Regulatory Considerations for Genetically Engineered Animals (Malini Wileman - US FDA)
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Transcript of Regulatory Considerations for Genetically Engineered Animals (Malini Wileman - US FDA)
8/20/2019 Regulatory Considerations for Genetically Engineered Animals (Malini Wileman - US FDA)
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\feujmtdry Kdnsabfrmtadns odr
Efnftakmjjy Fneanffrfb Mnahmjs
Hmjana Uajfhmn, H.[., S`.B.
Mnahmj Ladtfk`ndjdey Antfrbaskapjanmry Erdup
Kfntfr odr _ftfranmry HfbakanfOddb mnb Brue Mbhanastrmtadn
Efnf Fbatane td Hdbaoy Mnahmj Efndhfs odr
\fsfmrk` - [kafntao ak mnb Ft`akmj
Kdnsabfrmtadns
Bfkfhlfr 5-0, <3=6
8/20/2019 Regulatory Considerations for Genetically Engineered Animals (Malini Wileman - US FDA)
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Rdbmy‑s Srfsfntmtadn
• \fvafw Dvfrmjj \feujmtdry Srdkfss
‛ \fprasf [tmtutdry Mut`dratafs
‛ \feujmtdry Rraeefrs
‛ ANMB/NMBM \fvafw Srdkfss mt OBM-K_H
• Kmsf [tuby
‛ Mnahmj Hdbfjs do @uhmn Basfmsf
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[tmtutdry Mut`draty
Ofbfrmj Oddb, Brue, mnb Kdshftak Mkt (OB&K
Mkt)
• Srdbukts mrf rfeujmtfb7 ndt prdkfssfs
Nmtadnmj Fnvardnhfntmj Sdjaky Mkt (NFSM)
• Srdkfburmj7 drbfrs mefnkafs td fvmjumtf ahpmkts
do ‚mefnky mktadns‘
8/20/2019 Regulatory Considerations for Genetically Engineered Animals (Malini Wileman - US FDA)
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OB&K Mkt (Nfw Mnahmj Brue Srdvasadns)
• <= KO\ 6== mnb <= KO\ 6=2
• Srd`alats antrdbuktadn do unmpprdvfb brue antdkdhhfrkf
‛ Brues mrf bfoanfb ms mrtakjfs antfnbfb td• Bamendsf, kurf, hataemtf, trfmt, dr prfvfnt basfmsf
• Moofkt t`f strukturf dr mny ounktadn do t`f ldby• Fxfhptadn odr rfsfmrk`
‛ Anvfstaemtadn/Anvfstaemtadnmj Mnahmj
• Odr mpprdvmj, spdnsdr do mppjakmtadn hust bfhdnstrmtf
‛ [mofty td mnahmj ‛ Oddb smofty (odr oddb mnahmjs)
‛ Foofktavfnfss (bdfs t`f mrtakjf bd w`mt t`f spdnsdrkjmahs4)
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[tmtutdry Mut`draty Kjmraoafb an Euabmnkf
odr Anbustry =05*
• Kdvfrs mjj typfs do EF mnahmjs ‛ @fratmljf mnb ndn-`fratmljf rBNM kdnstrukts
• Bfoanatadn do ‚ mrtakjf‘
‛ rBNM kdnstrukt antfnbfb td moofkt t`f strukturf dr ounktadn do t`f mnahmj
• Mjj EF mnahmjs an m janfmef mrf kdvfrfb
• Fvfnt-lmsfb, kmsf-ly-kmsf fvmjumtadn
• Fnodrkfhfnt baskrftadn mnb mpprdvmj pmt`s
• Nfw Mnahmj Brue Mppjakmtadn (NMBM) hfmns hmnbmtdrympprdvmj pradr td hmrgftane
• Sdst-hmrgft survfajjmnkf*`ttp9//www.obm.edv/Mnahmj_ftfranmry/BfvfjdphfntMpprdvmjSrdkfss/EfnftakFneanffrane/EfnftakmjjyFneanffrfbMnahmjs/b
fomujt.`th
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Efndhf Fbatane
• Vsf do ‚ fneanffrfb nukjfmsfs‘ td foofkt
strukturf/ounktadn mjtfrmtadns
‛ Rmreftfb ansfrtadns
‛ [hmjj hutmtadns
• [ulstatutadns
• Bfjftadns ‛ Jmref hutmtadns
• Sfrkfptadn ms ‚ ndn-EF‘[mnbfr mnb Idune, Nmturf Ladtfk`ndjdey ;<, ;25‛;66 (<3=2)
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M Nmhf As Ndt m \feujmtdry Rraeefr
EHD….efnftakmjjy fneanffrfb……trmnsefnak……
efnf/efndhf fbatfb/ane……tfk`naqufs do hdbfrnladtfk`ndjdey
Mrf NDR rfeujmtdry traeefrs odr t`f Ofbfrmj Oddb, Brue mnbKdshftak Mkt.
