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涓庢绁炶禌璺戯細椤跺皷鎶曡祫浜哄拰浠栫殑鐗圭閮ㄩ槦

瀹為獙瀹よ祫璁綉鏃堕棿:2017-08-19 鐐瑰嚮: 娆?/span> 鐧惧害鎼滅储

銆愬璇汇€?/strong>鏈懆锛屼竴瀹跺悕涓篜revail Therapeutics鐨勫叕鍙告寮忔垚绔嬶紝鍏ㄥ姏寮€鍙戦拡瀵瑰笗閲戞.鐥呭拰鍏朵粬绁炵粡閫€琛屾€х柧鐥呯殑鐢熺墿鐤楁硶銆傜敨涓€鎴愮珛锛岃繖瀹跺叕鍙稿氨鍚稿紩浜嗕笟鍐呯殑澶ч噺鍏虫敞銆備竴鏂归潰锛屽畠鐨勬垚绔嬪緱鍒颁簡鍓嶆部鍩哄洜鐤?.....
TAG鏍囩锛?鑽槑搴峰痉 涓庢绁炶禌璺?/a> 椤跺皷鎶曡祫浜?/a> 鐗圭閮ㄩ槦

涔旂撼妫紝浣犳湁甯曢噾妫梾銆?/strong>鎴戜滑瀵硅繖涓粨鏋滄繁琛ㄩ仐鎲俱€?rdquo;

 

鍚埌璇婃柇鍚庯紝涔旂撼妫?middot;甯屽皵寮楁柉鍧︼紙Jonathan Silverstein锛夊崥澹ぇ涓洪渿鎯娿€傚湪鍖荤敓瀹h璇婃柇涔﹀墠锛屼粬鏄竴浣嶅叏鐞冭憲鍚嶇殑椋庨櫓鎶曡祫浜恒€傚湪鎶曡祫鍦堟湁20澶氬勾宸ヤ綔缁忓巻鐨勪粬锛屾槸濂ュ崥璧勬湰鐨勫叏鐞冪鍕熷叡鍚岃礋璐d汉銆傝嚜2012骞磋捣锛屼粬鏇存槸杩炵画6骞村叆閫夌甯冩柉鐨?ldquo;鍏ㄧ悆100鍚嶆渶浣冲垱鎶曚汉”姒滃崟銆傛姇璧勭敓娑噷锛屼箶绾虫.鎺ㄥ姩浜?0澶氱鏂拌嵂鍜屽尰鐤楀櫒姊颁笂甯傦紝鏀瑰彉浜嗗ぇ閲忔偅鑰呯殑鐢熸椿銆?/strong>

 

鑰屽浠婏紝鍙湁49宀佺殑浠栵紝绔熺劧鎴愪簡涓€鍚嶅笗閲戞.鐥呮偅鑰呫€?/strong>

 

鏇寸碂绯曠殑鏄紝浠栦綋鍐呭甫鏈夌殑涓€涓熀鍥犵獊鍙橈紝瀵艰嚧浠栫殑鐥呮儏蹇€熷彂灞曘€傚尰鐢熻锛岀洰鍓嶆病鏈夎兘澶熸不鐤椾粬鐨勬柊鑽€?/span>

 

涓庢绁炶禌璺戯細椤跺皷鎶曡祫浜哄拰浠栫殑鐗圭閮ㄩ槦

 

鏃犺嵂鍙不鐨勭粷鐥?/span>

 

甯曢噾妫梾鏃╁湪涓や釜涓栫邯浠ュ墠灏辨湁璁板綍銆?817骞达紝鑻卞浗鍖荤敓James Parkinson鍏堢敓璇︾粏鎻忚堪浜嗕竴绉嶅鎬殑鐤剧梾锛屽苟鎸囧嚭浜嗘偅鑰呯殑鍏稿瀷鐥囩姸——浠栦滑浼氬嚭鐜?ldquo;涓嶈嚜涓荤殑闇囬ⅳ锛屽苟浼撮殢鐫€鑲岃倝鍔涢噺涓嬮檷銆?rdquo;涓轰簡绾康杩欎釜鍙戠幇锛岃繖绉嶇柧鐥呬篃琚啝浠ヤ簡Parkinson鍏堢敓鐨勫姘忋€?/span>

