AAV9 CBA SMN1 gene therapy - AveXis

Drug Profile

AAV9 CBA SMN1 gene therapy - AveXis

Alternative Names: AVXS-101; ChariSMA; rAAV9 - AveXis; scAAV9.CB.SMN; SMA-based therapeutic - AveXis; Spinal muscular atrophy gene therapy - AveXis

Latest Information Update: 15 Nov 2016

Price : $50

At a glance

  • Originator AveXis
  • Developer AveXis; Nationwide Children's Hospital
  • Class Gene therapies; Spinal muscular atrophy gene therapies
  • Mechanism of Action Gene transference
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    No
  • On Fast track

    Fast track status is assigned by the US FDA so therapies with the potential to address unmet needs can move faster through development.

    No
  • New Molecular Entity No

Highest Development Phases

  • Phase I Spinal muscular atrophy

Most Recent Events

  • 11 Aug 2016 Interim efficacy and adverse events data from a phase I trial in Spinal muscular atrophy type 1 released by AveXis
  • 20 Jul 2016 AAV9 CBA SMN1 gene therapy - AveXis receives Breakthrough Therapy status for Spinal muscular atrophy (In infants, In neonates) in USA
  • 16 Mar 2016 AveXis plans a phase I trial for Spinal Muscular Atrophy (intrathecal delivery)
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