C12Y301/03048

COMPOSITIONS AND METHODS FOR MODULATING TTR EXPRESSION

Provided herein are oligomeric compounds with conjugate groups. In certain embodiments, the oligomeric compounds are conjugated to N-Acetylgalactosamine.

COMPOSITIONS AND METHODS FOR TREATING HEART DISEASE AND/OR INJURY
20210008148 · 2021-01-14 ·

A method of treating heart disease and/or injury in a subject includes administering to the subject a therapeutic agent that inhibits one or more of catalytic activity, signaling, and function of PTP.

Methods and Materials for Producing Polyols and Electron Rich Compounds

Methods and materials for producing polyols are provided comprising recombinant microorganisms expressing a Pyp1 polyol phosphatase. Also provided herein are methods and materials for producing electron rich compounds in recombinant microorganisms lacking the DET1 and/or PHO13 genes.

Compositions and methods for modulating apolipoprotein (a) expression

Provided herein are oligomeric compounds with conjugate groups targeting apoplipoprotein (a) [apo(a)]. In certain embodiments, the apo(a) targeting oligomeric compounds are conjugated to N-Acetylgalactosamine. Also disclosed herein are conjugated oligomeric compounds targeting apo(a) for use in decreasing apo(a) to treat, prevent, or ameliorate diseases, disorders or conditions related to apo(a) and/or Lp(a). Certain diseases, disorders or conditions related to apo(a) and/or Lp(a) include inflammatory, cardiovascular and/or metabolic diseases, disorders or conditions. The conjugated oligomeric compounds disclosed herein can be used to treat such diseases, disorders or conditions in an individual in need thereof.

Oligomeric Compounds Comprising Bicyclic Nucleotides and Uses Thereof

The present invention provides oligomeric compounds. Certain such oligomeric compounds are useful for hybridizing to a complementary nucleic acid, including but not limited, to nucleic acids in a cell. In certain embodiments, hybridization results in modulation of the amount activity or expression of the target nucleic acid in a cell.

ROBUST PANELS OF COLORECTAL CANCER BIOMARKERS
20200386759 · 2020-12-10 ·

Described herein are systems and methods for developing and utilizing assays for assessing health status such as colorectal cancer.

CHIMERIC TRANSMEMBRANE RECEPTORS AND USES THEREOF
20200384030 · 2020-12-10 ·

Provided herein are chimeric transmembrane receptors and methods of using them to regulate selective gene expression in cells (e.g., immune cells). For example, chimeric transmembrane receptors provided herein may be synthetic receptor-like protein tyrosine phosphatases (synPTPRs) that can regulate transcription of a heterologous target gene in a cell when bound by a target antigen present on a target cell.

CELL PENETRATING PEPTIDES AND METHODS OF MAKING AND USING THEREOF
20200385427 · 2020-12-10 ·

Disclosed herein are compounds having activity as cell penetrating peptides. In some examples, the compounds can comprise a cell penetrating peptide moiety and a cargo moiety. The cargo moiety can comprise one or more detectable moieties, one or more therapeutic moieties, one or more targeting moieties, or any combination thereof. In some examples, the cell penetrating peptide moiety is cyclic. In some examples, the cell penetrating peptide moiety and cargo moiety together are cyclic. In some examples, the cell penetrating peptide moiety is cyclic and the cargo moiety is appended to the cyclic cell penetrating peptide moiety structure. In some examples, the cargo moiety is cyclic and the cell penetrating peptide moiety is cyclic, and together they form a fused bicyclic system.

Cell penetrating peptides and methods of making and using thereof
10815276 · 2020-10-27 · ·

Disclosed herein are compounds having activity as cell penetrating peptides. In some examples, the compounds can comprise a cell penetrating peptide moiety and a cargo moiety. The cargo moiety can comprise one or more detectable moieties, one or more therapeutic moieties, one or more targeting moieties, or any combination thereof. In some examples, the cell penetrating peptide moiety is cyclic. In some examples, the cell penetrating peptide moiety and cargo moiety together are cyclic. In some examples, the cell penetrating peptide moiety is cyclic and the cargo moiety is appended to the cyclic cell penetrating peptide moiety structure. In some examples, the cargo moiety is cyclic and the cell penetrating peptide moiety is cyclic, and together they form a fused bicyclic system.

PHARMACEUTICAL COMPOSITION FOR TREATING OR PREVENTING PARKINSON'S DISEASE COMPRISING STT AS AN ACTIVE INGREDIENT

The present invention relates to a pharmaceutical composition comprising a STT compound as an active ingredient for the prevention or treatment of Parkinson's disease. STT showed neuroprotective effect and apoptosis recovery effect in the Parkinson's disease cell model, restored the reduced motility in the MPTP animal model, and was shown to significantly protect dopamine cells, so it can be used for the prevention and treatment of Parkinson's disease.