 

200骞村悗鐨勪粖澶╋紝闅忕潃鍏ㄧ悆鑰侀緞鍖栫殑涓嶆柇鍔犲墽锛屽笗閲戞.鐥呴€愭笎褰版樉鍑哄畠瀵硅€佸勾浜虹殑褰卞搷銆傛嵁浼拌锛屽叏涓栫晫65宀佷互涓婄殑鑰佸勾浜轰腑锛岀害鏈?-2%鐨勪汉鍙楁鐤剧梾鎵€鍥帮紝杩欎竴姣斾緥杩樻湁鍙兘杩涗竴姝ュ鍔犮€?strong>鐩墠锛屽笗閲戞.鐥呮槸鍏ㄤ笘鐣岃寖鍥村唴绗簩甯歌鐨勭缁忛€€琛屾€х柧鐥咃紝鎮h€呬汉鏁颁粎娆′簬闃垮吂娴烽粯鐥呫€?/strong>

 

 

涓庢绁炶禌璺戯細椤跺皷鎶曡祫浜哄拰浠栫殑鐗圭閮ㄩ槦

鈻插乏鏃嬪宸村彲缂撹В甯曢噾妫梾鐨勭棁鐘讹紝浣嗘棤娉曟彁渚涙牴娌?/span>锛堝浘鐗囨潵婧愶細缁村熀鐧剧锛?/span>

 

浠庣梾鐞嗕笂璁诧紝鎴戜滑鐭ラ亾甯曢噾妫梾鐨勮儗鍚庯紝鏄ぇ鑴戠粏鑳炵殑姝讳骸銆傚湪澶ц剳鍐咃紝涓€浜涚粏鑳炰細鍒嗘硨澶氬反鑳猴紝杩欏浜轰綋杩愬姩鑳藉姏鐨勫崗璋冩潵璇撮潪甯搁噸瑕併€傚綋杩欎簺缁嗚優姝讳骸鍚庯紝灏变細璇卞彂甯曢噾妫梾鐨勭棁鐘躲€傜洰鍓嶏紝宸︽棆澶氬反锛坙evodopa锛夌瓑鑽墿鍙互琛ュ厖澶ц剳涓殑澶氬反鑳哄惈閲忥紝缂撹В甯曢噾妫梾鐨勭棁鐘躲€傜劧鑰岋紝闀挎湡浣跨敤杩欐鑽墿鍙兘浼氬鑷磋繍鍔ㄨ兘鍔涙尝鍔紝鍦ㄤ竴娈垫椂闂村唴閫犳垚鎮h€呯殑涓ラ噸杩愬姩闅滅銆?strong>鏇撮噸瑕佺殑鏄紝杩欎簺鑾锋壒鐨勮嵂鐗╂不鏍囦笉娌绘湰銆傚畠浠病鏈変繚鎶ょ缁忕粏鑳炵殑鏁堟灉锛屼篃鏃犳硶闃绘绁炵粡鍏冪殑閫€鍖栦笌姝讳骸銆?/strong>

 

涔熷氨鏄锛屽湪鐩墠鐨勫尰瀛︽按骞充笅锛?strong>甯曢噾妫梾鏄竴绉嶆棤鑽彲娌荤殑缁濈棁銆?/strong>

 

涓嶅垢鐨勬槸锛屼箶绾虫.寰楃殑杩樹笉鏄櫘閫氱殑甯曢噾妫梾銆傚尰鐢熶滑鍙戠幇锛屼箶绾虫.浣撳唴鐨?em>GBA鍩哄洜鍑虹幇浜嗙獊鍙橈紝杩欏湪缇庡浗甯曢噾妫梾鎮h€呬腑鍑虹幇鐨勬瘮渚嬪ぇ绾﹀彧鏈?0%锛屽苟涓嶅父瑙併€傜劧鑰岋紝涓€鏃﹁繖涓熀鍥犲嚭鐜扮獊鍙橈紝甯曢噾妫梾鐨勮繘灞曞氨浼氭洿鍔犺繀鐚涖€?/strong>杩欎篃瑙i噴浜嗕负鍟ヤ箶绾虫.杩樹笉鍒?0宀侊紝灏辩‘璇婃偅鏈夊笗閲戞.鐥呫€?/span>

