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2024\u5e7412\u6708\uff0c\u4fe1\u5ff5\u91ab\u85e5\u5728\u7b2c66\u5c46\u7f8e\u570b\u8840\u6db2\u5b78\u6703\uff08ASH\uff09\u5e74\u6703\u6b63\u5f0f\u767c\u5e03\u2162\u671f\u81e8\u5e8a\u7814\u7a76\u7d50\u679c\u3002\u7d50\u679c\u986f\u793a\uff0c\u63a5\u53d7\u57fa\u56e0\u6cbb\u7642\u5f8c\u96a8\u8a2a52\u5468\u6642\uff0c\u53d7\u8a66\u8005\u5e74\u5316\u51fa\u8840\u7387\uff08ABR\uff09\u5747\u503c\u70ba0.6\uff0c\u5e73\u5747F\u2168\u6d3b\u6027\u9054\u523055.08 IU\/dL\uff08\u57fa\u65bcSynthaSIL aPTT\u8a66\u5291\u7684\u4e00\u671f\u6cd5\uff09\uff0c\u5916\u6e90\u6027F\u2168\u85e5\u7269\u7684\u5e73\u5747\u8f38\u8a3b\u6b21\u6578\u5f9e\u6cbb\u7642\u524d\u768458.2\u6b21\/\u5e74\u964d\u81f3\u6cbb\u7642\u5f8c\u76842.9\u6b21\/\u5e74\u300226\u540d\u53d7\u8a66\u8005\u4e2d\u768421\u4f4d\uff0880.8%\uff09\u5728\u6cbb\u7642\u5f8c\u672a\u51fa\u73fe\u51fa\u8840\u4e8b\u4ef6\uff0c\u4e14\u6240\u6709\u53d7\u8a66\u8005\u5747\u7121\u56b4\u91cd\u4e0d\u826f\u4e8b\u4ef6\u767c\u751f8<\/sup>\u3002\u8a72\u7814\u7a76\u4e2d\u6240\u6709\u60a3\u8005\u4ecd\u5728\u6301\u7e8c\u96a8\u8a2a\u904e\u7a0b\u4e2d\u3002<\/p>\n

\u53c2\u8003\u6587\u732e<\/p>\n

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[1] \u622a\u6b62\u81f32025\u5e744\u6708\u3002https:\/\/www.nmpa.gov.cn\/yaopin\/index.html<\/span><\/p>\n<\/td>\n<\/tr>\n

\n

[2] \u4e2d\u83ef\u91ab\u5b78\u6703\u8840\u6db2\u5b78\u5206\u6703\u8840\u6813\u8207\u6b62\u8840\u5b78\u7d44,\u4e2d\u570b\u8840\u53cb\u75c5\u5354\u4f5c\u7d44.\u8840\u53cb\u75c5\u6cbb\u7642\u4e2d\u570b\u6307\u5357(2020\u5e74\u7248).\u4e2d\u83ef\u8840\u6db2\u5b78\u96dc\u8a8c.2020;41(4):265-271<\/span><\/p>\n<\/td>\n<\/tr>\n

\n

[3]\u00a0Mortensen GL,et al. Haemophilia. 2018;862\u2013872<\/span><\/p>\n<\/td>\n<\/tr>\n

\n

[4] Srivastava A, et al. Haemophilia. 2020;001\u2013158.P95<\/span><\/p>\n<\/td>\n<\/tr>\n

\n

[5]\u00a0Xue F, et al. Lancet Haematol. 2022 Jul;9(7):e504-e513<\/span><\/p>\n<\/td>\n<\/tr>\n

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[6] Xue F, et al. N Engl J Med. 2022 Oct 27;387(17):1622-1624<\/span><\/p>\n<\/td>\n<\/tr>\n

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[7]\u00a0\u570b\u5bb6\u85e5\u54c1\u76e3\u7763\u7ba1\u7406\u5c40\u85e5\u54c1\u5be9\u8a55\u4e2d\u5fc3\u00a0https:\/\/www.cde.org.cn\/main\/xxgk\/listpage\/da6efd086c099b7fc949121166f0130c<\/a><\/span><\/p>\n<\/td>\n<\/tr>\n

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[8]\u00a0Feng X\uff0cet al. 2024 ASH. Poster 3582<\/span><\/p>\n<\/td>\n<\/tr>\n<\/tbody>\n<\/table><\/div>\n

