Interius BioTherapeutics Administers CAR Gene Therapy to First B-cell Malignancy Patient

Interius BioTherapeutics has announced the commencement of its first-in-human Phase 1 clinical trial of INT2104, an innovative in vivo chimeric antigen receptor (CAR) gene therapy. The company, which specializes in developing targeted, programmable vectors for precise genetic medicine delivery, disclosed that the initial participant in the INVISE study has been dosed. This study represents a significant milestone for Interius, marking the first clinical use of durable in vivo CAR therapy, designed to combat CD20-positive B cell malignancies.

Phil Johnson, M.D., President and CEO of Interius, emphasized the company's mission to provide novel therapeutic alternatives that do not necessitate preconditioning or lengthy manufacturing times. Johnson highlighted the breakthrough nature of their technology, which enables precise targeting of gene therapies to specific cells through intravenous administration. Preliminary studies have demonstrated successful depletion of B cells in both small and large animal models without the need for chemotherapy, and importantly, without causing cytokine release syndrome (CRS) or neurotoxicity. The interim clinical safety and proof-of-concept data are anticipated to be presented at a scientific meeting in the first half of 2025.

Dr. Michael Dickinson, MBBS, FRACP, Principal Investigator of the INVISE trial and Lead of the Aggressive Lymphoma Disease Group at Peter MacCallum Cancer Centre and Royal Melbourne Hospital, underscored the urgency for new, accessible cancer treatments. He noted the potential of in vivo CAR gene therapies to revolutionize cancer treatment with their faster, single-dose approach that alleviates the burden on both patients and healthcare facilities.

The INVISE trial, which stands for INjectable Vectors for In Situ Engineering, is designed to evaluate the safety of INT2104 administered via intravenous infusion in adults with refractory or relapsing B cell malignancies. This global study features a two-part, multicenter, open-label, single-dose design. The dose escalation portion of the trial aims to determine the appropriate dosage for future studies. The trial has received approval from the Human Research Ethics Committee (HREC) and clearance from the Australian Therapeutic Goods Administration (TGA).

INT2104 is an investigational gene therapy candidate that targets CD7-positive T and NK cells, delivering a CAR transgene to generate effector CAR-T and CAR-NK cells within the patient’s body. These CAR cells specifically target CD20-positive B cells to treat B cell malignancies. Unlike traditional ex vivo CAR-T therapies, INT2104 is an off-the-shelf, single-dose treatment administered systemically through intravenous infusion, eliminating the need for lymphodepletion or specialized equipment and training.

Interius BioTherapeutics is a clinical-stage biotechnology company focused on engineering targeted, programmable vectors for the precision delivery of genetic medicines. Their proprietary platform supports the development of differentiated off-the-shelf therapeutic modalities aimed at broad patient access. The company’s primary programs seek to address the limitations of current ex vivo CAR-T therapies by creating therapeutic CAR cells directly within the patient’s body, thereby avoiding preconditioning chemotherapy, manufacturing delays, and high costs.

The interim safety and proof-of-concept data will provide critical insights into the potential of INT2104 as a transformative therapy for patients with B cell malignancies, offering a new and accessible treatment option that could significantly change the landscape of cancer therapy.