Compositions, kits and methods for treatment of complement-related disorders

Abstract

Methods, compositions and kits for regulating complement activity or treating a complement activity disorder in a subject using soluble, membrane-independent CD59 protein, methods of assaying human macular degeneration (MD), and methods and kits for assaying potential therapeutic agents for treatment of human MD are provided herein.

1 - 102 . (canceled) 
     
     
         103 . A pharmaceutical composition for use in treating a macular degeneration in a human subject, the pharmaceutical composition comprising a membrane-independent CD59 protein and a pharmaceutically acceptable buffer, wherein the pharmaceutical composition is sufficiently pure for administration to a human subject. 
     
     
         104 . The pharmaceutical composition of  claim 103 , wherein the membrane-independent CD59 protein lacks a glycosyl phosphatidyl inositol (GPI) anchoring domain. 
     
     
         105 . The pharmaceutical composition of  claim 103 , wherein the membrane independent CD59 protein comprises about 90% identity to SEQ ID NO: 5. 
     
     
         106 . The pharmaceutical composition of  claim 103 , wherein the membrane independent CD59 protein comprises about 95% identity to SEQ ID NO: 5. 
     
     
         107 . The pharmaceutical composition of  claim 103 , wherein the membrane independent CD59 protein comprises about 99% identity to SEQ ID NO: 5. 
     
     
         108 . The pharmaceutical composition of  claim 103 , wherein the membrane independent CD59 protein is identical to SEQ ID NO: 5. 
     
     
         109 . The pharmaceutical composition of  claim 103 , wherein the membrane-independent CD59 protein does not further encode a fusion to a peptide or protein domain that binds to a cell membrane or membrane associated entity. 
     
     
         110 . The pharmaceutical composition of  claim 103 , formulated for a route of injection delivery that is at least one selected from the group of intra-ocular, subconjunctival, and subtenon. 
     
     
         111 . The pharmaceutical composition according to  claim 103 , formulated as an eye drop or an ointment. 
     
     
         112 . The pharmaceutical composition of claim  1 , further comprising at least one agent selected from the group consisting of: an anti-tumor, an antiviral, an antibacterial, an anti-mycobacterial, an anti-fungal, an anti-proliferative, and an anti-apoptotic. 
     
     
         113 . A kit for treating a macular degeneration in a subject comprising:
 a pharmaceutical composition comprising a membrane-independent CD59 protein, wherein the composition is sufficiently pure for administration to the subject;   instructions for use; and   a container.   
     
     
         114 . The kit of  claim 113 , wherein the membrane-independent CD59 protein lacks a glycosyl phosphatidyl inositol (GPI) anchoring domain. 
     
     
         115 . The kit of  claim 113 , wherein the membrane independent CD59 protein comprises about 90% identity to SEQ ID NO: 5. 
     
     
         116 . The kit of  claim 113 , wherein the membrane independent CD59 protein comprises about 95% identity to SEQ ID NO: 5. 
     
     
         117 . The kit of  claim 113 , wherein the membrane independent CD59 protein comprises about 99% identity to SEQ ID NO: 5. 
     
     
         118 . The kit of  claim 113 , wherein the membrane independent CD59 protein is identical to SEQ ID NO: 5. 
     
     
         119 . The kit of  claim 113 , wherein the membrane-independent CD59 protein does not further encode a fusion to a peptide or protein domain that binds to a cell membrane or membrane associated entity. 
     
     
         120 . The kit of  claim 113 , wherein the pharmaceutical composition is formulated for a route of injection delivery that is at least one selected from the group of intra-ocular, subconjunctival, and subtenon. 
     
     
         121 . The kit of  claim 113 , wherein the pharmaceutical composition is formulated as an eye drop or an ointment. 
     
     
         122 . The kit of  claim 113 , wherein the kit further comprises at least one agent selected from the group consisting of: an anti-tumor, an antiviral, an antibacterial, an anti-mycobacterial, an anti-fungal, an anti-proliferative, and an anti-apoptotic.