DETAILED ACTION
The present application, filed on or after March 16, 2013, is being examined under the first inventor to file provisions of the AIA .
Election/Restrictions
Applicant’s election without traverse of Group I, presently claims 1-5, 8-10, 12-16, and 20 in the reply filed on 7/22/2026. is acknowledged.
Claims 25-29 and 32 are withdrawn from further consideration pursuant to 37 CFR 1.142(b) as being drawn to a nonelected invention, there being no allowable generic or linking claim. Election was made without traverse in the reply filed on 7/22/2026.
Claims 1-5, 8-10, 12-16, and 20 are under consideration on the merits.
Claim Objections
Claims 25-29 and 32 are objected to, because these claims are withdrawn but were not amended in the instant reply to recite the “withdrawn” status identifier. See 37 C.F.R. § 1.121(c). Appropriate correction is required with the next reply.
Claim Rejections - 35 USC § 103
The following is a quotation of 35 U.S.C. 103 which forms the basis for all obviousness rejections set forth in this Office action:
A patent for a claimed invention may not be obtained, notwithstanding that the claimed invention is not identically disclosed as set forth in section 102, if the differences between the claimed invention and the prior art are such that the claimed invention as a whole would have been obvious before the effective filing date of the claimed invention to a person having
ordinary skill in the art to which the claimed invention pertains. Patentability shall not be negated by the manner in which the invention was made.
The factual inquiries for establishing a background for determining obviousness under 35 U.S.C. 103 are summarized as follows:
1. Determining the scope and contents of the prior art.
2. Ascertaining the differences between the prior art and the claims at issue.
3. Resolving the level of ordinary skill in the pertinent art.
4. Considering objective evidence present in the application indicating obviousness or nonobviousness.
Claims 1-3, 9, 10, 12-14, 16, and 20 are rejected under 35 U.S.C. 103 as being unpatentable over Do et al. (US 2020/0147241; cite # 20 in the IDS dated 5/31/2024).
Do teaches a method of treating Fabry disease in mice, the method comprising administering a single intravenous dose of about 2.5x 1013 GC/kg of AAVhu68.CB7.hGLAa human alpha-galactosidase A (hGLA) (various hGLA constructs, and comprising a CB7 promoter and beta-globin polyA) (Example 6), reading in-part on claims 1 and 20.
In separate embodiments, Do teaches SEQ ID NO: 5 which comprises D233C and I359C substitutions in human alpha-galactosidase A and is otherwise 100% identical to the polypeptide of SEQ ID NO: 2 of claim 1 and the polypeptide of SEQ ID NO: 7 encoded by generic nucleic acids ([0046] and Table 1a) and which is advantageous to stabilize dimers of human alpha-galactosidase A ([0007]), reading on claims 1, 2, and 4. Do teaches adding inverted terminal repeats to the vector to initiate viral DNA replication, packaging, and integration. ([0077]), reading on claim 1. SEQ ID NO: 5 of Do back-translated to nucleic acid sequence is 100% identical to SEQ ID NO: 4a, reading in-part on claim 3. Do teaches that the mice suffering from Fabry disease lack GLA (i.e. α-galactosidase) which causes globotriaosylceramide (aka GL3, GB3) buildup in-part in the kidneys ([0156]) and which causes kidney dysfunction and is then improved following treatment ([0026]), reading on the embodiment of post-symptomatic subjects for claim 9 and reading on claim 10. Do teaches treating late-onset Fabry disease ([0008]), reading on that embodiment of claim 9. Do teaches expression of hGLA in plasma/serum and kidney as measured by ([0164]-[0165]), reading on claim 12. Do teaches hGLA activity in the DRG, kidney, and plasma/serum as measured by a reduction of accumulated GB3 ([0156], [0162], and Fig. 6), reading on claims 13, 14, and 16.
