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N-Acetyl Leucine Treats Niemann-Pick Disease Type C with NPC1 Mutations

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Summary

The USPTO published patent application US20260097008A1 by inventor Michael Strupp covering methods of treating Niemann-Pick Disease Type C (NPC) or neurological symptoms thereof by administering N-acetyl leucine to subjects carrying NPC1 mutations. The application (No. 19352209) was filed October 7, 2025, and published April 9, 2026.

What changed

The USPTO published a patent application disclosing methods of treating Niemann-Pick Disease Type C (NPC) and associated neurological symptoms using N-acetyl leucine in patients harboring NPC1 gene mutations. The application claims therapeutic compositions and dosing regimens.

Affected parties including pharmaceutical companies, biotechnology firms, and research institutions developing NPC treatments should monitor this application for potential freedom-to-operate implications or licensing opportunities regarding N-acetyl leucine-based therapies for NPC patients with NPC1 mutations.

What to do next

  1. Monitor for patent grant or office action responses

Archived snapshot

Apr 10, 2026

GovPing captured this document from the original source. If the source has since changed or been removed, this is the text as it existed at that time.

← USPTO Patent Applications

ACETYL LEUCINE FOR USE IN TREATING NIEMANN-PICK DISEASE TYPE C

Application US20260097008A1 Kind: A1 Apr 09, 2026

Inventors

Michael STRUPP

Abstract

The present disclosure provides methods of treating NPC, or a neurological symptom thereof, in a subject by administering a therapeutically effective amount of N-acetyl leucine, wherein the subject has one or more NPC1 mutations.

CPC Classifications

A61K 31/198 A61K 31/445 A61P 25/28

Filing Date

2025-10-07

Application No.

19352209

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Last updated

Classification

Agency
USPTO
Instrument
Notice
Legal weight
Non-binding
Stage
Draft
Change scope
Minor
Document ID
US20260097008A1

Who this affects

Applies to
Pharmaceutical companies Healthcare providers Patients
Industry sector
3254 Pharmaceutical Manufacturing
Activity scope
Patent application Drug formulation Therapeutic method
Geographic scope
United States US

Taxonomy

Primary area
Intellectual Property
Operational domain
Legal
Topics
Pharmaceuticals Healthcare

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