Drug Profile

GNSC 001

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

Latest Information Update: 21 May 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

12 May 2025 GNSC 001 is still in phase-I development in Osteoarthritis(In the elderly, In adults) in USA (Intra-articular, Injection) (NCT05835895)
12 May 2025 Genascence completes a meeting with the US FDA for the design of a phase IIb/III trial in Osteoarthritis
12 May 2025 Updated adverse events and pharmacodynamics data from the phase Ib DONATELLO trial in Osteoarthritis released by Genascence Corporation

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