Rapafusyn

Rapafusyn Pharmaceuticals is a clinical-stage biopharmaceutical company, founded in 2015, developing therapies for diseases with high unmet medical needs. The company's scientific foundation stems from the work of its founder, Professor Jun O. Liu of Johns Hopkins University School of Medicine, a pioneer in the molecular glue field. His research, dating back to a groundbreaking discovery in 1991, formed the basis for intentionally designing non-degrading molecular glues, a departure from their previously serendipitous discovery.

The company's proprietary RapaGlue™ platform engineers cell-permeable, macrocyclic molecules that leverage the intracellular protein FKBP12 to form a complex with disease-related target proteins. This mechanism creates new protein-protein interactions to inhibit the target's native function, enabling the pursuit of what have been historically considered challenging or undruggable targets. The platform's modular architecture facilitates rapid optimization of drug-like properties, including potency and selectivity, accelerating the discovery process. Rapafusyn utilizes both DNA-encoded libraries (DELs) and arrayed libraries to identify novel therapeutic agents for intracellular and transmembrane proteins, such as transcription factors, ion channels, and transporters.

Rapafusyn's business model is focused on advancing its internal pipeline and pursuing partnerships with pharmaceutical companies. The company's lead program, RAP-0001, is a selective ENT1 inhibitor being developed for the prevention of Acute Kidney Injury (AKI) in patients undergoing cardiac surgery, a condition with no currently approved therapies. Other programs in the pipeline include candidates for oncology. The company is led by CEO Dr. Sean Hu. In June 2024, Rapafusyn announced it had raised a total of $28 million in its Series A financing round, led by 3E Bioventures Capital and Proxima Ventures Ltd., with participation from Lapam Capital, to advance its pipeline and platform capabilities.

Keywords: non-degrading molecular glues, RapaGlues, FKBP12, macrocyclic peptides, undruggable targets, protein-protein interactions, drug discovery platform, Acute Kidney Injury, RAP-0001, ENT1 inhibitor, Johns Hopkins University, Jun O. Liu, biopharmaceutical, clinical-stage, oncology therapeutics, cell-permeable macrocycles, DNA-encoded libraries, targeted protein modulation, novel therapeutics, hard-to-drug targets