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[{"orgOrder":0,"company":"Expression Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Agrees to Phase 1 Trial of ET3, Gene Therapy for Hemophilia A","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I"},{"orgOrder":0,"company":"Expression Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Expression Therapeutics Announces Success in Developing a Stem Cell Lentiviral Gene Therapy for Hemophagocytic Lymphohistiocytosis (HLH)","therapeuticArea":"Immunology","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"August 2020","url1":"","url2":"","graph1":"Immunology","graph2":"IND Enabling"}]
Find Clinical Drug Pipeline Developments & Deals by Expression Therapeutics
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Details:
Expression Therapeutics has developed a promising and potentially curative gene therapy candidate for familial hemophagocytic lymphohistiocytosis (HLH) type 3 (FHL3).
Lead Product(s):
Lentivirus-Hematopoietic Stem Cell
Therapeutic Area: Immunology
Product Name: Undisclosed
Highest Development Status: IND Enabling
Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
August 24, 2020
Details:
ET3 consists of autologous mobilized peripheral blood stem and progenitor cells transduced with a recombinant lentiviral vector, encoding a bioengineered coagulation factor VIII transgene designed for high‑level expression at low vector copy number.
Lead Product(s):
ET3
Therapeutic Area: Genetic Disease
Product Name: Undisclosed
Highest Development Status: Phase I
Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
May 26, 2020