Codon-optimized GLA gene and its use

JP2026090534APending Publication Date: 2026-06-024D MOLECULAR THERAPEUTICS INC
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Patent Information

Authority / Receiving Office
JP · JP
Patent Type
Applications
Current Assignee / Owner
4D MOLECULAR THERAPEUTICS INC
Filing Date
2026-02-27
Publication Date
2026-06-02

AI Technical Summary

Technical Problem

Current gene therapy approaches for Fabry disease, particularly targeting cardiomyocytes, face challenges due to inadequate transduction and insufficient uptake of recombinant alpha-galactosidase A (AGA) enzyme, leading to insufficient reduction of globotriaosylceramide (Gb3) accumulation and increased cardiovascular risk.

Method used

Development of codon-optimized nucleic acid molecules encoding human galactosidase A (AGA) protein, combined with modified AAV vectors, to enhance expression in cardiomyocytes and endothelial cells, using tissue-specific promoters and optimized codon usage to improve transduction efficiency.

Benefits of technology

The codon-optimized AGA expression significantly increases enzyme activity in target cells, reducing Gb3 levels and improving clinical outcomes by enhancing transduction efficiency and stability.

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Abstract

Providing a codon-optimized GLA gene and its use. [Solution] This disclosure provides a codon-optimized nucleotide sequence encoding human alpha-galactosidase A, a vector and host cells comprising the codon-optimized alpha-galactosidase A sequence, and a method for treating disorders such as Fabry disease, comprising the step of administering the codon-optimized sequence encoding human alpha-galactosidase A to a target. In some embodiments, the codon-optimized nucleic acid molecules described herein have an increased human codon adaptation index compared to that of wild-type GLA cDNA (GenBank accession number NM_000169.3; SEQ ID NO: 3).
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