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????????2024/4/20 9:19:04

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??????????????????Precision BioSciences???????????27??????????????????????????????(DMD)?????????????????芯??????????????????????

?????????????(DMD)?????????????????(Dys)?????????????????????????????????????????X?????????????????????????????????携?????????????DMD??????械????????1/6000-1/3500?????寤�??????15/100000-20/100000?????????????????4???????????????????卸?????????????????????12?????????????????械???校?????????20?????????????????????????????????

??????????DMD???????????????????????????????

???????????????DMD??????????????????????????????????????????????????胁???????????????????????????DMD????????????Sarepta Therapeutics???????????????FDA??2016??2019????????校??????Eteplirsen(Exondys 51)??Golodirse(Vyondys 53)?????????45/51/53??????????????????????????蟹????小?????????????????效????????????????????????????????????????????渭??????

??????????????????????DMD????????????????胁???????????DMD??????谐???????????????????????????????????????????????????????璺�???????????????????AAV?????榈�?????????????????????????????2600kb????????AAV???????????????????????宸�?????谩??????????????????????????????????????????????些???????薪????????AAV???????澹�??????????????????梅??????????????????????

??????????????????????绮�??DMD????????????????2016??????????????????Bamboo Therapeutics????DMD??????????蟹????????????5?路????????蟹????????????????????Sarepta Therapeutics??????芯????DMD????????SRP-9001?????????????????

????Grand View Research??????????2023?????DMD?????谐????????41.1?????????????41.3%??????????????????

????????????????????????????DMD???????????????

???????????????????芯??????

1????????Precision BioSciences

???????????Precision BioSciences?????????协?椋�?????????Precision??ARCUS????????????芯?????????????????????????????????????????斜?????DMD??????芯???????????Precision??????????????芯????泄?????芯?????(IND)?????????????????????????????????????

?????ARCUS??Precision????????????懈??????????????????????????????????????械?DNA?懈????????????????(????)?????(?贸?)???????????????????DNA??????I-CreI???????????????????????????槌�?????????????????????????????校?????????DNA???懈??????????懈?????????????????????????????????????????????????位??

????????ARCUS?????????????????????DMD?????????????????????????????????????????????(adRP)???????????????(FH)?????????????????????????????(ATTR)??????ARCUS????DMD?????????????????????45-55??????????????????????

??魏???????????????????????Precision??ARCUS????????????????????????????????????????????????????效??????蔚?????????????????????????????????????????????

2????????Bamboo Therapeutics

2020??11????????????????????????DMD???芯?????????????PF-06939926?????????FDA????????????????

??????????PF-06939926 Ib????????????????????9???????DMD?泻???????????????PF-06939926?????????????????????????????????????????????????12?????

PF-06939926???????2018????????Ib????????????????????????????????芯????????芯?????????????????芯???????????????????????PF-06939926????AAV9???澹�??????????姹�??????????????????????????????????????????????AAV9?????邪?????????????????????????????濉�

3??Sarepta??????

Sarepta Therapeutics??????????????????????????????DMD????????????SRP-9001??2020??9???Sarepta??????SRP-9001???芯?????????????4????????????4-7????2??????????????????????????SRP-9001??2??NSAA??????????????????????2???????????谢????SRP-9001???????????谩?

SRP-9001???????????????????????????????????????????????????????????????????????????DMD??????????????????????????(Dystrophin)?????未???SRP-9001???写???????效?????????2019??12?????????Sarepta????????28.5???????????协?椋�?????SRP-9001???????????????????????Sarepta????SRP-9001?????????????????????????SRP-9001???????

2020??7???SRP-9001???????FDA??????????DMD??????????????SRP-9001????FDA???????????????????SRP-9001???????????????????????????????

???DMD??????谢????????????????????????????Sarepta????????????械?????芯???????????????????????????????

???????????????纾�??

(2020/12/5 10:07:07 ???42978??)

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