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

ATLANTA, GA, USA I August 24, 2020 I Primary HLH is a family of devastating primary immune deficiencies with limited treatment options and no gene therapies under clinical testing. Expression Therapeutics has developed a promising and potentially curative gene therapy candidate for familial hemophagocytic lymphohistiocytosis (HLH) type 3 (FHL3). Untreated, FHL3 presents as a hyper-inflammatory state with multi-organ damage leading to premature death. Expression Therapeutics expects to rapidly progress candidates for other common forms of primary HLH as well.

"We are excited to announce this expansion of our gene and cell therapy pipeline beyond our lead stem cell lentiviral gene therapy candidate for hemophilia A that is entering Phase 1 clinical testing. Through ongoing research and development incorporating our core technology platforms, Expression Therapeutics has been able to rapidly generate promising therapeutic candidates for our HLH portfolio and several other critical disease areas with significant unmet clinical need," said Christopher Doering, Ph.D., Chief Scientific Officer of Expression Therapeutics.

Proof of concept for stem cell lentiviral gene therapy of FHL3 was demonstrated using primary patient cells and a genetic mouse model of FHL3. A key component in this success was the integration of one of Expression Therapeutics' core technology platforms that facilitates the rapid generation of transgenes with superior potency. Our lead candidate successfully restored exocytosis and cytolytic function to primary patient cells as well as a murine disease model strongly supporting the advancement of this pipeline product candidate.

"We believe there are three key aspects to FHL3 that make it a strong candidate for hematopoietic stem and progenitor cell (HSPC) – lentiviral vector (LV) gene therapy. First, preclinical and clinical studies suggest that less than 20% genetically corrected cells are required to reverse most FHL3 disease symptoms. Second, because of the autologous nature of stem cell-based lentiviral gene therapy, the grave risk of graft vs host disease is eliminated. Third, with stem cell-based lentiviral gene therapy there will be no wait time to find a sufficiently human leukocyte antigen-matched donor," said Trent Spencer, Ph.D., President of Expression Therapeutics.

According to Deanna Fournier, Executive Director of the Histiocytosis Association, "We are very excited about the possibilities this work offers. Our HLH community, and the entire histiocytosis community, is very hopeful and excited about the future!"

Expression Therapeutics is a biotechnology company based in Atlanta and Cincinnati. The current therapeutic pipeline includes advanced gene therapies for hemophilia, neuroblastoma, T-cell leukemia/lymphoma, acute myeloid leukemia (AML), and primary immunodeficiencies such as hemophagocytic lymphohistiocytosis (HLH).

SOURCE: Expression Therapeutics

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