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RC0125 AAV

Development stage: Preclinical
Lead developer: Rudacure
Modality: Gene Therapies
Administration: Subcutaneous

Overview

RC0125 AAV is an adeno-associated virus (AAV) vector-based gene therapy being developed by RudaCure (also known as LudaCure). It is designed to target the intracellular Transient Receptor Potential Vanilloid 4 (TRPV4) ion channel. The therapy is currently in the drug optimization stage of preclinical development, with initial development targeting rare orphan diseases such as Charcot-Marie-Tooth disease type 2C (CMT2C), skeletal dysplasia, scoliosis, and skeletal malformations. It has also demonstrated analgesic efficacy in animal models of osteoarthritis pain.

Targets

TRPV4 (Transient receptor potential cation channel subfamily V member 4)

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