Changeflow GovPing Pharma & Drug Safety BEST1 Vectors for Retinal Dystrophy Treatment
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BEST1 Vectors for Retinal Dystrophy Treatment

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Summary

The USPTO published patent application US20260098277A1 covering BEST1-targeted expression constructs, nucleic acid vectors, and pharmaceutical compositions for treating BEST1-associated retinal dystrophies including autosomal dominant bestrophinopathies. The application includes BEST1-targeted shRNAs, BEST1 coding sequences, promoters, and regulatory elements designed to improve functional BEST1 expression in subjects with BEST1 mutations. This published application represents an early-stage intellectual property filing with no granted rights or compliance obligations established.

What changed

The USPTO published patent application US20260098277A1 for BEST1 vectors and their uses in treating retinal dystrophies. The application covers expression constructs containing BEST1-targeted short hairpin RNAs, BEST1 coding sequences, promoters, and additional regulatory elements designed to improve functional BEST1 expression in subjects with BEST1 mutations and associated retinal dystrophies.

Affected parties—including pharmaceutical companies, biotech firms, and research institutions in the gene therapy or ocular therapeutics space—should monitor this application for potential licensing opportunities or freedom-to-operate considerations. This is a published patent application only; it does not grant rights or create compliance obligations. Parties developing competing therapies or seeking collaboration opportunities may wish to review the claims and track prosecution of this application through the USPTO examination process.

What to do next

  1. Monitor patent prosecution status

Archived snapshot

Apr 13, 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

BEST1 VECTORS AND USES THEREOF

Application US20260098277A1 Kind: A1 Apr 09, 2026

Inventors

Ricardo SALADANA-MEYER, Jin HUH, Jodi KENNEDY, Rosario FERNANDEZ GODINO

Abstract

Provided herein are expression constructs, nucleic acid vectors, pharmaceutical compositions, and methods for improved expression of functional BEST1 in subjects having mutations in BEST1 and associated retinal dystrophies. Improved expression constructs include BEST 1-targeted short hairpin RNAs (shRNAs), BEST1 coding sequences, promoters driving expression of shRNAs and coding sequences, and/or additional regulatory elements to improve BEST1 expression. Expression constructs, nucleic acid vectors and pharmaceutical compositions thereof, and methods of use thereof, can provide effective treatments for BEST1-associated disorders, such as autosomal dominant bestrophinopathies.

CPC Classifications

C12N 15/85 C12N 2800/107

Filing Date

2023-09-18

Application No.

19113376

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Last updated

Classification

Agency
USPTO
Published
April 9th, 2026
Instrument
Notice
Legal weight
Non-binding
Stage
Final
Change scope
Minor
Document ID
US20260098277A1

Who this affects

Applies to
Pharmaceutical companies Healthcare providers Medical device makers
Industry sector
3254.1 Biotechnology
Activity scope
Patent application Intellectual property registration Gene therapy research
Geographic scope
United States US

Taxonomy

Primary area
Intellectual Property
Operational domain
Legal
Topics
Public Health Healthcare

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