Interius BioTherapeutics, a leading biotechnology company focused on developing in vivo cell-specific gene medicines, presented preclinical data across six abstracts at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting, held in Boston, Massachusetts.
The data presented focused on INT2104, Interius’s lead candidate for treating B-cell malignancies. INT2104 is a targeted lentiviral vector-based therapy that utilizes CD7 targeting to specifically transduce T cells and NK cells upon intravenous (i.v.) administration, without the need for preconditioning chemotherapy.
In vivo studies conducted in humanized mice and cynomolgus macaques demonstrated that a single i.v. administration of INT2104 effectively transduced both T cells and NK cells, reprogramming them into functional chimeric antigen receptor (CAR) cells. Biodistribution and immunohistochemical analyses confirmed vector-targeting specificity. The in vivo generated autologous CAR cells selectively targeted CD20-positive cells, leading to B-cell depletion in both animal models. Notably, one macaque exhibited persistent B-cell aplasia for one year, with no detectable B cells in the peripheral circulation, spleen, bone marrow, or lymph nodes at autopsy.
A formal GLP toxicology study established a robust safety profile for INT2104. In 20 vector-treated cynomolgus macaques, no clinical signs or symptoms of toxicity were observed, and no laboratory abnormalities were recorded over the 180-day study period. These findings, together with an extensive preclinical data package, support the clinical entry of INT2104 for B-cell malignancies this year and the rapid development of the company’s second program for autoimmune diseases.
The ASGCT Annual Meeting is a leading international forum for the presentation and discussion of the latest advances in gene and cell therapy. Interius’s presentation of preclinical data on INT2104 highlights the company’s commitment to developing novel and effective gene therapies for the treatment of B-cell malignancies and autoimmune diseases.