Log In
BCIQ
Print this Print this
 

scAAV9.CB.SMN (AVXS-101) (formerly chariSMA)

  Manage Alerts
Collapse Summary General Information
Company RegenxBio Inc.
DescriptionGene therapy using RegenxBio's NAV rAAV9 vector technology to deliver the survival motor neuron (SMN) gene
Molecular Target
Mechanism of ActionGene therapy
Therapeutic ModalityGene therapy: Viral vector
Latest Stage of DevelopmentPhase I
Standard IndicationSpinal muscular atrophy (SMA)
Indication DetailsTreat infants with spinal muscular atrophy (SMA); Treat spinal muscular atrophy (SMA) type 1
Regulatory Designation U.S. - Breakthrough Therapy (Treat spinal muscular atrophy (SMA) type 1);
U.S. - Fast Track (Treat spinal muscular atrophy (SMA) type 1);
EU - Orphan Drug (Treat infants with spinal muscular atrophy (SMA))
PartnerAveXis Inc.

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

1

0

0

0


 Deals Details
DateCompanies InvolvedDeal HeadlineTotal Deal ValueTotal Upfront CashTotal Milestone

04/07/2014

Undisclosed

Undisclosed

Undisclosed

Get a free BioCentury trial today