                         SEQUENCE LISTING

<110>  Gemini Therapeutics Inc.
 
<120>  METHODS FOR TREATING PATIENTS HAVING CFI MUTATIONS WITH 
       RECOMBINANT CFI PROTEINS

<130>  GEM-014WO

<150>  62/925,066
<151>  2019-10-23

<160>  6     

<170>  PatentIn version 3.5

<210>  1
<211>  583
<212>  PRT
<213>  Homo sapiens

<400>  1

Met Lys Leu Leu His Val Phe Leu Leu Phe Leu Cys Phe His Leu Arg 
1               5                   10                  15      


Phe Cys Lys Val Thr Tyr Thr Ser Gln Glu Asp Leu Val Glu Lys Lys 
            20                  25                  30          


Cys Leu Ala Lys Lys Tyr Thr His Leu Ser Cys Asp Lys Val Phe Cys 
        35                  40                  45              


Gln Pro Trp Gln Arg Cys Ile Glu Gly Thr Cys Val Cys Lys Leu Pro 
    50                  55                  60                  


Tyr Gln Cys Pro Lys Asn Gly Thr Ala Val Cys Ala Thr Asn Arg Arg 
65                  70                  75                  80  


Ser Phe Pro Thr Tyr Cys Gln Gln Lys Ser Leu Glu Cys Leu His Pro 
                85                  90                  95      


Gly Thr Lys Phe Leu Asn Asn Gly Thr Cys Thr Ala Glu Gly Lys Phe 
            100                 105                 110         


Ser Val Ser Leu Lys His Gly Asn Thr Asp Ser Glu Gly Ile Val Glu 
        115                 120                 125             


Val Lys Leu Val Asp Gln Asp Lys Thr Met Phe Ile Cys Lys Ser Ser 
    130                 135                 140                 


Trp Ser Met Arg Glu Ala Asn Val Ala Cys Leu Asp Leu Gly Phe Gln 
145                 150                 155                 160 


Gln Gly Ala Asp Thr Gln Arg Arg Phe Lys Leu Ser Asp Leu Ser Ile 
                165                 170                 175     


Asn Ser Thr Glu Cys Leu His Val His Cys Arg Gly Leu Glu Thr Ser 
            180                 185                 190         


Leu Ala Glu Cys Thr Phe Thr Lys Arg Arg Thr Met Gly Tyr Gln Asp 
        195                 200                 205             


Phe Ala Asp Val Val Cys Tyr Thr Gln Lys Ala Asp Ser Pro Met Asp 
    210                 215                 220                 


Asp Phe Phe Gln Cys Val Asn Gly Lys Tyr Ile Ser Gln Met Lys Ala 
225                 230                 235                 240 


Cys Asp Gly Ile Asn Asp Cys Gly Asp Gln Ser Asp Glu Leu Cys Cys 
                245                 250                 255     


Lys Ala Cys Gln Gly Lys Gly Phe His Cys Lys Ser Gly Val Cys Ile 
            260                 265                 270         


Pro Ser Gln Tyr Gln Cys Asn Gly Glu Val Asp Cys Ile Thr Gly Glu 
        275                 280                 285             


Asp Glu Val Gly Cys Ala Gly Phe Ala Ser Val Thr Gln Glu Glu Thr 
    290                 295                 300                 


Glu Ile Leu Thr Ala Asp Met Asp Ala Glu Arg Arg Arg Ile Lys Ser 
305                 310                 315                 320 


Leu Leu Pro Lys Leu Ser Cys Gly Val Lys Asn Arg Met His Ile Arg 
                325                 330                 335     


Arg Lys Arg Ile Val Gly Gly Lys Arg Ala Gln Leu Gly Asp Leu Pro 
            340                 345                 350         


Trp Gln Val Ala Ile Lys Asp Ala Ser Gly Ile Thr Cys Gly Gly Ile 
        355                 360                 365             


Tyr Ile Gly Gly Cys Trp Ile Leu Thr Ala Ala His Cys Leu Arg Ala 
    370                 375                 380                 


Ser Lys Thr His Arg Tyr Gln Ile Trp Thr Thr Val Val Asp Trp Ile 
385                 390                 395                 400 