Regarding claims 1-3, it would have been obvious to a person of ordinary skill in the art before the invention was filed to substitute the unspecified hGLA of Example 6 of Do with a nucleic acid encoding for SEQ ID NO: 5 which is hGLA and comprises D233C and I359C substitutions in the methods of Do. Regarding claim 1, it would have been obvious to a person of ordinary skill in the art before the invention was filed to add the inverted terminal repeats of Do to the rAAV construct encoding for hGLA of Do. A person of ordinary skill in the art would have had a reasonable expectation of success to do so because Do expressly considers these alternative embodiments. Regarding claims 1-3, the skilled artisan would have been motivated to do so because Do expressly teaches that hGLA comprising D233C and I359C substitutions are predictably advantageous to improve the stability of hGLA dimers. Regarding claim 1, the skilled artisan would have been motivated to do so because Do teaches that inverted terminal
repeats are predictably advantageous to initiate viral DNA replication, packaging, and integration.
Therefore, the invention as a whole would have been prima facie obvious to a person of ordinary skill before the invention was filed.
Claim 4 is rejected under 35 U.S.C. 103 as being unpatentable over Do as applied to claim 1 above, and further in view of Choi et al. (MOLECULAR AND CELLULAR BIOLOGY, June 1991, p. 3070-3074; Reference U).
The teachings of Do are relied upon as set forth above. The CB7 promoter and beta-globin polyA of Do as cited above reads in-part on claim 4. Do further teaches adding a SV40 intron (claim 18), reading in-part on claim 4.
Choi teaches that the SV40 small-t intron confers a slight enhancement of transgene expression in mice (Fig. 3 and page 3073, paragraph starting “SV40 small-t intron confers…”), reading on claim 4.
Regarding claim 4, it would have been obvious to a person of ordinary skill in the art before the invention was filed to add the SV40 intron of Do to the rAAV construct encoding for hGLA of Do in view of Xu. A person of ordinary skill in the art would have had a reasonable expectation of success to do so because Do expressly considers the addition of SV40 intron. The skilled artisan would have been motivated to do so because Choi teaches that the addition of SV40 small-t intron confers a slight enhancement of transgene expression and so would predictably improve upon the rAAV vector and treatment of Fabry disease of Do.
Therefore, the invention as a whole would have been prima facie obvious to a person of ordinary skill before the invention was filed.
Claim 5 is rejected under 35 U.S.C. 103 as being unpatentable over Do as applied to claim 1 above, and further in view of Yasuda et al. (Molecular Therapy: Methods & Clinical Development Vol. 18 September 2020, 607-619; Reference V).
The teachings of Do are relied upon as set forth above.
Regarding claim 5, Do does not teach woodchuck hepatitis virus post-transcriptional regulator element (i.e. WPRE).
Yasuda teaches methods of treating Fabry disease in GLA-knockout mice, the method comprising administering varying dosages of AAV2/6-hGLA vectors comprising WPRE to the mice wherein WPRE increases transgene expression (Figure 1 and legend; the paragraph spanning pages 607-608) and wherein gb3 and lyso-gb3 accumulation in tissues is reduced (Figure 2C and legend; the first paragraph in the left column on page 609), reading on claim 5.
It would have been obvious to a person of ordinary skill in the art before the invention was filed to add the WPRE of Yasuda to the rAAV vector and treatment methods of Do. A person of ordinary skill in the art would have had a reasonable expectation of success to do so because Yasuda and Do are directed towards AAV vectors encoding for hGLA for treating Fabry disease in subjects in need thereof. The skilled artisan would have been motivated to do so because Yasuda teaches that the addition of WPRE increases transgene expression, and so would be predictably advantageous in the rAAV vector and treatment methods of Do.
Therefore, the invention as a whole would have been prima facie obvious to a person of ordinary skill before the invention was filed.
Claim 8 is rejected under 35 U.S.C. 103 as being unpatentable Do as applied to claim 1 above, and further in view of Vervecken (US 2018/0360991; Reference A).
