Gene therapy targeting mesenchymal stem cells
First written in FY2019. 10 of 18 entries say "Advanced", first in FY2020. Latest entry: 2026-10-07.
No other company in this theme has enough history to compare yet.
- FY2026-H1RestatedAnnual report
「In addition, stem cell gene therapy SR-GT1, aimed at providing a curative treatment for epidermolysis bullosa, was selected for the fiscal 2024 project conducted by the Japan Agency for Medical Research and Development (AMED), the “Project for Development of Core Technologies for the Industrialization of Regenerative Medicine and Gene Therapy (Project to Promote the Industrialization of Regenerative, Cellular and Gene Therapies).” The project aims to move swiftly to an investigator-initiated clinical trial by manufacturing investigational drugs for clinical application, building on the manufacturing system for gene-transduced cell products established under the fiscal 2022 Practical Research Project for Rare/Intractable Diseases and the advice received through the Pharmaceuticals and Medical Devices Agency (PMDA) regulatory science strategy consultation. Under the project, up to two-thirds of the expenses incurred may be received as an AMED subsidy, with the potential to receive a maximum total subsidy of ¥179 million over the 3 years from fiscal 2024 through fiscal 2026 (through March 2027).」
For SR-GT1, the development policy aimed at selection for an AMED project, manufacture of investigational drugs and transition to an investigator-initiated clinical trial was reconfirmed, along with the possibility of receiving a maximum subsidy of ¥179 million over the 3 years through March 2027. - FY2025RestatedAnnual report
「This is a treatment technology that enables regeneration-inducing medicine for severe hereditary diseases caused by gene deficiencies and other abnormalities. Using the Company's proprietary stem cell culture and conditioning technologies developed to date, it involves genetically editing the patient's own stem cells ex vivo, supplementing the deficient or mutated genes, and returning the cells to the patient's body as a curative regenerative cell therapy product.」
The development policy for a curative treatment technology involving gene transfer into patient-derived mesenchymal stem cells and their return to the body was reconfirmed. - FY2025-H1AdvancedAnnual report
「In addition, stem cell gene therapy SR-GT1, aimed at providing a curative treatment for epidermolysis bullosa, was selected for the fiscal 2024 project conducted by the Japan Agency for Medical Research and Development (AMED), the “Project for Development of Core Technologies for the Industrialization of Regenerative Medicine and Gene Therapy (Project to Promote the Industrialization of Regenerative, Cellular and Gene Therapies).” The project aims to move swiftly to an investigator-initiated clinical trial by manufacturing investigational drugs for clinical application, building on the manufacturing system for gene-transduced cell products established under the fiscal 2022 Practical Research Project for Rare/Intractable Diseases and the advice received through the Pharmaceuticals and Medical Devices Agency (PMDA) regulatory science strategy consultation. Under the project, up to two-thirds of the expenses incurred may be received as an AMED subsidy, with the potential to receive a maximum total subsidy of ¥179 million over 3 years.」
The stem cell gene therapy development code was identified as SR-GT1, and specific development stages were presented, including selection for an AMED project, manufacture of investigational drugs and transition to an investigator-initiated clinical trial. The possibility of receiving a maximum subsidy of ¥179 million over 3 years was also stated. - FY2024RestatedAnnual report
「This is a curative regenerative cell therapy product that involves genetically editing the patient's own stem cells ex vivo, supplementing the deficient or mutated genes, and returning the cells to the patient's body.」
The development policy for a curative treatment technology involving gene transfer into patient-derived mesenchymal stem cells and their return to the body was reconfirmed. - FY2024-Q3RestatedAnnual report
「The Company's stem cell gene therapy under development in collaboration with Osaka University, with development code PJ5, is a curative treatment technology for epidermolysis bullosa. It is based on a proprietary technology for collecting mesenchymal stem cells from blisters in patients with epidermolysis bullosa, efficiently introducing the type VII collagen gene into patient skin-derived mesenchymal stem cells using a lentiviral vector, and returning the cells to the blisters to enable sustained delivery of type VII collagen. Skin tissue models of epidermolysis bullosa were created using patient-derived skin cells, and blisters were artificially formed by suction. In the models created by administering mesenchymal stem cells carrying the type VII collagen gene to the same area as the blisters, the type VII collagen protein was supplied extensively to the basement membrane region, and no blisters formed. In addition, intrab blister administration demonstrated high engraftment capability in vivo compared with other administration routes.」
