Introduction

At RAER Therapeutics, we develop innovative therapies for rare diseases. Leveraging a unique, rapid, and cost-effective screening platform to develop novel and as well as repurposed leads, we address significant unmet medical needs in a market with limited effective treatments. We have advanced projects in RASopathies, TAUopathies, and Adenoid Cystic Carcinoma.

Utilizing Drosophila, mouse and other models to screen promising repurposed compounds and novel protected compounds

By modeling disease mechanisms through genetically engineered Drosophila models and combining robotic high-throughput screening, this approach rapidly tests thousands of compounds to identify potential candidate molecules. These are subsequently validated for safety and efficacy in mammalian models such as mice before advancing to clinical trials. This highly efficient and low-cost workflow significantly shortens drug development timelines and offers new therapeutic possibilities for nearly 7,000 rare diseases. RAER's workflow encompasses the entire process, from constructing genetically engineered Drosophila models and conducting robotic high-throughput screening to mammalian model validation and ultimately advancing candidates to clinical trials.

Using fruit flies, mice and other models to rapidly and accurately identify potential candidate drugs

Systematically establishing the Drosophila model as a cornerstone in precision drug discovery for rare diseases, challenging the traditional screening paradigm dominated by cell lines and mouse models. Its original perspective posits that by leveraging evolutionarily conserved disease pathways, Drosophila serves as a holistic in vivo system capable of efficiently modeling complex human disease phenotypes. This approach enables a "Fly-to-Bedside" translational path, offering a novel theoretical framework for tackling rare diseases that lack adequate animal models. In terms of research methodology, a significant innovation lies in constructing a unique technological platform that integrates high-throughput robotic screening in Drosophila, mammalian validation, and chemical evolution. This system seamlessly connects low-cost, large-scale initial screening in flies with rigorous preclinical validation, substantially enhancing the efficiency of the entire pipeline from compound discovery and lead optimization to identifying clinical candidates. It pioneers a new paradigm for drug development characterized by speed, cost-effectiveness, and scalability.

Project Gains International Recognition

The research was published in the high-impact peer-reviewed journal iScience (Das et al., 2021), detailing the creation of 24 Drosophila models for RASopathies. The publication in a Cell Press journal reflects the scientific rigor and innovation of the achievement, which has gained peer recognition.

The most significant recognition of this approach lies in its clinical translation. Patients with adenoid cystic carcinoma have already received treatments discovered through this platform, and larger-scale clinical trials are currently underway, preliminarily validating its feasibility and potential in practical applications.