Interius BioTherapeutics Doses First Patient with in vivo CAR Gene Therapy for B-cell Malignancies

INT2104 is a novel in vivo chimeric antigen receptor (CAR) gene therapy designed to generate CAR-T and CAR-NK cells in the patient’s body to target CD20-expressing malignant B cells. Interim safety and proof-of-concept data to be presented at a scientific meeting in 1H 2025.

Interius BioTherapeutics, a clinical-stage company engineering targeted, programmable vectors for the precision delivery of genetic medicines, announced that the first study participant has been dosed in INVISE, its first-in-human Phase 1 clinical trial of INT2104, a first-in-class gene therapy that delivers a CAR transgene to generate effector CAR-T and CAR-NK cells in vivo for the targeting of CD20-positive B cells for the treatment of B-cell malignancies.

“Interius was founded with a sense of urgency to provide patients with a new therapeutic option that does not require preconditioning or long manufacturing and wait times. Interius’s breakthrough technology allows precise targeting of gene therapies to specific cells via intravenous administration. This milestone marks the first time that a durable in vivo CAR therapy has been used in the clinic.

INT2104 has the potential to overcome ex vivo CAR therapy challenges with a single-dose, off-the-shelf, widely accessible therapy for the treatment of B cell malignancies enabled by Interius’s programmable platform.

In our preclinical studies, we achieved successful B cell depletion in small and large animal models without chemotherapy and observed no signs of cytokine release syndrome (CRS) or neurotoxicity. We look forward to sharing interim clinical safety and proof-of-concept data at a scientific meeting next year.” – said Phil Johnson, President and CEO of Interius.

“The need for new, accessible treatment options for patients with cancer is critical. Innovative approaches, like in vivo CAR gene therapies, have the potential to transform how we treat cancer, offering faster, single-dose solutions that are less burdensome on patients and treatment centres.” – Michael Dickinson, INVISE Principal Investigator

About INVISE

INVISE (INjectable Vectors for In Situ Engineering) is a first-in-human Phase 1 clinical trial evaluating the safety of INT2104 intravenous infusion in adults with refractory/relapsing B cell malignancies. The study is a global, two-part, multicenter, open-label, single dose design with a dose escalation portion designed to inform the dose of INT2104 to be used in the dose confirmation part of the trial and future studies.

INVISE has been granted Human Research Ethics Committee (HREC) approval and Clinical Trial Notification (CTN) clearance by the Australian Therapeutic Goods Administration (TGA). Additional information and enrollment criteria may be found on clinicaltrials.gov (NCT06539338).

About INT2104

INT2104 is a wholly-owned investigational gene therapy candidate, which specifically targets CD7-positive T and NK cells and delivers a CAR transgene to create effector CAR-T and CAR-NK cells in vivo. The CAR cells target CD20-positive B cells for the treatment of B cell malignancies. Unlike ex vivo CAR-T therapies, INT2104 is an off-the-shelf, single dose treatment, administered systemically through intravenous infusion without the need for lymphodepletion or for any special equipment or training.

About Interius BioTherapeutics

Interius BioTherapeutics is a clinical stage biotechnology company engineering targeted, programmable vectors for the precision delivery of genetic medicines to treat an array of challenging diseases in oncology, autoimmunity and beyond. Leveraging a proprietary platform, Interius has created a differentiated off-the-shelf therapeutic modality designed for broad patient access.

The Company’s lead programs aim to overcome the current limitations of ex vivo chimeric antigen receptor (CAR) T-cell therapy by creating therapeutic CAR cells directly in the patient’s body, avoiding preconditioning chemotherapy, manufacturing bottlenecks, and high costs.

For more information, visit interiusbio.com