ASC618

ASC618 (also known as AAV2/8 HCB-ET3-LCO BDD FVIII) is an experimental gene therapy for Hemophilia A. It was developed by Applied StemCell Therapeutics and is delivered via a hybrid of adeno-associated virus types 2 and 8. The gene therapy is hoped to be more effective than earlier gene therapies for hemophilia A.^[1]^[2]^[3]

References

^ Guzzardo, Gianna M; Sidonio, Jr, Robert; Callaghan, Michael U; Regling, Katherine (2 November 2022). "Early stage clinical trials for the treatment of hemophilia A". Expert Opinion on Investigational Drugs. 31 (11): 1169–1186. doi:10.1080/13543784.2022.2138742. PMID 36265129. S2CID 253044102.
^ Gong, Jie; Wang, Hao-Lin; Chang, Lung-Ji (4 May 2022). "Molecular therapeutics of hemophilia A and B". Expert Review of Hematology. 15 (5): 431–441. doi:10.1080/17474086.2022.2075339. PMID 35523283. S2CID 248554428.
^ Pipe, Steven W.; Gonen-Yaacovi, Gil; Segurado, Oscar G. (2 September 2022). "Hemophilia A gene therapy: current and next-generation approaches". Expert Opinion on Biological Therapy. 22 (9): 1099–1115. doi:10.1080/14712598.2022.2002842. PMID 34781798.

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[1] Guzzardo, Gianna M; Sidonio, Jr, Robert; Callaghan, Michael U; Regling, Katherine (2 November 2022). "Early stage clinical trials for the treatment of hemophilia A". Expert Opinion on Investigational Drugs. 31 (11): 1169–1186. doi:10.1080/13543784.2022.2138742. PMID 36265129. S2CID 253044102.

[2] Gong, Jie; Wang, Hao-Lin; Chang, Lung-Ji (4 May 2022). "Molecular therapeutics of hemophilia A and B". Expert Review of Hematology. 15 (5): 431–441. doi:10.1080/17474086.2022.2075339. PMID 35523283. S2CID 248554428.

[3] Pipe, Steven W.; Gonen-Yaacovi, Gil; Segurado, Oscar G. (2 September 2022). "Hemophilia A gene therapy: current and next-generation approaches". Expert Opinion on Biological Therapy. 22 (9): 1099–1115. doi:10.1080/14712598.2022.2002842. PMID 34781798.

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