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Clinical data | |
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Other names |
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Routes of administration | Infusion |
Pharmacokinetic data | |
Elimination half-life | 2.9-11 min |
Identifiers | |
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CAS Number |
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PubChem CID | |
PubChem SID | |
DrugBank | |
ChEBI | |
Chemical and physical data | |
Formula | C150H188N57O97P15 |
Molar mass | 4806.062 g·mol−1 |
3D model ( JSmol) | |
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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.
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]
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]
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. [14] [15] In 2018, a Phase I clinical trial found that BC-007 was well-tolerated, with no serious adverse events reported. [2] [12] Phase IIa trials demonstrated that BC-007 could neutralize the activity of functional autoantibodies in most subjects treated. [16]
BC-007 is under investigation as a possible agent for treating disorders of long COVID. [17]
![]() | |
Clinical data | |
---|---|
Other names |
|
Routes of administration | Infusion |
Pharmacokinetic data | |
Elimination half-life | 2.9-11 min |
Identifiers | |
| |
CAS Number |
|
PubChem CID | |
PubChem SID | |
DrugBank | |
ChEBI | |
Chemical and physical data | |
Formula | C150H188N57O97P15 |
Molar mass | 4806.062 g·mol−1 |
3D model ( JSmol) | |
| |
|
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.
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]
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]
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. [14] [15] In 2018, a Phase I clinical trial found that BC-007 was well-tolerated, with no serious adverse events reported. [2] [12] Phase IIa trials demonstrated that BC-007 could neutralize the activity of functional autoantibodies in most subjects treated. [16]
BC-007 is under investigation as a possible agent for treating disorders of long COVID. [17]