On August 30, 2019 Esco Ventures reported the launch of Carmine Therapeutics (Press release, Carmine Therapeutics, AUG 30, 2019, View Source [SID1234567821]). Carmine Therapeutics plans to establish a site in Cambridge, Massachusetts and to expand in the United States at the end of 2019 .
Carmine Therapeutics is developing novel types of gene therapies that utilize extracellular vesicles (EVs) produced in the laboratory from red blood cells (RBCs) and that solve many unmet medical needs.
The foundational work behind this revolutionary platform technology was published in 2018 in Nature Communications 9:2359. Red blood cell extracellular vesicles (RBCEVs) are homogeneous, 150-nanometer vesicles that are produced physiologically by RBCs as they age. Several features make RBCEVs an ideal delivery vehicle for gene therapy. First, they are homogenous and can be produced in huge amounts at low cost. Second, RBCs are routinely used for blood transfusion and their secreted vesicles are biocompatible and non-immunogenic. Third, RBCEVs can be frozen and thawed multiple times for long-term storage and can be administered repeatedly without adverse effects. Fourth, RBCEVs have a unique natural biodistribution and can deliver cargoes to several organs in addition to the liver, including bone marrow cells. Lastly, they can be used to target specific cells and tissues using covalently attached peptides or single chain antibodies.
With the REGENT Platform – a proprietary RBCEV isolation, engineering and manufacturing platform of Carmine Therapeutics, we are generating a pipeline of next-generation gene therapies in the areas of hematology, oncology, and immunology.
Using our proprietary technology, RBCEVs can be loaded efficiently with nucleic acids up to 10 kilobases in size and they can deliver their payload efficiently both to cultured cells and to tissues and tumors in vivo. The powerful combination of a robust vector and a unique payload enables us to overcome most of the limitations of existing gene therapies in the clinic. Carmine Therapeutics has exclusively in-licensed and is also developing a comprehensive portfolio of intellectual properties encompassing the key aspects of the REGENT Platform and REGENT product candidates.