April 2nd of each year is "World Autism Awareness Day," aiming to increase public understanding, acceptance, and support for Autism Spectrum Disorder (ASD). Autism is not a single disease but a collection of diverse neurodevelopmental conditions, referred to as a "spectrum." Its core features primarily manifest in differences in social communication, a potentially narrow range of interests, and repetitive patterns of behavior or thinking. According to relevant research reports, the prevalence of ASD among children aged 6-12 in China is approximately 0.7%.

In the "valley of death" of drug development, over 80% of preclinical candidate drugs fail in human trials, with "species differences" being a key factor in the inaccurate prediction of efficacy. How can we observe physiological and pharmacological responses in mice during the preclinical stage that are closer to those in humans? Humanized mice are the core technological platform for solving this challenge.

Principles of Gene Knock-in (KI) Technology The core of gene knock-in technology lies in utilizing the cell's own DNA repair mechanisms to achieve precise integration of exogenous genes or specific mutations.

The innovative drug development landscape is defined by cutthroat competition and mounting pressure to reverse declining R&D efficiency. For biopharma stakeholders, the speed of candidate compound screening, the timeline of target validation, and overall R&D cycles, costs and success rates are inextricably linked. As a cornerstone of preclinical in vivo validation, humanized mouse models are make-or-break for downstream target screening and verification—with model development speed and quality directly dictating research momentum.

Gene knockout (KO) refers to a gene editing technique that permanently inactivates the function of a specific gene in an organism's genome through targeted deletion, insertion, or replacement, mediated by homologous recombination or nuclease-induced DNA double-strand break repair mechanisms.