Drug intelligence / Profile preview

CC2008

Development stage
Preclinical
Lead developer
环码生物
Modality
Long Non-coding RNA (lncRNA) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
01

Overview

CC2008 is an investigational circular RNA (circRNA) therapeutic candidate developed by Shanghai CirCode Biomed for the treatment of Gaucher disease. It belongs to a novel class of RNA-based medicines that utilize circularized RNA transcripts to provide more stable and long-lasting protein expression compared to traditional linear mRNA. CC2008 is engineered to encode the human glucocerebrosidase (GBA) enzyme, serving as a protein replacement therapy to address the underlying enzyme deficiency in Gaucher disease patients. By leveraging the proprietary circRNA platform, the drug aims to improve therapeutic efficacy and potentially reduce the frequency of administration by resisting exonuclease-mediated degradation. CC2008 is currently in the preclinical stage of development.

02

Targets

GBA1 (Glucosylceramidase)

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