivers improved or enhanced therapeutic effects. Also provided are therapeutic compositions and the use thereof comprising the functionally enhanced derivative effector cells alone, or with antibodies or checkpoint inhibitors in combination therapies.

Abstract: Disclosed herein are chimeric antigen receptor (CAR) polypeptides that can be used with adoptive cell transfer having endodomains with a customized number of immunoreceptor tyrosine-based activation motifs (ITAMs). In some embodiments, the endodomain has a backbone of a receptor endodomain, such as CD8, CD3?, CD3?, CD3?, CD3?, CD32 (Fc gamma RIIa), DAP10, DAP12, CD79a, CD79b, Fc?RI?, Fc?RIII?, Fc?RI? (FCERIB), and Fc?RI? (FCERIG). In particular embodiments disclosed herein, the backbone is a DAP12 backbone. The disclosed endodomain is engineered to express at least one heterologous ITAM, including 1, 2, 3, 4, 5, or 6 ITAMs.

Abstract: Methods of using polypeptides to modulate transforming growth factor-? (TGF?) signaling (e.g., TGF? receptors, antibodies or antigen-binding fragments thereof that specifically bind TGF? or a TGF? receptor) are provided. Compositions comprising the antibodies or fragments thereof and methods of using the same for treatment of diseases involving TGF? activity are provided. Nucleic acids, recombinant expression vectors, host cells, antigen binding fragments, and pharmaceutical compositions comprising these antigen binding agents and fragments thereof are also disclosed. The invention also provides therapeutic methods for utilizing the TGF? signaling modulators are provided herein.

Abstract: Provided herein are mutants of estrogen receptor alpha ligand binding domain (ER-LBD), and chimeric proteins including such mutant ER-LBD. Also provided are methods of modulating transcription and modulating localization of such chimeric proteins.

Abstract: The present invention includes compositions and methods for an DPP6 specific chimeric antigen receptor (CAR). In certain embodiments the DPP6 specific CAR is expressed on a T regulatory cell. In certain embodiments, the DPP6 specific CAR is used to treat type 1 diabetes.

Abstract: Provided are B cell-activating factor receptor (BAFF-R)-binding molecules, in particular, to human antibodies specific for BAFF-R, including antibody fragments. The present disclosure further relates to recombinant receptors, including chimeric antigen receptors (CARs) that contain such antibodies or fragments, and polynucleotides that encode the antibodies, antigen-binding fragments or receptors specific for BAFF-R. Also provided are CARs which contain extracellular binding domains that bind to BAFF-R and B-lymphocyte antigen CD19 (CD19), genetically engineered cells expressing such CARs, and uses thereof in adoptive cell therapy.

Abstract: High purity magnetosomes produced by magnetotactic bacteria, wherein the metallic composition of the magnetosome is characterized by a high level of iron purity, preferentially not achieved through a chemical synthesis. The high purity magnetosomes are further characterized by having a content of Cd, Pb, Ni, Co, Al, Mn, Zn, Li, and/or Cu.

Abstract: This disclosure relates to the use of an oxygen-containing liquid as a counter measure against vesicants.

Abstract: Compositions and methods of using and manufacturing an oral pharmaceutical anhydrous suspension are provided. The anhydrous suspension is suitable to suspend active pharmaceutical ingredients (APIs), improve the stability of oral pharmaceutical suspensions, and help in the formation of a thermo-reversible gel and shear thinning to keep the APIs suspended. The anhydrous suspension disclosed herein may include glyceryl distearate NF (e.g., in an amount of from about 0.1 wt. % to about 10 wt. %) and stearoyl polyoxyl-32 glycerides NF (e.g., in an amount of from about 0.1 wt. % to about 10 wt. %). In embodiments, the anhydrous suspension also includes glycerin (e.g., in an amount of about 5 wt. %). The anhydrous suspension may also include one or more of medium chain triglycerides NF, vitamin E acetate (DL) USP liquid (1 IU/mg), flavoring agent (e.g., a sweeter or other flavoring agent), ascorbyl palmitate NF, and other components.

Abstract: Provided herein are compounds, such as compounds of Formula (I), and pharmaceutically acceptable salts thereof, and compositions, methods, uses, and kits thereof. The compounds provided herein are lipids useful for delivery of agents, including polynucleotides such as mRNA, for the treatment and/or prevention of various diseases and conditions (e.g., genetic diseases, proliferative diseases, hematological diseases, neurological diseases, liver diseases, spleen diseases, lung diseases, painful conditions, psychiatric disorders, musculoskeletal diseases, metabolic disorders, inflammatory diseases, and autoimmune diseases). Also provided herein are methods of synthesis of compounds of Formulae (I) and (II).

Abstract: A solidified melt extrudate comprising an active pharmaceutical ingredient in an amorphous form and a combination of at least two polymers.

Abstract: Described herein is a composition and method of preventing COVID-19 with lipid-peptide fusion antiviral therapy.