The teachings of Do are relied upon as set forth above.
Regarding claim 8, Do does not teach treating pre-symptomatic subjects (e.g. prophylactic treatment).
Vervecken teaches methods of treating Fabry disease, the method comprising administering an effective dosage of an AAV vector encoding for an enzyme having increased α-galactosidase (α-gal A) activity ([0010] and [0487]-[0489]), reading on claim 8. Vervecken teaches prophylactic/preventative administration of the enzyme having increased α-galactosidase (α-gal A) activity to prevent or lessen the incidence of Fabry disease ([0427]), reading on claim 8.
It would have been obvious to a person of ordinary skill in the art before the invention was filed to prophylactically/preventatively treat the subjects of Do suffering from Fabry disease of Do in view of Vervecken. A person of ordinary skill in the art would have had a reasonable expectation of success to do so because both Do and Vervecken are directed in-part towards administering an effective dosage of an AAV vector for an enzyme having α-galactosidase activity to treat Fabry disease in subjects. The skilled artisan would have been motivated to do so because Vervecken teaches that prophylactic/preventative administration of an enzyme with α-gal A activity would be predictably advantageous to prevent or lessen the incidence of Fabry disease, thus improving the treatment methods of Do.
Therefore, the invention as a whole would have been prima facie obvious to a person of ordinary skill before the invention was filed.
Claim 15 is rejected under 35 U.S.C. 103 as being unpatentable over Do as applied to claims 1 and 14 above, and further in view of Germain (Orphanet Journal of Rare Diseases 2010, 5:30, 49 pages; Reference W).
The teachings of Do are relied upon as set forth above.
Regarding claim 15, Do does not teach the embodiment of measuring creatinine to determine improved kidney function.
Germain teaches measuring blood urea nitrogen (BUN) and serum creatinine as a baseline and at regular intervals to monitor the kidney symptoms and progression of Fabry disease (page 5, the first full paragraph and Table 3), reading on claim 15.
It would have been obvious to a person of ordinary skill in the art before the invention was filed to further measure blood urea nitrogen (BUN) and serum creatinine in the subjects of Do suffering from Fabry disease in view of Germain. A person of ordinary skill in the art would have had a reasonable expectation of success to do so because both Do and Germain are in-part directed towards subjects suffering from Fabry disease, The skilled artisan would have been motivated to do so because Germain teaches that measuring blood urea nitrogen (BUN) and serum creatinine as a baseline and at regular intervals is predictably advantageous to monitor the kidney symptoms and progression of Fabry disease, and so it then logically flows that measuring blood urea nitrogen (BUN) and serum creatinine at regular intervals would improve upon Do’s methods to monitor for improvements in kidney functions in the treated subjects.
Therefore, the invention as a whole would have been prima facie obvious to a person of ordinary skill before the invention was filed.
Conclusion
No claims are allowed. No claims are free of the art.
Any inquiry concerning this communication or earlier communications from the examiner should be directed to SEAN C BARRON whose telephone number is (571)270-5111. The examiner can normally be reached 7:30am-3:30pm EDT/EST (M-F).
Examiner interviews are available via telephone, in-person, and video conferencing using a USPTO supplied web-based collaboration tool. To schedule an interview, applicant is encouraged to use the USPTO Automated Interview Request (AIR) at http://www.uspto.gov/interviewpractice.
If attempts to reach the examiner by telephone are unsuccessful, the examiner’s supervisor, Sharmila Landau can be reached at 571-272-0614. The fax phone number for the organization where this application or proceeding is assigned is 571-273-8300.
Information regarding the status of published or unpublished applications may be obtained from Patent Center. Unpublished application information in Patent Center is available to registered users. To file and manage patent submissions in Patent Center, visit: https://patentcenter.uspto.gov. Visit https://www.uspto.gov/patents/apply/patent-center for more information about Patent Center and https://www.uspto.gov/patents/docx for information about filing in DOCX format. For additional questions, contact the Electronic Business Center (EBC) at 866-217-9197 (toll-free). If you would like assistance from a USPTO Customer Service Representative, call 800-786-9199 (IN USA OR CANADA) or 571-272-1000.
/Sean C. Barron/Primary Examiner, Art Unit 1653
SEQ ID NO: 2
GenCore version 6.5.3
Copyright (c) 1993 - 2026 Biocceleration Ltd.
OM protein - protein search, using sw model
Run on: September 3, 2026, 12:13:51 ; Search time 230 Seconds
(without alignments)
3617.567 Million cell updates/sec
Title: US-18-712-323-2
Perfect score: 2351
Sequence: 1 MQLRNPELHLGCALALRFLA..........TGTVLLQLENTMQMSLKDLL 429
Scoring table: BLOSUM62
Gapop 10.0 , Gapext 0.5
Searched: 17250840 unique seqs, 1939488216 residues
Total number of hits satisfying chosen parameters: 46
Minimum DB seq length: 1
Maximum DB seq length: 40000
Post-processing: Minimum Match 95%
Maximum Match 100%
Listing first 45 summaries
Database : Published_Applications_AA_Main:*
RESULT 19
US-16-598-960-5
(NOTE: this sequence has 4 duplicates in the database searched.
See complete list at the end of this report)
Sequence 5, US/16598960
Publication No. US20200147241A1
GENERAL INFORMATION
APPLICANT: AMICUS THERAPEUTICS, INC.
TITLE OF INVENTION: DISULFIDE BOND STABILIZED POLYPEPTIDE COMPOSITIONS AND
TITLE OF INVENTION: METHODS OF USE
FILE REFERENCE: 36003-704.201
CURRENT APPLICATION NUMBER: US/16/598,960
CURRENT FILING DATE: 2019-10-10
PRIOR APPLICATION NUMBER: 62/744,069
PRIOR FILING DATE: 2018-10-10
NUMBER OF SEQ ID NOS: 34
SEQ ID NO 5
LENGTH: 429
TYPE: PRT
ORGANISM: Artificial Sequence
FEATURE:
NAME/KEY: source
OTHER INFORMATION: /note="Description of Artificial Sequence: Synthetic
polypeptide"
Query Match 99.4%; Score 2337; Length 429;
Best Local Similarity 99.5%;
Matches 427; Conservative 0; Mismatches 2; Indels 0; Gaps 0;
Qy 1 MQLRNPELHLGCALALRFLALVSWDIPGARALDNGLARTPTMGWLHWERFMCNLDCQEEP 60
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Db 1 MQLRNPELHLGCALALRFLALVSWDIPGARALDNGLARTPTMGWLHWERFMCNLDCQEEP 60
Qy 61 DSCISEKLFMEMAELMVSEGWKDAGYEYLCIDDCWMAPQRDSEGRLQADPQRFPHGIRQL 120
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Db 61 DSCISEKLFMEMAELMVSEGWKDAGYEYLCIDDCWMAPQRDSEGRLQADPQRFPHGIRQL 120
Qy 121 ANYVHSKGLKLGIYADVGNKTCAGFPGSFGYYDIDAQTFADWGVDLLKFDGCYCDSLENL 180
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Db 121 ANYVHSKGLKLGIYADVGNKTCAGFPGSFGYYDIDAQTFADWGVDLLKFDGCYCDSLENL 180
Qy 181 ADGYKHMSLALNRTGRSIVYSCEWPLYMWPFQKPNYTEIRQYCNHWRNFADIDDSWKSIK 240
|||||||||||||||||||||||||||||||||||||||||||||||||||| |||||||
