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Spinraza, nusinersen (IONIS-SMNRx) (formerly ISIS-SMNRx)

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Company Ionis Pharmaceuticals Inc.
DescriptionAntisense oligonucleotide that modulates splicing of survival of motor neuron 2 centromeric (SMN2) mRNA
Molecular Target Survival of motor neuron 2 centromeric (SMN2)
Mechanism of ActionAntisense therapy
Therapeutic ModalityNucleic acid: Linear RNA: Antisense
Latest Stage of DevelopmentRegistration
Standard IndicationSpinal muscular atrophy (SMA)
Indication DetailsTreat infantile-onset spinal muscular atrophy (SMA); Treat later-onset spinal muscular atrophy (SMA); Treat spinal muscular atrophy (SMA) in children; Treat spinal muscular atrophy (SMA) in children and infants
Regulatory Designation U.S. - Fast Track (Treat spinal muscular atrophy (SMA) in children);
U.S. - Orphan Drug (Treat spinal muscular atrophy (SMA) in children);
EU - Accelerated Assessment (Treat infantile-onset spinal muscular atrophy (SMA))
PartnerBiogen Inc.

 Product Details
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Collapse Summary Deals Information
Total Number of DealsTotal Deal ValueTotal Upfront CashTotal Milestone Payments

1

$299.0M

0

$270.0M


 Deals Details
DateCompanies InvolvedDeal HeadlineTotal Deal ValueTotal Upfront CashTotal Milestone

01/09/2012

$299.0M

0

$270.0M

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