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Publication: The 10th Oligonucleotide Therapy Approved: Golodirsen for Duchenne Muscular Dystrophy

February 11, 2020 | MARY ANN LIEBERT, INC.

On December 12, 2019, the Food and Drug Administration (FDA) granted accelerated approval to Sarepta Therapeutics for golodirsen to treat Duchenne muscular dystrophy (DMD) patients with eligible mutations (www.drugs.com/newdrugs/fda-approves-vyondys-53-golodirsen-duchenne-muscular-dystrophy-dmd-patients-amenable-skipping-exon-5119.html). This brings the number of FDA and/or European Medicines Agency (EMA) approved oligonucleotide drugs to 10 and the number of oligonucleotide drugs for treatment of DMD to 2 (Table 1). The approval is less controversial than that of previously approved eteplirsen, but it was still not a smooth ride.


society-of-GTH

Publication: Chemical Diversity of Locked Nucleic Acid-Modified Antisense Oligonucleotides

2020 | AMERICAN SOCIETY OF CELL & GENE THERAPY

The identification of molecules that can modulate RNA or protein function and the subsequent chemical and structural optimization to refine such molecules into drugs is a key activity in drug discovery.


ChemRxiv

Publication: Enantiodivergent Formation of C–P Bonds: Synthesis of P-Chiral Phosphines and Methyl-phosphonate Oligonucleotides

JANUARY 17, 2020 | CHEMRXIV

A simple limonene-derived P(V)-based reagent for the modular, scalable, and stereospecific synthesis of chiral phosphines and methyl-phosphonate oligonucleotide (MPO) building blocks is presented.


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