• ClinicalTrials.gov Identifier: NCT04418414¹
• Phase 1, Single Group Assignment, Non-randomized, open label trial¹

The status of the trial was reported on ClinicalTrials.gov¹ as follows

• Not yet recruiting *,
• Last Update Posted: May 23, 2022

• Male subjects who are >= 18 years of age
• Diagnosis of severe hemophilia A (<1 IU/dL FVIII activity) based on one-stage coagulation assay
• Documented history of more than 150 days of FVIII treatment

• History of spontaneous central nervous system bleeding within the last 5 years
• Subjects who have had prior cellular based therapy or gene editing/ gene therapy including a previous stem cell transplant

Serotype of the lentiviral vector not reported / manufacturing platform unknown

Hybrid human/porcine B-domain deleted FVIII variant (ET3, HP47)^2, 3, 4

Lentiviral FVIII gene therapy targeting expression in Macrophages

Hematopoietic Stem Cell Transplantation (HSCT) of CD34+ cells transduced with CD68-ET3 lentiviral vector (encoding human FVIII gene)

Dose for the 7 participants not reported

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Not applicable

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Not reported

1. Hematopoietic Stem Cell Transplantation Gene Therapy for Treatment of Severe Hemophilia A. Available at: Study Details | Hematopoietic Stem Cell Transplantation Gene Therapy for Treatment of Severe Hemophilia A | ClinicalTrials.gov
2. Brown, H.C., et al., Bioengineered coagulation factor VIII enables long-term correction of murine hemophilia A following liver-directed adeno-associated viral vector delivery. Mol Ther Methods Clin Dev, 2014. 1: p. 14036. Bioengineered coagulation factor VIII enables long-term correction of murine hemophilia A following liver-directed adeno-associated viral vector delivery - PubMed (nih.gov)
3. Doering, C.B., et al., Identification of porcine coagulation factor VIII domains responsible for high level expression via enhanced secretion. J Biol Chem, 2004. 279(8): p. 6546-52. 37. Identification of Porcine Coagulation Factor VIII Domains Responsible for High Level Expression via Enhanced Secretion* - Journal of Biological Chemistry (jbc.org)
4. Doering, C.B., et al., Preclinical Development of a Hematopoietic Stem and Progenitor Cell Bioengineered Factor VIII Lentiviral Vector Gene Therapy for Hemophilia A. Hum Gene Ther, 2018. 29(10): p. 1183-1201. Preclinical Development of a Hematopoietic Stem and Progenitor Cell Bioengineered Factor VIII Lentiviral Vector Gene Therapy for Hemophilia A - PMC (nih.gov)

CD68, Cluster of Differentiation 68 Marker for monocyte lineage and circulating macrophages; ET3, FVIII variant with porcine A1 and ap-A3 domain sequences; LV; Lentivirus