Abstract: This invention relates to dual activity compounds and uses thereof for the treatment of type 2 diabetes in patients who are overweight or obese.

Abstract: The present invention is related to the field of pulmonary therapeutics. In particular, the compositions described herein are used in methods of treating lung disease and injury including but not limited to acute respiratory distress syndrome (ARDS), COVID infection, cytokine storms, sepsis and related conditions. These compositions include, but are not limited to, peptide variants and compositions that inhibit activity of a receptor complex formed by triggering receptors expressed on myeloid cells (TREM; i.e., TREM-1, TREM-2, TREM-3 or TREM-4) and DNAX activation protein of 12 kDa (DAP 12).

Abstract: Disclosed are POEGMA-aptamer conjugates with a reduced or eliminated host-immune response. An example conjugate includes an aptamer conjugated to a POEGMA having a plurality of side chains, where each side chain includes 1 to 6 monomers of ethylene glycol repeated in tandem. Also disclosed are methods of making the conjugate and methods of using the conjugate. An example method of use includes a method of controlling coagulation in a subject.

Abstract: The present invention relates to dosing regimens of peptide conjugates of toporsomcrase I inhibitors (e.g., a peptide conjugate of the topoisomerase I inhibitor exatecan), which is useful for the treatment of diseases such as cancer.

Abstract: Disclosed herein are methods for treating head and neck squamous cell carcinoma (HNSCC) in a subject in need thereof, comprising administering IL-2 conjugates in combination with cetuximab.

Abstract: The present invention relates to compositions and methods for effective delivery of a therapeutic agent to a subject using a delivery vehicle comprising a domain to evade the subject's immune response. In some embodiments, the present invention relates to compositions and methods for targeted delivery of a therapeutic agent to a subject using a delivery vehicle comprising a domain to evade the subject's immune response and a domain for targeting a specific cell type. The invention also relates to methods of use of the compositions of the invention for the treatment of diseases and disorders, including the treatment of diseases and disorders in subjects having an inflammatory or autoimmune disease or disorder.

Abstract: The present disclosure relates to cell penetrating anti-DNA antibodies conjugated to a pay load and methods of delivering these antibody conjugates to the brain. Compositions comprising these antibody conjugates may be useful for detecting and treating disease.

Abstract: The present disclosure demonstrates that the amount of soluble folate receptor alpha (FR?) present in a cancer patient is a strong predictor of the efficacy of FR?-targeting therapies. Surprisingly, increased levels of soluble FR? are associated with improved outcomes. Accordingly, the present disclosure provides methods for treating cancer in patients with soluble FR? and methods for identifying a cancer as likely to respond to an anti-FR? therapy based on soluble FR? levels.

Abstract: The present invention relates to antibody-drug conjugates comprising an antibody that binds to CEACAM5 conjugated to a drug, such as a Topoisomerase I inhibitor. Also provided herein are methods of treating cancer comprising administering such ADCs.

Abstract: The present disclosure provides antibodies and antigen-binding fragments that target transferrin receptor. Also provided is the use of these antibodies and antigen-binding fragments as carriers to deliver therapeutic molecules across the blood-brain barrier.

Abstract: The present disclosure provides a method to translocate a molecule across cellular membrane by using a transferrin-binding protein. The transferrin-binding protein is capable of specifically binding to transferrin and not disturbing the interaction between transferrin and transferrin receptor.

Abstract: The present disclosure relates to cell penetrating anti-DNA antibodies conjugated to a payload and methods of delivering these antibody conjugates to the brain. Compositions comprising these antibody conjugates may be useful for detecting and treating disease.

Abstract: The present invention relates to conjugates targeting uPARAP, in particular antibody-drug conjugates (ADCs) comprising monoclonal antibodies directed against the N-terminal region of uPARAP, and their use in delivery of active agents to cells and tissues expressing uPARAP. The invention further relates to the use of said ADCs in the treatment of diseases involving uPARAP expressing cells, such as cancer.

Abstract: An antibody-drug conjugate targeting a poliovirus receptor-like molecule 4 (Nectin-4). The antibody-drug conjugate can be used for preparing a drug for treating Nectin-4-related diseases. The antibody-drug conjugate has strong targeting properties to Nectin-4 and a strong endocytosis effect via the target, and has an excellent tumor-killing effect.

Abstract: The invention relates to EGFR targeting Fc antigen binding fragment-drug conjugates (EGFR Fcab-drug conjugates) and the use of the EGFR Fcab-drug conjugates of the present invention for the treatment and/or prevention of hyperproliferative diseases and disorders in mammals, especially humans, and pharmaceutical compositions containing such EGFR Fcab-drug conjugates. Further, the invention relates to EGFR Fcab-label conjugates and diagnostic compositions containing such EGFR Fcab-label conjugates.

Abstract: Compounds and compositions are disclosed in which a Drug Unit is linked to a targeting Ligand Unit through a self-stabilizing Linker Unit from which a drug compound or active drug moiety is released at the targeted site of action. Methods for treating diseases characterized by the targeted abnormal cells, such as cancer or an autoimmune disease using the compounds and compositions of the invention are also disclosed.

