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CAP 001

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Drug Profile

CAP 001

Alternative Names: CAP-001

Latest Information Update: 01 Jun 2022

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At a glance

  • Originator Capsida Biotherapeutics
  • Class Gene therapies
  • Mechanism of Action Gene transference; Iduronate sulfatase replacements
  • 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

  • Preclinical Mucopolysaccharidosis II

Most Recent Events

  • 18 May 2022 Capsida Biotherapeutics has patent protection for adeno-associated virus compositions for targeted gene therapy in the US
  • 18 May 2022 Preclinical trials in Mucopolysaccharidosis II in USA (IV)
  • 18 May 2022 Pharmacokinetics and pharmacodynamics data from a preclinical trial in Mucopolysaccharidosis II released by Capsida Biotherapeutics

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