Research programme: rare disease mRNA therapeutics - Elpidera

Drug Profile

Research programme: rare disease mRNA therapeutics - Elpidera

Alternative Names: Antibody messenger RNA therapeutics™; mRNA-3283; mRNA-3630; mRNA-3704; mRNA-3927; MT-004; MT-026; MT-033; MT-039; MT-059; MT-088; MT-104

Latest Information Update: 16 Jul 2018

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At a glance

  • Originator Moderna Therapeutics
  • Developer Moderna Therapeutics; National Human Genome Research Institute
  • Class RNA
  • Mechanism of Action Alpha-galactosidase stimulants; Methylmalonyl CoA mutase stimulants; Protein synthesis stimulants
  • Orphan Drug Status

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

    Yes - Methylmalonic acidaemia
  • New Molecular Entity Yes
  • Available For Licensing Yes

Highest Development Phases

  • Preclinical Fabry's disease; Methylmalonic acidaemia; Phenylketonuria
  • Research Acidaemia
  • No development reported Haemophilia

Most Recent Events

  • 11 Jul 2018 Preclinical trials in Fabry's disease in USA (Parenteral)
  • 11 Jul 2018 Preclinical trials in Phenylketonuria in USA (Parenteral)
  • 11 Jul 2018 mRNA 3704 receives Orphan Drug status for Methylmalonic acidaemia in Europe (Before July 2018)
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