Preparative Synthesis of an R (P)-Guanosine-3',5'-Cyclic Phosphorothioate Analogue, a Drug Candidate for the Treatment of Retinal Degenerations

R(P)-鸟苷-3',5'-环状硫代磷酸酯类似物的制备合成,一种用于治疗视网膜变性的候选药物

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Abstract

Cyclic guanosine monophosphorothioate analogue 1a is currently showing potential as a drug for the treatment of inherited retinal neurodegenerations. To support ongoing preclinical and clinical work, we have developed a diastereoselective synthesis via cyclization and sulfurization of the nucleoside 5'-H-phosphonate monoester, which affords the desired R (P)-3',5'-cyclic phosphorothioate in 9:1 ratio to the undesired S (P)-diastereomer. This route was made viable as a result of the silyl protection sequence used, which achieved >80% selectivity for 2',5'-hydroxyls over 3',5'-hydroxyls. Finally, the chromatography-free process allowed for a scale-up, as intermediates and the final product were isolated by crystallization to give 125 g of 1a (13.8% total yield) with over 99.9% HPLC purity.

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