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Research programme: motor neuron disease gene therapy - Oxford BioMedica

Drug Profile

Research programme: motor neuron disease gene therapy - Oxford BioMedica

Alternative Names: Amyotrophic lateral sclerosis gene therapy - Oxford BioMedica; MoNuDin; OXB-103; SMN-1G; Spinal muscular atrophy gene therapy - Oxford BioMedica

Latest Information Update: 24 Oct 2018

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At a glance

  • Originator Oxford BioMedica; The Cleveland Clinic Foundation
  • Developer Oxford BioMedica
  • Class Gene therapies; Spinal muscular atrophy gene therapies
  • Mechanism of Action Gene transference; Survival of motor neuron 1 protein expression stimulants; Vascular endothelial growth factor A expression stimulants
  • Orphan Drug Status

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

  • New Molecular Entity No
  • Available For Licensing Yes - Amyotrophic lateral sclerosis; Motor neuron disease

Highest Development Phases

  • Preclinical Amyotrophic lateral sclerosis
  • Discontinued Spinal muscular atrophy

Most Recent Events

  • 24 Oct 2018 Preclinical development in Amyotrophic lateral sclerosis is ongoing in United Kingdom (Oxford BioMedica pipeline, October 2018)
  • 24 Oct 2018 Discontinued - Preclinical for Spinal muscular atrophy in United Kingdom (IM)
  • 04 Nov 2017 No recent reports of development identified for preclinical development in Amyotrophic-lateral-sclerosis in United Kingdom (IM, Injection)
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