{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,28]],"date-time":"2026-10-28T12:49:27Z","timestamp":1793191767844,"version":"4.5.0"},"reference-count":49,"publisher":"American Society of Hematology","issue":"26","content-domain":{"domain":["ashpublications.org"],"crossmark-restriction":true},"short-container-title":[],"published-print":{"date-parts":[[2025,12,25]]},"abstract":"<jats:sec>\n                    <jats:title>Abstract<\/jats:title>\n                    <jats:p>The recent approval of adeno-associated virus (AAV)\u2013based gene therapies for hemophilia A (HA) represents a major advancement in the management of this X-linked bleeding disorder, offering multiyear bleed protection and improved quality of life over factor VIII (FVIII) replacement. However, challenges remain, including concerns over long-term durability of expression and the difficulty of packaging the oversized FVIII transgene into AAV vectors. To address these limitations, we developed AAV8-Bi8, a liver-directed gene therapy encoding Bi8, a novel 54.5-kilodalton FVIII-mimetic antibody. Bi8 is expressed as a compact, single-chain tandem, single-chain fragment variable, and is delivered via a 4.4-kilobase expression cassette packaged within AAV8 capsids, well within the vector packaging capacity. In vitro, Bi8 demonstrated FVIII-mimetic activity, and effectively corrected FVIII-deficient human plasma to levels comparable with emicizumab, the current market standard. In vivo, a single administration of AAV8-Bi8 in FVIII-deficient mice resulted in dose-dependent, durable expression of Bi8, complete phenotypic correction of bleeding, and therapeutic equivalence to both emicizumab-treated and wild-type animals. Importantly, no toxicity or antidrug antibody responses were observed. This approach, based on delivering FVIII-mimetic antibodies through AAV rather than truncated FVIII transgenes, could provide a more flexible and efficient platform for gene therapy in HA. AAV8-Bi8 has the potential to offer sustained, lifelong hemostatic control, including in patients who have developed inhibitors to FVIII.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1182\/blood.2024027709","type":"journal-article","created":{"date-parts":[[2025,9,22]],"date-time":"2025-09-22T21:54:47Z","timestamp":1758578087000},"page":"3175-3187","update-policy":"https:\/\/doi.org\/10.1182\/blood.2019cm0000","source":"Crossref","is-referenced-by-count":6,"title":["Alternative AAV gene therapy for hemophilia A using expression of Bi8, a novel single-chain FVIII-mimetic antibody"],"prefix":"10.1182","volume":"146","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-5503-5337","authenticated-orcid":false,"given":"Vincent","family":"Muczynski","sequence":"first","affiliation":[{"name":"1Haematology Department, Cancer Institute, University College London, London, United Kingdom"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9080-6336","authenticated-orcid":false,"given":"Olivier D.","family":"Christophe","sequence":"additional","affiliation":[{"name":"2H\u00e9mostase Inflammation Thrombose, HITh U1176, Universit\u00e9 Paris-Saclay, Institut National de la Sant\u00e9 et de la Recherche Medicale, Le Kremlin-Bic\u00eatre, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lewis","family":"Tanner","sequence":"additional","affiliation":[{"name":"1Haematology Department, Cancer Institute, University College London, London, United Kingdom"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Charlotte","family":"Vayssiere","sequence":"additional","affiliation":[{"name":"1Haematology Department, Cancer Institute, University College London, London, United Kingdom"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alice","family":"Gu\u00e9rin","sequence":"additional","affiliation":[{"name":"2H\u00e9mostase Inflammation Thrombose, HITh U1176, Universit\u00e9 Paris-Saclay, Institut National de la Sant\u00e9 et de la Recherche Medicale, Le Kremlin-Bic\u00eatre, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8271-6795","authenticated-orcid":false,"given":"Caterina","family":"Casari","sequence":"additional","affiliation":[{"name":"2H\u00e9mostase Inflammation Thrombose, HITh U1176, Universit\u00e9 Paris-Saclay, Institut National de la Sant\u00e9 et de la Recherche Medicale, Le Kremlin-Bic\u00eatre, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4115-7867","authenticated-orcid":false,"given":"Jenny","family":"McIntosh","sequence":"additional","affiliation":[{"name":"1Haematology Department, Cancer Institute, University College London, London, United Kingdom"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Doyoung","family":"Lee","sequence":"additional","affiliation":[{"name":"1Haematology Department, Cancer Institute, University College London, London, United Kingdom"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7064-8767","authenticated-orcid":false,"given":"Gavin","family":"Ling","sequence":"additional","affiliation":[{"name":"1Haematology Department, Cancer Institute, University College London, London, United Kingdom"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0484-991X","authenticated-orcid":false,"given":"Satyen","family":"Gohil","sequence":"additional","affiliation":[{"name":"1Haematology Department, Cancer Institute, University College London, London, United Kingdom"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7937-3429","authenticated-orcid":false,"given":"Peter J.","family":"Lenting","sequence":"additional","affiliation":[{"name":"2H\u00e9mostase Inflammation Thrombose, HITh U1176, Universit\u00e9 Paris-Saclay, Institut National de la Sant\u00e9 et de la Recherche Medicale, Le Kremlin-Bic\u00eatre, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8955-2909","authenticated-orcid":false,"given":"Edward G. 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