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

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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‘

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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/

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