Research programme: genetic disorder therapeutics - BioBlast Pharma

Drug Profile

Research programme: genetic disorder therapeutics - BioBlast Pharma

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

Latest Information Update: 14 Jul 2015

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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.

  • New Molecular Entity No

Highest Development Phases

  • Preclinical Friedreich's ataxia; Inborn urea cycle disorders; Spinal muscular atrophy

Most Recent Events

  • 22 Jun 2015 Pharmacodynamics data from a preclinical trial in spinal muscular atrophy released by BioBlast Pharma
  • 22 Jun 2015 BioBlast plans a phase IIa trial for Spinal muscular atrophy in USA
  • 26 Mar 2015 Pharmacodynamics data from preclinical trial in Friedreich's ataxia released by BioBlast
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