0288-0066WO1 US 63/455,867 2023-03-29 PHARMA CINQ, LLC LUO, Tianci VECTOR ENCODING ROD-DERIVED CONE VIABILITY FACTOR AND HUMAN IgK SIGNAL SEQUENCE 15 5349 DNA PAT misc_feature 1..5349 note Synthetic: pAAV.hIgK.hRdCVF1L Shuttle vector sequence for mammalian expression cassette of human RdCVF1L (-Met) and human IgK signal sequence (+Asp) into rAAV source 1..5349 mol_type other DNA organism synthetic construct cctgcaggcagctgcgcgctcgctcgctcactgaggccgcccgggcaaagcccgggcgtcgggcgacctttggtcgcccggcctcagtgagcgagcgagcgcgcagagagggagtggccaactccatcactaggggttcctgcggccgcacgcgtggagctagttattaatagtaatcaattacggggtcattagttcatagcccatatatggagttccgcgttacataacttacggtaaatggcccgcctggctgaccgcccaacgacccccgcccattgacgtcaataatgacgtatgttcccatagtaacgtcaatagggactttccattgacgtcaatgggtggagtatttacggtaaactgcccacttggcagtacatcaagtgtatcatatgccaagtacgccccctattgacgtcaatgacggtaaatggcccgcctggcattatgcccagtacatgaccttatgggactttcctacttggcagtacatctacgtattagtcatcgctattaccatggtgatgcggttttggcagtacatcaatgggcgtggatagcggtttgactcacggggatttccaagtctccaccccattgacgtcaatgggagtttgttttgcaccaaaatcaacgggactttccaaaatgtcgtaacaactccgccccattgacgcaaatgggcggtaggcgtgtacggtgggaggtctatataagcagagctcgtttagtgaaccgtcagatcgcctggagacgccatccacgctgttttgacctccatagaagacaccgggaccgatccagcctccgcggattcgaatcccggccgggaacggtgcattggaacgcggattccccgtgccaagagtgacgtaagtaccgcctatagagtctataggcccacaaaaaatgctttcttcttttaatatacttttttgtttatcttatttctaatactttccctaatctctttctttcagggcaataatgatacaatgtatcatgcctctttgcaccattctaaagaataacagtgataatttctgggttaaggcaatagcaatatttctgcatataaatatttctgcatataaattgtaactgatgtaagaggtttcatattgctaatagcagctacaatccagctaccattctgcttttattttatggttgggataaggctggattattctgagtccaagctaggcccttttgctaatcatgttcatacctcttatcttcctcccacagctcctgggcaacgtgctggtctgtgtgctggcccatcactttggcaaagaattgggattcgaacatcgattgaattcgagccaccatggacatgagggtccctgctcagctcctggggctcctgctgctctggctctcaggtgccagatgtgatgccagcctgttcagcggccggatcctgatcaggaacaacagcgaccaggacgagctggacaccgaggccgaagtgagcaggaggctggagaacagactggtgctgctgttctttggcgccggagcctgccctcagtgccaggccttcgtgcccatcctgaaggatttctttgtgaggctgaccgacgagttctacgtgctgagagccgcccagctggccctggtgtatgtgagccaggacagcaccgaggagcagcaggacctgttcctgaaggacatgcccaagaagtggctgttcctgcccttcgaggacgacctgagaagagacctgggcaggcagttcagcgtggagagactgcccgccgtggtggtgctgaagcctgatggcgacgtgctgaccagagatggcgccgacgagatccagagactgggcaccgcctgcttcgccaactggcaggaggccgccgaggtcctggacagaaacttccagctgcccgaggatctggaggatcaggagcccagatccctgaccgagtgcctgaggcggcacaagtacagagtggagaaggccgccagaggcggcagagaccctggcggcggaggaggagaggagggcggagccggcggactgttctgatgagctagcaccggttgtacaagtcaagcggccaaccctccctagatctacgggtggcatccctgtgacccctccccagtgcctctcctggccctggaagttgccactccagtgcccaccagccttgtcctaataaaattaagttgcatcattttgtctgactaggtgtccttctataatattatggggtggaggggggtggtatggagcaaggggcaagttgggaagacaacctgtagggcctgcggggtctattgggaaccaagctggagtgcagtggcacaatcttggctcactgcaatctccgcctcctgggttcaagcgattctcctgcctcagcctcccgagttgttgggattccaggcatgcatgaccaggctcagctaatttttgtttttttggtagagacggggtttcaccatattggccaggctggtctccaactcctaatctcaggtgatctacccaccttggcctcccaaattgctgggattacaggcgtgaaccactgctcccttccctgtccttctgattttgtaggtaaccacgtgcggaccgagcggccgcaggaacccctagtgatggagttggccactccctctctgcgcgctcgctcgctcactgaggccgggcgaccaaaggtcgcccgacgcccgggctttgcccgggcggcctcagtgagcgagcgagcgcgcagctgcctgcaggggcgcctgatgcggtattttctccttacgcatctgtgcggtatttcacaccgcatacgtcaaagcaaccatagtacgcgccctgtagcggcgcattaagcgcggcgggtgtggtggttacgcgcagcgtgaccgctacacttgccagcgccctagcgcccgctcctttcgctttcttcccttcctttctcgccacgttcgccggctttccccgtcaagctctaaatcgggggctccctttagggttccgatttagtgctttacggcacctcgaccccaaaaaacttgatttgggtgatggttcacgtagtgggccatcgccctgatagacggtttttcgccctttgacgttggagtccacgttctttaatagtggactcttgttccaaactggaacaacactcaaccctatctcgggctattcttttgatttataagggattttgccgatttcggcctattggttaaaaaatgagctgatttaacaaaaatttaacgcgaattttaacaaaatattaacgtttacaattttatggtgcactctcagtacaatctgctctgatgccgcatagttaagccagccccgacacccgccaacacccgctgacgcgccctgacgggcttgtctgctcccggcatccgcttacagacaagctgtgaccgtctccgggagctgcatgtgtcagaggttttcaccgtcatcaccgaaacgcgcgagacgaaagggcctcgtgatacgcctatttttataggttaatgtcatgataataatggtttcttagacgtcaggtggcacttttcggggaaatgtgcgcggaacccctatttgtttatttttctaaatacattcaaatatgtatccgctcatgagacaataaccctgataaatgcttcaataatattgaaaaaggaagagtatgagtattcaacatttccgtgtcgcccttattcccttttttgcggcattttgccttcctgtttttgctcacccagaaacgctggtgaaagtaaaagatgctgaagatcagttgggtgcacgagtgggttacatcgaactggatctcaacagcggtaagatccttgagagttttcgccccgaagaacgttttccaatgatgagcacttttaaagttctgctatgtggcgcggtattatcccgtattgacgccgggcaagagcaactcggtcgccgcatacactattctcagaatgacttggttgagtactcaccagtcacagaaaagcatcttacggatggcatgacagtaagagaattatgcagtgctgccataaccatgagtgataacactgcggccaacttacttctgacaacgatcggaggaccgaaggagctaaccgcttttttgcacaacatgggggatcatgtaactcgccttgatcgttgggaaccggagctgaatgaagccataccaaacgacgagcgtgacaccacgatgcctgtagcaatggcaacaacgttgcgcaaactattaactggcgaactacttactctagcttcccggcaacaattaatagactggatggaggcggataaagttgcaggaccacttctgcgctcggcccttccggctggctggtttattgctgataaatctggagccggtgagcgtgggtctcgcggtatcattgcagcactggggccagatggtaagccctcccgtatcgtagttatctacacgacggggagtcaggcaactatggatgaacgaaatagacagatcgctgagataggtgcctcactgattaagcattggtaactgtcagaccaagtttactcatatatactttagattgatttaaaacttcatttttaatttaaaaggatctaggtgaagatcctttttgataatctcatgaccaaaatcccttaacgtgagttttcgttccactgagcgtcagaccccgtagaaaagatcaaaggatcttcttgagatcctttttttctgcgcgtaatctgctgcttgcaaacaaaaaaaccaccgctaccagcggtggtttgtttgccggatcaagagctaccaactctttttccgaaggtaactggcttcagcagagcgcagataccaaatactgtccttctagtgtagccgtagttaggccaccacttcaagaactctgtagcaccgcctacatacctcgctctgctaatcctgttaccagtggctgctgccagtggcgataagtcgtgtcttaccgggttggactcaagacgatagttaccggataaggcgcagcggtcgggctgaacggggggttcgtgcacacagcccagcttggagcgaacgacctacaccgaactgagatacctacagcgtgagctatgagaaagcgccacgcttcccgaagggagaaaggcggacaggtatccggtaagcggcagggtcggaacaggagagcgcacgagggagcttccagggggaaacgcctggtatctttatagtcctgtcgggtttcgccacctctgacttgagcgtcgatttttgtgatgctcgtcaggggggcggagcctatggaaaaacgccagcaacgcggcctttttacggttcctggccttttgctggccttttgctcacatgt 2752 DNA PAT misc_feature 1..2752 note Synthetic: rAAV2.hIgK.hRdCVF1L vector genomic DNA source 1..2752 mol_type other DNA organism synthetic construct cctgcaggcagctgcgcgctcgctcgctcactgaggccgcccgggcaaagcccgggcgtcgggcgacctttggtcgcccggcctcagtgagcgagcgagcgcgcagagagggagtggccaactccatcactaggggttcctgcggccgcacgcgtggagctagttattaatagtaatcaattacggggtcattagttcatagcccatatatggagttccgcgttacataacttacggtaaatggcccgcctggctgaccgcccaacgacccccgcccattgacgtcaataatgacgtatgttcccatagtaacgtcaatagggactttccattgacgtcaatgggtggagtatttacggtaaactgcccacttggcagtacatcaagtgtatcatatgccaagtacgccccctattgacgtcaatgacggtaaatggcccgcctggcattatgcccagtacatgaccttatgggactttcctacttggcagtacatctacgtattagtcatcgctattaccatggtgatgcggttttggcagtacatcaatgggcgtggatagcggtttgactcacggggatttccaagtctccaccccattgacgtcaatgggagtttgttttgcaccaaaatcaacgggactttccaaaatgtcgtaacaactccgccccattgacgcaaatgggcggtaggcgtgtacggtgggaggtctatataagcagagctcgtttagtgaaccgtcagatcgcctggagacgccatccacgctgttttgacctccatagaagacaccgggaccgatccagcctccgcggattcgaatcccggccgggaacggtgcattggaacgcggattccccgtgccaagagtgacgtaagtaccgcctatagagtctataggcccacaaaaaatgctttcttcttttaatatacttttttgtttatcttatttctaatactttccctaatctctttctttcagggcaataatgatacaatgtatcatgcctctttgcaccattctaaagaataacagtgataatttctgggttaaggcaatagcaatatttctgcatataaatatttctgcatataaattgtaactgatgtaagaggtttcatattgctaatagcagctacaatccagctaccattctgcttttattttatggttgggataaggctggattattctgagtccaagctaggcccttttgctaatcatgttcatacctcttatcttcctcccacagctcctgggcaacgtgctggtctgtgtgctggcccatcactttggcaaagaattgggattcgaacatcgattgaattcgagccaccatggacatgagggtccctgctcagctcctggggctcctgctgctctggctctcaggtgccagatgtgatgccagcctgttcagcggccggatcctgatcaggaacaacagcgaccaggacgagctggacaccgaggccgaagtgagcaggaggctggagaacagactggtgctgctgttctttggcgccggagcctgccctcagtgccaggccttcgtgcccatcctgaaggatttctttgtgaggctgaccgacgagttctacgtgctgagagccgcccagctggccctggtgtatgtgagccaggacagcaccgaggagcagcaggacctgttcctgaaggacatgcccaagaagtggctgttcctgcccttcgaggacgacctgagaagagacctgggcaggcagttcagcgtggagagactgcccgccgtggtggtgctgaagcctgatggcgacgtgctgaccagagatggcgccgacgagatccagagactgggcaccgcctgcttcgccaactggcaggaggccgccgaggtcctggacagaaacttccagctgcccgaggatctggaggatcaggagcccagatccctgaccgagtgcctgaggcggcacaagtacagagtggagaaggccgccagaggcggcagagaccctggcggcggaggaggagaggagggcggagccggcggactgttctgatgagctagcaccggttgtacaagtcaagcggccaaccctccctagatctacgggtggcatccctgtgacccctccccagtgcctctcctggccctggaagttgccactccagtgcccaccagccttgtcctaataaaattaagttgcatcattttgtctgactaggtgtccttctataatattatggggtggaggggggtggtatggagcaaggggcaagttgggaagacaacctgtagggcctgcggggtctattgggaaccaagctggagtgcagtggcacaatcttggctcactgcaatctccgcctcctgggttcaagcgattctcctgcctcagcctcccgagttgttgggattccaggcatgcatgaccaggctcagctaatttttgtttttttggtagagacggggtttcaccatattggccaggctggtctccaactcctaatctcaggtgatctacccaccttggcctcccaaattgctgggattacaggcgtgaaccactgctcccttccctgtccttctgattttgtaggtaaccacgtgcggaccgagcggccgcaggaacccctagtgatggagttggccactccctctctgcgcgctcgctcgctcactgaggccgggcgaccaaaggtcgcccgacgcccgggctttgcccgggcggcctcagtgagcgagcgagcgcgcagctgcctgcagg 234 AA PAT REGION 1..234 note Synthetic: Amino acid Sequence of Human RdCVF1L (-Met) and Human IgK signal sequence (+Asp) source 1..234 mol_type protein organism synthetic construct MDMRVPAQLLGLLLLWLSGARCDASLFSGRILIRNNSDQDELDTEAEVSRRLENRLVLLFFGAGACPQCQAFVPILKDFFVRLTDEFYVLRAAQLALVYVSQDSTEEQQDLFLKDMPKKWLFLPFEDDLRRDLGRQFSVERLPAVVVLKPDGDVLTRDGADEIQRLGTACFANWQEAAEVLDRNFQLPEDLEDQEPRSLTECLRRHKYRVEKAARGGRDPGGGGGEEGGAGGLF 233 AA PAT REGION 1..233 note Synthetic: Amino acid Sequence of Human RdCVF1L (-Met) and Human IgK signal sequence source 1..233 mol_type protein organism synthetic construct MDMRVPAQLLGLLLLWLSGARCASLFSGRILIRNNSDQDELDTEAEVSRRLENRLVLLFFGAGACPQCQAFVPILKDFFVRLTDEFYVLRAAQLALVYVSQDSTEEQQDLFLKDMPKKWLFLPFEDDLRRDLGRQFSVERLPAVVVLKPDGDVLTRDGADEIQRLGTACFANWQEAAEVLDRNFQLPEDLEDQEPRSLTECLRRHKYRVEKAARGGRDPGGGGGEEGGAGGLF 131 AA PAT REGION 1..131 note Synthetic: Amino acid Sequence of Human RdCVF1S (-Met) and Human IgK Signal Sequence (+Asp) source 1..131 mol_type protein organism synthetic construct MDMRVPAQLLGLLLLWLSGARCDASLFSGRILIRNNSDQDELDTEAEVSRRLENRLVLLFFGAGACPQCQAFVPILKDFFVRLTDEFYVLRAAQLALVYVSQDSTEEQQDLFLKDMPKKWLFLPFEDDLRR 130 AA PAT REGION 1..130 note Synthetic: Amino acid Sequence of Human RdCVF1S (-Met) and Human IgK Signal Sequence source 1..130 mol_type protein organism synthetic construct MDMRVPAQLLGLLLLWLSGARCASLFSGRILIRNNSDQDELDTEAEVSRRLENRLVLLFFGAGACPQCQAFVPILKDFFVRLTDEFYVLRAAQLALVYVSQDSTEEQQDLFLKDMPKKWLFLPFEDDLRR 23 AA PAT REGION 1..23 note Synthetic: Amino acid Sequence of Human IgK Signal Sequence (+Asp) source 1..23 mol_type protein organism synthetic construct MDMRVPAQLLGLLLLWLSGARCD 22 AA PAT REGION 1..22 note Synthetic: Amino acid Sequence of Human IgK Signal Sequence source 1..22 mol_type protein organism synthetic construct MDMRVPAQLLGLLLLWLSGARC 23 AA PAT REGION 1..23 note Synthetic: Amino acid Sequence of Human IgK Signal Sequence I source 1..23 mol_type protein organism synthetic construct METDTLLWVLLLWVPGSTGDDIV 2752 DNA PAT misc_feature 