Our Pipeline

Indication

Chronic Traumatic Spinal Cord Injury (SCI) patients classified as AIS grade A or B. AIS-A refers to complete sensory and motor paralysis, while AIS-B involves partial sensory loss with complete motor paralysis.

• -Chronic Traumatic Spinal Cord Injury (SCI)
• -AIS-A or B grade patients (complete motor paralysis)

Modality

AAV-based in vivo gene therapy.

Mode of Action

STUP-001 is designed to directly convert somatic cells into functional motor and sensory neural cell in vivo. This process, known as Direct Lineage Reprogramming (DLR), aims to rebuild neural circuits in lesion site.

Key Data

• •In a preclinical SCI disease model, STUP-001 demonstrated a more than 30% improvement in the BBB motor function score.
• •STUP-001 administered group showed significant recovery of both motor (MEP) and sensory (SSEP) evoked potentials, indicating the restoration of functional neural pathways.
• •STUP-001 successfully induced the formation of new, myelinated neurons and showed evidence of synaptic connections between reprogrammed neurons and existing ones.
• •In GLP toxicity studies, STUP-001 was confirmed to be non-toxic, with no target organ toxicity observed even at high doses (500X).

Status

A Phase I/IIa clinical trial was approved by the South Korean Ministry of Food and Drug Safety (MFDS) in Mar 2025. The first patient enrollment was conducted in Aug 2025.

Indication

Parkinson's disease.

Modality

AAV9-based in vivo gene therapy.

Mode of Action

The therapy targets degenerated dopaminergic neurons within the brain. It is designed to restore the function of these malfunctioning cells by delivering a proprietary Cell Resetting Factor. This Cell Resetting Technology (CRT) aims to fundamentally "reset" the neurons back to a healthy, functional state, thereby revitalizing both pre- and post-synaptic activity. This approach differs from conventional gene therapies that only supplement deficient proteins.

Key Data

• •In 6-OHDA-treated primary dopaminergic neurons, STUP-002 promoted axonal elongation, enhanced dendritic branching, and restored key electrophysiological properties, including action potential firing rates and resting membrane potential, to levels comparable to healthy controls.
• •In toxin-induced PD models, STUP-002 revitalized dopaminergic neurons and improved motor function.
• •Ex vivo  and  in vivo  recordings showed rescued neuronal morphology.
• •Transcriptomic analyses (RNA-seq, scRNA-seq and spatial transcriptomics) showed that STUP-002 treatment largely normalized the gene expression profiles of PD neurons, shifting them closer to the transcriptional landscape of healthy controls.

Status

Preclinical development.