His Pro Asp Leu Lys Arg Ile Val Ile Glu Tyr Val Asp Arg Ile Ile 
                405                 410                 415     


Phe His Glu Asn Tyr Asn Ala Gly Thr Tyr Gln Asn Asp Ile Ala Leu 
            420                 425                 430         


Ile Glu Met Lys Lys Asp Gly Asn Lys Lys Asp Cys Glu Leu Pro Arg 
        435                 440                 445             


Ser Ile Pro Ala Cys Val Pro Trp Ser Pro Tyr Leu Phe Gln Pro Asn 
    450                 455                 460                 


Asp Thr Cys Ile Val Ser Gly Trp Gly Arg Glu Lys Asp Asn Glu Arg 
465                 470                 475                 480 


Val Phe Ser Leu Gln Trp Gly Glu Val Lys Leu Ile Ser Asn Cys Ser 
                485                 490                 495     


Lys Phe Tyr Gly Asn Arg Phe Tyr Glu Lys Glu Met Glu Cys Ala Gly 
            500                 505                 510         


Thr Tyr Asp Gly Ser Ile Asp Ala Cys Lys Gly Asp Ser Gly Gly Pro 
        515                 520                 525             


Leu Val Cys Met Asp Ala Asn Asn Val Thr Tyr Val Trp Gly Val Val 
    530                 535                 540                 


Ser Trp Gly Glu Asn Cys Gly Lys Pro Glu Phe Pro Gly Val Tyr Thr 
545                 550                 555                 560 


Lys Val Ala Asn Tyr Phe Asp Trp Ile Ser Tyr His Val Gly Arg Pro 
                565                 570                 575     


Phe Ile Ser Gln Tyr Asn Val 
            580             


<210>  2
<211>  583
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  SEQ ID NO: 2 Exemplary CFI Amino Acid Sequence

<400>  2

Met Lys Leu Leu His Val Phe Leu Leu Phe Leu Cys Phe His Leu Arg 
1               5                   10                  15      


Phe Cys Lys Val Thr Tyr Thr Ser Gln Glu Asp Leu Val Glu Lys Lys 
            20                  25                  30          


Cys Leu Ala Lys Lys Tyr Thr His Leu Ser Cys Asp Lys Val Phe Cys 
        35                  40                  45              


Gln Pro Trp Gln Arg Cys Ile Glu Gly Thr Cys Val Cys Lys Leu Pro 
    50                  55                  60                  


Tyr Gln Cys Pro Lys Asn Gly Thr Ala Val Cys Ala Thr Asn Arg Arg 
65                  70                  75                  80  


Ser Phe Pro Thr Tyr Cys Gln Gln Lys Ser Leu Glu Cys Leu His Pro 
                85                  90                  95      


Gly Thr Lys Phe Leu Asn Asn Gly Thr Cys Thr Ala Glu Gly Lys Phe 
            100                 105                 110         


Ser Val Ser Leu Lys His Gly Asn Thr Asp Ser Glu Gly Ile Val Glu 
        115                 120                 125             


Val Lys Leu Val Asp Gln Asp Lys Thr Met Phe Ile Cys Lys Ser Ser 
    130                 135                 140                 


Trp Ser Met Arg Glu Ala Asn Val Ala Cys Leu Asp Leu Gly Phe Gln 
145                 150                 155                 160 


Gln Gly Ala Asp Thr Gln Arg Arg Phe Lys Leu Ser Asp Leu Ser Ile 
                165                 170                 175     


Asn Ser Thr Glu Cys Leu His Val His Cys Arg Gly Leu Glu Thr Ser 
            180                 185                 190         


Leu Ala Glu Cys Thr Phe Thr Lys Arg Arg Thr Met Gly Tyr Gln Asp 
        195                 200                 205             


Phe Ala Asp Val Val Cys Tyr Thr Gln Lys Ala Asp Ser Pro Met Asp 
    210                 215                 220                 


Asp Phe Phe Gln Cys Val Asn Gly Lys Tyr Ile Ser Gln Met Lys Ala 
225                 230                 235                 240 


Cys Asp Gly Ile Asn Asp Cys Gly Asp Gln Ser Asp Glu Leu Cys Cys 
                245                 250                 255     


