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BC-007
DNA-based experimental drug From Wikipedia, the free encyclopedia
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BC-007, whose international nonproprietary name is Rovunaptabin,[1] is an oligonucleotide aptamer, a synthetic DNA compound designed to bind other chemicals.[2] BC-007 is in early-stage clinical trials as a lead compound intended for the potential treatment of heart failure or long COVID.
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History
Since the 1990s, the binding of G protein coupled receptors to autoantibodies (GPCR-AABs) was investigated as a possible factor in the pathology of several diseases, including heart disease.[3][4] In parallel, treatment strategies to remove GPCR-AABs were investigated, initially using proteins or peptides to bind the antibodies.[5][6]
In 2012, scientists from the Max Delbrück Center and the Charité Heart Center obtained a patent in the United States for using aptamers as a therapy or diagnosis of autoimmune diseases.[7] Beginning in 2013, the research group focused on the treatment of dilated cardiomyopathy in people positive for beta-1 adrenergic receptor autoantibodies.[8][9] In 2015–16, scientists reported that two aptamers might bind and inhibit GPCR-AABs.[10][11]
The biotechnology company Berlin Cures pursued the development of the aptamer with the nucleotide sequence GGT TGG TGT GGT TGG under the codename BC-007 for the inhibition of autoantibodies in cardiomyopathy.[12]
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Properties
BC-007 is a 15-nucleotide single-stranded DNA molecule consisting of nine unmodified deoxy-guanosines and six corresponding deoxythymidines with the sequence 5'-GGT TGG TGT GGT TGG-3'.[2] Its three-dimensional structure allows it to wrap around the target structure of G-protein-coupled receptor autoantibodies and neutralize their activity.[2]
BC-007 is synthetic, enabling it to be produced in high volumes quickly.[13] It is stable and suited for long-term storage.[13] It has shown no side effects in early clinical studies, and does not trigger immunological responses.[2][13] As it is water soluble, it can be formulated as inhalation or as nasal spray.[13] In some human studies, it was given by intravenous infusion, displaying an in vivo half-life in blood of about 4 minutes.[2]
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References
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