Spinal cord injury (SCI) is defined for the last few decades as patients getting wheelchair-bound and under medication for a lifetime. SCI is damage to the tight bundle of nerves that communicate signals from the brain to and from the rest of the organs. SCI can be caused by direct injury to the spinal cord or from damage to the tissue and bones that surround the spinal cord. This damage can result in temporary or permanent impairments in motion, sensation and body functions below the site of injury.
Motor vehicle accidents and catastrophic falls are the most common causes of SCI in worldwide.
Complete spinal cord injury causes permanent damage to the area of the spinal cord that is affected. Paraplegia or tetraplegia are major results of complete SCI.
Incomplete spinal cord injury is partial damage of spinal cord. The ability to move depends on the area of the spine injured and the severity of the injury.
Medical battery for SCI is ultimately limited which often results in heightened frustration of the caregivers who land up with no solid solution to the condition yet.
STUP-001, the innovative gene therapy (GT) medication, unties the knot for spinal cord injured
patient through our motor sensory neuron reprogramming technology.
Cell fate conversion using virus transduction system in of great interest in the field of Gene Therapy. Among them, direct lineage reprogramming (DLR) have been spot lighten because of its high drug safety. DLR is the technology which is converting the somatic cell to somatic cells patients need.
Recently, DLR technology has been evaluating as future innovative gene therapy technology which can provide the solution for incurable neuro-disease. STUP-001 is our first launching AAV-based in vivo gene therapy medication for complete or incomplete spinal cord injury (SCI) patients. It is based on the motor sensory neuron direct lineage reprogramming (DLR) using our developed factor Z. We have successfully generated electro-functional motor sensory neurons with high efficiency. We hope our gene therapy (GT) medication, STUP-001, brings hope to every spinal cord injury (SCI) patient in the world.