OPC1
for Spinal Cord Injury
OPC1 is an oligodendrocyte progenitor cell therapy that is planned to be evaluated in a device safety study, the DOSED study, in subacute and chronic spinal cord injury patients.
OPC1 is an oligodendrocyte progenitor cell (OPC) transplant therapy designed to provide clinically meaningful recovery in, and improvements to, motor function in individuals with spinal cord injuries (SCIs). OPCs are naturally occurring precursors to the cells that provide electrical insulation for nerve axons in the form of a myelin sheath. SCI most often occurs when the spinal cord is subjected to a severe crush or contusion injury and typically results in severe functional impairment, including limb paralysis, aberrant pain signaling, and loss of bladder control and other body functions. In the U.S., there are approximately 18,000 new spinal cord injuries annually and over 300,000 patients in total living with spinal cord injuries. There currently are no FDA-approved drugs or interventions specifically for the treatment of SCI. The clinical development of OPC1 has been partially funded by a $14.3 million grant from the California Institute for Regenerative Medicine (CIRM). OPC1 has received Regenerative Medicine Advanced Therapy (RMAT) designation and Orphan Drug designation from the U.S. Food and Drug Administration (FDA).
OPC1 has an extensive long-term safety profile and has been tested in two clinical trials to date: a five-patient Phase 1 safety trial in acute thoracic SCI, where all active subjects have been followed for at least 13 years; and a 25-patient Phase 1/2a multicenter dose-escalation trial in subacute cervical SCI, where all active subjects have been evaluated for at least 7 years. Long-term safety monitoring is ongoing for both studies, with no unexpected serious adverse events attributable to the OPC1 transplant being reported to date.
In February 2025, Lineage initiated the DOSED (Delivery of Oligodendrocyte Progenitor Cells (OPCs) for Spinal Cord Injury: Evaluation of a Novel Device) clinical study. The DOSED study will evaluate the safety and utility of a novel delivery device developed to deliver OPC1 directly to the area of injury in patients with SCI. The DOSED study will enroll both subacute (between 21 to 42 days following injury) and chronic (between 1 to 5 years following injury) SCI patients.
Part 1
In 2016, Chris Block was paralyzed in a bicycling accident. In 2023, he is an avid scuba diver, horseback rider and, importantly, has his independence.
Part 2
In 2016, Chris Block was paralyzed in a bicycling accident. In 2023, he is an avid scuba diver, horseback rider and, importantly, has his independence.
In 2016, Jake Javier was paralyzed from the neck down. In late 2022, he is set to graduate from Duke University with his Master’s Degree in Biomedical Engineering, with plans to help those impacted by neurological injuries or diseases.
Highlighting the progress made by Kris Boesen and Lucas Lindner, both of whom received Lineage’s OPC1 cell therapy following traumatic spinal cord injury (SCI).
Publications
Market Opportunity
Learn More About Spinal Cord Injury and Lineage’s OPC1 Cell Therapy
There are approximately 18,000 new spinal cord injuries annually in the U.S. (NSCIC SCI Facts and Figures at a Glance (2019), and there are currently no drugs approved by the FDA specifically for the treatment of SCI.
Clinical Opportunity
OPC1 has been tested in two clinical trials to date; a five patient Phase 1 study in acute thoracic spinal cord injury, where all subjects were followed for at least 10 years, and a 25 patient Phase 1/2a study in subacute cervical spinal cord injury, where all subjects were evaluated for at least 2 years (the “SCiStar trial”). The clinical development of OPC1 has been partially funded by a $14.3 million grant from the California Institute for Regenerative Medicine.
Expected Recovery1 vs OPC1: Motor Function Gains
In November 2019, we reported positive results from the ongoing SciStar study of OPC1, where the overall safety profile of OPC1 has remained excellent with robust motor recovery in upper extremities maintained through Year 2 patient follow-ups available to date. In July 2020, we reported that following the transfer of OPC1 manufacturing to our cGMP manufacturing facility in Israel, key process improvements have been developed and implemented. Lineage also strengthened its patent position in order to protect the OPC1 processes, product and composition, and methods of use. In December 2020, Lineage reported it has developed an enhanced differentiation process, leading to major improvements in production and quality of its OPC1 cell therapy drug product.
In February 2021, we announced that we had entered into an exclusive option and license agreement with Neurgain Technologies, Inc., a medical device company that is commercializing technology developed by neurosurgeons at the University of California San Diego. Under the terms of the agreement, the Company and Neurgain will collaborate on the clinical testing of Neurgain’s novel Parenchymal Delivery Injection (“PDI”) system, which is designed to allow for the administration of cells to the spinal cord without stopping the patient’s respiration. Elimination of the need to stop respiration during surgery is expected to reduce the complexity, risk, and variability of administering cells to the area of injury.
View our pipeline to see our other programs in development.
References:
1. Steeves JD, Lammertse DP, Kramer JL, Kleitman N, Kalsi-Ryan S, Jones L, Curt A, Blight AR, Anderson KD. Outcome Measures for Acute/Subacute Cervical Sensorimotor Complete (AIS-A) Spinal Cord Injury During a Phase 2 Clinical Trial. Top Spinal Cord Inj Rehabil. 2012 Winter;18(1):1-14. doi: 10.1310/sci1801-1. Epub 2012 Jan 31. PMID: 23239927; PMCID: PMC3519288.
2. Fessler, R. G., Ehsanian, R., Liu, C. Y., Steinberg, G. K., Jones, L., Lebkowski, J. S., Wirth, E. D., III, & McKenna, S. L. (2022). A phase 1/2a dose-escalation study of oligodendrocyte progenitor cells in individuals with subacute cervical spinal cord injury, Journal of Neurosurgery: Spine (published online ahead of print 2022). Retrieved Aug 19, 2022, from https://thejns.org/spine/view/journals/j-neurosurg-spine/aop/article-10.3171-2022.5.SPINE22167/article-10.3171-2022.5.SPINE22167.xml