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Onasemnogene abeparvovec - Novartis Gene Therapies/Nationwide Children's Hospital

Drug Profile

Onasemnogene abeparvovec - Novartis Gene Therapies/Nationwide Children's Hospital

Alternative Names: AAV9-CBA-SMN1-gene-therapy-Novartis Gene Therapies; Adeno-associated-serotype-9-chicken-beta-actin-survival-motor-neuron-gene-therapy-Novartis Gene Therapies; AVXS 101; ChariSMA™; OAV-101; onasemno-gene abepar-vovec; onasemnogene abeparvovec-xioi; scAAV9.CB.SMN; SMA1-gene-therapy-Novartis Gene Therapies; SMN1-gene-therapy-Novartis Gene Therapies; SMNT-gene-therapy-Novartis Gene Therapies; Spinal-muscular-atrophy-gene-therapy-Novartis Gene Therapies; Survival-motor-neuron-1-gene-therapy-Novartis Gene Therapies; T-BCD541-gene-therapy-Novartis Gene Therapies; Telomeric-SMN-gene-therapy-Novartis Gene Therapies; ZOLGENSMA

Latest Information Update: 06 Jun 2022

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At a glance

  • Originator Nationwide Children's Hospital
  • Developer AveXis; Novartis Gene Therapies
  • Class Gene therapies; Spinal muscular atrophy gene therapies
  • Mechanism of Action Gene transference; Survival of motor neuron 1 protein replacements
  • Orphan Drug Status

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

    Yes - Spinal muscular atrophy
  • New Molecular Entity No

Highest Development Phases

  • Marketed Spinal muscular atrophy

Most Recent Events

  • 28 Apr 2022 NCT05335876-Feedback incorporated
  • 28 Apr 2022 Novartis plans phase IIIb STRENGTH trial in Spinal muscular atrophy (In children) in September 2022 (Intrathecal, Injection) (NCT05386680)
  • 02 Apr 2022 Efficacy and adverse events data from the phase III SPR1NT trial in Spinal muscular atrophy presented at the 74th Annual Meeting of the American Academy of Neurology 2022 (AAN-2022)
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