Basic Information

Gene symbol IL1RN Synonyms CRMO2, DIRA, ICIL-1RA, IL-1RN, IL-1ra, IL-1ra3, IL1F3, IL1RA, IRAP, MVCD4 Type of gene protein-coding
Gene perturbation-related omics dataset PerturbAtlas
Description interleukin 1 receptor antagonist

GTO ID GTC3423
Trial ID NCT05835895
Disease Knee Osteoarthritis
Altered gene IL1RN
Therapeutic/Target gene Therapeutic gene
TherapyGene transfer
Treatment GNSC-001|sc-rAAV2.5IL-1Ra
PhasePhase1
Recruitment statusActive, Not Recruiting
TitleA Phase 1b, Randomized, Double-Blinded, Placebo-Controlled Dose Ranging Study to Evaluate Safety, Tolerability and Pharmacodynamics of a Single Intra-articular Injection of GNSC-001 Gene Therapy in Subjects With Osteoarthritis of the Knee
Year2023
CountryUnited States
Company sponsorGenascence Corporation
Other ID(s)CPRO-22-001|CLIN2-14265
Vector information
Vectoradeno-associated virus
Constructsc-rAAV2.5IL-1Ra
Vector typeself-complementary recombinant adeno-associated virus serotype 2/5 (sc-rAAV2/5) vector
Transgene/Inserted genehuman IL-1Ra cDNA
Regulatory elementthe human cytomegalovirus (CMV) immediate early promoter
Vector production methodthe triple plasmid transfection method in HEK293 cells

Clinical Result

Cohort 1
Administration route intraarticular injection
Pts 67
Age Adult, Older_Adult
References PMID: 26817025

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