Db 181 ADGYKHMSLALNRTGRSIVYSCEWPLYMWPFQKPNYTEIRQYCNHWRNFADICDSWKSIK 240
Qy 241 SILDWTSFNQERIVDVAGPGGWNDPDMLVIGNFGLSWNQQVTQMALWAIMAAPLFMSNDL 300
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Db 241 SILDWTSFNQERIVDVAGPGGWNDPDMLVIGNFGLSWNQQVTQMALWAIMAAPLFMSNDL 300
Qy 301 RHISPQAKALLQDKDVIAINQDPLGKQGYQLRQGDNFEVWERPLSGLAWAVAMINRQEIG 360
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||| |
Db 301 RHISPQAKALLQDKDVIAINQDPLGKQGYQLRQGDNFEVWERPLSGLAWAVAMINRQECG 360
Qy 361 GPRSYTIAVASLGKGVACNPACFITQLLPVKRKLGFYEWTSRLRSHINPTGTVLLQLENT 420
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Db 361 GPRSYTIAVASLGKGVACNPACFITQLLPVKRKLGFYEWTSRLRSHINPTGTVLLQLENT 420
Qy 421 MQMSLKDLL 429
|||||||||
Db 421 MQMSLKDLL 429
SEQ ID NO: 4
GenCore version 6.5.3
Copyright (c) 1993 - 2026 Biocceleration Ltd.
OM nucleic - protein search, using frame_plus_n2p model
Run on: September 4, 2026, 12:40:48 ; Search time 458 Seconds
(without alignments)
10900.093 Million cell updates/sec
Title: US-18-712-323-4
Perfect score: 2402
Sequence: 1 atgcaactgagaaatcctga..........tgagcctgaaggacctgctg 1287
Scoring table: BLOSUM62
Xgapop 10.0 , Xgapext 0.5
Ygapop 10.0 , Ygapext 0.5
Fgapop 6.0 , Fgapext 7.0
Delop 6.0 , Delext 7.0
Searched: 17250840 unique seqs, 1939488216 residues
Total number of hits satisfying chosen parameters: 33
Minimum DB seq length: 1
Maximum DB seq length: 40000
Post-processing: Minimum Match 95%
Maximum Match 100%
Listing first 45 summaries
Command line parameters:
-THR=4 -MODEL=frame+_n2p.model -DEV=SOFT -Q=/tmp/query.filtered.fasta
-DB=/abss/application/config/db/pubpaa__le40000.farm_22May2026 -QFMT=fastan
-SUFFIX=rapbm -START=1 -END=-1 -MATRIX=blosum62 -TRANS=human40.cdi -LIST=45
-DOCALIGN=200 -THR_SCORE=pct -THR_MAX=100 -THR_MIN=95 -ALIGN=45 -MODE=LOCAL
-OUTFMT=pto -NORM=pto -RUNMODE=sse2 -HEAPSIZE=0 -MINLEN=1 -MAXLEN=40000
-MINDBSEQLEN=1 -MAXDBSEQLEN=40000 -NO_MMAP -LONGLOG -DBSEGSIZE=-1 -N=2
-XGAPOP=10 -XGAPEXT=0.5 -FGAPOP=6 -FGAPEXT=7 -YGAPOP=10 -YGAPEXT=0.5 -DELOP=6
-DELEXT=7 -BAND_WIDTH=100
Database : Published_Applications_AA_Main:*
RESULT 1
US-16-598-960-5
(NOTE: this sequence has 4 duplicates in the database searched.
See complete list at the end of this report)
Sequence 5, US/16598960
Publication No. US20200147241A1
GENERAL INFORMATION
APPLICANT: AMICUS THERAPEUTICS, INC.
