Drug intelligence / Profile preview

HLA-null platelets

Development stage
Unknown
Lead developer
Megakaryon
Modality
Recombinant Proteins and Enzymes, iPSCs → Pluripotent Stem Cells → Stem Cell Therapies → Cell Therapies
Administration
Intravenous
01

Overview

HLA-null platelets are laboratory-produced human platelets that lack expression of human leukocyte antigen (HLA) class I molecules. These are typically generated from induced pluripotent stem cells (iPSCs) by knocking out the β2-microglobulin gene or other components essential for surface expression of HLA-I. The absence of HLA-I prevents recognition and destruction by anti-HLA antibodies and reduces activation by natural killer (NK) cells, making these "universal" donor products suitable for patients with platelet transfusion refractoriness due to alloimmunization against conventional platelet antigens. Mechanistically, these engineered cells evade immune-mediated clearance while retaining normal hemostatic function in preclinical models. The primary indication is treatment of thrombocytopenia in patients who have developed immune refractoriness to standard platelet transfusions[1][5].

Other names
HLA class I knockout plateletsiPSC-derived HLA-null plateletsuniversal donor platelets

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