Bioengineering Life-Altering Gene & Cell Therapies
Expression Therapeutics is a clinical-stage biotechnology company pioneering a curative gene therapy for hemophilia A and best-in-class cell therapies for neuroendocrine tumors. Our work is powered by proprietary AI-based technological breakthroughs and an integrated 43,000 sq ft GMP manufacturing facility.
SSTR2 & PTK7 for Neuroendocrine Tumors
Cutting-Edge Science Designed for Impact
ET3 for Hemophilia A
ET3 for Hemophilia A
ET3 is a next-generation, autologous lentiviral gene therapy designed to restore durable Factor VIII production for people with Hemophilia A. By engineering a patient’s own CD34+ stem cells with our optimized vector, ET3 aims to deliver sustained FVIII levels and long-lasting bleed protection. Early Phase 1 trial results show consistent FVIII expression, elimination of spontaneous bleeds, and a strong safety profile.
SSTR2 & PTK7 for NETs
We use engineered gamma delta (γδ) T cells equipped with non-signaling CARs to deliver potent, controlled tumor targeting with a favorable safety profile. We are advancing SSTR2 and PTK7 CAR-T therapies for neuroendocrine tumors (NETs), with label expansion for pediatric neuroblastoma. This approach aims to combine effective tumor clearance with reduced risk of long-lived persistence or off-target toxicity.
Control from
Discovery to Delivery
Control from Discovery to Delivery
Our 43,000 sq ft manufacturing facility in Cincinnati, Ohio is equipped with advanced modular cleanrooms, GLP labs, and an experienced team led by industry pioneer William Swaney giving us full control of lentiviral and cell therapy manufacturing. Powered by our proprietary ECO and LentET technologies, we are able to accelerate development from IND through commercialization.
Leadership
We bring decades of experience in biotechnology, with a proven track record of developing FDA-approved therapies at global leaders such as Novartis, Pfizer, Bayer, and Amgen. Our team combines deep research and development expertise from leading academic medical centers with specialized knowledge in oncology, hematology, and advanced gene and cell therapy. Together, we are committed to driving innovation that transforms patient care.
Our Technology
Expression Cassette
Optimization (eCO)
ECO is our proprietary AI-driven platform that engineers transgenes for precise, tissue-directed expression across mRNA, AAV, and lentiviral modalities. By optimizing sequence design for potency, stability, and safety, ECO consistently delivers 2–8× higher expression with reduced off-target activity and immunogenicity.
LentET: Proprietary
Lentiviral Delivery
LentET is our next-generation lentiviral vector production system, engineered for higher titers, enhanced safety, and seamless integration with ECO-optimized transgenes. Built on the same AI-driven design principles as ECO, it unifies design, engineering, and manufacturing into a streamlined, clinically scalable workflow.