TITLE OF INVENTION: DISULFIDE BOND STABILIZED POLYPEPTIDE COMPOSITIONS AND
TITLE OF INVENTION: METHODS OF USE
FILE REFERENCE: 36003-704.201
CURRENT APPLICATION NUMBER: US/16/598,960
CURRENT FILING DATE: 2019-10-10
PRIOR APPLICATION NUMBER: 62/744,069
PRIOR FILING DATE: 2018-10-10
NUMBER OF SEQ ID NOS: 34
SEQ ID NO 5
LENGTH: 429
TYPE: PRT
ORGANISM: Artificial Sequence
FEATURE:
NAME/KEY: source
OTHER INFORMATION: /note="Description of Artificial Sequence: Synthetic
polypeptide"
Alignment Scores:
Length: 429
Score: 2359.00 Matches: 429
Percent Similarity: 100.0% Conservative: 0
Best Local Similarity: 100.0% Mismatches: 0
Query Match: 98.2% Indels: 0
Gaps: 0
US-18-712-323-4 (1-1287) x US-16-598-960-5 (1-429)
Qy 1 ATGCAACTGAGAAATCCTGAACTGCACCTGGGCTGCGCCCTGGCTCTGAGATTTCTGGCT 60
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Db 1 MetGlnLeuArgAsnProGluLeuHisLeuGlyCysAlaLeuAlaLeuArgPheLeuAla 20
Qy 61 CTGGTGTCCTGGGACATCCCTGGCGCTAGAGCCCTGGATAACGGCCTGGCCAGAACACCT 120
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Db 21 LeuValSerTrpAspIleProGlyAlaArgAlaLeuAspAsnGlyLeuAlaArgThrPro 40
Qy 121 ACAATGGGCTGGCTGCACTGGGAGAGATTCATGTGCAACCTGGACTGCCAAGAGGAACCC 180
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Db 41 ThrMetGlyTrpLeuHisTrpGluArgPheMetCysAsnLeuAspCysGlnGluGluPro 60
Qy 181 GACAGCTGCATCAGCGAGAAGCTGTTCATGGAAATGGCCGAGCTGATGGTGTCCGAAGGC 240
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Db 61 AspSerCysIleSerGluLysLeuPheMetGluMetAlaGluLeuMetValSerGluGly 80
Qy 241 TGGAAGGACGCCGGCTACGAGTACCTGTGCATCGACGACTGTTGGATGGCCCCTCAGAGA 300
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Db 81 TrpLysAspAlaGlyTyrGluTyrLeuCysIleAspAspCysTrpMetAlaProGlnArg 100
Qy 301 GACTCTGAGGGCAGACTGCAGGCCGATCCTCAGAGATTTCCCCACGGCATTAGACAGCTG 360
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Db 101 AspSerGluGlyArgLeuGlnAlaAspProGlnArgPheProHisGlyIleArgGlnLeu 120
Qy 361 GCCAACTACGTGCACAGCAAGGGCCTGAAGCTGGGCATCTACGCCGACGTGGGCAACAAG 420
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Db 121 AlaAsnTyrValHisSerLysGlyLeuLysLeuGlyIleTyrAlaAspValGlyAsnLys 140
Qy 421 ACCTGTGCCGGCTTTCCTGGCAGCTTCGGCTACTACGATATCGACGCCCAGACCTTCGCC 480
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Db 141 ThrCysAlaGlyPheProGlySerPheGlyTyrTyrAspIleAspAlaGlnThrPheAla 160
Qy 481 GATTGGGGAGTCGATCTGCTGAAGTTCGACGGCTGCTACTGCGACAGCCTGGAAAATCTG 540
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Db 161 AspTrpGlyValAspLeuLeuLysPheAspGlyCysTyrCysAspSerLeuGluAsnLeu 180
Qy 541 GCCGACGGCTACAAGCACATGTCTCTGGCCCTGAATCGGACCGGCAGATCCATCGTGTAC 600
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Db 181 AlaAspGlyTyrLysHisMetSerLeuAlaLeuAsnArgThrGlyArgSerIleValTyr 200
Qy 601 AGCTGCGAGTGGCCCCTGTACATGTGGCCCTTCCAGAAGCCTAACTACACCGAGATCAGA 660
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Db 201 SerCysGluTrpProLeuTyrMetTrpProPheGlnLysProAsnTyrThrGluIleArg 220
