Drug intelligence / Profile preview

NUDT21 siRNA

Development stage
Phase 1
Lead developer
Fudan University
Modality
Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intravitreal
01

Overview

NUDT21 siRNA is a small interfering RNA (siRNA) therapeutic designed to target and modulate the expression of the NUDT21 gene (nudix hydrolase 21). The drug acts by silencing NUDT21, which plays a key role in alternative polyadenylation and mRNA processing. In preclinical and early clinical studies, targeting NUDT21 with siRNA has been shown to promote tumor apoptosis by regulating the 3' untranslated region (UTR) plus tail of SMC1A, thereby weakening tumor cell proliferative activity and achieving tumor control. This approach is being investigated as a targeted therapy for refractory retinoblastoma, aiming to increase anti-tumor activity while reducing side effects compared to conventional chemotherapy[1][3]. The drug is under development at Fudan University Affiliated Eye & ENT Hospital.

02

Targets

CPSF3 (Cleavage and polyadenylation specificity factor 3)

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