The invention relates to a combination of a TLR-9 agonist and a conjugate of Formula (I) or pharmaceutically acceptable salt thereof. (Formula (I))

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siRNA compositions are provided that contain gemcitabine (GEM) in place of cytosine moieties within the siRNA sequence. Pharmaceuticals compositions containing these siRNA molecules, and methods of using the compositions for treating diseases such as cancer are provided.

Provided herein are certain nucleic acids (e.g., double stranded siRNA molecules), as well as conjugates that comprise a targeting moiety, a double stranded siRNA, and optional linking groups. Certain embodiments also provide synthetic methods useful for preparing the conjugates. The conjugates are useful to treat certain diseases, such as liver fibrosis, e.g., in the setting of NASH or ASH.

The present disclosure relates to glyconucleic acids, such as glycoRNA and glycoDNA described herein. Provided are glycosylated ribonucleic acid (glycoRNA)-related methods and compositions.

Complexes comprising a nucleic acid-guided endonuclease, a sequence-specific targeting nucleic acid and an amphipathic helical peptide are provided. Compositions and methods for delivery of complexes comprising a nucleic acid-guided endonuclease, a sequence-specific targeting nucleic acid and an amphipathic helical peptide to mammals for both research and therapeutic use are provided. Methods of treating or reducing one or more symptoms of type 2 diabetes, prediabetes and/or gestational diabetes are provided.

This disclosure relates to an improved process for the preparation of carbohydrate conjugates. The disclosure also relates to carbohydrate conjugated iRNA agents comprising these carbohydrate conjugates, which have improved purity and are advantageous for the in vivo delivery of the iRNA agents.

Biologically active compounds and their methods of preparation are provided that may be used as photosensitizers for diagnostic and therapeutic applications, particularly for PDT of cancer, infections and other hyperproliferative diseases, fluorescence diagnosis and PDT treatment of non-tumorous indications such as arthritis, inflammatory diseases, viral or bacterial infections, dermatological, otorhinolaryngology disorders, ophthalmological or urological disorders. As the compounds exhibit also toxicity against targets (tumor cells, bacteria, inflammation-related cells) without light these biologically active compounds may also be used for the light-independent treatment of such indications. Embodiments also include methods to synthesize boron dipyrromethene complex structures incorporating a substituted 2,3,5,6-tetrafluorophenyl-dipyrromethene (2,3,5,6-tetrafluorophenyldipyrrin) unit or a substituted 3-nitrophenyl-dipyrromethene (3-nitrophenyl-dipyrrin) unit. Amphiphilic compounds with increased anti-tumour and anti-bacterial efficacy are also provided. Specifically, this is achieved by substitution with bromine atoms and sugar moieties.

The present invention provides a conjugate of an oligonucleotide having a nucleic acid sequence expected to have a pharmacological effect in hepatic parenchymal cells with a biantennary GalNAc unit, or a pharmaceutically acceptable salt thereof, and a medicament or the like containing the same as an active component.

A method for synthesizing cell membrane-biomimetic nanotherapeutics can include coating core particles with cell membrane materials using flash nanocomplexation (FNC). FNC is a turbulent mixing and self-assembly method that can produce cell membrane-coated nanotherapeutics in a reproducible and scalable manner. The FNC-produced cell membrane-coated particles demonstrate lower aggregation, polydispersity, and zeta potential, than nanoparticles prepared by conventional coating methods, such as conventional bulk-sonication. As such, the present method achieves more complete, homogeneous and controllable coating than conventional bulk-sonication methods.

The invention relates to a liquid oral formulation of bumetanide for the treatment of autism, more particularly for the improvement of the Autism Spectrum Disorder (ASD) core symptoms, tuberous sclerosis complex, fragile X syndrome, Rett syndrome, Down syndrome, cancer and particularly gliomas, spinal cord lesions, chronic pain, brain trauma, cerebrovascular infarcts, various types of epilepsies, and also acute lung injury such as pneumonias or Severe Acute Respiratory Disease due to Coronavirus-2 (SARS-CoV-2). Said formulation is especially designed for paediatric populations. The invention relates also to a posology for the administration of said liquid oral formulation.