Takara Bio Inc. is pleased to announce that the clinical trial notification for the NY-ESO-1・siTCRTM gene therapy (TBI-1301; mipetresgene autoleucel) (Protocol No.1301-04) has been submitted to the Pharmaceuticals and Medical Devices Agency (PMDA), and the required regulatory review has been completed. Following approval by the Institutional Review Boards (IRBs) of the participating institutions, enrollment of patients and administration of the investigational product are scheduled to commence.
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Title |
Multicenter Study of TBI-1301 (INN: mipetresgene autoleucel; Mip-cel) for NY-ESO-1 Antigen-Positive Synovial Sarcoma |
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Phase |
Phase III |
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Indication |
Unresectable advanced or recurrent synovial sarcoma |
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Design |
Non-controlled, multicenter |
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Enrollment |
5 patients |
Details of the study will be published on the following websites:
・ClinicalTrials.gov：https://clinicaltrials.gov/
・Japan Registry of Clinical Trials（jRCT）：https://jrct.mhlw.go.jp/search
As announced on May 13, 2025, in the press release titled “Redesign of the Regulatory Strategy for NY-ESO-1・siTCR™ Gene Therapy in Japan”, Takara Bio has revised its regulatory and development strategy. The approach has shifted from seeking conditional and time-limited approval to pursuing full approval based on the results of the confirmatory study. This strategy change was made in consultation with the PMDA.
TBI-1301 has been designated by the Ministry of Health, Labour and Welfare as a “Regenerative Medicine Product for Rare Diseases” and is also eligible for expedited review under the “Sakigake Designation System”. Upon completion of the confirmatory study, Takara Bio plans to submit an application for manufacturing and marketing approval, utilizing the priority review pathway through the Sakigake Comprehensive Evaluation Consultation. Takara’s goal is to deliver a new treatment option within five years for synovial sarcoma, a rare and refractory cancer with limited therapeutic alternatives.
Takara Bio remains committed to advancing innovative gene and cell processing technologies and contributing to public health by broadly providing these technologies. The impact of this progress on the financial results for the fiscal year ending March 31, 2026 is expected to be minimal.
