Polynucleotides for treating GCase deficiency-associated diseases

JP2025534899A5Pending Publication Date: 2026-10-14LINGYI BIOTECH CO LTD
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Patent Information

Application Number
JP2025520670
Authority / Receiving Office
JP · JP
Patent Type
Applications
Current Assignee / Owner
Priority Date
2022-10-08
Filing Date
2023-10-07
Publication Date
2026-10-14

AI Technical Summary

Technical Problem

Current enzyme replacement therapies for Gaucher disease are costly and require frequent injections, and there is a need for a more effective therapeutic expression vector that allows high-level and stable expression of β-glucocerebrosidase (GCase) to address the underlying GCase deficiency.

Method used

Development of optimized polynucleotide sequences and expression constructs that encode GBA1, including codon-optimized sequences and inclusion of signal peptides, untranslated introns, and transcriptional control elements, packaged in AAV vectors for efficient GCase expression in target tissues.

Benefits of technology

The optimized polynucleotide sequences and AAV vectors achieve higher levels of GCase expression and activity, providing stable GCase activity and increased bioavailability, effectively reducing substrate accumulation in Gaucher disease models.

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Abstract

The present disclosure provides codon-optimized GBA1 polynucleotides that encode GCase proteins in which some coding sequences deviate from wild-type. The disclosure further provides expression constructs, vectors, viral particles, or compositions of the disclosed polynucleotides, as well as methods and uses of these polynucleotides, expression constructs, vectors, viral particles, or compositions, including the treatment of diseases or conditions associated with GCase deficiency.
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