Suzhou Xueji Biotech’s XJ-MK-002 Gets FDA Orphan Drug Designation for CAMT

China-based cell therapy developer Suzhou Xueji Biotechnology Co., Ltd’s (HemaCell) XJ-MK-002, described as the world’s first platelet-related cell therapy, received orphan drug designation (ODD) from the US Food and Drug Administration (FDA) last week. The designation is for the treatment of congenital amegakaryocytic thrombocytopenia (CAMT), a rare hereditary bone marrow failure syndrome. Concurrently, a pre-IND application has been filed with the FDA for XJ-MK-001, HemaCell’s first-in-class cell therapy to treat thrombocytopenia.

Therapy Differentiation and Advantages
These therapies, which do not require gene editing, are differentiated from hematopoietic stem cells through simulated developmental processes. This approach is noted for being safe, reliable, and demonstrating significant therapeutic effects. The products feature stable production processes, short cycles, and low infusion doses, enabling sustained and efficient recovery of platelet counts.-Fineline Info & Tech