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Process Development for Nusinersen: An Orphan Drug Used to Treat Spinal Muscular Atrophy (SMA)
Temp 9

DST
March 27, 2023

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Spinal Muscular Atrophy
Invitro study

Quantitative data
• An indigenous synthetic process route/technology for the development of generic Nusinersen. • Innovative Elements of the Project: Improved synthesis of 2’-O-MOE phosphoramidite monomers through appropriate modification ofreported procedures. • Shortened solid phase synthesis cycle by avoiding a separate capping step; achieving sulfurizationand capping in the same step through re-circulation of reagents.

Nusinersen, Orphan drug, Spinal Muscular Atrophy
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Dr. Moneesha Fernandes/None/CSIR-NML, Pune