Qy 661 CAGTACTGCAACCACTGGCGGAACTTCGCCGACATCTGCGATAGCTGGAAGTCCATCAAG 720
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Db 221 GlnTyrCysAsnHisTrpArgAsnPheAlaAspIleCysAspSerTrpLysSerIleLys 240
Qy 721 AGCATCCTGGACTGGACCAGCTTCAATCAAGAGCGGATCGTGGACGTGGCAGGACCTGGC 780
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Db 241 SerIleLeuAspTrpThrSerPheAsnGlnGluArgIleValAspValAlaGlyProGly 260
Qy 781 GGATGGAACGATCCTGACATGCTGGTCATCGGCAACTTCGGCCTGAGCTGGAACCAGCAA 840
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Db 261 GlyTrpAsnAspProAspMetLeuValIleGlyAsnPheGlyLeuSerTrpAsnGlnGln 280
Qy 841 GTGACCCAGATGGCCCTGTGGGCCATTATGGCCGCTCCTCTGTTCATGAGCAACGACCTG 900
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Db 281 ValThrGlnMetAlaLeuTrpAlaIleMetAlaAlaProLeuPheMetSerAsnAspLeu 300
Qy 901 AGACACATCAGCCCTCAGGCCAAGGCTCTGCTGCAGGACAAGGATGTGATCGCTATCAAC 960
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Db 301 ArgHisIleSerProGlnAlaLysAlaLeuLeuGlnAspLysAspValIleAlaIleAsn 320
Qy 961 CAGGATCCTCTGGGCAAGCAGGGCTACCAGCTGAGACAGGGCGACAATTTCGAAGTGTGG 1020
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Db 321 GlnAspProLeuGlyLysGlnGlyTyrGlnLeuArgGlnGlyAspAsnPheGluValTrp 340
Qy 1021 GAAAGACCCCTGAGCGGACTGGCTTGGGCCGTCGCCATGATCAACCGGCAAGAGTGCGGC 1080
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Db 341 GluArgProLeuSerGlyLeuAlaTrpAlaValAlaMetIleAsnArgGlnGluCysGly 360
Qy 1081 GGCCCCAGATCCTACACAATCGCCGTGGCCAGTCTCGGCAAAGGCGTGGCATGTAATCCC 1140
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Db 361 GlyProArgSerTyrThrIleAlaValAlaSerLeuGlyLysGlyValAlaCysAsnPro 380
Qy 1141 GCCTGCTTCATCACACAGCTGCTGCCCGTGAAGAGAAAGCTGGGCTTTTACGAGTGGACC 1200
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Db 381 AlaCysPheIleThrGlnLeuLeuProValLysArgLysLeuGlyPheTyrGluTrpThr 400
Qy 1201 AGCAGACTGCGGAGCCACATCAATCCTACCGGCACAGTGCTGCTGCAGCTGGAAAACACC 1260
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Db 401 SerArgLeuArgSerHisIleAsnProThrGlyThrValLeuLeuGlnLeuGluAsnThr 420
Qy 1261 ATGCAGATGAGCCTGAAGGACCTGCTG 1287
|||||||||||||||||||||||||||
Db 421 MetGlnMetSerLeuLysAspLeuLeu 429
SEQ ID NO: 7
GenCore version 6.5.3
Copyright (c) 1993 - 2026 Biocceleration Ltd.
OM protein - protein search, using sw model
Run on: September 3, 2026, 12:18:28 ; Search time 828 Seconds
(without alignments)
1004.880 Million cell updates/sec
Title: US-18-712-323-7
Perfect score: 2359
Sequence: 1 MQLRNPELHLGCALALRFLA..........TGTVLLQLENTMQMSLKDLL 429
Scoring table: BLOSUM62
Gapop 10.0 , Gapext 0.5
Searched: 17250840 unique seqs, 1939488216 residues
Total number of hits satisfying chosen parameters: 43
Minimum DB seq length: 1
Maximum DB seq length: 40000
Post-processing: Minimum Match 95%
Maximum Match 100%
Listing first 45 summaries
Database : Published_Applications_AA_Main:*
RESULT 1
US-16-598-960-5
(NOTE: this sequence has 4 duplicates in the database searched.
