HN-2301: An Investigational In Vivo mRNA-Based CAR-T Cell Therapy for Autoimmune Diseases

I. Executive Summary

HN-2301 is an investigational therapeutic candidate being developed by Shenzhen MagicRNA Biotechnology Co., Ltd. It represents an innovative approach in the field of cell therapy, specifically designed as an in vivo generated Chimeric Antigen Receptor T (CAR-T) cell therapy. The core technology involves the delivery of messenger RNA (mRNA) encoding a CD19-targeting CAR, utilizing the company's proprietary Engineered Cell-targeted Lipid Nanoparticle (EnC-LNP) delivery platform. This method aims to transiently reprogram a patient's own T cells within their body to recognize and eliminate CD19-expressing B cells.

The primary therapeutic indications for HN-2301 are B-cell mediated autoimmune diseases, with initial clinical investigations focusing on Systemic Lupus Erythematosus (SLE) and Myasthenia Gravis (MG). Preclinical studies, including those in non-human primates and murine models of SLE, have reportedly demonstrated successful in vivo CAR-T cell generation and B-cell depletion, with therapeutic effects observed in disease models. HN-2301 is currently advancing through IND-enabling studies and into early-phase clinical trials in China.

This therapeutic modality holds the potential to overcome several limitations associated with traditional ex vivo CAR-T cell therapies, such as complex manufacturing processes, high costs, and the need for patient lymphodepletion. By leveraging transient mRNA expression and targeted LNP delivery, HN-2301 aims to offer a safer, more accessible, and potentially scalable treatment option for patients with severe autoimmune conditions. The ongoing and upcoming clinical trial results, along with further elucidation of its proprietary EnC-LNP technology, will be critical in determining the ultimate clinical utility and positioning of HN-2301 in the evolving landscape of autoimmune therapies.