ET3 / Expression Therap - LARVOL DELTA

Factor VIII Expression from a Novel F8 Transgene through a Lentiviral Vector Transduced CD34+ Autologous Hematopoietic Stem Cells for Gene Therapy of Severe Hemophilia Α: Final Results from a Phase 1 Clinical Trial (ASH 2024) - P1 | "They received hematopoietic stem cell transplantation (HSCT) with autologous G-CSF- and plerixafor-mobilized CD34+ peripheral blood cells collected by apheresis and transduced ex-vivo with the CD68-ET3-LV vector and cryo-preserved. Study participants were conditioned with myeloablative doses of treosulfan and fludarabine before infusion of a target dose of >5x106 cells/kg transduced CD34+ cells...During the engraftment period, participants received recombinant FVIII (rFVIII) targeting a trough level of >50% FVIII activity along with standard supportive care for HSCT...The results from this first-in-human phase 1 clinical trial of a lentiviral vector transduced HSC-based gene therapy for hemophilia A validates this novel approach with no major safety concerns identified. There is stable expression of FVIII up to 2 years and a positive correlation of VCN in PB with plasma levels of FVIII."

Clinical • Gene therapy • P1 data • Viral vector • Bone Marrow Transplantation • Gene Therapies • Hemophilia • Hemophilia A • Neutropenia • Rare Diseases • Thrombocytopenia • CD14 • CD34 • CD68

Hematopoietic Stem Cell Transplantation Gene Therapy for Treatment of Severe Hemophilia A (clinicaltrials.gov) - P1 | N=6 | Completed | Sponsor: Christian Medical College, Vellore, India | Recruiting ➔ Completed | Trial completion date: Jan 2039 ➔ Jun 2024 | Trial primary completion date: Jan 2025 ➔ Jun 2024

Gene therapy • Trial completion • Trial completion date • Trial primary completion date • Bone Marrow Transplantation • Gene Therapies • Hematological Disorders • Hemophilia • Rare Diseases • Transplantation • CD34 • CD68

Hematopoietic Stem Cell Transplantation Gene Therapy for Treatment of Severe Hemophilia A (clinicaltrials.gov) - P1 | N=7 | Not yet recruiting | Sponsor: Expression Therapeutics, LLC | Trial completion date: Apr 2039 ➔ Aug 2039 | Trial primary completion date: Apr 2026 ➔ Aug 2029

Gene therapy • Trial completion date • Trial primary completion date • Viral vector • Bone Marrow Transplantation • Gene Therapies • Hematological Disorders • Hemophilia • Rare Diseases • Transplantation • CD34 • CD68

Hematopoietic Stem Cell Transplantation Gene Therapy for Treatment of Severe Hemophilia A (clinicaltrials.gov) - P1 | N=7 | Not yet recruiting | Sponsor: Expression Therapeutics, LLC | Trial primary completion date: Apr 2025 ➔ Apr 2026

Trial primary completion date • Bone Marrow Transplantation • Gene Therapies • Hematological Disorders • Hemophilia • Rare Diseases • Transplantation • CD34 • CD68

Hematopoietic Stem Cell Transplantation Gene Therapy for Treatment of Severe Hemophilia A (clinicaltrials.gov) - P1 | N=9 | Recruiting | Sponsor: Christian Medical College, Vellore, India | Not yet recruiting ➔ Recruiting

Enrollment open • Bone Marrow Transplantation • Gene Therapies • Hematological Disorders • Hemophilia • Rare Diseases • Transplantation • CD34 • CD68

Expression Therapeutics announces IND approval by the FDA for hemophilia A gene therapy (PRNewswire) - "Expression Therapeutics has announced that it has received clearance by the United States Food and Drug Administration (FDA) to proceed following review of its Investigational New Drug Application (IND) for clinical testing of its novel lentiviral vector-based gene therapy — ET3 — for hemophilia A....In the ET3 trial, subjects will be preconditioned with low-dose stem and immune cell suppressing agents prior to receiving a single infusion of ET3....Expects to initiate a Phase 1 clinical trial — titled ET3-201 — at Emory University and enroll patients shortly."

IND • New P1 trial • Hemophilia