Methods of Treating Transthyretin (TTR) Mediated Amyloidosis
AssigneeAlnylam Pharmaceuticals, Inc.
InventorBrian Bettencourt
Life of this patent
Estimating the term…
Abstract
Disclosed herein are methods for reducing or arresting an increase in a Neuropathy Impairment Score (NIS) or a modified NIS (mNIS+7) in a human subject by administering an effective amount of a transthyretin (TTR)-inhibiting composition.
Claims23
-38. (canceled)
A method of treating polyneuropathy in a patient with hereditary transthyretin-mediated amyloidosis (hATTR), the method comprising administering a dose of a TTR siRNA with lipid excipients to the patient,
- wherein the TTR siRNA consists of a sense strand consisting of SEQ ID NO:
- 1 and an antisense strand consisting of SEQ ID NO:2;
- and
- the lipid excipients consist of DLin-MC3-DMA ((6Z, 9Z, 28Z, 31Z)-heptatriaconta-6, 9, 28, 31-tetraen-19-yl-4-(dimethylamino) butanoate), DSPC ((1,2-Distearoyl-sn-Glycero-3-Phosphocholine), cholesterol, and PEG2000-C-DMG (((R)-methoxy-PEG2000-carbamoyl-di-O-myristyl-sn-glyceride) in isotonic phosphate buffered saline;
- wherein the dose is 0.3 mg siRNA per kg body weight, and the TTR siRNA with lipid excipients is administered via IV infusion once every three weeks, and the patient receives a premedication before infusion to reduce the risk of infusion-related reactions.
A method of treating polyneuropathy in a patient with hereditary transthyretin-mediated amyloidosis (hATTR), the method comprising administering a dose of a TTR siRNA with lipid excipients to the patient,
- wherein the TTR siRNA consists of a sense strand consisting of SEQ ID NO:
- 1 and an antisense strand consisting of SEQ ID NO:2;
- and
- the lipid excipients consist of DLin-MC3-DMA ((6Z, 9Z, 28Z, 31Z)-heptatriaconta-6, 9, 28, 31-tetraen-19-yl-4-(dimethylamino) butanoate), DSPC ((1,2-Distearoyl-sn-Glycero-3-Phosphocholine), cholesterol, and PEG2000-C-DMG (((R)-methoxy-PEG2000-carbamoyl-di-O-myristyl-sn-glyceride) in isotonic phosphate buffered saline;
- wherein the dose is 0.3 mg siRNA per kg body weight, and the TTR siRNA with lipid excipients is administered via IV infusion once every three weeks, and the patient receives a premedication before infusion to reduce the risk of infusion-related reactions, whereby the method reduces a Neuropathy Impairment Score (NIS) or a modified NIS (mNIS+7) or arrests an increase in a NIS or a mNIS+7 in the patient.
Dependencies are inferred from the claim text.