Expression Therapeutics develops gene and cell therapies for severe genetic diseases and cancers. They utilize proprietary bioengineering platforms, including optimized expression cassettes and viral vectors, to enhance therapeutic outcomes for conditions like hemophilia and certain leukemias.
Funding
Funding not disclosed
Founders
Product
Problem
Developing curative therapies for severe genetic diseases and certain cancers presents significant challenges in optimizing gene delivery and ensuring therapeutic efficacy. Existing treatment paradigms for conditions like bleeding disorders, primary immunodeficiencies, and aggressive childhood cancers often involve complex, lifelong management with limited long-term success.
Solution
Expression Therapeutics is a clinical-stage company focused on developing novel gene and cell therapies for serious genetic diseases and oncology indications. The company leverages proprietary bioengineering platforms, including its ECO (Expression Cassette Optimization) technology, to enhance transgene expression and therapeutic outcomes. Their approach utilizes both lentiviral (LV) and adeno-associated viral (AAV) vectors for gene delivery, tailored to specific disease targets. For oncology, Expression Therapeutics employs a proprietary gamma delta (γδ) T cell platform with non-signaling chimeric antigen receptor technology designed for targeted, short-term cytotoxicity without long-term T cell ablation risks. This multi-platform strategy aims to provide potentially curative treatments for conditions such as hemophilia A, hemophagocytic lymphohistiocytosis (HLH), neuroblastoma, and various leukemias and lymphomas.
Target Audience
The primary target audience includes patients with severe genetic disorders and specific types of cancer, as well as healthcare providers and institutions seeking advanced therapeutic options for these conditions.
Features
- **ECO (Expression Cassette Optimization) Platform**: Utilizes bioinformatics and AI to design optimized transgene expression cassettes for enhanced therapeutic efficacy.
- **Lentiviral (LV) and Adeno-Associated Viral (AAV) Vector Technology**: Employed for gene delivery in indications including bleeding disorders and primary immunodeficiencies.
- **Autologous Hematopoietic Stem Cell (HSC) Gene Therapy**: For inherited disorders like Hemophilia A and HLH, involving ex vivo transduction of CD34+ cells.
- **Proprietary Gamma Delta (γδ) T Cell Oncology Platform**: Features non-signaling chimeric antigen receptor (CAR) technology for targeted cancer cell cytotoxicity.
- **Allogeneic Cell Therapy Development**: Focuses on developing off-the-shelf cell therapies for indications such as neuroblastoma, T cell leukemia/lymphoma, and acute myeloid leukemia (AML).
- **cGMP Manufacturing Capabilities**: Operates a 43,000 sq ft facility capable of producing LV and AAV vectors, cell therapy products, and recombinant proteins under current Good Manufacturing Practices.
- **mRNA Technology for CAR-γδ T Cells**: Engineering gamma delta T cells with novel CAR designs for enhanced therapeutic potential in hematologic malignancies.