UHKT Launches Phase I Trial of Linea DNA-Enabled CAR T-Cell Therapy for AML

Key Insights

• The State Institute for Drug Control of the Czech Republic has approved a Phase I clinical trial for UHKT-CAR123-01, a CD123-specific autologous CAR T-cell therapy.
• UHKT-CAR123-01 utilizes Applied DNA's Linea DNA in its manufacturing process, aiming to reduce costs and timelines associated with CAR T-cell production.
• The trial targets relapsed and/or refractory acute myeloid leukemia (AML), addressing a critical unmet need due to high treatment failure rates with current palliative options.

The Institute of Hematology and Blood Transfusion (UHKT) in Prague has initiated a Phase I clinical trial of UHKT-CAR123-01, an investigational CD123-specific autologous CAR T-cell therapy, following approval from the State Institute for Drug Control of the Czech Republic (SÚKL). This therapy is intended for patients with relapsed and/or refractory acute myeloid leukemia (AML). The trial marks a significant step in leveraging synthetic DNA technology to improve CAR T-cell therapy manufacturing.

Addressing AML Treatment Challenges

AML is a hematologic malignancy characterized by a high rate of treatment failure. Current treatment options are often limited to palliative care, highlighting the urgent need for innovative therapeutic approaches. CAR T-cell therapies represent a promising avenue, but their widespread clinical use has been hindered by high costs and lengthy manufacturing processes, largely due to the reliance on viral vectors.

Linea DNA's Role in Streamlining CAR T-Cell Production

UHKT-CAR123-01 aims to overcome these challenges by employing Applied DNA's Linea DNA in a non-viral workflow. This approach seeks to reduce both manufacturing costs and timelines. Preclinical data has demonstrated that UHKT's Linea DNA-empowered process enables the rapid production of substantial and cost-efficient CAR T-cell yields with high potency.

Expert Perspectives

"The use of Linea DNA illustrates our innovative approach to finding new and best-in-class treatments for patients with relapsed or refractory AML," stated Dr. Jan Vydra, principal investigator of the UHKT-CAR123 clinical trial.

Pavel Otáhal, Ph.D., scientific project leader at UHKT, added, "The Linea DNA platform enables the very rapid abiotic production of expression vectors usable for highly effective electroporation-based CAR-T manufacturing compared to plasmid-based vectors... This is an incredible milestone for UHKT and one that we could not have achieved without the commitment of the Applied DNA team."

Applied DNA's Perspective

Applied DNA CEO Dr. James A. Hayward commented, "We congratulate UHKT on their progress into the clinic. Their accomplishment is also a significant milestone for Linea DNA as we look to support additional customers expected to initiate clinical trials in calendar 2025."