 

 

涓庢绁炶禌璺戯細椤跺皷鎶曡祫浜哄拰浠栫殑鐗圭閮ㄩ槦

鈻蹭箶绾虫.鐩镐俊锛屼粬鐨勫姫鍔涙湁鏈涙敼鍙樺叏鐞?0涓囧悕鎮h€呯殑鐢熸椿锛堝浘鐗囨潵婧愶細绂忓竷鏂級

 

鏃╂湡璇婃柇鐥呮病鏈夊甫鏉ユ彁鏃╂不鐤楃殑甯屾湜銆?strong>涓?em>GBA鍩哄洜绐佸彉鐩稿叧鐨勫笗閲戞.鐥呮槸杩欑鐤剧梾鐨勬櫄鏈熷舰鎬侊紝鏋佷负鑷村懡銆傚埆璇存不鎰堬紝鎴戜滑鍑犱箮娌℃湁寤剁紦鐥呮儏杩涘睍鐨勬墜娈点€?/strong>

 

“鎴戞槸涓€鍚嶉闄╂姇璧勫锛岄闄╂姇璧勫閮芥槸瑙e喅闂鐨勯珮鎵嬶紝”涔旂撼妫锛?ldquo;杩欎釜闂涓嶄粎浠呭奖鍝嶅埌浜嗘垜锛岃繕褰卞搷鍒颁簡鍏ㄧ悆10涓囧悕鎮h€呫€?rdquo;韬唤鎰忓鍑虹幇杞彉鐨勪箶绾虫.锛屽喅瀹氫笌姝荤璧涜窇銆?/span>

 

鍩哄洜鐤楁硶

 

“褰撲綘鑷繁鎴愪负涓€鍚嶆偅鑰咃紝浣犲鐤剧梾鐮旂┒鐨勮瑙掑氨瀹屽叏涓嶅悓浜嗭紝浣犳兂瑕佺煡閬撲粈涔堢枟娉曡兘娌绘剤鐤剧梾銆?/strong>”涔旂撼妫銆?/span>

 

鎯宠娌绘剤鐤剧梾锛岄鍏堣鐭ラ亾鐤剧梾鑳屽悗鐨勬満鐞嗐€傚湪浜轰綋涓紝GBA鍩哄洜璐熻矗缂栫爜鐢熸垚钁$硸鑴戣嫹鑴傞叾锛坓lucocerebrosidase锛孏Case锛夎泲鐧斤紝杩欑铔嬬櫧鍦ㄦ憾閰朵綋涓湡浣滅敤锛屽娓呴櫎缁嗚優涓殑鍨冨溇鑷冲叧閲嶈銆傚綋瀹冨彂鐢熺獊鍙樻椂锛孏Case鐨勮泲鐧芥椿鎬у氨浼氫笅闄嶏紝鐢卞畠璐熻矗闄嶈В鐨勭硸鑴傦紝浠ュ強涓€浜涢敊璇姌鍙犵殑铔嬬櫧姘村钩灏变細鍗囬珮锛岃浣夸骇鐢熷宸磋兒鐨勭粏鑳炴浜★紝璁╂偅鑰呭嚭鐜板笗閲戞.鐥呯棁鐘躲€?/span>

 

 

涓庢绁炶禌璺戯細椤跺皷鎶曡祫浜哄拰浠栫殑鐗圭閮ㄩ槦

鈻睱TI-291鏄竴娆炬湁鏈涘GBA鍩哄洜绐佸彉鐩稿叧鐨勫笗閲戞.鐥呮彁渚涙不鐤楃殑鏂拌嵂锛堝浘鐗囨潵婧愶細MedKoo锛?/span>

 

鐩墠锛屼竴浜涘尰鑽叕鍙告鍦ㄩ拡瀵硅繖涓€绐佸彉锛岀爺鍙戞不鐤楀笗閲戞.鐥呯殑鏂拌嵂銆傝濡傦紝涓€娆惧彨鍋歀TI-291鐨勬柊鑽湁鏈涜兘澧炲姞澶ц剳鍐匞Case鐨勮泲鐧芥椿鎬э紝鏈夋湜涓鸿繖浜涘笗閲戞.鐥呮偅鑰呭甫鏉ユ敼鍠勩€傜洰鍓嶏紝瀹冭繕澶勪簬鏃╂湡涓村簥涓€?/span>

