Expression Therapeutics, LLC
Expression Therapeutics is a fully integrated clinical stage gene and cell therapy company dedicated to developing novel, potentially curative therapeutics for deadly childhood and adult genetic diseases. Their innovative bioengineering platforms enable optimized gene therapies targeting bleeding disorders, primary immunodeficiencies, and cancer, utilizing both lentiviral and adeno-associated viral vectors. The company also has a proprietary oncology platform based on non-signaling chimeric antigen receptor technology to develop allogeneic cell therapies for neuroblastoma, leukemia, lymphoma, and acute myeloid leukemia. With a state-of-the-art manufacturing facility, they support rapid development and production of gene and cell therapies.
Industries
Nr. of Employees
small (1-50)
Expression Therapeutics, LLC
Products
Ex vivo CD34+ hematopoietic stem cell lentiviral gene therapy for hemophilia A
Autologous apheresis-derived CD34+ enrichment, lentiviral transduction, and reinfusion to enable durable endogenous production of coagulation factor VIII.
Autologous lentiviral gene therapy for hemophagocytic lymphohistiocytosis (HLH)
Autologous LV gene transfer into patient cells with expression-optimized transgenes intended to correct underlying genetic defects in HLH.
γδ T cell therapy for neuroblastoma
Engineered gamma-delta T cell product intended to provide short-term, tumor-specific cytotoxicity in combination with standard-of-care chemotherapy and immunotherapy for neuroblastoma.
Engineered γδ T cell products with transient CAR expression for leukemia/lymphoma and AML
mRNA-engineered γδ T cells expressing chimeric antigen receptors designed for treatment of B- and T-cell leukemias/lymphomas and acute myeloid leukemia.
Ex vivo CD34+ hematopoietic stem cell lentiviral gene therapy for hemophilia A
Autologous apheresis-derived CD34+ enrichment, lentiviral transduction, and reinfusion to enable durable endogenous production of coagulation factor VIII.
Autologous lentiviral gene therapy for hemophagocytic lymphohistiocytosis (HLH)
Autologous LV gene transfer into patient cells with expression-optimized transgenes intended to correct underlying genetic defects in HLH.
γδ T cell therapy for neuroblastoma
Engineered gamma-delta T cell product intended to provide short-term, tumor-specific cytotoxicity in combination with standard-of-care chemotherapy and immunotherapy for neuroblastoma.
Engineered γδ T cell products with transient CAR expression for leukemia/lymphoma and AML
mRNA-engineered γδ T cells expressing chimeric antigen receptors designed for treatment of B- and T-cell leukemias/lymphomas and acute myeloid leukemia.
Services
End-to-end CDMO for viral vectors and cell therapies
Contract development and GMP manufacturing services including analytical and process development, early-phase and commercial-scale production, and QA-managed batch records.
Research and feasibility viral vector production
Small-scale production services using shake flasks, T225 vessels, and multi-stack systems for early-stage research and feasibility studies.
Pilot and engineering run support
Pilot/engineering runs at partial or full scale to support process transfer and scale-up activities.
Clinical/GMP vector manufacturing (LV, RV, AAV)
Manufacture of clinical-grade lentiviral, retroviral, and AAV vector lots with upstream capacities described (e.g., 60L/120L for lentiviral, up to ~30L for retroviral, stacked systems for AAV).
End-to-end CDMO for viral vectors and cell therapies
Contract development and GMP manufacturing services including analytical and process development, early-phase and commercial-scale production, and QA-managed batch records.
Research and feasibility viral vector production
Small-scale production services using shake flasks, T225 vessels, and multi-stack systems for early-stage research and feasibility studies.
Pilot and engineering run support
Pilot/engineering runs at partial or full scale to support process transfer and scale-up activities.
Clinical/GMP vector manufacturing (LV, RV, AAV)
Manufacture of clinical-grade lentiviral, retroviral, and AAV vector lots with upstream capacities described (e.g., 60L/120L for lentiviral, up to ~30L for retroviral, stacked systems for AAV).
Expertise Areas
- Gene therapy development
- Viral vector manufacturing and scale-up
- Ex vivo hematopoietic stem cell therapies
- Allogeneic cell therapy engineering (γδ T cells)
Key Technologies
- Lentiviral vector production
- Adeno-associated virus (AAV) production
- Retroviral vector production
- Ex vivo CD34+ hematopoietic stem cell modification