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AAV NDP Gene Therapy for NDP-Related Diseases Including Norrie Disease and Retinopathy

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Summary

USPTO published patent application US20260091131A1 by University of Massachusetts inventors disclosing AAV constructs encoding wildtype or codon-optimized NDP gene sequences for treating Norrie disease, diabetic maculopathy, retinopathy, and other NDP-related retinal diseases. The application, filed October 10, 2023, covers methods of administration including intravitreal injection for retinal neovascularization and exudation conditions.

What changed

The USPTO published a patent application disclosing AAV (adeno-associated virus) gene therapy constructs containing NDP (noriccin) gene sequences for treating Norrie disease and related beta-catenin signaling retinal diseases. The application claims both wildtype and codon-optimized NDP nucleic acid sequences packaged in AAV particles, along with methods for treating conditions including retinopathy of prematurity, diabetic maculopathy, Coats disease, familial exudative vitreoretinopathy, and age-related hearing loss.

Patent applications are informational publications; they do not impose compliance deadlines or operational requirements on third parties. Biotech companies, pharmaceutical firms, and researchers developing gene therapies for ocular diseases should review this publication for potential freedom-to-operate implications or licensing opportunities.

Archived snapshot

Apr 2, 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

PRODUCTS AND METHODS FOR USE IN TREATING NDP-RELATED DISEASES

Application US20260091131A1 Kind: A1 Apr 02, 2026

Inventors

Jane SOWDEN, Valda PAUZUOLYTE, James WAWRZYNSKI, Aara PATEL

Abstract

The invention relates to a construct and AAV particle comprising a wildtype NDP nucleic acid sequence or a codon-optimised NDP nucleic acid sequence. The invention further relates to products and methods for the treatment of one or more of Norrie disease, age related hearing loss, diabetic maculopathy and retinopathy, retinal neovascularisation, retinal exudation, retinopathy of prematurity (ROP), Familial exudative vitreoretinopathy (FEVR), Coats disease, and other NDP-related diseases, or other beta catenin signalling-related retinal diseases.

CPC Classifications

A61K 48/005 A61K 9/0019 A61K 9/0048 A61K 48/0075 A61K 48/0083 A61P 27/12 A61P 27/16 C07K 14/475 C12N 15/86 A61K 38/00 C12N 2750/14143 C12N 2750/14145 C12N 2800/22

Filing Date

2023-10-10

Application No.

19120184

View original document →

Named provisions

NDP Gene Therapy AAV Constructs NDP-Related Disease Treatment Beta Catenin Signalling Retinal Diseases

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Last updated

Classification

Agency
USPTO
Published
April 2nd, 2026
Instrument
Notice
Legal weight
Non-binding
Stage
Draft
Change scope
Minor
Document ID
US20260091131A1

Who this affects

Applies to
Pharmaceutical companies Healthcare providers Drug manufacturers
Industry sector
3254 Pharmaceutical Manufacturing 3254.1 Biotechnology 3345 Medical Device Manufacturing
Activity scope
Biotechnology Pharmaceutical Research Gene Therapy
Geographic scope
United States US

Taxonomy

Primary area
Intellectual Property
Operational domain
Legal
Topics
Healthcare Pharmaceuticals

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