张锋进军 in vivo CAR-T，临床前数据优异，即将开展人体临床试验，利用LNP-mRNA治疗自身免疫病

Core Insights - Aera Therapeutics presented preclinical data for its candidate therapy AERA-109 at the 10th CAR-TCR Summit, targeting various B cell-mediated autoimmune diseases, showing significant B cell depletion in humanized mouse models and non-human primates [3][19][22] - The company plans to advance AERA-109 into clinical development by mid-2026, utilizing a proprietary targeted lipid nanoparticle (tLNP) delivery platform for in vivo CAR-T cell therapy [3][22] Company Overview - Aera Therapeutics was founded by CRISPR pioneer Professor Zhang Feng and other executives from Alnylam Pharmaceuticals, completing a $193 million financing round in 2023 [4][5] - The company's vision is to unlock the potential of gene therapies and transform human health by overcoming existing delivery technology limitations [4] Technology and Platforms - Aera Therapeutics has developed three proprietary delivery platforms: targeted lipid nanoparticles (tLNP), antibody-oligonucleotide conjugates (AOC), and protein nanoparticles (PNP) [12][14][15] - The tLNP platform is designed to target cell types and tissues beyond the liver, enhancing therapeutic applications [14] - AOC combines the targeting ability of antibodies with the programmable functionality of oligonucleotides, aiming to expand applications beyond traditional immunology and oncology [15] - PNP is a disruptive delivery platform that utilizes human endogenous proteins for gene therapy delivery, offering modularity and scalability [10][15] Research and Development - AERA-109 utilizes the tLNP platform to deliver CAR mRNA directly to CD8+ T cells in vivo, eliminating the need for pre-treatment and enabling the generation of CAR-T cells within the body [19][20] - Preclinical results demonstrated dose-dependent CAR-T cell generation and significant B cell depletion in both humanized mouse models and non-human primates [20][21] - The therapy showed good tolerability with no significant adverse clinical manifestations or changes in hematological or serum chemistry parameters [22]