Lys Ala Cys Gln Gly Lys Gly Phe His Cys Lys Ser Gly Val Cys Ile 
            260                 265                 270         


Pro Ser Gln Tyr Gln Cys Asn Gly Glu Val Asp Cys Ile Thr Gly Glu 
        275                 280                 285             


Asp Glu Val Gly Cys Ala Gly Phe Ala Ser Val Ala Gln Glu Glu Thr 
    290                 295                 300                 


Glu Ile Leu Thr Ala Asp Met Asp Ala Glu Arg Arg Arg Ile Lys Ser 
305                 310                 315                 320 


Leu Leu Pro Lys Leu Ser Cys Gly Val Lys Asn Arg Met His Ile Arg 
                325                 330                 335     


Arg Lys Arg Ile Val Gly Gly Lys Arg Ala Gln Leu Gly Asp Leu Pro 
            340                 345                 350         


Trp Gln Val Ala Ile Lys Asp Ala Ser Gly Ile Thr Cys Gly Gly Ile 
        355                 360                 365             


Tyr Ile Gly Gly Cys Trp Ile Leu Thr Ala Ala His Cys Leu Arg Ala 
    370                 375                 380                 


Ser Lys Thr His Arg Tyr Gln Ile Trp Thr Thr Val Val Asp Trp Ile 
385                 390                 395                 400 


His Pro Asp Leu Lys Arg Ile Val Ile Glu Tyr Val Asp Arg Ile Ile 
                405                 410                 415     


Phe His Glu Asn Tyr Asn Ala Gly Thr Tyr Gln Asn Asp Ile Ala Leu 
            420                 425                 430         


Ile Glu Met Lys Lys Asp Gly Asn Lys Lys Asp Cys Glu Leu Pro Arg 
        435                 440                 445             


Ser Ile Pro Ala Cys Val Pro Trp Ser Pro Tyr Leu Phe Gln Pro Asn 
    450                 455                 460                 


Asp Thr Cys Ile Val Ser Gly Trp Gly Arg Glu Lys Asp Asn Glu Arg 
465                 470                 475                 480 


Val Phe Ser Leu Gln Trp Gly Glu Val Lys Leu Ile Ser Asn Cys Ser 
                485                 490                 495     


Lys Phe Tyr Gly Asn Arg Phe Tyr Glu Lys Glu Met Glu Cys Ala Gly 
            500                 505                 510         


Thr Tyr Asp Gly Ser Ile Asp Ala Cys Lys Gly Asp Ser Gly Gly Pro 
        515                 520                 525             


Leu Val Cys Met Asp Ala Asn Asn Val Thr Tyr Val Trp Gly Val Val 
    530                 535                 540                 


Ser Trp Gly Glu Asn Cys Gly Lys Pro Glu Phe Pro Gly Val Tyr Thr 
545                 550                 555                 560 


Lys Val Ala Asn Tyr Phe Asp Trp Ile Ser Tyr His Val Gly Arg Pro 
                565                 570                 575     


Phe Ile Ser Gln Tyr Asn Val 
            580             


<210>  3
<211>  1807
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  SEQ ID NO: 3- Exemplary Codon Optimized Human Complement Factor I

<400>  3
gcggccgcca ccatgaaact gctgcatgtc tttctgctgt ttctgtgctt ccatctgcgc       60

