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FDA Approves First Gene Therapy for AADC Deficiency

2/3/2025

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Summary

Story summary

On November 13, 2024, the FDA made a groundbreaking decision by approving Kebilidi, the first-ever brain-delivered AAV gene therapy for AADC deficiency. This innovative treatment enhances motor function and significantly boosts quality of life by directly delivering the DDC gene into the brain. Meanwhile, Spur Therapeutics is gearing up for a Phase 3 trial of FLT201, targeting Gaucher disease, set to begin in late 2025.

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Story summary

On November 13, 2024, the FDA made a groundbreaking decision by approving Kebilidi, the first-ever brain-delivered AAV gene therapy for AADC deficiency. This innovative treatment enhances motor function and significantly boosts quality of life by directly delivering the DDC gene into the brain. Meanwhile, Spur Therapeutics is gearing up for a Phase 3 trial of FLT201, targeting Gaucher disease, set to begin in late 2025.