See complete list at the end of this report)
Sequence 5, US/16598960
Publication No. US20200147241A1
GENERAL INFORMATION
APPLICANT: AMICUS THERAPEUTICS, INC.
TITLE OF INVENTION: DISULFIDE BOND STABILIZED POLYPEPTIDE COMPOSITIONS AND
TITLE OF INVENTION: METHODS OF USE
FILE REFERENCE: 36003-704.201
CURRENT APPLICATION NUMBER: US/16/598,960
CURRENT FILING DATE: 2019-10-10
PRIOR APPLICATION NUMBER: 62/744,069
PRIOR FILING DATE: 2018-10-10
NUMBER OF SEQ ID NOS: 34
SEQ ID NO 5
LENGTH: 429
TYPE: PRT
ORGANISM: Artificial Sequence
FEATURE:
NAME/KEY: source
OTHER INFORMATION: /note="Description of Artificial Sequence: Synthetic
polypeptide"
Query Match 100.0%; Score 2359; Length 429;
Best Local Similarity 100.0%;
Matches 429; Conservative 0; Mismatches 0; Indels 0; Gaps 0;
Qy 1 MQLRNPELHLGCALALRFLALVSWDIPGARALDNGLARTPTMGWLHWERFMCNLDCQEEP 60
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Db 1 MQLRNPELHLGCALALRFLALVSWDIPGARALDNGLARTPTMGWLHWERFMCNLDCQEEP 60
Qy 61 DSCISEKLFMEMAELMVSEGWKDAGYEYLCIDDCWMAPQRDSEGRLQADPQRFPHGIRQL 120
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Db 61 DSCISEKLFMEMAELMVSEGWKDAGYEYLCIDDCWMAPQRDSEGRLQADPQRFPHGIRQL 120
Qy 121 ANYVHSKGLKLGIYADVGNKTCAGFPGSFGYYDIDAQTFADWGVDLLKFDGCYCDSLENL 180
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Db 121 ANYVHSKGLKLGIYADVGNKTCAGFPGSFGYYDIDAQTFADWGVDLLKFDGCYCDSLENL 180
Qy 181 ADGYKHMSLALNRTGRSIVYSCEWPLYMWPFQKPNYTEIRQYCNHWRNFADICDSWKSIK 240
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Db 181 ADGYKHMSLALNRTGRSIVYSCEWPLYMWPFQKPNYTEIRQYCNHWRNFADICDSWKSIK 240
Qy 241 SILDWTSFNQERIVDVAGPGGWNDPDMLVIGNFGLSWNQQVTQMALWAIMAAPLFMSNDL 300
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Db 241 SILDWTSFNQERIVDVAGPGGWNDPDMLVIGNFGLSWNQQVTQMALWAIMAAPLFMSNDL 300
Qy 301 RHISPQAKALLQDKDVIAINQDPLGKQGYQLRQGDNFEVWERPLSGLAWAVAMINRQECG 360
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Db 301 RHISPQAKALLQDKDVIAINQDPLGKQGYQLRQGDNFEVWERPLSGLAWAVAMINRQECG 360
Qy 361 GPRSYTIAVASLGKGVACNPACFITQLLPVKRKLGFYEWTSRLRSHINPTGTVLLQLENT 420
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Db 361 GPRSYTIAVASLGKGVACNPACFITQLLPVKRKLGFYEWTSRLRSHINPTGTVLLQLENT 420
Qy 421 MQMSLKDLL 429
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Db 421 MQMSLKDLL 429