ttctgcaagg tcacttacac ttctcaggag gatctggtcg agaagaagtg tctggccaag      120

aagtacacac acctgagctg cgacaaggtg ttctgtcagc cttggcagag atgcatcgag      180

ggcacctgcg tgtgcaagct gccttaccag tgcccaaaga acggaaccgc cgtgtgcgca      240

acaaatcggc ggagctttcc aacatattgc cagcagaaga gcctggagtg tctgcacccc      300

ggcaccaagt tcctgaacaa tggcacctgc acagccgagg gcaagttttc tgtgagcctg      360

aagcacggca acacagatag cgagggcatc gtggaggtga agctggtgga ccaggataag      420

accatgttca tctgtaagag ctcctggtcc atgagggagg caaacgtggc atgcctggat      480

ctgggattcc agcagggagc agacacacag aggcgcttta agctgtccga cctgtctatc      540

aatagcaccg agtgcctgca cgtgcactgt aggggcctgg agacatccct ggcagagtgc      600

accttcacaa agcggagaac catgggctac caggactttg ccgacgtggt gtgctatacc      660

cagaaggccg atagcccaat ggacgatttc tttcagtgcg tgaacggcaa gtatatctcc      720

cagatgaagg cctgcgacgg catcaatgac tgtggcgatc agtctgacga gctgtgctgt      780

aaggcctgtc agggcaaggg cttccactgc aagagcggcg tgtgcatccc ttcccagtac      840

cagtgcaacg gcgaggtgga ttgtatcaca ggagaggacg aagtgggatg cgctgccgcc      900

agacacccaa ccatccaggg ctttgcctct gtggcccagg aggagacaga gatcctgaca      960

gccgacatgg atgccgagag gcgccggatc aagtctctgc tgcccaagct gagctgcggc     1020

gtgaagaata ggatgcacat cagaaggaag cgcatcgtgg gaggcaagag ggcacagctg     1080

ggcgatctgc cttggcaggt ggccatcaag gacgcctctg gcatcacctg cggcggcatc     1140

tacatcggag gatgttggat cctgaccgca gcacactgcc tgagagcaag caagacacac     1200

aggtatcaga tttggaccac agtggtggat tggatccacc cagacctgaa gagaatcgtg     1260

atcgagtacg tggataggat catcttccac gagaactaca atgccggcac atatcagaac     1320

gacatcgccc tgatcgagat gaagaaggat ggcaataaga aggactgtga gctgccacgc     1380

tccatccctg catgcgtgcc ctggagcccc tatctgttcc agcccaacga tacctgtatc     1440

gtgtccggct ggggccgcga gaaggacaat gagcgggtgt tttctctgca gtggggcgag     1500

gtgaagctga tctccaactg ttctaagttc tacggcaatc ggttttatga gaaggagatg     1560

gagtgcgccg gcacctacga tggcagcatc gacgcctgta agggcgattc cggaggacca     1620

ctggtgtgca tggacgcaaa caatgtgaca tacgtgtggg gcgtggtgtc ctggggcgag     1680

aattgcggca agccagagtt tcccggcgtg tataccaagg tggccaacta ttttgattgg     1740

atttcctacc atgtcgggag accattcatt tcacagtata acgtgtaata agcttggatc     1800

cagatct                                                               1807


<210>  4
<211>  1807
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  SEQ ID NO: 4- Exemplary Non-Codon Optimized Human Complement 
       Factor I

<400>  4
gcggccgcca ccatgaagct tcttcatgtt ttcctgttat ttctgtgctt ccacttaagg       60

