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Gene therapy is a one\u2010time treatment that can elevate factor levels for many years and minimize or eliminate the need for clotting factor concentrate (CFC) replacement therapy. However, there is a paucity of reports on gene therapy efforts in countries outside of North America or Europe, especially in low\u2010and\u2010middle\u2010income countries (LMIC). All indications are that gene therapy will be one of standard care treatments for haemophilia in the future. Still, it may not be accessible to many countries due to various barriers and challenges. At the same time, each country may formulate solutions that may be used globally.<\/jats:p><\/jats:sec><jats:sec><jats:title>Aim<\/jats:title><jats:p>To summarize the approaches taken to establish haemophilia gene therapy in Japan, China, India, South Africa, and Brazil, and to describe the US\u2010initiated multi\u2010LMIC haemophilia gene therapy development program to include Peru, Vietnam, Thailand, Nepal, and Sri Lanka.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>A review of related published information or as accessible by each country's author.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Different starting conditions, differing input and level of support from the multitude of stakeholders, and strong leadership have led to various approaches for facilitating research and developing needed infrastructure and regulatory and financing models. Gene therapy programs are at various stages of development and include both adeno\u2010associated viral and lentiviral vectors.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>Global partnerships and collaboration, exchange of knowledge and experience, and alignment of processes across borders will promote further progress towards global access to gene therapy for haemophilia.<\/jats:p><\/jats:sec>","DOI":"10.1111\/hae.14512","type":"journal-article","created":{"date-parts":[[2022,5,6]],"date-time":"2022-05-06T10:07:08Z","timestamp":1651831628000},"page":"61-67","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":11,"title":["Haemophilia gene therapy\u2014Update on new country initiatives"],"prefix":"10.1111","volume":"28","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-2258-3687","authenticated-orcid":false,"given":"Ulrike M.","family":"Reiss","sequence":"first","affiliation":[{"name":"Department of Hematology St.\u00a0Jude Children's Research Hospital  Memphis Tennessee USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5781-7669","authenticated-orcid":false,"given":"Johnny","family":"Mahlangu","sequence":"additional","affiliation":[{"name":"Haemophilia Comprehensive Care Center National Health Laboratory Service and University of the Witwatersrand Charlotte Maxeke Johannesburg Academic Hospital  Johannesburg South Africa"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5082-6394","authenticated-orcid":false,"given":"Tsukasa","family":"Ohmori","sequence":"additional","affiliation":[{"name":"Department of Biochemistry Jichi Medical University School of Medicine  Shimotsuke Tochigi Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5938-0675","authenticated-orcid":false,"given":"Margareth C.","family":"Ozelo","sequence":"additional","affiliation":[{"name":"Department of Internal Medicine School of Medical Sciences Haemophilia Treatment Center \u2018Cl\u00e1udio Luiz Pizzigatti Corr\u00eaa\u2019 Hemocentro UNICAMP University of Campinas UNICAMP  Campinas Brazil"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5032-5020","authenticated-orcid":false,"given":"Alok","family":"Srivastava","sequence":"additional","affiliation":[{"name":"Centre for Stem Cell Research Unit of inStem Bengaluru, and Department of Hematology Christian Medical College  Vellore India"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Lei","family":"Zhang","sequence":"additional","affiliation":[{"name":"State Key Laboratory of Experimental Hematology National Clinical Research Center for Blood Diseases Haihe Laboratory of Cell Ecosystem CAMS Key Laboratory of Gene Therapy for Blood Diseases Tianjin Key Laboratory of Blood Disease Gene Therapy Institute of Hematology &amp; Blood Diseases Hospital Chinese Academy of Medical Sciences &amp; Peking Union Medical College  Tianjin China"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2022,5,6]]},"reference":[{"key":"e_1_2_6_2_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood.2019003777"},{"key":"e_1_2_6_3_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.13862"},{"key":"e_1_2_6_4_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.blre.2020.100759"},{"key":"e_1_2_6_5_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14391"},{"key":"e_1_2_6_6_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymthe.2020.06.001"},{"key":"e_1_2_6_7_1","doi-asserted-by":"publisher","DOI":"10.1002\/rth2.12586"},{"key":"e_1_2_6_8_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13059-019-1907-9"},{"key":"e_1_2_6_9_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.omtn.2020.03.015"},{"key":"e_1_2_6_10_1","doi-asserted-by":"publisher","DOI":"10.1002\/sctm.19-0261"},{"key":"e_1_2_6_11_1","article-title":"National library of medicine","author":"NIH U.S","year":"2021","journal-title":"ClinicalTrials.gov"},{"key":"e_1_2_6_12_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.13673"},{"key":"e_1_2_6_13_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14420"},{"key":"e_1_2_6_14_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14080"},{"key":"e_1_2_6_15_1","article-title":"Project entrusted by Ministry of Health, Labour and Welfare","author":"Japan Foundation for AIDS Prevention","year":"2020","journal-title":"Nationwide Survey on Coagulation Disorders"},{"key":"e_1_2_6_16_1","unstructured":"TakedaniH&Hemophilia QOL Research Committee. 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