4 results about "Blood Disorder" patented technology

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.

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.