                         SEQUENCE LISTING

<110>  AveXis Inc.
       KASPAR, Brian
       KASPAR, Allan
       FOUST, Kevin
       FUGERE, Martin
       PARK, Eunhye
       THOMSEN, Gretchen
       ZUO, Fengrong
 
<120>  Cell-Based Assay For Measuring Drug Product Potency

<130>  AVEX-004/001WO

<150>  62/682,263
<151>  2018-06-08

<160>  4     

<170>  PatentIn version 3.5

<210>  1
<211>  2359
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  SMN vector sequence

<400>  1
ctgcgcgctc gctcgctcac tgaggccgcc cgggcaaagc ccgggcgtcg ggcgaccttt       60

ggtcgcccgg cctcagtgag cgagcgagcg cgcagagagg gagtggaatt cacgcgtgga      120

tctgaattca attcacgcgt ggtacctctg gtcgttacat aacttacggt aaatggcccg      180

cctggctgac cgcccaacga cccccgccca ttgacgtcaa taatgacgta tgttcccata      240

gtaacgccaa tagggacttt ccattgacgt caatgggtgg agtatttacg gtaaactgcc      300

cacttggcag tacatcaagt gtatcatatg ccaagtacgc cccctattga cgtcaatgac      360

ggtaaatggc ccgcctggca ttatgcccag tacatgacct tatgggactt tcctacttgg      420

cagtacatct actcgaggcc acgttctgct tcactctccc catctccccc ccctccccac      480

ccccaatttt gtatttattt attttttaat tattttgtgc agcgatgggg gcgggggggg      540

ggggggggcg cgcgccaggc ggggcggggc ggggcgaggg gcggggcggg gcgaggcgga      600

gaggtgcggc ggcagccaat cagagcggcg cgctccgaaa gtttcctttt atggcgaggc      660

ggcggcggcg gcggccctat aaaaagcgaa gcgcgcggcg ggcgggagcg ggatcagcca      720

ccgcggtggc ggcctagagt cgacgaggaa ctgaaaaacc agaaagttaa ctggtaagtt      780

tagtcttttt gtcttttatt tcaggtcccg gatccggtgg tggtgcaaat caaagaactg      840

ctcctcagtg gatgttgcct ttacttctag gcctgtacgg aagtgttact tctgctctaa      900

aagctgcgga attgtacccg cggccgatcc accggtccgg aattcccggg atatcgtcga      960

cccacgcgtc cgggccccac gctgcgcacc cgcgggtttg ctatggcgat gagcagcggc     1020

ggcagtggtg gcggcgtccc ggagcaggag gattccgtgc tgttccggcg cggcacaggc     1080

cagagcgatg attctgacat ttgggatgat acagcactga taaaagcata tgataaagct     1140

gtggcttcat ttaagcatgc tctaaagaat ggtgacattt gtgaaacttc gggtaaacca     1200

aaaaccacac ctaaaagaaa acctgctaag aagaataaaa gccaaaagaa gaatactgca     1260

gcttccttac aacagtggaa agttggggac aaatgttctg ccatttggtc agaagacggt     1320

tgcatttacc cagctaccat tgcttcaatt gattttaaga gagaaacctg tgttgtggtt     1380

tacactggat atggaaatag agaggagcaa aatctgtccg atctactttc cccaatctgt     1440

gaagtagcta ataatataga acagaatgct caagagaatg aaaatgaaag ccaagtttca     1500

acagatgaaa gtgagaactc caggtctcct ggaaataaat cagataacat caagcccaaa     1560

tctgctccat ggaactcttt tctccctcca ccacccccca tgccagggcc aagactggga     1620

ccaggaaagc caggtctaaa attcaatggc ccaccaccgc caccgccacc accaccaccc     1680

cacttactat catgctggct gcctccattt ccttctggac caccaataat tcccccacca     1740

cctcccatat gtccagattc tcttgatgat gctgatgctt tgggaagtat gttaatttca     1800

tggtacatga gtggctatca tactggctat tatatgggtt ttagacaaaa tcaaaaagaa     1860

ggaaggtgct cacattcctt aaattaagga gaaatgctgg catagagcag cactaaatga     1920

caccactaaa gaaacgatca gacagatcta gaaagcttat cgataccgtc gactagagct     1980

cgctgatcag cctcgactgt gccttctagt tgccagccat ctgttgtttg cccctccccc     2040

gtgccttcct tgaccctgga aggtgccact cccactgtcc tttcctaata aaatgaggaa     2100

attgcatcgc attgtctgag taggtgtcat tctattctgg ggggtggggt ggggcaggac     2160

