BEST1 Vectors for Retinal Dystrophy Treatment
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
- Monitor patent prosecution status
Archived snapshot
Apr 13, 2026GovPing 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.
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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