Cambridge, Massachusetts Patent of the Year – 2024/2025
STRM.bio Incorporated has been awarded the 2024/2025 Patent of the Year for their groundbreaking ‘Compositions and methods related to megakaryocyte-derived extracellular vesicles’. Their invention, detailed in U.S. Patent No. 12090234, this innovation leverages naturally occurring extracellular vesicles (EVs) from megakaryocytes – platelet-producing cells – to deliver gene therapies directly to bone marrow stem cells.
Unlike traditional gene delivery methods, which often rely on viral vectors or lipid nanoparticles, STRM.bio’s approach utilizes megakaryocyte-derived EVs as a non-viral, off-the-shelf delivery system. These EVs are engineered to carry therapeutic cargo, such as DNA or RNA, and are capable of targeting hematopoietic stem and progenitor cells (HSPCs) in the bone marrow. This targeted delivery minimizes off-target effects and enhances the precision of gene therapies.
Preclinical studies in mice and non-human primates have demonstrated that these EVs preferentially accumulate in the bone marrow following intravenous administration, with minimal distribution to other organs like the liver or spleen. Importantly, the platform has shown no adverse effects on platelet counts, coagulation parameters, or liver and kidney function, indicating its safety for repeated dosing.
STRM.bio’s patented technology represents a significant advancement in gene therapy, offering a safer, more efficient method for treating rare blood disorders and potentially other diseases requiring targeted gene delivery.
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