Cell therapy, a cutting-edge therapeutic approach, involves transplanting healthy human cells to replace or repair diseased or damaged cells, thereby restoring function. Cell therapies include blood transfusions, where healthy blood cells and platelets from a donor are infused into the patient’s bloodstream. Another cell therapy is the infusion of hematopoietic stem cells to stimulate bone marrow production. A crucial distinction between these two methods lies in the use of somatic versus stem cells. Somatic cells are mature, specialized cells such as brain, muscle, or even photoreceptor cells, while stem cells are versatile, early-stage cells capable of differentiating into different cell types.
BlueRock Therapeutics, a clinical-stage cell therapy company, has recently announced a collaboration and exclusive licensing agreement with FUJIFILM Cellular Dynamics and Opsis Therapeutics to develop and commercialize OpCT-001. OpCT-001, a stem cell-derived cell therapy candidate, holds promise for treating primary photoreceptor diseases. This innovative therapy involves treating patients with a cellular therapy that has been induced from pluripotent stem cells, with the aim of replacing damaged photoreceptor cells with healthy ones. Furthermore, BlueRock Therapeutics has disclosed plans to submit an Investigational New Drug (IND) application to the FDA in 2024, seeking approval to initiate human clinical trials.
What this means for Usher syndrome: The exclusive licensing agreement and anticipated IND filing in 2024 indicates that OpCT-001 may soon be ready for evaluation in human clinical trials and offers new hope to USH patients that vision restoration through the replacement of damaged photoreceptor cells may become a reality one day.
