US 2025/0375378 A1Published application

mRNA LIPID NANOPARTICLES FOR THE TREATMENT OF NIEMANN-PICK TYPE C DISEASE

OwnerMark Schultz, Anna Schwendeman +1inventors

InventorsMark Schultz, Anna Schwendeman, Troy Halseth

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Abstract

Composition and methods for treating Niemann-Pick disease type C are disclosed, utilizing lipid nanoparticles (LNPs) encapsulating mRNA sequences coding for NPC1 and/or NPC2 proteins.

Claims20

Dependent claims

A composition comprising lipid nanoparticles (LNPs) encapsulating a mRNA sequence coding for NPC1 protein and/or NPC2 protein, wherein the LPNs comprise a combination of SM-102, DMG-PEG 2000, DSPC and cholesterol.

Dependencies are inferred from the claim text.

Facts

Application: 19/229,666Filed 5 Jun 2025
Priority: 5 Jun 2024
Published: 11 Dec 2025
Term ends: Not estimatedThe source records don’t support an estimate.

Classification10 codes

A61K 9/1271
Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant: non-conventional liposomes,…
A61K 9/5123
Excipients; Inactive ingredients: organic compounds, e.g. fats, sugars
A61K 9/5192
Nanocapsules; Nanoparticles: processes
A61K 31/7105
Compounds having three or more nucleosides or nucleotides: natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine,…

Family

Application63/656,343Us provisional application · filed 5 Jun 2024
US 2025/0375378 A1This patent

From the US filing history. Foreign family members aren’t listed.

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