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Ai-derived Nanoparticle Lysosomeclevable Long-acting Cysteamine Prodrug for Nephropathic Cystinosis

Year: 2026

Type: article

Abstract: A rare lysosomal storage disorder caused by CTNS gene mutations leading to cystine accumulation is called Cystinosis Nephropathy. Current oral cysteamine therapies require frequent dosing and demonstrate limited bioavailability. To design and evaluate an AI-guided lysosome-cleavable cysteamine prodrug for sustained subcutaneous administration.A cysteamine–Peptidepolymer conjugate was synthesized and evaluated using in vitro release studies, extracellular matrix modeling, and pilot in vivo pharmacokinetics in Sprague Dawley rats. JEL®Conjugate #2 demonstrated sustained release up to 120 hours in vitro with controlled burst release. In vivo studies showed prolonged systemic exposure through 24 hours.The AI-guided lysosome-cleavable prodrug demonstrates proof-of-concept for extended-release cysteamine delivery.

Source: Advanced Nanoscience and Technology An International Journal

DOI: 10.5121/antj.2026.12101

Authors: Michael Sekar, Deepakraja Rajendran, Anand Paul Sekar, Ramasamy Paulmurugan

Institutions: Avaya (United States), Stanford University

Language: English

Topic: Biomedical Research and Pathophysiology

Subfield: Pathology and Forensic Medicine

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: diamond

OpenAlex ID: W7154694334

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