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[d w`mt as t`f rfeujmtdry traeefr
R`f tfrh ‚ brue‘ hfmns
‛ (M) mrtakjfs rfkdenazfb an t`f dooakamj Vnatfb [tmtfsS`mrhmkdpdfam, dooakamj @dhdfdpmt`ak S`mrhmkdpdfam do t`f
Vnatfb [tmtfs, dr dooakamj Nmtadnmj Odrhujmry, dr mny
suppjfhfnt td mny do t`fh7 mnb
‛ (L) mrtakjfs antfnbfb odr usf an t`f bamendsas, kurf, hataemtadn,trfmthfnt, dr prfvfntadn do basfmsf an hmn dr dt`fr mnahmjs7
mnb
‛ (K) mrtakjfs (dt`fr t`mn oddb) antfnbfb td moofkt t`f strukturf
dr mny ounktadn do t`f ldby do hmn dr dt`fr mnahmjs7 mnb ‛ (B) mrtakjfs antfnbfb odr usf ms m kdhpdnfnt do mny mrtakjf
spfkaoafb an kjmusf (M), (L), dr (K).
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K_H‑s ANMB/NMBM \fvafw Srdkfss
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Srdbukt Bfoanatadn
Bfskralfs t`f kdnstrukt an
t`f mnahmj, mnb prdpdsfbkjmah ms lmsas odr `mzmrb
abfntaoakmtadn.
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Hdjfkujmr K`mrmktfrazmtadn9 Kdnstrukt
Mrf t`frf sfqufnkfs jagfjy td
kdntman pdtfntamj `mzmrbs td
t`f mnahmj, ̀ uhmns dr mnahmjskdnsuhane oddb ordh t`mt
mnahmj, dr t`f fnvardnhfnt4
f.e., hdlajazfmljf sfqufnkfs
ordh varusfs fnbfhak an t`mt
spfkafs4
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Hdjfkujmr K`mrmktfrazmtadn9EF Mnahmj Janfmef
Bdfs t`f ansfrtadn do t`f rBNM
kdnstrukt pdsf m ̀ mzmrb td t`fmnahmj, ̀ uhmns dr t`f
fnvardnhfnt4
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S`fndtypak K`mrmktfrazmtadnBarfkt mnb anbarfkt rasgs pdsfb td
t`f EF mnahmj4
(f.e., kmn survfyane t`f `fmjt` mnb
dt`fr p`fndtypak k`mrmktfrastaks dot`f mnahmj anodrh us wat` rfspfkt td
rasg td t`f mnahmj mnb pdtfntamj
`uhmn oddb smofty kdnkfrns4)
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Burmlajaty Mssfsshfnt mnb Sjmn
Mrf t`f efndtypf
dr p`fndtypf do
t`f mnahmjk`mneane dvfr t`f
jaofspmn an m wmy
t`mt wdujb moofkt
t`f rasg(s)
mssdkamtfb wat`t`f prdbukt4
As t`frf m pjmn odr
hdnatdrane
stmlajaty tdmntakapmtf dr
abfntaoy t`dsf
k`mnefs4
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Oddb/Offb [mofty AO ANRFNBFB
OD\
ODDB/OFFB\asg do barfkt dr
anbarfkt mbvfrsfdutkdhfs
mssdkamtfb wat`
t`f kdnsuhptadn
do t`f EF mnahmj
ms oddb dr offb4
AO NDRANRFNBFB OD\
ODDB/OFFBFvabfnkf prdvabfb
td bfhdnstrmtf
t`mt anvfstaemtadnmjdr pdst-
kdhhfrkamjazfb
EF mnahmjs wajj ndt
fntfr t`f oddb
suppjy4
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Fnvardnhfntmj [mofty
Barfkt dr anbarfkt foofktsordh antrdbuktadn do t`f EF
mnahmj antd t`f
fnvardnhfnt4
Lmsas odr smtasoyane NFSMrfquarfhfnts.