agcaaggggg aggattggga agacaatagc aggcatgctg gggagagatc gatctgagga     2220

acccctagtg atggagttgg ccactccctc tctgcgcgct cgctcgctca ctgaggccgg     2280

gcgaccaaag gtcgcccgac gcccgggctt tgcccgggcg gcctcagtga gcgagcgagc     2340

gcgcagagag ggagtggcc                                                  2359


<210>  2
<211>  1621
<212>  DNA
<213>  Homo sapiens

<400>  2
ccacaaatgt gggagggcga taaccactcg tagaaagcgt gagaagttac tacaagcggt       60

cctcccggcc accgtactgt tccgctccca gaagccccgg gcggcggaag tcgtcactct      120

taagaaggga cggggcccca cgctgcgcac ccgcgggttt gctatggcga tgagcagcgg      180

cggcagtggt ggcggcgtcc cggagcagga ggattccgtg ctgttccggc gcggcacagg      240

ccagagcgat gattctgaca tttgggatga tacagcactg ataaaagcat atgataaagc      300

tgtggcttca tttaagcatg ctctaaagaa tggtgacatt tgtgaaactt cgggtaaacc      360

aaaaaccaca cctaaaagaa aacctgctaa gaagaataaa agccaaaaga agaatactgc      420

agcttcctta caacagtgga aagttgggga caaatgttct gccatttggt cagaagacgg      480

ttgcatttac ccagctacca ttgcttcaat tgattttaag agagaaacct gtgttgtggt      540

ttacactgga tatggaaata gagaggagca aaatctgtcc gatctacttt ccccaatctg      600

tgaagtagct aataatatag aacagaatgc tcaagagaat gaaaatgaaa gccaagtttc      660

aacagatgaa agtgagaact ccaggtctcc tggaaataaa tcagataaca tcaagcccaa      720

atctgctcca tggaactctt ttctccctcc accacccccc atgccagggc caagactggg      780

accaggaaag ccaggtctaa aattcaatgg cccaccaccg ccaccgccac caccaccacc      840

ccacttacta tcatgctggc tgcctccatt tccttctgga ccaccaataa ttcccccacc      900

acctcccata tgtccagatt ctcttgatga tgctgatgct ttgggaagta tgttaatttc      960

atggtacatg agtggctatc atactggcta ttatatgggt ttcagacaaa atcaaaaaga     1020

aggaaggtgc tcacattcct taaattaagg agaaatgctg gcatagagca gcactaaatg     1080

acaccactaa agaaacgatc agacagatct ggaatgtgaa gcgttataga agataactgg     1140

cctcatttct tcaaaatatc aagtgttggg aaagaaaaaa ggaagtggaa tgggtaactc     1200

ttcttgatta aaagttatgt aataaccaaa tgcaatgtga aatattttac tggactcttt     1260

tgaaaaacca tctgtaaaag actggggtgg gggtgggagg ccagcacggt ggtgaggcag     1320

ttgagaaaat ttgaatgtgg attagatttt gaatgatatt ggataattat tggtaatttt     1380

atggcctgtg agaagggtgt tgtagtttat aaaagactgt cttaatttgc atacttaagc     1440

atttaggaat gaagtgttag agtgtcttaa aatgtttcaa atggtttaac aaaatgtatg     1500

tgaggcgtat gtggcaaaat gttacagaat ctaactggtg gacatggctg ttcattgtac     1560

tgtttttttc tatcttctat atgtttaaaa gtatataata aaaatattta attttttttt     1620

a                                                                     1621


<210>  3
<211>  294
<212>  PRT
<213>  Homo sapiens

<400>  3

Met Ala Met Ser Ser Gly Gly Ser Gly Gly Gly Val Pro Glu Gln Glu 
1               5                   10                  15      


Asp Ser Val Leu Phe Arg Arg Gly Thr Gly Gln Ser Asp Asp Ser Asp 
            20                  25                  30          


Ile Trp Asp Asp Thr Ala Leu Ile Lys Ala Tyr Asp Lys Ala Val Ala 
        35                  40                  45              


Ser Phe Lys His Ala Leu Lys Asn Gly Asp Ile Cys Glu Thr Ser Gly 
    50                  55                  60                  


Lys Pro Lys Thr Thr Pro Lys Arg Lys Pro Ala Lys Lys Asn Lys Ser 
65                  70                  75                  80  


