Elapegademase

• CAT Number: I048028
• CAS Number: 1709806-75-6
• Molecular Formula: C10H20N2O5
• Molecular Weight: 248.3
• Purity: 98%
• Synonyms: Revcovi; Elapegademase-lvlr; Elapegademase [INN]
• Target: ADA
• Target Protein: XNGDIOBPVDBNQH-QMMMGPOBSA-N
• Appearance: Solid
• IUPAC Name: (2S)-2-amino-6-(2-methoxyethoxycarbonylamino)hexanoic acid
• InChI: InChI=1S/C10H20N2O5/c1-16-6-7-17-10(15)12-5-3-2-4-8(11)9(13)14/h8H,2-7,11H2,1H3,(H,12,15)(H,13,14)/t8-/m0/s1
• SMILES: COCCOC(=O)NCCCCC(C(=O)O)N

Elapegademase is an investigational enzyme replacement therapy (ERT) developed to treat adenosine deaminase severe combined immunodeficiency (ADA-SCID), a rare genetic disorder. ADA-SCID is characterized by a deficiency of the enzyme adenosine deaminase, crucial for immune cell function. Elapegademase is a PEGylated recombinant adenosine deaminase enzyme, modified with polyethylene glycol (PEG) to enhance stability and prolong activity. It aims to replace the missing enzyme in ADA-SCID patients. Administered by intramuscular injection, elapegademase offers a potentially long-lasting treatment option, addressing the underlying cause of ADA-SCID. Clinical trials are underway to assess its safety and efficacy.