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Guangzhou Kebilidi Medical Technology

South Plainfield, New Jersey, United States

Ownership: Private
Employees: ~1100
Development stage: Other

Overview

Kebilidi (eladocagene exuparvovec-tneq) demonstrated improvement in motor milestones in 8 of 12 severely affected pediatric patients, with approval based on accelerated approval pathway requiring confirmatory postmarketing studies. It is the first brain-administered gene therapy in the U.S. for AADC deficiency, with similar approvals in Europe as Upstaza.[1][2][3][4][7][10][11]

Therapeutic areas: Rare Diseases
Modalities: Gene therapyBiologicAdeno-associated virus (AAV) vector-based gene therapy
Industry: Biotech

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