\u95dc\u65bc\u4fe1\u5ff5\u91ab\u85e5<\/b><\/p>\n

\u4fe1\u5ff5\u91ab\u85e5\u96c6\u5718\uff08Belief BioMed Inc.\uff09\uff0c\u662f\u4e00\u5bb6\u96c6\u57fa\u56e0\u6cbb\u7642\u7522\u54c1\u7814\u767c\u3001\u751f\u7522\u548c\u81e8\u5e8a\u61c9\u7528\u70ba\u4e00\u9ad4\u7684\u9ad8\u79d1\u6280\u4f01\u696d\u3002\u516c\u53f8\u81f4\u529b\u65bc\u901a\u904e\u5b89\u5168\u9ad8\u6548\u7684\u75c5\u6bd2\u8f09\u9ad4\u6280\u8853\u70ba\u56b4\u91cd\u907a\u50b3\u75be\u75c5\u548c\u6162\u6027\u75be\u75c5\u63d0\u4f9b\u66f4\u52a0\u6709\u6548\u7684\u5275\u65b0\u6027\u57fa\u56e0\u7642\u6cd5\u3002\u516c\u53f8\u7814\u767c\u4e86\u6578\u767e\u7a2e\u8f09\u9ad4\u95dc\u9375\u6280\u8853\uff0c\u5305\u62ecHEK293\u7d30\u80de\u61f8\u6d6e\u7121\u8840\u6e05\u57f9\u990a\u5de5\u85dd\u548c\u5168\u5c64\u6790\u898f\u6a21\u5316\u7d14\u5316\u5de5\u85dd\uff0c\u4e26\u5efa\u7acb\u4e86\u57fa\u56e0\u6cbb\u7642\u85e5\u7269\u5546\u696d\u5316\u751f\u7522\u5e73\u81fa\u3002\u516c\u53f8\u5728\u9776\u5411\u4e0d\u540c\u7d44\u7e54\u7684AAV\u65b0\u578b\u8863\u6bbc\u3001\u9ad8\u6548\u7684\u8f49\u57fa\u56e0\u8868\u9054\u76d2\u8a2d\u8a08\u3001\u5148\u9032\u7684\u81e8\u5e8a\u7d1a\u8f09\u9ad4\u88fd\u9020\u5de5\u85dd\u7b49\u9818\u57df\u9032\u884c\u4e86\u5168\u9762\u5e03\u5c40\u3002\u540c\u6642\u9084\u5efa\u7acb\u4e86\u8c50\u5bcc\u7684\u7814\u767c\u7ba1\u7dda\uff0c\u6cbb\u7642\u9818\u57df\u8986\u84cb\u8840\u53cb\u75c5\u3001\u675c\u6c0f\u808c\u71df\u990a\u4e0d\u826f\u7665\u3001\u5e15\u91d1\u68ee\u75c5\u3001\u9aa8\u95dc\u7bc0\u708e\u7b49\u672a\u88ab\u6eff\u8db3\u81e8\u5e8a\u9700\u6c42\u7684\u75be\u75c5\uff0c\u591a\u500b\u7522\u54c1\u7ba1\u7dda\u5df2\u7d93\u9032\u5165\u81e8\u5e8a\u7814\u7a76\u968e\u6bb5\u6216IND\u7533\u5831\u968e\u6bb5\uff0c\u5176\u91dd\u5c0d\u8840\u53cb\u75c5B\u7684\u57fa\u56e0\u7642\u6cd5\u7684\u65b0\u85e5\u5df2\u7372\u4e2d\u570b\u570b\u5bb6\u85e5\u54c1\u76e3\u7763\u7ba1\u7406\u5c40\u6279\u6e96\u4e0a\u5e02\u3002<\/p>\n

\u66f4\u591a\u4fe1\u606f\uff0c\u656c\u8acb\u8a2a\u554f\uff1awww.beliefbiomed.com\u3002<\/p>\n

\u95dc\u65bc\u6b66\u7530<\/b><\/p>\n

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\u66f4\u591a\u4fe1\u606f\uff0c\u656c\u8acb\u8a2a\u554f\uff1ahttps:\/\/www.takeda.com\u3002<\/p>\n

