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Research programme: genetic disorder therapeutics - Enlivex Therapeutics

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Drug Profile

Research programme: genetic disorder therapeutics - Enlivex Therapeutics

Alternative Names: BB-FA; BB-OTC; BBrm 01; BBrm02; BBrm1; Intrathecal azithromycin - Enlivex Therapeutics

Latest Information Update: 22 May 2020

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At a glance

  • Originator Bioblast Pharma
  • Class Macrolides; Proteins; Small molecules
  • Mechanism of Action Frataxin protein replacements; Gene expression modulators; Ornithine carbamoyltransferase replacements; Survival of motor neuron 2 protein expression stimulants
  • Orphan Drug Status

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

    No
  • New Molecular Entity No

Highest Development Phases

  • No development reported Friedreich's ataxia; Inborn urea cycle disorders; Spinal muscular atrophy

Most Recent Events

  • 19 Nov 2018 Bioblas Pharma has been merged into Enlivex Therapeutics to form Enlivex Therapeutics
  • 28 Sep 2018 No recent reports of development identified for preclinical development in Friedreich's ataxia in Israel (Parenteral)
  • 28 Sep 2018 No recent reports of development identified for preclinical development in Inborn-urea-cycle-disorders in Israel (Parenteral)

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