RJK002

RJK002 is an investigational adeno-associated virus (AAV)-based gene therapy developed by **RJK Biopharma** for the treatment of **Amyotrophic Lateral Sclerosis (ALS)**. The therapy is designed to be administered via a single **intrathecal injection**. It aims to address the underlying pathology of ALS by targeting the abnormal accumulation and aggregation of **TAR DNA-binding protein 43 (TDP-43)**, a hallmark protein found in the majority of ALS cases. By delivering a genetic payload that modulates TDP-43 levels or function, RJK002 seeks to preserve motor neuron function and slow disease progression. It is currently being evaluated in Phase 1 clinical trials to assess its safety, tolerability, and preliminary efficacy, including its impact on viral load and biomarkers in the serum and cerebrospinal fluid.

Other names

Adeno-associated virus vector-based gene therapy delivering a therapeutic transgene of RJK002