Abstract: Provided herein are therapeutic agents having specificity for having inhibitory activity against cancer cells that overexpress human epidermal growth factor receptor (HER) genes, including therapeutic agents comprising one or more HER-targeting peptides, pharmaceutical compositions comprising such therapeutic agents, and methods of using such compositions to treat or prevent a cancer or other disease condition associated with HER overexpression.

Abstract: It was the object of the present invention to provide RNA with increased stability and translation efficiency and means for obtaining such RNA. It should be possible to obtain increased grades of expression by using said RNA in gene therapy approaches.

Abstract: RNA molecules comprising a guide sequence portion having 17-20 nucleotides in the sequence of 17-20 contiguous nucleotides set forth in any one of SEQ ID NOs: 1-1990 and compositions, methods, and uses thereof.

Abstract: The present invention provides an artificial nuclease comprising a DNA-binding domain and a function domain linked to each other via a polypeptide consisting of 35 to 55 amino acid residues wherein amino acid residues at two sites in a DNA-binding module contained in a DNA-binding domain exhibit a mode of repetition that is different for every four DNA-binding modules; a vector for expressing said artificial nuclease; a vector library for preparing said vector; and a vector set for preparing said vector library.

Abstract: Provided are novel peptides, methods of their preparation and their use in controlled release of pharmaceutically active compounds.

Abstract: Recombinant bacterial cells are provided that comprise a stable non-canonical amino acid translation pathway. In some aspects, the bacteria comprise nucleic acids encoding a non-canonical amino acid translation pathway (e.g., a tRNA for incorporation of a non-canonical amino acid, such selenocysteine); a marker polypeptide that includes the non-canonical amino acid. Recombinant tRNA and selection marker coding sequences are likewise provided.

Abstract: Modified capsid proteins, isolated polynucleotides, methods for the preparation of modified capsid proteins, recombinant viral particles, recombinant expression systems for the generation of modified viral particles, and methods of gene editing and regulation are provided herein.

Abstract: In various embodiments, the present invention provides a process for separating target proteins from non-target proteins in a sample comprising increasing the concentration of the target proteins and non-target proteins in the sample and subsequently delivering the concentrated sample to a chromatography device. In other embodiments, the invention relates to a process for increasing the capacity of a chromatography device for a target protein by delivering a concentrated sample comprising the target protein to a chromatography device.

Abstract: The invention features methods for treating or preventing pulmonary diseases, including acute respiratory distress syndrome (ARDS) and pneumonia, in a subject in need thereof that involve administering to the subject inter-alpha inhibitor proteins (I?Ips), including, e.g., inter-alpha inhibitor (I?I) and/or pre-alpha inhibitor (P?I).

Abstract: Disclosed herein are recombinant adenoviruses with one or more nucleotide sequences inserted between two viral transcription units, formulations comprising the recombinant adenoviruses, and methods of treatment using the recombinant adenoviruses. In some embodiments, the one or more nucleotide sequences are inserted in an IX-E2 insertion site and/or an L5-E4 insertion site.

Abstract: Among the various aspects of the present disclosure is the provision of methods of suppressing Plasmodium parasite growth or infectivity or treating malaria comprising administering a polybasic antimalarial agent. The polybasic peptides and polymers provided can be used for the treatment of Plasmodium falciparum and other plasmodium species that cause human malaria.

Abstract: Provided herein are compositions comprising multi domain peptide (MDP) hydrogels where the peptides that constitute the hydrogel have at least one N6-(1-iminoethyl)-lysine side chain. Also provided are hydrogels that further comprise a STING agonist, an immune checkpoint inhibitor, and/or an anti-cancer therapy. Also provided are methods of using such compositions in the treatment of cancer.

Abstract: The present invention relates to natural or natural-like analogues of insect adipokinetic hormone (AKH) neuropeptide [Pyr]-LTFTSSWGG-[NH2] (SEQ ID NO:2) having activity against insects, for example hemipteran, dipteran, lepidopteran and/or coleopteran insects, such as aphids, moths and fruit flies, and their use as insect control agents (e.g. insecticides) and plant protection agents.

Abstract: The present invention provides a continuous upstream manufacturing process for the production of bispecific antibody products, which comprise at least two binding domains. The process comprises at least the steps of (i) providing in a perfusion bioreactor at least one mammalian cell culture, which is capable of expressing the bispecific antibody product, (ii) growing the mammalian cell culture at a first perfusion rate until a set point viable cell density is reached, and (iii) maintaining perfusion culture at a second perfusion rate, wherein the bispecific antibody product concentration in the bioreactor is kept below a threshold value. The bispecific antibody product is then subject to subsequent downstream processing. Moreover, the invention provides a bispecific antibody product produced by the continuous upstream manufacturing process.

Abstract: The invention relates to an anti-thrombotic molecule having both antiplatelet and anticoagulant (APAC) activity; its use as a medicament; its selective configuration and use as an anticoagulant and platelet inhibitor, or its selective configuration and use predominantly as either an anticoagulant or a platelet inhibitor; and a method for its production.

Abstract: Methods and compositions for rapid development of reporter lines utilizing safe harbor sites in iPSCS, as well as other progenitor cells, pluripotent and multipotent stem cells and differentiated cells, and multiple Lox sites are provided.

Abstract: Disclosed herein are factor H variants, vectors comprising said factor H variants, and pharmaceutical compositions comprising the same for treatment of disorders.

Abstract: The present disclosure provides nucleic acid molecules comprising a first inverted terminal repeat (ITR), a second ITR, and a genetic cassette encoding a miRNA and/or a therapeutic protein. In certain embodiments, the therapeutic protein comprises a clotting factor, e.g., a FVIII polypeptide, a FIX polypeptide, or a fragment thereof. In some embodiments, the first ITR and/or the second ITR is an ITR of a non-adeno-associated virus (AAV). The present disclosure also provides methods of treating bleeding disorders such as hemophilia comprising administering to the subject the nucleic acid molecule or a polypeptide encoded thereby.

Abstract: Disclosed herein is a technology to perform programmable RNA editing at single-nucleotide resolution using RNA-targeting CRISPR/Cas9. This approach, which Applicants have termed “Cas9-directed RNA editing” or “CREDIT,” provides a means to reversibly alter genetic information in a temporal manner, unlike traditional CRISPR/Cas9 driven genomic engineering which relies on permanently altering DNA sequence.

Abstract: The present disclosure provides polynucleotide cassettes, expression vectors and methods for the expression of a gene in mammalian cells to provide gene therapy for pyruvate kinase deficiency.