RENATUS UNVEILS NEXT-GENERATION SINGLE-ISOMER CYCLODEXTRIN

Hydroxypropyl-β-cyclodextrin (HPβCD) is the most widely used cyclodextrin in pharmaceutical applications and has long been recognized for its ability to modulate cholesterol. Originally adopted as a pharmaceutical excipient, it later demonstrated therapeutic potential through its capacity to mobilize and redistribute accumulated cholesterol in cells. Since its first clinical administration in Niemann–Pick Type C (NPC) patients in 2009, HPβCD has been evaluated in multiple human trials, including those for Alzheimer’s disease and atherosclerosis, where cholesterol dysregulation plays a key pathogenic role.

Despite its clinical utility, HPβCD presents several well-recognized limitations: (1) it is a heterogeneous mixture composed of millions of positional isomers, (2) it requires high therapeutic doses (often exceeding grams per kilogram), and (3) it exhibits a narrow safety margin. The heterogeneity, in particular, poses a critical CMC (Chemistry, Manufacturing, and Controls) challenge—making it difficult to ensure batch-to-batch consistency, define precise quality attributes, and establish clear structure–activity relationships (SAR) necessary for target engagement and mechanism elucidation.

In response to these long-standing challenges, Renatus, a biotechnology company pioneering next-generation cholesterol modulators, announced today it has filed a new patent application covering a novel class of single-isomer cyclodextrins. Unlike HPβCD, which contains millions of molecular variants, a single-isomer cyclodextrin has one defined and uniform structure. The single-isomer design provides unprecedented control over purity, reproducibility, and molecular characterization, overcoming the key limitation of HPβCD. Furthermore, the single isomers demonstrate up to 50-fold higher efficacy in cholesterol solubilization compared to HPβCD, suggesting that lower effective doses and an improved safety margin may be achievable.

Renatus CEO Heegon Kim stated, “This single-isomer technology represents the next phase of cyclodextrin innovation, overcoming the molecular complexity of HPβCD and delivering a cleaner, more uniform compound to patients. In addition to the superior molecular quality as single isomers, these candidates exhibit markedly enhanced cholesterol solubilization efficacy. Together, these advances position our single-isomer cyclodextrins as a promising next-generation alternative to HPβCD, with the potential to unlock broader therapeutic utility across cholesterol-driven diseases.”

Kim further added, “Our research has already demonstrated that single-isomer cyclodextrins can effectively reduce cellular cholesterol. We are actively advancing these candidates in diseases where HPβCD has previously shown therapeutic potential, including atherosclerosis and Alzheimer’s disease. In parallel, we are exploring additional indications such as cancer and fatty liver disease, where dysregulated cholesterol metabolism plays a key role. Ultimately, we aim to reshape the therapeutic use of cyclodextrins through disease-optimized single-isomer molecules.”

About HPβCD

Hydroxypropyl-β-cyclodextrin (HPβCD) has been clinically explored for its ability to mobilize intracellular cholesterol, beginning with its first administration to Niemann–Pick Type C (NPC) patients in 2009 under compassionate-use programs. Since then, HPβCD has progressed into both intrathecal and intravenous clinical studies, including the development of VTS-270 (Adrabetadex) and Trappsol® Cyclo™. Beyond NPC, HPβCD has also been investigated in other cholesterol-related conditions including atherosclerosis and Alzheimer’s disease. While clinical studies have shown signs of therapeutic potential, no HPβCD-based therapy has yet received regulatory approval.

About Renatus

Renatus is a biotechnology company developing next-generation cholesterol modulators designed to address the underlying lipid dysregulation that drives a wide range of diseases including atherosclerosis, Alzheimer’s disease, chronic kidney disease, metabolic dysfunction-associated steatohepatitis (MASH), and cancer. The company focuses on translating innovative cyclodextrin chemistry into safer and more effective therapeutic options, aiming to broaden the clinical utility of cyclodextrins and deliver meaningful benefits to patients who require cholesterol modulation. For more information, please visit www.renatustx.com

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