Drug intelligence / Profile preview

REC-01

Development stage
Unknown
Lead developer
PuREC
Modality
Stem Cell Therapies → Cell Therapies
Administration
Intravenous
01

Overview

REC-01 is a high-purity mesenchymal stem cell (MSC) product derived from donor bone marrow, developed by PuREC Co., Ltd. using its proprietary cell selection technology. It is specifically designed for the treatment of severe pediatric hypophosphatasia (HPP), a rare genetic disorder characterized by defective bone mineralization due to a deficiency in tissue-nonspecific alkaline phosphatase (TNSALP). REC-01 is intended to be administered alongside umbilical cord blood transplantation (UCBT) to provide a sustainable source of alkaline phosphatase-producing cells. The product is currently being evaluated in a Phase I/IIa clinical trial in Japan for patients who are already receiving asfotase alfa but require additional therapeutic support.

Other names
high-purity mesenchymal stem cells

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