Gln Lys Lys Asn Thr Ala Ala Ser Leu Gln Gln Trp Lys Val Gly Asp 
                85                  90                  95      


Lys Cys Ser Ala Ile Trp Ser Glu Asp Gly Cys Ile Tyr Pro Ala Thr 
            100                 105                 110         


Ile Ala Ser Ile Asp Phe Lys Arg Glu Thr Cys Val Val Val Tyr Thr 
        115                 120                 125             


Gly Tyr Gly Asn Arg Glu Glu Gln Asn Leu Ser Asp Leu Leu Ser Pro 
    130                 135                 140                 


Ile Cys Glu Val Ala Asn Asn Ile Glu Gln Asn Ala Gln Glu Asn Glu 
145                 150                 155                 160 


Asn Glu Ser Gln Val Ser Thr Asp Glu Ser Glu Asn Ser Arg Ser Pro 
                165                 170                 175     


Gly Asn Lys Ser Asp Asn Ile Lys Pro Lys Ser Ala Pro Trp Asn Ser 
            180                 185                 190         


Phe Leu Pro Pro Pro Pro Pro Met Pro Gly Pro Arg Leu Gly Pro Gly 
        195                 200                 205             


Lys Pro Gly Leu Lys Phe Asn Gly Pro Pro Pro Pro Pro Pro Pro Pro 
    210                 215                 220                 


Pro Pro His Leu Leu Ser Cys Trp Leu Pro Pro Phe Pro Ser Gly Pro 
225                 230                 235                 240 


Pro Ile Ile Pro Pro Pro Pro Pro Ile Cys Pro Asp Ser Leu Asp Asp 
                245                 250                 255     


Ala Asp Ala Leu Gly Ser Met Leu Ile Ser Trp Tyr Met Ser Gly Tyr 
            260                 265                 270         


His Thr Gly Tyr Tyr Met Gly Phe Arg Gln Asn Gln Lys Glu Gly Arg 
        275                 280                 285             


Cys Ser His Ser Leu Asn 
    290                 


<210>  4
<211>  736
<212>  PRT
<213>  Adeno-associated virus

<400>  4

Met Ala Ala Asp Gly Tyr Leu Pro Asp Trp Leu Glu Asp Asn Leu Ser 
1               5                   10                  15      


Glu Gly Ile Arg Glu Trp Trp Ala Leu Lys Pro Gly Ala Pro Gln Pro 
            20                  25                  30          


Lys Ala Asn Gln Gln His Gln Asp Asn Ala Arg Gly Leu Val Leu Pro 
        35                  40                  45              


Gly Tyr Lys Tyr Leu Gly Pro Gly Asn Gly Leu Asp Lys Gly Glu Pro 
    50                  55                  60                  


Val Asn Ala Ala Asp Ala Ala Ala Leu Glu His Asp Lys Ala Tyr Asp 
65                  70                  75                  80  


Gln Gln Leu Lys Ala Gly Asp Asn Pro Tyr Leu Lys Tyr Asn His Ala 
                85                  90                  95      


Asp Ala Glu Phe Gln Glu Arg Leu Lys Glu Asp Thr Ser Phe Gly Gly 
            100                 105                 110         


Asn Leu Gly Arg Ala Val Phe Gln Ala Lys Lys Arg Leu Leu Glu Pro 
        115                 120                 125             


Leu Gly Leu Val Glu Glu Ala Ala Lys Thr Ala Pro Gly Lys Lys Arg 
    130                 135                 140                 


Pro Val Glu Gln Ser Pro Gln Glu Pro Asp Ser Ser Ala Gly Ile Gly 
145                 150                 155                 160 


Lys Ser Gly Ala Gln Pro Ala Lys Lys Arg Leu Asn Phe Gly Gln Thr 
                165                 170                 175     


Gly Asp Thr Glu Ser Val Pro Asp Pro Gln Pro Ile Gly Glu Pro Pro 
            180                 185                 190         


Ala Ala Pro Ser Gly Val Gly Ser Leu Thr Met Ala Ser Gly Gly Gly 
        195                 200                 205             


Ala Pro Val Ala Asp Asn Asn Glu Gly Ala Asp Gly Val Gly Ser Ser 
    210                 215                 220                 


Ser Gly Asn Trp His Cys Asp Ser Gln Trp Leu Gly Asp Arg Val Ile 
225                 230                 235                 240 


Thr Thr Ser Thr Arg Thr Trp Ala Leu Pro Thr Tyr Asn Asn His Leu 
                245                 250                 255     


