Remix Therapeutics

Remix Therapeutics, founded in 2020, aims to transform patients’ lives through the modulation of RNA processing. The company develops small molecule therapies that address the underlying drivers of disease. Central to its efforts is the proprietary REMaster™ drug discovery platform, which identifies druggable target sites and active chemical matter to bring new medicines to patients. Remix Therapeutics has built an extensive transcriptome database leveraging machine learning to identify modulatable sites across diverse target areas, identifying more than 1.6 million novel exon targets in the mRNA degradation space alone. The company has also developed a proprietary genetic toolkit to validate that these novel exon targets can be modulated in cells, leading to functional outcomes on gene and protein expression. Additionally, Remix Therapeutics has a proprietary chemical library enriched for compounds that can interact with RNA processing mechanisms. The company’s clinical development programs are designed by industry experts and informed by patients and clinicians to advance relevant and impactful therapies. REM-422, an oral small molecule mRNA degrader, is currently in Phase 1 clinical trials for the treatment of Adenoid Cystic Carcinoma (ACC) and Acute Myeloid Leukemia/Myelodysplastic Syndromes (AML/MDS). REM-422 targets MYB, a transcription factor known to be an oncogenic driver of multiple solid tumors and hematological malignancies, and functions by facilitating the incorporation of poison exons within the mRNA transcript, leading to nonsense-mediated decay (NMD) of the transcript. This process is designed to induce the reduction of MYB mRNA and protein expression, providing antitumor activity in MYB-dependent human tumor models. REM-422 has been granted Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of ACC and AML. The company has announced collaborations and license agreements with Roche and Johnson & Johnson Innovation Medicine (formerly Janssen) for the discovery and development of small molecule therapeutics that modulate RNA processing. Located at 100 Forge Road, Suite 400, Watertown, USA 02472, Remix Therapeutics continues to advance multiple drug discovery programs towards the clinic.