1..2752 note Synthetic: Nucleotide Sequence of Human RdCVF1L (-Met) and Human IgK Signal Sequence I source 1..2752 mol_type other DNA organism synthetic construct cctgcaggcagctgcgcgctcgctcgctcactgaggccgcccgggcaaagcccgggcgtcgggcgacctttggtcgcccggcctcagtgagcgagcgagcgcgcagagagggagtggccaactccatcactaggggttcctgcggccgcacgcgtggagctagttattaatagtaatcaattacggggtcattagttcatagcccatatatggagttccgcgttacataacttacggtaaatggcccgcctggctgaccgcccaacgacccccgcccattgacgtcaataatgacgtatgttcccatagtaacgtcaatagggactttccattgacgtcaatgggtggagtatttacggtaaactgcccacttggcagtacatcaagtgtatcatatgccaagtacgccccctattgacgtcaatgacggtaaatggcccgcctggcattatgcccagtacatgaccttatgggactttcctacttggcagtacatctacgtattagtcatcgctattaccatggtgatgcggttttggcagtacatcaatgggcgtggatagcggtttgactcacggggatttccaagtctccaccccattgacgtcaatgggagtttgttttgcaccaaaatcaacgggactttccaaaatgtcgtaacaactccgccccattgacgcaaatgggcggtaggcgtgtacggtgggaggtctatataagcagagctcgtttagtgaaccgtcagatcgcctggagacgccatccacgctgttttgacctccatagaagacaccgggaccgatccagcctccgcggattcgaatcccggccgggaacggtgcattggaacgcggattccccgtgccaagagtgacgtaagtaccgcctatagagtctataggcccacaaaaaatgctttcttcttttaatatacttttttgtttatcttatttctaatactttccctaatctctttctttcagggcaataatgatacaatgtatcatgcctctttgcaccattctaaagaataacagtgataatttctgggttaaggcaatagcaatatttctgcatataaatatttctgcatataaattgtaactgatgtaagaggtttcatattgctaatagcagctacaatccagctaccattctgcttttattttatggttgggataaggctggattattctgagtccaagctaggcccttttgctaatcatgttcatacctcttatcttcctcccacagctcctgggcaacgtgctggtctgtgtgctggcccatcactttggcaaagaattgggattcgaacatcgattgaattcgagccaccatggagacggatacgttgctgtgggtcctcttgctgtgggtcccgggctctacaggagatgacatagtcgccagcctgttcagcggccggatcctgatcaggaacaacagcgaccaggacgagctggacaccgaggccgaagtgagcaggaggctggagaacagactggtgctgctgttctttggcgccggagcctgccctcagtgccaggccttcgtgcccatcctgaaggatttctttgtgaggctgaccgacgagttctacgtgctgagagccgcccagctggccctggtgtatgtgagccaggacagcaccgaggagcagcaggacctgttcctgaaggacatgcccaagaagtggctgttcctgcccttcgaggacgacctgagaagagacctgggcaggcagttcagcgtggagagactgcccgccgtggtggtgctgaagcctgatggcgacgtgctgaccagagatggcgccgacgagatccagagactgggcaccgcctgcttcgccaactggcaggaggccgccgaggtcctggacagaaacttccagctgcccgaggatctggaggatcaggagcccagatccctgaccgagtgcctgaggcggcacaagtacagagtggagaaggccgccagaggcggcagagaccctggcggcggaggaggagaggagggcggagccggcggactgttctgatgagctagcaccggttgtacaagtcaagcggccaaccctccctagatctacgggtggcatccctgtgacccctccccagtgcctctcctggccctggaagttgccactccagtgcccaccagccttgtcctaataaaattaagttgcatcattttgtctgactaggtgtccttctataatattatggggtggaggggggtggtatggagcaaggggcaagttgggaagacaacctgtagggcctgcggggtctattgggaaccaagctggagtgcagtggcacaatcttggctcactgcaatctccgcctcctgggttcaagcgattctcctgcctcagcctcccgagttgttgggattccaggcatgcatgaccaggctcagctaatttttgtttttttggtagagacggggtttcaccatattggccaggctggtctccaactcctaatctcaggtgatctacccaccttggcctcccaaattgctgggattacaggcgtgaaccactgctcccttccctgtccttctgattttgtaggtaaccacgtgcggaccgagcggccgcaggaacccctagtgatggagttggccactccctctctgcgcgctcgctcgctcactgaggccgggcgaccaaaggtcgcccgacgcccgggctttgcccgggcggcctcagtgagcgagcgagcgcgcagctgcctgcagg 