The introduction of the type VII collagen gene into patient-derived mesenchymal stem cells, delivery to the basement membrane through intrab blister administration, suppression of blister formation and high engraftment capability were reconfirmed. - FY2024-Q1RestatedAnnual report
「The Company's stem cell gene therapy under development in collaboration with Osaka University, with development code PJ5, is a curative treatment technology for epidermolysis bullosa. It is based on a proprietary technology for collecting mesenchymal stem cells from blisters in patients with epidermolysis bullosa, efficiently introducing the type VII collagen gene into patient skin-derived mesenchymal stem cells using a lentiviral vector, and returning the cells to the blisters to enable sustained delivery of type VII collagen.」
The development policy for a curative treatment technology involving introduction of the type VII collagen gene into patient-derived mesenchymal stem cells was reconfirmed. - FY2023AdvancedAnnual report
「Skin tissue models of epidermolysis bullosa were created using patient-derived skin cells, and blisters were artificially formed by suction. In the models created by administering mesenchymal stem cells carrying the type VII collagen gene to the same area as the blisters, the type VII collagen protein was supplied extensively to the basement membrane region, and no blisters formed. In addition, intrab blister administration demonstrated high engraftment capability in vivo compared with other administration routes.」
For stem cell gene therapy, delivery of type VII collagen to the basement membrane region, suppression of blister formation and high engraftment capability following intrab blister administration were confirmed. - FY2023-Q2AdvancedAnnual report
「Compared with other administration routes, intrab blister administration has demonstrated high engraftment capability in vivo.」
A new research finding was reported that intrab blister administration in stem cell gene therapy produces higher engraftment capability than other administration routes. - FY2023-Q1RestatedAnnual report
「The Company's stem cell gene therapy under development in collaboration with Osaka University, with development code PJ5, is a curative treatment technology for epidermolysis bullosa. It is based on a proprietary technology for collecting mesenchymal stem cells from blisters in patients with epidermolysis bullosa, efficiently introducing the type VII collagen gene into patient skin-derived mesenchymal stem cells using a lentiviral vector, and returning the cells to the blisters to enable sustained delivery of type VII collagen.」
The development policy for a curative treatment technology involving introduction of the type VII collagen gene into patient-derived mesenchymal stem cells was reconfirmed. - FY2022RestatedAnnual report
「Skin tissue models of epidermolysis bullosa were created using patient-derived skin cells, and blisters were artificially formed by suction. In the models created by administering mesenchymal stem cells carrying the type VII collagen gene to the same area as the blisters, it was confirmed that no blisters formed.」
The research finding that introduction of the type VII collagen gene into mesenchymal stem cells prevents blister formation in an epidermolysis bullosa model was reconfirmed. - FY2022-Q3AdvancedAnnual report
「Skin tissue models of epidermolysis bullosa were created using patient-derived mesenchymal stem cells, and blisters were artificially formed by suction. In the skin tissue models created using mesenchymal stem cells into which the type VII collagen gene had been introduced, no blisters formed.」
For gene therapy involving introduction of the type VII collagen gene into patient-derived mesenchymal stem cells, it was confirmed that no blisters formed in skin tissue models of epidermolysis bullosa. A plan was also presented to participate as a joint research company in AMED's Practical Research Project for Rare/Intractable Diseases from April 2022. - FY2020AdvancedAnnual report
「Experiments involving transplantation into animals of mesenchymal stem cells into which normal genes had been incorporated using gene-editing technology confirmed that the target protein, type VII collagen, was stably produced at sufficient levels in the transplanted animals, demonstrating that the expected mechanism of action functions as intended.」
For gene therapy targeting mesenchymal stem cells, animal studies confirmed stable production of the target protein and functioning of the expected mechanism of action. - FY2019First statedAnnual report
「This is a curative regenerative cell therapy product that involves genetically editing the patient's own stem cells ex vivo, supplementing the deficient or mutated genes, and returning the cells to the patient's body. ※32」
The development target is a therapeutic technology in which the patient's own stem cells are collected, genetically edited ex vivo and then returned to the patient.