Английская Википедия:BC-007
Шаблон:Short description Шаблон:Use American English Шаблон:Infobox drug
BC-007 is an oligonucleotide aptamer, a synthetic DNA compound designed to bind other chemicals.[1] BC-007 is in early-stage clinical trials as a lead compound intended for the potential treatment of heart failure or long COVID. The international nonproprietary name is rovunaptabin.[2]
History
Since the 1990s, GPCR autoantibodies were investigated as possible factors 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 2013, scientists from the Max Delbrück Center and the Charité Heart Center reported using aptamers as a treatment for dilated cardiomyopathy in people positive for beta-1 adrenergic receptor AABs.[7][8] In 2015–16, scientists reported that two aptamers might bind GPCR-AABs, possibly resulting in an inhibition of GPCR-AABs.[9][10]
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.[11]
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'.[1] Its three-dimensional structure allows it to wrap around the target structure of functionally active G-protein-coupled receptor autoantibodies and neutralize their activity.[1]
BC-007 can be synthetically produced, and is considered safe.[1][11] In preliminary human studies, it was given by intravenous infusion and had an in vivo plasma half-life of around 4 minutes.[1]
Research
Heart failure
The removal of pathogenic functional autoantibodies through a medical blood purification procedure, known as immunoadsorption, can stabilize heart function in people with dilated cardiomyopathy who are awaiting heart transplantation.[12][13] In 2018, a Phase I clinical trial found that BC-007 was well-tolerated, with no serious adverse events reported.[1][11] Phase IIa trials demonstrated that BC-007 could neutralize the activity of functional autoantibodies in most subjects treated.[14]
Long COVID
BC-007 is under investigation as a possible agent for treating disorders of long COVID.[15]
References
External links
- ↑ 1,0 1,1 1,2 1,3 1,4 1,5 Шаблон:Cite book
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- ↑ 11,0 11,1 11,2 Шаблон:Cite web
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