234 AA PAT REGION 1..234 note Synthetic: Amino acid Sequence of Human RdCVF1L (-Met) and Human IgK Signal Sequence I source 1..234 mol_type protein organism synthetic construct METDTLLWVLLLWVPGSTGDDIVASLFSGRILIRNNSDQDELDTEAEVSRRLENRLVLLFFGAGACPQCQAFVPILKDFFVRLTDEFYVLRAAQLALVYVSQDSTEEQQDLFLKDMPKKWLFLPFEDDLRRDLGRQFSVERLPAVVVLKPDGDVLTRDGADEIQRLGTACFANWQEAAEVLDRNFQLPEDLEDQEPRSLTECLRRHKYRVEKAARGGRDPGGGGGEEGGAGGLF 471 DNA PAT misc_feature 1..471 note Synthetic: Nucleotide Sequence of RdCVF2L source 1..471 mol_type other DNA organism synthetic construct atggttgacattctgggcgagcggcacctggtgacctgtaagggcgcgacggtggaggccgaggcggcgctgcagaacaaggtggtggcactgtacttcgcggcggcccggtgcgcgccgagccgcgacttcacgccgctgctctgcgacttctatacggcgctggtggccgaggcgcggcggcccgcgcccttcgaagtggtcttcgtgtcagccgacggcagctcccaggagatgctggacttcatgcgcgagctgcatggcgcctggctggcgctgcccttccacgacccctaccggcatgagctgaggaagaggtacaacgtcacagccatccccaagcttgtgattgtgaaacaaaatggggaggtcatcaccaacaaagggcggaagcagatccgggaacgggggttggcctgcttccaggactgggtggaggcggccgatatcttccagaatttctccgtttga 156 AA PAT REGION 1..156 note Synthetic: Amino Acid Sequence of RdCVF2L source 1..156 mol_type protein organism synthetic construct MVDILGERHLVTCKGATVEAEAALQNKVVALYFAAARCAPSRDFTPLLCDFYTALVAEARRPAPFEVVFVSADGSSQEMLDFMRELHGAWLALPFHDPYRHELRKRYNVTAIPKLVIVKQNGEVITNKGRKQIRERGLACFQDWVEAADIFQNFSV 537 DNA PAT misc_feature 1..537 note Synthetic: Codon Optimized Nucleotide Sequence of RdCVF2L (-Met) and human IgK Signal Sequence (+Asp) source 1..537 mol_type other DNA organism synthetic construct atggacatgagggtccctgctcagctcctggggctcctgctgctctggctctcaggtgccagatgtgatgttgatatactgggagaaaggcatcttgtaacatgcaaaggggctacagtggaagctgaggctgcacttcagaacaaagtggtggctctgtatttcgctgctgcccggtgcgctccctcacgggactttacccccttgctgtgtgacttttacaccgccctggttgcggaagctagaaggccagccccttttgaggtggttttcgtcagtgctgatggttcaagccaagagatgctggactttatgagagagctgcatggcgcttggctcgcgttgccctttcacgacccctatagacacgagttgagaaagcggtacaacgtcaccgcaataccgaagcttgttatcgtaaaacaaaacggtgaggttatcaccaataaaggccgcaaacaaattcgcgagagaggcctggcgtgttttcaggattgggtggaagccgcagacatctttcagaacttttcagtgtga 178 AA PAT REGION 1..178 note Synthetic: Amino Acid Sequence of RdCVF2L (-Met) and human IgK Signal Sequence (+Asp) source 1..178 mol_type protein organism synthetic construct MDMRVPAQLLGLLLLWLSGARCDVDILGERHLVTCKGATVEAEAALQNKVVALYFAAARCAPSRDFTPLLCDFYTALVAEARRPAPFEVVFVSADGSSQEMLDFMRELHGAWLALPFHDPYRHELRKRYNVTAIPKLVIVKQNGEVITNKGRKQIRERGLACFQDWVEAADIFQNFSV