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Research programme: enzyme replacement therapies - Valerion Therapeutics

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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: 28 Oct 2021

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At a glance

  • Originator 4s3 Bioscience
  • Developer Valerion Therapeutics
  • Class Antineoplastics; 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.

    No
  • New Molecular Entity Yes

Highest Development Phases

  • No development reported Congenital structural myopathies; Lafora disease; Myotonic dystrophy; Neurofibromatosis 1

Most Recent Events

  • 28 Oct 2021 No recent reports of development identified for preclinical development in Lafora disease in USA (Intraventricular, Injection)
  • 28 Aug 2020 No recent reports of development identified for research development in Neurofibromatosis-1 in USA
  • 28 Aug 2018 No recent reports of development identified for preclinical development in Congenital structural myopathies in USA (Parenteral)

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