Research programme: neurodegenerative disorders therapeutics - BioMarin

Drug Profile

Research programme: neurodegenerative disorders therapeutics - BioMarin

Alternative Names: Compound 106; Compound 109; Compound 123; Compound 136; Compound 44; Compound 4b; Compound 526; Compound 531; Compound 9b; RGFA 8

Latest Information Update: 03 Feb 2014

Price : $50

At a glance

  • Originator The Scripps Research Institute
  • Developer BioMarin Pharmaceutical; Repligen Corporation
  • Class Small molecules
  • Mechanism of Action Histone deacetylase inhibitors; Transcription factor 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 Yes

Highest Development Phases

  • Preclinical Friedreich's ataxia; Huntington's disease
  • Discontinued Spinal muscular atrophy

Most Recent Events

  • 21 Jan 2014 License to The Scripps Research Institute’s histone deacetylase inhibitors transferred to BioMarin
  • 01 Jan 2014 Discontinued - Preclinical for Spinal muscular atrophy in USA (unspecified route) before January 2014
  • 21 Mar 2012 Compounds are still in preclinical studies for Huntington's disease in USA
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