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DFT 383

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Drug Profile

DFT 383

Alternative Names: DFT-383

Latest Information Update: 14 May 2025

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At a glance

  • Originator Novartis Pharmaceuticals
  • Class Gene therapies
  • Mechanism of Action CTNS 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 Yes
  • Available For Licensing Yes

Highest Development Phases

  • Phase I/II Cystinosis

Most Recent Events

  • 30 Apr 2025 Phase-I/II clinical trials in Cystinosis (In children) in USA (Parenteral) (NCT06910813)
  • 31 Mar 2025 Novartis plans the phase I/II CYStem trial for Nephropathic Cystinosis (In children) in (Parenteral), in March 2025 (NCT06910813)
  • 26 Apr 2024 DFT 383 is available for licensing as of 26 Apr 2024. https://www.novartis.com/partnering

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