Basic Information

Gene symbol CEP290 Synonyms 3H11Ag, BBS14, CT87, JBTS5, LCA10, MKS4, NPHP6, POC3, SLSN6, rd16 Type of gene protein-coding
Gene perturbation-related omics dataset PertOrg
Description centrosomal protein 290

GTO ID GTC0683
Trial ID 2020-000535-45
Disease Leber Congenital Amaurosis
Altered gene CEP290
Therapeutic/Target gene Target gene
TherapyASO
Treatment Sepofarsen|QR-110
PhasePhase2|Phase3
Recruitment statusCompleted
TitleAn Open-Label, Dose Escalation and Double-Masked, Randomized, Controlled Study to Evaluate the Safety and Tolerability of Sepofarsen in Pediatric Subjects <8 Years of Age with Leber Congenital Amaurosis Type 10 (LCA10) due to the c.2991 +1655A>G (p.Cys998X) mutation
Year2020
CountryBelgium
Company sponsorProQR Therapeutics IV B.V.
Vector information
VectorNo vector was used

Clinical Result

Cohort1: dose level 1
Administration route intravitreal injection
Dosage 0.8 mg/ml
Age Child
Cohort2: dose level 2
Administration route intravitreal injection
Dosage 1.6 mg/ml
Age Child

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