Editing Haemophilia (section)

===Gene therapy===
In those with severe haemophilia, [[gene therapy]] may reduce symptoms to those that a person with mild or moderate haemophilia might have.<ref name=Pey2016>{{cite journal|last1=Peyvandi|first1=F|last2=Garagiola|first2=I|last3=Young|first3=G|title=The past and future of haemophilia: diagnosis, treatments, and its complications.|journal=Lancet|date=9 July 2016|volume=388|issue=10040|pages=187–97|pmid=26897598|doi=10.1016/s0140-6736(15)01123-x|s2cid=21945676}}</ref> The best results have been found in haemophilia B.<ref name=Pey2016/> In 2016 early stage human research was ongoing with a few sites recruiting participants.<ref name=Pey2016/> In 2017 a gene therapy trial on nine people with haemophilia A reported that high doses did better than low doses.<ref name="pmid29224506">{{cite journal | vauthors = Rangarajan S, Walsh L, Lester W, Perry D, Madan B, Laffan M, Yu H, Vettermann C, Pierce GF, Wong WY, Pasi KJ | title = AAV5-Factor VIII Gene Transfer in Severe Hemophilia A | journal = N. Engl. J. Med. | volume = 377 | issue = 26 | pages = 2519–30 | date = December 2017 | pmid = 29224506 | doi = 10.1056/NEJMoa1708483 | hdl = 10044/1/57163 | url = http://qmro.qmul.ac.uk/xmlui/handle/123456789/31880 | doi-access = free | hdl-access = free }}</ref><ref name="pmid29224412">{{cite journal | vauthors = van den Berg HM | title = A Cure for Hemophilia within Reach | journal = N. Engl. J. Med. | volume = 377 | issue = 26 | pages = 2592–3 | date = December 2017 | pmid = 29224412 | doi = 10.1056/NEJMe1713888}}</ref> It is not currently an accepted treatment for haemophilia.<ref name=NIH2013Tx/>

In July 2022 results of a gene therapy candidate for [[haemophilia B]] called  FLT180 were announced, it works using an [[adeno-associated virus]] (AAV) to restore the clotting [[factor IX]] (FIX) protein, normal levels of the protein were observed with low doses of the therapy but immunosuppression was necessitated to decrease the risk of vector-related immune responses.<ref>{{Cite journal |last1=Chowdary |first1=Pratima |last2=Shapiro |first2=Susan |last3=Makris |first3=Mike |last4=Evans |first4=Gillian |last5=Boyce |first5=Sara |last6=Talks |first6=Kate |last7=Dolan |first7=Gerard |last8=Reiss |first8=Ulrike |last9=Phillips |first9=Mark |last10=Riddell |first10=Anne |last11=Peralta |first11=Maria R. |date=21 July 2022 |title=Phase 1–2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia B |journal=New England Journal of Medicine |volume=387 |issue=3 |pages=237–247 |doi=10.1056/NEJMoa2119913 |pmid=35857660 |s2cid=250697905 |issn=0028-4793|doi-access=free }}</ref><ref>{{Cite web |title=Novel gene therapy could reduce bleeding risk for haemophilia patients |url=https://www.sciencedaily.com/releases/2022/07/220720193711.htm |access-date=3 August 2022 |website=ScienceDaily |language=en}}</ref><ref>{{Cite news |date=21 July 2022 |title=Transformational therapy cures haemophilia B |language=en-GB |work=BBC News |url=https://www.bbc.com/news/health-62240061 |access-date=3 August 2022}}</ref>

In November 2022, the first gene therapy treatment for haemophilia B was approved by the U.S. Food and Drug Administration, called Hemgenix. It is a single-dose treatment that gives the patient the genetic information required to produce Factor IX.<ref>{{cite web|title=HEMGENIX |url=https://www.fda.gov/vaccines-blood-biologics/vaccines/hemgenix |website=U.S. Food and Drug Administration |date=4 January 2023 |access-date=17 March 2024}}</ref>

In June 2023, the FDA approved the first gene therapy treatment for haemophilia A, called Roctavian. It was only approved for patients with severe cases, but it has been shown to reduce yearly bleeding episodes by 50%. It works similarly to Hemgenix, being administered by intravenous infusion that contains a gene for Factor VIII.<ref>{{cite web |title=FDA Approves First Gene Therapy for Adults with Severe Hemophilia A |url=https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-adults-severe-hemophilia#:~:text=Today%2C%20the%20U.S.%20Food%20and,by%20an%20FDA%2Dapproved%20test. |website=U.S. Food and Drug Administration | date=30 June 2023 |access-date=16 March 2024}}</ref>