 

涔旂撼妫苟涓嶆弧瓒充簬鐜扮姸銆備綔涓轰竴鍚嶈祫娣辨姇璧勪汉锛屼粬鐭ラ亾锛?strong>瑙e喅甯曢噾妫梾闅鹃鐨勬渶浣虫柟寮忎箣涓€锛屽氨鏄彫闆嗛鍩熷唴鐨勯《灏栦笓瀹讹紝璁╀粬浠湞鍚屼竴涓柟鍚戝姫鍔涖€?/strong>

 

鏈懆锛屼粬鍗忓姪鎴愮珛浜嗕竴瀹跺悕涓篜revail Therapeutics鐨勫叕鍙革紝鏃ㄥ湪鍙戠幇鍜屽紑鍙戝垱鏂扮殑鐢熺墿鐤楁硶锛屾不鐤楀笗閲戞.鐥呭拰鍏朵粬绁炵粡閫€琛屾€х柧鐥呫€傚熀鍥犵枟娉曞湪杩欏鍏徃閲屾湁鐫€閲嶈鐨勫湴浣嶃€?/span>

 


鈻茶吅鐩稿叧鐥呮瘨锛圓AV锛夌殑宸ヤ綔鍘熺悊锛堣棰戞潵婧愶細REGENXBIO锛?/strong>

 

鍗忓姪涔旂撼妫紑鍙戝熀鍥犵枟娉曠殑鍏抽敭锛屾槸鑽槑搴峰痉闆嗗洟鍚堜綔浼欎即REGENXBIO鍏徃銆傝繖瀹跺叕鍙稿湪鍩轰簬鑵虹梾姣掞紙adeno-associated virus锛孉AV锛夌殑鍩哄洜杞戒綋鎶€鏈笂鏈夌潃鏋佷负鐙埌涔嬪锛屾槸AAV鍩哄洜鐤楁硶鐨勯鍐涗紒涓氥€傚湪鑳屽悗涓鸿繖瀹跺叕鍙告彁渚涙妧鏈敮鎸佺殑锛屾槸鍩哄洜鐤楁硶鐨勫厛椹变箣涓€瑭瑰鏂?middot;濞佸皵妫紙James Wilson锛夋暀鎺?/strong>銆備粬涔熸槸杩欏鍏徃鐨勭瀛﹀垱濮嬩汉銆?/span>

 

涓庢绁炶禌璺戯細椤跺皷鎶曡祫浜哄拰浠栫殑鐗圭閮ㄩ槦

鐩稿叧闃呰锛?/span>鏄庢槦绉戝瀹朵竴澶滄垚涓虹姜浜猴紝浠栫敤16骞磋繘琛岃嚜鎴戞晳璧?/strong>

 

“浣滀负鍩哄洜鐤楁硶鐨勯鍐涗紒涓氫箣涓€锛屾垜浠緢楂樺叴鑳戒笌Prevail灞曞紑鍚堜綔锛屼负缃规偅璇稿涓ラ噸绁炵粡閫€琛屾€х柧鐥呯殑鎮h€呭紑鍙戞湁鏈涙敼鍙樼敓娲荤殑鐤楁硶銆備粬浠€ラ渶鏇村ソ鐨勬不鐤楁柟妗堬紝” REGENXBIO鐨勬€昏鍏奸甯墽琛屽畼Ken Mills鍏堢敓璇撮亾锛?ldquo;鎴戜滑鐨勬効鏅槸鍒╃敤NAV鎶€鏈钩鍙帮紝寮€鍙戞垚鍔熺殑鍒涙柊鐤楁硶锛岄€犵缃规偅涓ラ噸鐤剧梾鐨勬偅鑰呫€侾revail鐨勬垚绔嬫槸鍏朵腑鐨勯噸瑕侀儴鍒嗐€?rdquo;

 

 