Abstract: The present disclosure pertains to polypeptides (in particular, polypeptides comprising Eph receptor domain(s), i.e., Eph receptor-derived polypeptides), nanovesicles (e.g., extracellular vesicles (EVs) and hybridosomes) comprising such polypeptides. Said polypeptides can act as membrane bound protein scaffolds to which molecules of interest can be attached. The polypeptides and nanovesicles can be used in targeting, therapeutic and/or diagnostic applications. Also provided are nucleic acids and expression vectors encoding such polypeptides as well as cells expressing said polypeptides. Further provided are methods for producing nanovesicles comprising such polypeptides. Compositions comprising such polypeptides or nanovesicles as well as their uses are also described.

Abstract: Disclosed are fibrin-specific nanogels. The fibrin-specific nanogels can comprise a fibrin binding moiety conjugated to a polymeric matrix (e.g., a crosslinked polyacrylamide matrix). By varying the morphology of fibrin-specific nanogels (e.g., by varying the crosslinker density and/or the three-dimensional structure of the fibrin-specific nanogels), fibrin-specific materials which mimic the biophysical characteristics of platelets can be formed. The resulting materials can be used in a variety of biomedical applications (e.g., for drug delivery, to promote wound healing, to treat thrombotic events, and to treat coagulative disorders).

Abstract: Ligand-bound gold clusters and compositions comprising the ligand-bound gold clusters are used for treating depression and manufacturing a medicament for treatment of depression. Methods for treating depression.

Abstract: The application describes methods and compositions comprising ceDNA vectors useful for the expression of antibodies and antigen-binding fragments thereof in a cell, tissue or subject, and methods of treatment and/or prevention of various diseases, disorders and cancers.

Abstract: Provided herein are lipids having the Formula I or Formula la, and pharmaceutically acceptable salts thereof, wherein R?, R1, R2, R3, R4, R5, R6a, R6b, X1, X2, and n are as defined herein for Formula I and Formula la, respectively. Also provided herein are lipid nanoparticle (LNP) compositions comprising lipid having the Formula I or la and a capsid-free, non-viral vector (e.g., ceDNA). In one aspect of any of the aspects or embodiments herein, these LNPs can be used to deliver a capsid-free, non-viral DNA vector to a target site of interest (e.g., cell, tissue, organ, and the like).

Abstract: The disclosure features compositions and methods for the treatment of sensorineural hearing loss and auditory neuropathy, particularly forms of the disease that are associated with mutations in otoferlin (OTOF), by way of OTOF gene therapy. The disclosure provides a variety of compositions that include a first nucleic acid vector that contains a polynucleotide encoding an N-terminal portion of an OTOF protein and a second nucleic acid vector that contains a polynucleotide encoding a C-terminal portion of an OTOF protein. These vectors can be used to increase the expression of OTOF in a subject, such as a human subject suffering from sensorineural hearing loss.

Abstract: Provided herein are compositions, systems, and methods comprising effector proteins, effector partners, and uses thereof. These effector proteins may be characterized as CRISPR-associated (Cas) proteins. Various compositions, systems, and methods of the present disclosure may leverage the activities of these effector proteins for the modification, detection, and/or engineering of nucleic acids.

Abstract: Provided in the present disclosure include targeted therapeutics for regulating gene transcription at a specific locus (or loci). Such targeted therapeutics can be used as novel genomic therapeutics for treating and/or preventing a disease or disorder that is tightly associated with the locus where the gene transcription is reprogrammed using the present systems. The regulatory system comprises a macromolecular complex of transcription effector proteins that is directed to a locus using CRISPR-dCas9.

Abstract: A bacterial targeting vector and use thereof in tumor treatment, in particular, a bacterial targeting vector carrying a cytokine such as interleukin 6 and interleukin 10 or carrying a polynucleotide such as mRNA of the cytokine and use thereof in tumor treatment. An attenuated chassis bacterium capable of targeting solid tumors can be used as a delivery carrier of a tumor therapy molecule, and achieve expression and controllable release of a human/murine cytokine, thereby achieving anti-tumor effects.

Abstract: Described herein are variant adeno-associated virus (AAV) capsid polypeptides and gene therapeutics thereof for use in the treatment or prevention of hearing loss.

Abstract: The disclosure features compositions and methods for the treatment of inner ear dysfunction, such as hearing loss or vestibular dysfunction, that reduce inflammatory or cell-mediated immune toxicity in the inner ear, thereby improving transduction and therapeutic efficacy. The disclosure provides a variety of compositions that include a nucleic acid vector that contains a polynucleotide encoding a therapeutic agent operably linked to a ubiquitous promoter and inhibitor of inflammatory or immune cell signaling. The disclosed compositions and methods can be used to increase expression of the therapeutic agent in a subject, such as a human subject suffering from an inner ear dysfunction, while minimizing undesirable immune activation resulting from off-target expression of the target protein in immune cells of the inner ear.