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Research programme: gene-encoded enzyme replacement therapy - SmartPharm Therapeutics

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Drug Profile

Research programme: gene-encoded enzyme replacement therapy - SmartPharm Therapeutics

Latest Information Update: 29 Jul 2024

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At a glance

  • Originator SmartPharm Therapeutics
  • Class Gene therapies
  • Mechanism of Action Enzyme replacements; Gene transference
  • 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 Lysosomal storage diseases

Most Recent Events

  • 29 Jul 2024 Preclinical development is ongoing for Lysosomal storage diseases in USA (Parenteral, Injection) (SmartPharm Therapeutics pipeline, July 2024)
  • 28 Oct 2023 No recent reports of development identified for preclinical development in Lysosomal storage diseases in USA (Parenteral, Injection)
  • 01 Sep 2020 SmartPharm Therapeutics has been acquired by Sorrento Therapeutics

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