Cre-Lox AAV Gene Therapy Reconstitution System for Large Gene Delivery
Summary
The USPTO published patent application US20260098279A1 for a Cre-Lox AAV gene therapy system enabling delivery of therapeutic genes up to 16kb using up to four adeno-associated virus vectors with sequence-specific recombination. The invention by inventor Seongjin Seo includes novel lox site embodiments for near-unidirectional recombination. Applications include treatment of genetic disorders such as IFT140-associated retinitis pigmentosa and other retinal degeneration conditions.
What changed
The USPTO published a patent application for a Cre-Lox DNA recombination system enabling large gene delivery via adeno-associated virus (AAV) vectors exceeding their standard packaging capacity. The invention employs up to four AAV vectors with novel lox site embodiments supporting reconstitution of therapeutic genes up to 16kb in predetermined arrangements.
Affected parties in the gene therapy R&D sector should monitor this application's prosecution for potential freedom-to-operate considerations when developing large-gene AAV therapies. The claimed platform technology for treating retinal disorders including IFT140-associated retinitis pigmentosa may affect patent landscapes for similar gene therapy approaches.
What to do next
- Monitor for patent grant status
Archived snapshot
Apr 12, 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.
RECONSTITUTION OF LARGE GENES VIA CRE-LOX DNA RECOMBINATION IN ADENO-ASSOCIATED VIRUS VECTORS
Application US20260098279A1 Kind: A1 Apr 09, 2026
Inventors
Seongjin Seo
Abstract
The described technology pertains to gene therapy methodologies, specifically techniques and systems for delivering therapeutic genes of substantial size that exceed the packaging capacity of adeno-associated virus (AAV) vectors. The disclosed approach employs up to four AAV vectors and the CRE-lox DNA recombination system, utilizing novel lox site embodiments that allow sequence-specific and near-unidirectional recombination. This method supports efficient reconstitution of therapeutic genes up to 16 kb in a predetermined arrangement. Applications include the delivery of genes such as IFT140, PCDH15, CEP290, and CDH23 for addressing genetic disorders, including retinal degeneration. The described technology demonstrates successful production of full-length proteins in mammalian cells and mouse retinas, with therapeutic efficacy observed in an IFT140-associated retinitis pigmentosa mouse model. The CRE-lox approach offers a flexible platform for addressing AAV's packaging constraints, enabling effective gene therapy for large genes.
CPC Classifications
C12N 15/86 A61K 48/0016 A61P 27/02 C12N 9/1241 C12N 2750/14143 C12N 2830/34 C12N 2830/50
Filing Date
2025-10-08
Application No.
19353409
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