涓庢绁炶禌璺戯細椤跺皷鎶曡祫浜哄拰浠栫殑鐗圭閮ㄩ槦

鈻?/span>涓轰簡鏀诲厠甯曢噾妫梾锛屼箶绾虫.鍗氬+鎷涘嫙浜嗕竴鎵规澃鍑虹殑浜烘墠锛孉beliovich鏁欐巿灏辨槸鍏朵腑涔嬩竴锛堝浘鐗囨潵婧愶細alector锛?/span>

 

涔旂撼妫粍寤轰簡涓€鏀拡瀵瑰笗閲戞.鐥呯殑‘娴疯惫绐佸嚮闃?rsquo;銆?/strong>” 鍝ヤ鸡姣斾簹澶у锛圕olumbia University锛夌殑甯曢噾妫梾涓撳Asa Abeliovich鏁欐巿璇撮亾銆備粬涔熸槸Prevail鍏徃鐨勫叡鍚屽垱濮嬩汉涔嬩竴銆?/span>

 

鏀诲厠鐤剧梾锛屾垜浠彲浠ュ仛浠€涔堬紵

 

杩戝嚑骞达紝鐢熺墿鍖昏嵂琛屼笟鏈夌潃蹇€熷彂灞曘€?017骞存埅姝㈣嚦浠婃棩锛?strong>缇庡浗FDA宸茬粡鎵瑰噯浜?a href="http://mp.weixin.qq.com/s?__biz=MzAwMDA5NTIxNQ==&mid=2649962841&idx=1&sn=b2fa411c5b6ed17e28d947f334d74e56&chksm=82e9bfd9b59e36cfd6a3705292ea7d75838bef999e0368b41cede7c60197a144c34f8145f0a6&scene=21#wechat_redirect" style="color: rgb(0, 112, 192);" target="_blank">28娆炬柊鑽?/a>锛屾不鐤楃檶鐥囥€佽倽鐐庛€佺被椋庢箍鎬у叧鑺傜値銆佸鍙戞€х‖鍖栫棁绛夊绉嶇柧鐥呫€傜劧鑰岋紝涓栫晫涓婅澶氱柧鐥呰繕娌℃湁鑽墿鍙敤浜庢不鐤椼€傚彟澶栵紝璁稿鑽墿鍙兘鎺у埗鐥呮儏锛屾棤娉曞交搴曟不鎰堛€傚彲浠ユ兂璞★紝鐩墠鎮h€呬滑鐨勫尰鐤楅渶姹傚苟娌℃湁寰楀埌婊¤冻銆?/span>

 

 

涓庢绁炶禌璺戯細椤跺皷鎶曡祫浜哄拰浠栫殑鐗圭閮ㄩ槦

鈻?/span>涔旂撼妫悜鎴戜滑灞曠ず浜嗚涓氫汉澹兘濡備綍鍚戠柧鐥呭彂璧峰啿閿嬶紙鍥剧墖鏉ユ簮锛欳NBC锛?/span>

 

涔旂撼妫粰浜嗘垜浠竴涓粷浣崇殑妗堜緥銆?strong>閫氳繃韬竟鑳借皟鍔ㄧ殑璧勬簮锛屼粬姹囪仛浜嗛《灏栧鑰呭拰鏂拌嵂鐮斿彂浜哄憳锛屽埄鐢ㄦ渶鏂扮殑鎶€鏈紝鍚戠柧鐥呭彂璧锋寫鎴樸€?/strong>鍊樿嫢鎴愬姛锛岃繖娆惧熀鍥犵枟娉曟湁鏈涗负甯曢噾妫梾鐨勬不鐤楀甫鏉ュ彉闈┿€?/span>

 

鍏跺疄鍦ㄥ垱绔嬪叕鍙稿墠锛屼箶绾虫.灏卞湪娌荤枟甯曢噾妫梾涓婂仛鍑轰簡璁稿鍔姏銆備粬涓庡瀛愭垚绔嬩簡涓€涓潪鐩堝埄鎬х粍缁囷紝骞跺嫙寰椾簡瓒呰繃1600涓囩編鍏冪殑璧勯噾銆傚埄鐢ㄨ繖绗旇祫閲戯紝涔旂撼妫笇鏈涘畬鎴愪笁涓富瑕佺洰鏍囷細涓€锛屾壘鍒拌兘澶熷欢缂?em>GBA鍩哄洜绐佸彉鐩稿叧鐨勫笗閲戞.鐥呰繘灞曠殑鏂规硶锛涗簩锛屽彂鐜板拰寮€鍙戜竴绉嶅啀鐢熺枟娉曪紝寮ヨˉ甯曢噾妫梾缁欏ぇ鑴戦€犳垚鐨勬崯澶憋紱涓夛紝鎵惧埌涓€绉嶈兘澶熼闃茬柧鐥呬骇鐢熺殑鏂规硶銆?/span>