Tyr Lys Gln Ile Ser Asn Ser Thr Ser Gly Gly Ser Ser Asn Asp Asn 
            260                 265                 270         


Ala Tyr Phe Gly Tyr Ser Thr Pro Trp Gly Tyr Phe Asp Phe Asn Arg 
        275                 280                 285             


Phe His Cys His Phe Ser Pro Arg Asp Trp Gln Arg Leu Ile Asn Asn 
    290                 295                 300                 


Asn Trp Gly Phe Arg Pro Lys Arg Leu Asn Phe Lys Leu Phe Asn Ile 
305                 310                 315                 320 


Gln Val Lys Glu Val Thr Asp Asn Asn Gly Val Lys Thr Ile Ala Asn 
                325                 330                 335     


Asn Leu Thr Ser Thr Val Gln Val Phe Thr Asp Ser Asp Tyr Gln Leu 
            340                 345                 350         


Pro Tyr Val Leu Gly Ser Ala His Glu Gly Cys Leu Pro Pro Phe Pro 
        355                 360                 365             


Ala Asp Val Phe Met Ile Pro Gln Tyr Gly Tyr Leu Thr Leu Asn Asp 
    370                 375                 380                 


Gly Ser Gln Ala Val Gly Arg Ser Ser Phe Tyr Cys Leu Glu Tyr Phe 
385                 390                 395                 400 


Pro Ser Gln Met Leu Arg Thr Gly Asn Asn Phe Gln Phe Ser Tyr Glu 
                405                 410                 415     


Phe Glu Asn Val Pro Phe His Ser Ser Tyr Ala His Ser Gln Ser Leu 
            420                 425                 430         


Asp Arg Leu Met Asn Pro Leu Ile Asp Gln Tyr Leu Tyr Tyr Leu Ser 
        435                 440                 445             


Lys Thr Ile Asn Gly Ser Gly Gln Asn Gln Gln Thr Leu Lys Phe Ser 
    450                 455                 460                 


Val Ala Gly Pro Ser Asn Met Ala Val Gln Gly Arg Asn Tyr Ile Pro 
465                 470                 475                 480 


Gly Pro Ser Tyr Arg Gln Gln Arg Val Ser Thr Thr Val Thr Gln Asn 
                485                 490                 495     


Asn Asn Ser Glu Phe Ala Trp Pro Gly Ala Ser Ser Trp Ala Leu Asn 
            500                 505                 510         


Gly Arg Asn Ser Leu Met Asn Pro Gly Pro Ala Met Ala Ser His Lys 
        515                 520                 525             


Glu Gly Glu Asp Arg Phe Phe Pro Leu Ser Gly Ser Leu Ile Phe Gly 
    530                 535                 540                 


Lys Gln Gly Thr Gly Arg Asp Asn Val Asp Ala Asp Lys Val Met Ile 
545                 550                 555                 560 


Thr Asn Glu Glu Glu Ile Lys Thr Thr Asn Pro Val Ala Thr Glu Ser 
                565                 570                 575     


Tyr Gly Gln Val Ala Thr Asn His Gln Ser Ala Gln Ala Gln Ala Gln 
            580                 585                 590         


Thr Gly Trp Val Gln Asn Gln Gly Ile Leu Pro Gly Met Val Trp Gln 
        595                 600                 605             


Asp Arg Asp Val Tyr Leu Gln Gly Pro Ile Trp Ala Lys Ile Pro His 
    610                 615                 620                 


Thr Asp Gly Asn Phe His Pro Ser Pro Leu Met Gly Gly Phe Gly Met 
625                 630                 635                 640 


Lys His Pro Pro Pro Gln Ile Leu Ile Lys Asn Thr Pro Val Pro Ala 
                645                 650                 655     


Asp Pro Pro Thr Ala Phe Asn Lys Asp Lys Leu Asn Ser Phe Ile Thr 
            660                 665                 670         


Gln Tyr Ser Thr Gly Gln Val Ser Val Glu Ile Glu Trp Glu Leu Gln 
        675                 680                 685             


Lys Glu Asn Ser Lys Arg Trp Asn Pro Glu Ile Gln Tyr Thr Ser Asn 
    690                 695                 700                 


Tyr Tyr Lys Ser Asn Asn Val Glu Phe Ala Val Asn Thr Glu Gly Val 
705                 710                 715                 720 


Tyr Ser Glu Pro Arg Pro Ile Gly Thr Arg Tyr Leu Thr Arg Asn Leu 
                725                 730                 735     


