Potential medical applications of Geron Patent US9133233 B2

[Fishermangents]

-- With thanks to Hoosier (SA) --

This patent seems to be relevant for the 8K that just has been filed.

Patent number: US9133233 B2
Inventors: Sergei Gryaznov, Krisztina Pongracz
Patent owner: Geron Corporation
Publicized: 15 September2015
Expiration: March 2027
Publication type: prolongation


Summary
The present disclosure relates to RNA amidates and thioamidates useful for RNA interference applications. The RNA amidates and thioamidates contain at least one internucleoside linkage chosen from ribo-N3′→P5′ phosphoramidate (NP) and ribo-N3′→P5′ thiophosphoramidate (NPS) linkages, and optionally further containing at least one covalently conjugated lipid moiety. Compositions comprising the amidates and thioamidates are disclosed, as are methods for their use in modulating gene expression.

== List of potential medical applications (under Summary of the Invention, starting at Column 7; Line 5) ==

For example, the compounds disclosed herein can be designed and used to modulate or block: Hepatitis B virus (HBV) and Hepatitis C virus (HCV) protein expression, and can thus be used to treat diseases associated with HBV and HCV such as, for example, cirrhosis, liver failure, and hepatocellular carcinoma; Ras gene expression, such as K-Ras (associated with colon and pancreatic carcinomas), H-Ras (associated with leukemias), and/or N-Ras expression; HIV-1 and HER2 gene expression, the latter of which is associated with breast and ovarian cancers; expression of vascular endothelial growth factor and/or vascular endothelial growth factor receptors, such as VEGFR1 and/or VEGFR2, for the purpose of, e.g., preventing, treating, controlling disorders and conditions related to angiogenesis, including but not limited to cancer, tumor angiogenesis, or ocular indications, such as diabetic retinopathy, or age-related macular degeneration, proliferative diabetic retinopathy, hypoxia-induced angiogenisis, rheumatoid arthritis, psoriasis, wound healing, endometriosis, endometrial carcinoma, gynecologic bleeding disorders, irregular menstrual cycles, ovulation, premenstrual syndrome (PMS), and menopausal dysfunction; beta-secretase (BACE), PIN-1, presenillin-1 (PS-1) and presenillin-2 (PS-2) polypeptide and polynucleotide targets, associated with Alzheimer's disease; expression of NOGO and NOGO receptor genes, and the expression of genes encoding the IkB kinase IKK complex, for example IKK-alpha, IKK-beta, or IKK-gamma and/or a protein kinase PKR protein; expression of kinases which phosphorylate Cdc25 S216, such as Chk1 (checkpoint kinase 1) enzyme, Chk2 (Cds1) and C-Tak1; and expression of the T-cell co-stimulatory adapter protein GRID (Grb2-related with Insert Domain).

In an exemplary therapeutic application, a 19 to 23 nucleotide riboamidate, such as a 21 to 23 nucleotide riboamidate, is introduced into a mammal or mammalian cells, for example a human or human cells, in order to mediate RNA interference in the mammal or mammalian cells, such as to prevent or treat a disease or undesirable condition. In this method, a gene (or genes) that cause or contribute to the disease or undesirable condition is targeted and a riboamidate complementary to the mRNA of the gene targeted for degradation is introduced into the cell or organism. The cell or organism is maintained under conditions under which degradation of the corresponding mRNA occurs, thereby mediating RNA interference of the mRNA in the gene in the cell or organism.

link to the patent: https://www.google.com/patents/US9133233

[Fishermangents]

Summarizing the examples of medial applications:

cirrhosis
liver failure
hepatocellular carcinoma
colon and pancreatic carcinomas
leukemias
breast and ovarian cancers
cancer
diabetic retinopathy
proliferative diabetic retinopathy
hypoxia-induced angiogenisis
rheumatoid arthritis
psoriasis
wound healing
endometriosis
endometrial carcinoma
gynecologic bleeding disorders
irregular menstrual cycles
premenstrual syndrome (PMS)
menopausal dysfunction
Alzheimer's disease