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GNSC 001

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Drug Profile

GNSC 001

Alternative Names: AAVIL-1Ra; GNSC-001; IL-1Ra; Sc-rAAV2.5IL-1Ra

Latest Information Update: 21 Jul 2025

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At a glance

  • Originator Mayo Clinic
  • Class Antirheumatics; Gene therapies
  • Mechanism of Action Gene transference; Interleukin 1 receptor antagonists
  • 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

  • Phase I Osteoarthritis

Most Recent Events

  • 16 Jul 2025 GNSC 001 receives Regenerative Medicine Advanced Therapy (RMAT) status for Osteoarthritis in USA
  • 11 Jun 2025 Updated adverse events and pharmacodynamics data from the phase Ib DONATELLO trial in Osteoarthritis presented at the 26th Annual Congress of the European League Against Rheumatism (EULAR-2025)
  • 12 May 2025 GNSC 001 is still in phase-I development in Osteoarthritis(In the elderly, In adults) in USA (Intra-articular, Injection) (NCT05835895)

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