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    Expression Therapeutics Announces Success in Developing a Stem Cell Lentiviral Gene Therapy for Hemophagocytic Lymphohistiocytosis (HLH)

    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 EnablingProduct 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

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    FDA Agrees to Phase 1 Trial of ET3, Gene Therapy for Hemophilia A

    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 IProduct 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

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