\u8072\u660e<\/b><\/p>\n

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GL,et al. Haemophilia. 2018;862\u2013872 Srivastava A, et al. Haemophilia. 2020;001\u2013158.P95 \u00a0Xue F, et al. Lancet Haematol. 2022 Jul;9(7):e504-e513 Xue F, et al. N Engl J Med. 2022 Oct 27;387(17):1622-1624 \u00a0\u570b\u5bb6\u85e5\u54c1\u76e3\u7763\u7ba1\u7406\u5c40\u85e5\u54c1\u5be9\u8a55\u4e2d\u5fc3\u00a0https:\/\/www.cde.org.cn\/main\/xxgk\/listpage\/da6efd086c099b7fc949121166f0130c \u00a0Feng X\uff0cet al. 2024 ASH. 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\u66f4\u591a\u4fe1\u606f\uff0c\u656c\u8acb\u8a2a\u554f\uff1awww.beliefbiomed.com\u3002 \u95dc\u65bc\u6b66\u7530 \u6b66\u7530\u88fd\u85e5\u4ee5"\u70ba\u4eba\u985e\u5275\u9020\u5065\u5eb7\u751f\u6d3b\uff0c\u70ba\u4e16\u754c\u7de0\u9020\u7f8e\u597d\u672a\u4f86"\u70ba\u4f7f\u547d\u3002\u6211\u5011\u5c08\u8a3b\u65bc\u6d88\u5316\u548c\u708e\u7665\u6027\u75be\u75c5\u3001\u7f55\u898b\u75c5\u3001\u8840\u6db2\u88fd\u54c1\u3001\u816b\u7624\u3001\u795e\u7d93\u79d1\u5b78\u53ca\u75ab\u82d7\u7b49\u95dc\u9375\u6cbb\u7642\u9818\u57df\uff0c\u8457\u529b\u7814\u767c\u4e26\u70ba\u60a3\u8005\u5e36\u4f86\u7a81\u7834\u6027\u7684\u5275\u65b0\u7642\u6cd5\u3002\u6211\u5011\u7684\u76ee\u6a19\u662f\u651c\u624b\u5408\u4f5c\u5925\u4f34\uff0c\u6253\u9020\u52d5\u614b\u5316\u548c\u591a\u6a23\u5316\u7684\u7522\u54c1\u7ba1\u7dda\uff0c\u4e0d\u65b7\u6539\u5584\u60a3\u8005\u9ad4\u9a57\uff0c\u4e26\u62d3\u5c55\u5c0d\u524d\u6cbf\u6027\u6cbb\u7642\u65b9\u6848\u7684\u63a2\u7d22\u3002 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GL,et al. Haemophilia. 2018;862\u2013872 Srivastava A, et al. Haemophilia. 2020;001\u2013158.P95 \u00a0Xue F, et al. Lancet Haematol. 2022 Jul;9(7):e504-e513 Xue F, et al. N Engl J Med. 2022 Oct 27;387(17):1622-1624 \u00a0\u570b\u5bb6\u85e5\u54c1\u76e3\u7763\u7ba1\u7406\u5c40\u85e5\u54c1\u5be9\u8a55\u4e2d\u5fc3\u00a0https:\/\/www.cde.org.cn\/main\/xxgk\/listpage\/da6efd086c099b7fc949121166f0130c \u00a0Feng X\uff0cet al. 2024 ASH. Poster 3582 \u95dc\u65bc\u4fe1\u5ff5\u91ab\u85e5 \u4fe1\u5ff5\u91ab\u85e5\u96c6\u5718\uff08Belief BioMed 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\u00a0Mortensen GL,et al. Haemophilia. 2018;862\u2013872 Srivastava A, et al. Haemophilia. 2020;001\u2013158.P95 \u00a0Xue F, et al. Lancet Haematol. 2022 Jul;9(7):e504-e513 Xue F, et al. N Engl J Med. 2022 Oct 27;387(17):1622-1624 \u00a0\u570b\u5bb6\u85e5\u54c1\u76e3\u7763\u7ba1\u7406\u5c40\u85e5\u54c1\u5be9\u8a55\u4e2d\u5fc3\u00a0https:\\\/\\\/www.cde.org.cn\\\/main\\\/xxgk\\\/listpage\\\/da6efd086c099b7fc949121166f0130c \u00a0Feng X\uff0cet al. 2024 ASH. 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GL,et al. Haemophilia. 2018;862\u2013872 Srivastava A, et al. Haemophilia. 2020;001\u2013158.P95 \u00a0Xue F, et al. Lancet Haematol. 2022 Jul;9(7):e504-e513 Xue F, et al. N Engl J Med. 2022 Oct 27;387(17):1622-1624 \u00a0\u570b\u5bb6\u85e5\u54c1\u76e3\u7763\u7ba1\u7406\u5c40\u85e5\u54c1\u5be9\u8a55\u4e2d\u5fc3\u00a0https:\/\/www.cde.org.cn\/main\/xxgk\/listpage\/da6efd086c099b7fc949121166f0130c \u00a0Feng X\uff0cet al. 2024 ASH. 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Haemophilia. 2018;862\u2013872 Srivastava A, et al. Haemophilia. 2020;001\u2013158.P95 \u00a0Xue F, et al. Lancet Haematol. 2022 Jul;9(7):e504-e513 Xue F, et al. N Engl J Med. 2022 Oct 27;387(17):1622-1624 \u00a0\u570b\u5bb6\u85e5\u54c1\u76e3\u7763\u7ba1\u7406\u5c40\u85e5\u54c1\u5be9\u8a55\u4e2d\u5fc3\u00a0https:\/\/www.cde.org.cn\/main\/xxgk\/listpage\/da6efd086c099b7fc949121166f0130c \u00a0Feng X\uff0cet al. 2024 ASH. 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Haemophilia. 2018;862\u2013872 Srivastava A, et al. Haemophilia. 2020;001\u2013158.P95 \u00a0Xue F, et al. Lancet Haematol. 2022 Jul;9(7):e504-e513 Xue F, et al. N Engl J Med. 2022 Oct 27;387(17):1622-1624 \u00a0\u570b\u5bb6\u85e5\u54c1\u76e3\u7763\u7ba1\u7406\u5c40\u85e5\u54c1\u5be9\u8a55\u4e2d\u5fc3\u00a0https:\/\/www.cde.org.cn\/main\/xxgk\/listpage\/da6efd086c099b7fc949121166f0130c \u00a0Feng X\uff0cet al. 2024 ASH. 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