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Kjmah _mjabmtadn
Bdfs t`f EF mnahmj
hfft t`f kjmahfstmljas`fb an t`f
prdbukt bfoanatadn4
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U`mt as t`f jagfja`ddb do
• Antrdbukfb dr mjtfrfb sfqufnkfs rfkdhlanane wat` fnbdefndussfqufnkfs/fnbfhak varusfs td pdsf anofktadus rasg td `uhmns drmnahmjs
• Fbaljf prdbukts ordh anvfstaemtadnmj mnahmjs fntfrane oddb suppjy
wat`dut mut`drazmtadn ‛ \fkdrb gffpane
‛ Mnahmj AB/trmkgane
‛ Kdntmanhfnt
‛ Mpprdpramtf baspdsatadn• Barfkt dr anbarfkt unantfnbfb foofkts dn mnahmj `fmjt`
• As t`f kjmah vmjab mnb burmljf4
Sdtfntamj \asg-Lmsfb ]ufstadns odrEF Mnahmj Hdbfjs do @uhmn Basfmsf
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@ypdt`ftakmj Kmsf [tuby9Sae Hdbfj do @uhmn Kmnkfr
• Hdbfj9 @fratmljf satf spfkaoak ansfrtadn do t`f `uhmn jhp ESK\efnf moofktane fxprfssadn jfvfjs do m k`fhdganf rfkfptdr. Vsfouj
odr stubafs do shmjj kfjj june kmnkfr, mhdne dt`fr kmnkfrs.
• [`drt bfskraptadn do fvfnt (kdnstrukt an t`f janfmef)
‛ [atf spfkaoak ansfrtadn vam RMJFNs ‛ [atf abfntaoafb
‛ Ojmngane sfqufnkfs (~ = gl mt ;‑ mnb 6‑ fnbs)
‛ Nuhlfr do kdpafs
‛ [fqufnkf (ms kdhpmrfb td kdnstrukt mjdnf)
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@ypdt`ftakmj Kmsf [tuby9Kmttjf Mjtfrfb odr Basfmsf \fsastmnkf
• Hdbfj9 @fratmljf satf spfkaoak ansfrtadn antd Lds tmurus do mLuoomjd luoomjd efnf odr rfsastmnkf td hmstatas.
• [`drt bfskraptadn do fvfnt (kdnstrukt an t`f janfmef)
‛ [atf spfkaoak ansfrtadn vam m sfqufnkf-spfkaoak nukjfmsf
‛ [atf abfntaoafb• Ojmngane sfqufnkfs (~ = gl mt ;‑ mnb 6‑ fnbs)
‛ Nuhlfr do kdpafs
‛ [fqufnkf an t`f mnahmj (ms kdhpmrfb td kdnstrukt mjdnf)
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…Kdntanufb
• S`fndtypak k`mrmktfrazmtadn
‛ Mnahmj ‚smofty‘
• Erdwt` k`mrmktfrastaks
• \fprdbuktavf kmpmkaty
• Mlndrhmjatafs
• Ljddb k`fhastry• Srfsfnkf do mjtfrfb prdtfan an satu
‛ Mnahmj `fmjt`/`uslmnbry
• Bfskraptadn do kdnbatadns, kmrf
• Mooarhmtavf bmajy mnahmj dlsfrvmtadn rfkdrbs
• _ftfranmry trfmthfnt rfpdrts
• Ladsurvfajjmnkf
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….Kdntanufb
• Kjmah _mjabmtadn (Foofktavfnfss)
[tmtutdry rfquarfhfnt9 [ulstmntamj fvabfnkf odr t`f foofkt unbfr t`fprfskralfb kdnbatadns do usf <= V.[.K ;?3l(b)(=), w`frf
‚[ulstmntamj fvabfnkf‘ hfmns
‛ fvabfnkf kdnsastane do dnf dr hdrf mbfqumtf mnb wfjj-kdntrdjjfb anvfstaemtadns
• An t`f tmreft mnahmj
• Jmldrmtdry mnahmjs, oafjb, ladfquavmjfnkf, dr an vatrd stubafs
‛ dn t`f lmsas do w`ak` qumjaoafb fxpfrts kdujb kdnkjubf t`mt t`f
rfeujmtfb mrtakjf wajj `mvf t`f antfnbfb foofkt unbfr t`f
kdnbatadns do usf an t`f jmlfjane. <= K.O.\. 6=2.2(m).
<;
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R@MNG QDV
<2
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Br. Jmrasm \ubfngd
OBM‑s \asg Mnmjysas Srdkfss odr Ladtfk`ndjdey
Srdbukts
Bfkfhlfr 1t`, =39;3 m.h. wflanmr dn V[
\feujmtadns dn Ladtfk`ndjdey
`ttp9//nms-satfs.dre/efnf-bravfs/<3=6/==/=2/wflanmr-us-
rfeujmtadns/
<6