ACE 031 1mg
ACE-031 is an investigational (experimental) drug that is designed to increase muscle size and strength by blocking proteins that slow down muscle growth. ACE-031 is a protein drug that is administered by subcutaneous injection.
ACE-031 (ACVR2B) 1mg Polypeptide Hormones Myostatin Inhibitor ACE 031 for Muscle Growth
Description:
40.5 kDa protein containing 368 amino acid residues of the ActRIIb-hFc recombinant protein.
MDAMKRGLCCVLLLCGAVFVSPGASGRGEAETRECIYYNANWELERTN QSGLERCEGEQDKRLHCYASWRNSSGTIELVKKGCWLDDFNCYDRQEC VATEENPQVYFCCCEGNFCNERFTHLPEAGGPEVTYEPPPTAPTGGGTH TCPPCPAPELLGGPSVFLFPPKPKDTLMISRTPEVTCVVVDVSHEDPEVKF NWYVDGVEVHNAKTKPREEQYNSTYRVVSVLTVLHQDWLNGKEYKCK VSNKALPVPIEKTISKAKGQPREPQVYTLPPSREEMTKNQVSLTCLVKGF YPSDIAVEWESNGQPENNYKTTPPVLDSDGSFFLYSKLTVDKSRWQQG
NVFSCSVMHEALHNHYTQKSLSLSPGK
Unit:1mg/vial ,10vial/kit
Synonyms:ACE-031, ActRIIb-hFc, ACVR2B, activin receptor type IIB and human immunoglobulin Fc region construct, myostatin inhibitor.
Physical Appearance:
Sterile Filtered White lyophilized (freeze-dried) powder.
Specificity:The amino acid sequence of the recombinant human ActRIIb-hFc is 100% homologous to the amino acid sequence of the human ActRIIb receptor and immunoglobulin Fc region proteins.
Solubility:Add 1 ml of sterile diluent and let the lyophilized pellet dissolve completely.
Stability:Store lyophilized protein at -20 °C. Aliquot the product after reconstitution to avoid repeated freezing/thawing cycles. Reconstituted protein can be stored at 4 °C for a limited period of time. The lyophilized protein remains stable until the expiry date when stored at -20 °C.
Ace-031, also known as ACVR2B, is a soluble form of activin type IIB receptor, which is an inhibitor of myostatin and other naturally occurring proteins that limit muscle growth. This compound was genetically developed to bind to myostatin before it can attach to its receptor and halt muscle growth. The two Biopharma companies that worked on this compound are Acceleron and Shire.
Development of ACE-031 is fully discussed in Diverting an Unwanted Protein, in the April 2011 issue of Quest, MDA’s quarterly research and health magazine.
Companies hope to resume ACE-031 testing after safety issues fully explored
Acceleron and Shire are completing studies of ACE-031 in rats and monkeys and will analyze the human and animal data to gain an understanding of the observed adverse events. The companies say they hope to resume testing ACE-031 in humans after these analyses have been obtained and reviewed by the U.S. Food and Drug Administration (FDA) and Health Canada.
What is ACE-031?
ACE-031 is an investigational (experimental) drug that is designed to increase muscle size and strength by blocking proteins that slow down muscle growth. ACE-031 is a protein drug that is administered by subcutaneous injection.
How does ACE-031 work on muscle?
Muscle growth is regulated by proteins in the body that serve as “on” or “off” switches for muscle production. ACE-031 acts by reducing the “off” signal that stops muscle production, thus muscles may become larger and stronger.
Why is ACE-031 being studied in Duchenne Muscular Dystrophy (DMD)?
ACE-031 may promote muscle growth and improve muscle quality, which may compensate for loss of muscle strength and function due to disease-related muscle damage that occurs in DMD. Because it acts on muscle, ACE-031 has the potential to treat all DMD patients, regardless of their type of dystrophin mutation.
What does ACE-031 do in animal models of DMD?
In animal studies, including healthy animals and animal models of DMD, ACE-031 has been shown to increase the size of muscles and decrease the amount of abnormal scarring in muscle. ACE-031 has also been shown to increase the protein utrophin, which may partially make up for the lack of dystrophin, which causes DMD. The significance of this effect on utrophin is not yet known.
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