Research programme: enzyme replacement therapies - Valerion Therapeutics

Drug Profile

Research programme: enzyme replacement therapies - Valerion Therapeutics

Alternative Names: 3E10Fab-GAA - Valerion Therapeutics; 3E10fv-MTM1 fusion protein - Valerion Therapeutics; 4s3-001; 4s3-002; 4s3-004; 4s3-005; VAL 0524; VAL-0411; VAL-0417; VAL-0620; VAL-1205

Latest Information Update: 07 Sep 2017

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At a glance

  • Originator 4s3 Bioscience
  • Developer Valerion Therapeutics
  • Class Enzymes; Proteins
  • Mechanism of Action Enzyme replacements; Protein replacements; Tumour suppressor protein modulators
  • Orphan Drug Status

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

  • New Molecular Entity Yes

Highest Development Phases

  • Preclinical Congenital structural myopathies; Myoclonic epilepsies; Myotonic dystrophy
  • Research Neurofibromatoses

Most Recent Events

  • 06 Sep 2017 Preclinical trials in Myoclonic epilepsies (Lafora disease) in USA (Intraventricular)
  • 26 Jul 2016 Early research in Myoclonic epilepsies in USA (unspecified route) (Alopexx pipelines, July 2016)
  • 26 Jul 2016 Preclinical development is ongoing for Congenital structural myopathies and Myotonic dystrophy in USA (Alopexx pipeline, July 2016)
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