ttttgcaagg tcacttatac atctcaagag gatctggtgg agaaaaagtg cttagcaaaa      120

aaatatactc acctctcctg cgataaagtc ttctgccagc catggcagag atgcattgag      180

ggcacctgtg tttgtaaact accgtatcag tgcccaaaga atggcactgc agtgtgtgca      240

actaacagga gaagcttccc aacatactgt caacaaaaga gtttggaatg tcttcatcca      300

gggacaaagt ttttaaataa cggaacatgc acagccgaag gaaagtttag tgtttccttg      360

aagcatggaa atacagattc agagggaata gttgaagtaa aacttgtgga ccaagataag      420

acaatgttca tatgcaaaag cagctggagc atgagggaag ccaacgtggc ctgccttgac      480

cttgggtttc aacaaggtgc tgatactcaa agaaggttta agttgtctga tctctctata      540

aattccactg aatgtctaca tgtgcattgc cgaggattag agaccagttt ggctgaatgt      600

acttttacta agagaagaac tatgggttac caggatttcg ctgatgtggt ttgttataca      660

cagaaagcag attctccaat ggatgacttc tttcagtgtg tgaatgggaa atacatttct      720

cagatgaaag cctgtgatgg tatcaatgat tgtggagacc aaagtgatga actgtgttgt      780

aaagcatgcc aaggcaaagg cttccattgc aaatcgggtg tttgcattcc aagccagtat      840

caatgcaatg gtgaggtgga ctgcattaca ggggaagatg aagttggctg tgcagcagct      900

agacatccta caattcaagg ctttgcatct gtggctcaag aagaaacaga aattttgact      960

gctgacatgg atgcagaaag aagacggata aaatcattat tacctaaact atcttgtgga     1020

gttaaaaaca gaatgcacat tcgaaggaaa cgaattgtgg gaggaaagcg agcacaactg     1080

ggagacctcc catggcaggt ggcaattaag gatgccagtg gaatcacctg tgggggaatt     1140

tatattggtg gctgttggat tctgactgct gcacattgtc tcagagccag taaaactcat     1200

cgttaccaaa tatggacaac agtagtagac tggatacacc ccgaccttaa acgtatagta     1260

attgaatacg tggatagaat tattttccat gaaaactaca atgcaggcac ttaccaaaat     1320

gacatcgctt tgattgaaat gaaaaaagac ggaaacaaaa aagattgtga gctgcctcgt     1380

tccatccctg cctgtgtccc ctggtctcct tacctattcc aacctaatga tacatgcatc     1440

gtttctggct ggggacgaga aaaagataac gaaagagtct tttcacttca gtggggtgaa     1500

gttaaactaa taagcaactg ctctaagttt tacggaaatc gtttctatga aaaagaaatg     1560

gaatgtgcag gtacatatga tggttccatc gatgcctgta aaggggactc tggaggcccc     1620

ttagtctgta tggatgccaa caatgtgact tatgtctggg gtgttgtgag ttggggggaa     1680

aactgtggaa aaccagagtt cccaggtgtt tacaccaaag tggccaatta ttttgactgg     1740

attagctacc atgtaggaag gccttttatt tctcagtaca atgtataata agcttggatc     1800

cagatct                                                               1807


<210>  5
<211>  1752
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  SEQ ID NO: 5: Exemplary CFI Nucleotide Sequence

<400>  5
atgaagcttc ttcatgtttt cctgttattt ctgtgcttcc acttaaggtt ttgcaaggtc       60