 

杩欓兘涓嶆槸鑳藉杞绘槗瀹屾垚鐨勭洰鏍囷紝浣嗘€诲緱鏈変汉鍘诲紑杈熶竴鏉℃柊閬撹矾銆傛鍍忎箶绾虫.璇寸殑閭f牱锛?strong>“濡傛灉鎴戦兘涓嶅幓鍋氳繖浜涗簨锛岃皝鍙堣兘鍋氬憿锛?rdquo;

 

 

涓庢绁炶禌璺戯細椤跺皷鎶曡祫浜哄拰浠栫殑鐗圭閮ㄩ槦

 

涔旂撼妫浉淇★紝鍙涓嶆斁寮冨笇鏈涳紝濂囪抗鎬讳細鍙戠敓銆?/strong>浠栬锛屼粬杩欎竴鐢熸渶澶х殑鎴愬氨锛屽氨鏄姇璧勫紑鍙戜簡涓€娆惧彨鍋歋trensiq鐨勬柊鑽紝瀹冭兘娌荤枟涓€绉嶇綍瑙佺殑閬椾紶鐥呫€傛湁涓€鍚嶅皬濂冲鍦ㄥ嚑鍛ㄥぇ鐨勬椂鍊欙紝灏辫璇婃柇鎮f湁杩欑鐤剧梾锛屽尰鐢熻濂规渶澶氳繕鏈夊嚑涓湀鑳芥椿銆備絾缁忚繃Strensiq鐨勬不鐤楋紝杩欏悕灏忓コ瀛╁凡缁?宀佷簡銆?/strong>鍜屽叾浠栧悓榫勪汉涓€鏍凤紝濂硅皟鐨€屾椿娉笺€?/span>

 

“鎴戣杩囧杩瑰彂鐢燂紝”涔旂撼妫锛?ldquo;鐜板湪锛屾槸鏃跺€欏垱閫犲彟涓€涓杩逛簡銆?rdquo;

 

鍚庤

 

鏂拌嵂鐮斿彂涔嬭矾浠庢潵閮戒笉鏄竴鏉″潶閫旓紝绁炵粡閫€琛屾€х柧鐥呯殑鏂拌嵂鐮斿彂鏇存槸鍏呮弧鑽嗘锛屽け璐ョ巼鏋侀珮銆?/span>涔熸鍥犱负濡傛锛岃繖浜涢毦娌荤殑鐤剧梾锛屾墠闇€瑕佸悇浣嶅悓浠佷滑鍏卞悓浼稿嚭鎻存墜锛屽紑鍒涙柊鐨勫巻鍙层€傛垜浠湡寰呭叏涓栫晫鐨勭瀛﹀銆佹姇璧勪汉銆佺爺鍙戜汉鍛樿兘澶熼綈蹇冨崗鍔涳紝鍏卞悓鎵撻€犱竴涓仴搴风殑鏈潵锛?ldquo;璁╁ぉ涓嬫病鏈夐毦鍋氱殑鑽紝闅炬不鐨勭梾”銆?/span>

 

鍙傝€冭祫鏂欙細

[1] Venture capitalist wages war against Parkinson's, searches for miracle after his own diagnosis

[2] Silverstein-backed startup will test gene therapy for Parkinson's

[3] The Silverstein Foundation, REGENXBIO, and OrbiMed Launch Prevail Therapeutics

[4] Meet The Top VC In A Race To Find His Own Parkinson's Cure

[5] The Silverstein Foundation锛孧ichael J. Fox Foundation瀹樼綉

锛堟湰鏂囨潵婧愶細鑽槑搴峰痉 锛?

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