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Lysosomal Storage Disorder

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Breaking the Cystine Barrier: Long-term Safety and Efficacy of CTNS-RD-04 Gene Therapy in Cystinosis
Posted inHematology-Oncology Nephrology news

Breaking the Cystine Barrier: Long-term Safety and Efficacy of CTNS-RD-04 Gene Therapy in Cystinosis

Posted by MedXY By MedXY 02/22/2026
A phase 1-2 trial shows that autologous hematopoietic stem-cell gene therapy (CTNS-RD-04) safely reduces lysosomal cystine levels in patients with cystinosis, potentially offering a definitive alternative to lifelong cysteamine therapy.
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