STUP-001

STUP-001 is an investigational **AAV-based in vivo gene therapy** developed for the treatment of **spinal cord injury (SCI)**, specifically targeting chronic SCI classified as AIS-A or B. The therapy utilizes **direct lineage reprogramming (DLR)** technology to convert resident **astrocytes into functional motor sensory neurons** within the spinal cord, aiming to restore neuronal function and improve motor activity. The vector employs a proprietary factor, referred to as "factor Z," to induce this cell conversion. Preclinical studies have demonstrated successful astrocyte-to-neuron reprogramming and significant improvements in motor function. STUP-001 is in **phase 1 and phase 2 clinical trials** to evaluate its safety and exploratory efficacy in humans. The drug is developed by **Stand Up Therapeutics** under Dr. Junsang Yoo, with manufacturing support from **VectorBuilder**[1][3][5][7][9][10].

Brand names

STUP-001STUP001STUP 001

Other names

STUP-001STUP001STUP 001