Recombinant adeno-associated virus vectors for treatment of Fabry disease
By developing codon-optimized α-Gal A variants and constructing chimeric promoters to build rAAV vectors, the problem of poor efficacy of ERT in treating Fabry disease has been solved, achieving efficient and economical gene therapy, and significantly improving the enzyme activity of α-Gal A and the convenience of treatment.
Patent Information
- Application Number
- CN202480070058.5
- Authority / Receiving Office
- CN · China
- Patent Type
- Applications(China)
- Current Assignee / Owner
- Priority Date
- 2023-11-03
- Filing Date
- 2024-11-01
- Publication Date
- 2026-06-05
AI Technical Summary
Existing enzyme replacement therapy (ERT) has limited effectiveness in treating Fabry disease (FD) patients, is costly, requires frequent injections, and some patients develop resistance, thus failing to effectively stop disease progression.
We developed codon-optimized sequences encoding α-Gal A and α-Gal A variants with enhanced activity, combined them with chimeric promoters, and constructed recombinant adeno-associated virus (rAAV) vectors for delivering α-Gal A to target tissues to achieve durable therapeutic effects.
This approach enables sustained therapeutic effects with a single dose, improving convenience and cost-effectiveness for FD patients, significantly enhancing α-Gal A enzyme activity, and reducing treatment frequency and cost.