acttatacat ctcaagagga tctggtggag aaaaagtgct tagcaaaaaa atatactcac      120

ctctcctgcg ataaagtctt ctgccagcca tggcagagat gcattgaggg cacctgtgtt      180

tgtaaactac cgtatcagtg cccaaagaat ggcactgcag tgtgtgcaac taacaggaga      240

agcttcccaa catactgtca acaaaagagt ttggaatgtc ttcatccagg gacaaagttt      300

ttaaataacg gaacatgcac agccgaagga aagtttagtg tttccttgaa gcatggaaat      360

acagattcag agggaatagt tgaagtaaaa cttgtggacc aagataagac aatgttcata      420

tgcaaaagca gctggagcat gagggaagcc aacgtggcct gccttgacct tgggtttcaa      480

caaggtgctg atactcaaag aaggtttaag ttgtctgatc tctctataaa ttccactgaa      540

tgtctacatg tgcattgccg aggattagag accagtttgg ctgaatgtac ttttactaag      600

agaagaacta tgggttacca ggatttcgct gatgtggttt gttatacaca gaaagcagat      660

tctccaatgg atgacttctt tcagtgtgtg aatgggaaat acatttctca gatgaaagcc      720

tgtgatggta tcaatgattg tggagaccaa agtgatgaac tgtgttgtaa agcatgccaa      780

ggcaaaggct tccattgcaa atcgggtgtt tgcattccaa gccagtatca atgcaatggt      840

gaggtggact gcattacagg ggaagatgaa gttggctgtg caggctttgc atctgtgact      900

caagaagaaa cagaaatttt gactgctgac atggatgcag aaagaagacg gataaaatca      960

ttattaccta aactatcttg tggagttaaa aacagaatgc acattcgaag gaaacgaatt     1020

gtgggaggaa agcgagcaca actgggagac ctcccatggc aggtggcaat taaggatgcc     1080

agtggaatca cctgtggggg aatttatatt ggtggctgtt ggattctgac tgctgcacat     1140

tgtctcagag ccagtaaaac tcatcgttac caaatatgga caacagtagt agactggata     1200

caccccgacc ttaaacgtat agtaattgaa tacgtggata gaattatttt ccatgaaaac     1260

tacaatgcag gcacttacca aaatgacatc gctttgattg aaatgaaaaa agacggaaac     1320

aaaaaagatt gtgagctgcc tcgttccatc cctgcctgtg tcccctggtc tccttaccta     1380

ttccaaccta atgatacatg catcgtttct ggctggggac gagaaaaaga taacgaaaga     1440

gtcttttcac ttcagtgggg tgaagttaaa ctaataagca actgctctaa gttttacgga     1500

aatcgtttct atgaaaaaga aatggaatgt gcaggtacat atgatggttc catcgatgcc     1560

tgtaaagggg actctggagg ccccttagtc tgtatggatg ccaacaatgt gacttatgtc     1620

tggggtgttg tgagttgggg ggaaaactgt ggaaaaccag agttcccagg tgtttacacc     1680

aaagtggcca attattttga ctggattagc taccatgtag gaaggccttt tatttctcag     1740

tacaatgtat aa                                                         1752


<210>  6
<211>  1752
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  SEQ ID NO: 6- Exemplary CFI Nucleotide Sequence

<400>  6
atgaagcttc ttcatgtttt cctgttattt ctgtgcttcc acttaaggtt ttgcaaggtc       60

acttatacat ctcaagagga tctggtggag aaaaagtgct tagcaaaaaa atatactcac      120

ctctcctgcg ataaagtctt ctgccagcca tggcagagat gcattgaggg cacctgtgtt      180

tgtaaactac cgtatcagtg cccaaagaat ggcactgcag tgtgtgcaac taacaggaga      240

agcttcccaa catactgtca acaaaagagt ttggaatgtc ttcatccagg gacaaagttt      300

ttaaataacg gaacatgcac agccgaagga aagtttagtg tttccttgaa gcatggaaat      360

acagattcag agggaatagt tgaagtaaaa cttgtggacc aagataagac aatgttcata      420

tgcaaaagca gctggagcat gagggaagcc aacgtggcct gccttgacct tgggtttcaa      480

caaggtgctg atactcaaag aaggtttaag ttgtctgatc tctctataaa ttccactgaa      540

tgtctacatg tgcattgccg aggattagag accagtttgg ctgaatgtac ttttactaag      600

agaagaacta tgggttacca ggatttcgct gatgtggttt gttatacaca gaaagcagat      660

tctccaatgg atgacttctt tcagtgtgtg aatgggaaat acatttctca gatgaaagcc      720

tgtgatggta tcaatgattg tggagaccaa agtgatgaac tgtgttgtaa agcatgccaa      780

ggcaaaggct tccattgcaa atcgggtgtt tgcattccaa gccagtatca atgcaatggt      840

gaggtggact gcattacagg ggaagatgaa gttggctgtg caggctttgc atctgtggct      900

caagaagaaa cagaaatttt gactgctgac atggatgcag aaagaagacg gataaaatca      960

ttattaccta aactatcttg tggagttaaa aacagaatgc acattcgaag gaaacgaatt     1020

gtgggaggaa agcgagcaca actgggagac ctcccatggc aggtggcaat taaggatgcc     1080

agtggaatca cctgtggggg aatttatatt ggtggctgtt ggattctgac tgctgcacat     1140

tgtctcagag ccagtaaaac tcatcgttac caaatatgga caacagtagt agactggata     1200

caccccgacc ttaaacgtat agtaattgaa tacgtggata gaattatttt ccatgaaaac     1260

tacaatgcag gcacttacca aaatgacatc gctttgattg aaatgaaaaa agacggaaac     1320

aaaaaagatt gtgagctgcc tcgttccatc cctgcctgtg tcccctggtc tccttaccta     1380

ttccaaccta atgatacatg catcgtttct ggctggggac gagaaaaaga taacgaaaga     1440

gtcttttcac ttcagtgggg tgaagttaaa ctaataagca actgctctaa gttttacgga     1500

aatcgtttct atgaaaaaga aatggaatgt gcaggtacat atgatggttc catcgatgcc     1560

tgtaaagggg actctggagg ccccttagtc tgtatggatg ccaacaatgt gacttatgtc     1620

tggggtgttg tgagttgggg ggaaaactgt ggaaaaccag agttcccagg tgtttacacc     1680

aaagtggcca attattttga ctggattagc taccatgtag gaaggccttt tatttctcag     1740

tacaatgtat aa                                                         1752


