19 results about "Blood Disorder" patented technology

Disclosed are novel compounds that are antagonists of the human GPR39 protein. Disclosed are pharmaceutical compositions of antagonists of the human GPR39 protein and methods of use. In particular, disclosed herein are methods of using the antagonists to treat diseases or conditions, including cardiovascular conditions, endocrine system and hormone disorders, cancer disorders, metabolic diseases, gastrointestinal and liver diseases, blood disorders, neurological disorders, and respiratory diseases.

Described herein are compounds that activate pyruvate kinase, pharmaceutical compositions, and methods of using the same. The compounds are represented by Formula (I), wherein R 1 , R 2 , R a , R b , R j , R k , and Q are as defined herein.

Lentiviral vector (LV) formulations, and pharmaceutical compositions comprising such LV formulations, with improved stability and suitable for systemic administration are provided. Methods for treating disorders, especially blood disorders, using systemic administration of LV formulations are also provided.

Provided herein are compounds and compositions thereof for activating nuclear factor erythroid 2-related factor 2 (Nrf2) for treating red blood cell diseases and inflammatory diseases.

Devices and methods for reducing or limiting bleeding in an animal by stimulating the spleen with focused ultrasound (FUS). The devices and methods can be used to treat blood disorders such as hemophilia, or to reduce bleeding during surgery or due to trauma. The methods can be administered to a patient non-invasively by applying ultrasound energy transcutaneously.

Provided herein are antibodies, compositions, and methods of use thereof. The disclosed antibodies bind to von Willebrand factor in blood plasma of a subject. Such antibodies are useful for methods of treatment, such methods of treating one or more blood disorders characterized by excessive bleeding. Also provided herein are methods of treating blood disorders by administering a combination of an anti-VWF antibody and von Willebrand Factor.

The present invention relates to an integration-defective lentiviral vector (IDLV) comprising a nucleic acid, the said nucleic acid comprising, between a 5′ LTR sequence and a 3′ LTR sequence, at least one nucleus export signaling sequence; at least one nucleic acid sequence of interest; and at least one nuclease site. The invention further relates to an isolated cell comprising said IDLV, a pharmaceutical composition comprising said IDLV or said isolated cell, and their pharmaceutical use in the treatment of a disease selected from the group consisting of immune diseases, viral infections, tumors and blood diseases; and / or a disease caused by the lack of a protein or by the presence of an aberrant non-functional one in an individual in need thereof.

Disclosed herein are novel compounds that are antagonists of the human GPR39 protein. Pharmaceutical compositions and methods of use of antagonists of human GPR39 protein are disclosed. In particular, disclosed herein are methods of treating diseases or conditions, including cardiovascular conditions, endocrine system and hormone disorders, cancer disorders, metabolic disorders, gastrointestinal and liver disorders, hematological disorders, neurological disorders, and respiratory disorders, using the antagonists.

The present disclosure includes compositions possessing a recombinant H-ferritin homopolymer that has a plurality of H-ferritin subunits with an amino acid sequence of SEQ ID NO: 1. Methods for treating an iron-related condition in a subject and for treating a neurological or blood disorder in a subject with such compositions are also disclosed.

The present disclosure provides pyrimidine nucleoside compounds, including torcitabine, thymidine, deoxyuridine, deoxycytidine and uridine, and methods to treat telomere biology disorders (TBDs) and aging-related diseases, including hematological disorder, liver disease, or hepatic fibrosis, comprising administering to a subject diagnosed with said telomere biology disorder a therapeutically effective amount of said compounds.

Disclosed herein are methods of expanding hematopoietic stem cells (HSC) ex vivo and their uses thereof, the methods comprising administrating a radical trapping antioxidant (RTA) to the HSC isolated from a subject, wherein the RTA blocks ferroptosis in the HSC and increases HSC expansion. Also disclosed herein are methods of treating blood disorders, such as, for example, sickle cell disease or β-thalassemia. Further disclosed is a kit for use of HSC expansion.

The present invention relates to products and compositions and their uses. In particular, the invention relates to nucleic acid products that interfere with or inhibit the expression of the TMPRSS6 gene in combination with one or more iron chelators and possibly other active agents, as well as to therapeutic uses such as the treatment of hemochromatosis, porphyria, and blood disorders such as beta-thalassemia, sickle cell anemia, and transfusion iron overload or myelodysplastic syndrome, as well as infections and mortality not related to relapses associated with bone marrow transplantation.

Provided herein are antibodies, compositions, and methods of use thereof. The disclosed antibodies bind to a von Willebrand factor in the plasma of a subject. Such antibodies are useful in methods of treatment, such as methods of treating one or more blood conditions characterized by excessive bleeding.

This disclosure relates in part to lipid nanoparticles (LNPs) comprising a payload molecule (i.e., a nucleic acid molecule and / or a therapeutic agent), and methods of using thereto to deliver the payload molecule in vivo to target cells or target tissues. In some embodiments, the payload molecule comprises a peptide, a nucleic acid molecule, an enzyme, or any combination thereof. In some embodiments, the target cells are hematopoietic stem cells (HSCs). In some embodiments, this disclosure relates to the use of the lipid nanoparticles described herein for the treatment, prevention, and / or improvement of diseases and / or conditions in a subject, including but not limited to blood disorders.

Described herein is a compound of Formula I or a pharmaceutically acceptable salt thereof: wherein Ring A R1, R2, a, b, and n are as defined herein. Also described is a method of treating a subject having a disorder in need of treatment, comprising inhibiting aminolevulinic acid synthase 2 (ALAS2) in the subject by administering a compound of Formula (I) or a pharmaceutically acceptable salt thereof. Disorders that are of particular interest are blood disorders, such as porphyria and anemia.