WiNK Therapeutics, a biotech startup spun out of the University of Miami Miller School of Medicine, is pioneering a novel RNA aptamer technology to combat diabetes. The company's approach targets the underlying causes of both Type 1 and Type 2 diabetes by delivering therapeutics directly to pancreatic beta cells, potentially offering a long-sought-after cure.
Addressing Beta Cell Loss in Diabetes
Type 1 diabetes is characterized by the autoimmune destruction of insulin-producing beta cells, while Type 2 diabetes involves insulin resistance and eventual beta cell failure. In both conditions, the loss of functional beta cells leads to impaired blood sugar control, necessitating insulin injections or other medications. WiNK Therapeutics is focusing on a two-pronged strategy: regenerating existing beta cells and preventing their destruction by the immune system.
RNA Aptamers: A Targeted Approach
Dr. Paolo Serafini, co-founder of WiNK Therapeutics and associate professor at the University of Miami, developed RNA aptamers that can selectively bind to beta cells. These aptamers act as delivery vehicles, carrying therapeutic payloads directly to the target cells. This targeted approach minimizes off-target effects and maximizes the therapeutic impact on beta cells.
In a 2022 publication in Nature Communications, Dr. Serafini's team demonstrated the effectiveness of aptamers in targeting beta cells. WiNK Therapeutics is now refining this technology to deliver molecules that can regenerate and protect these cells.
Regenerating and Protecting Beta Cells
WiNK's strategy involves silencing a gene that normally inhibits cell division, a mechanism inspired by cancer research. By selectively silencing this gene in beta cells using RNA aptamers, the company aims to stimulate the regeneration of insulin-producing cells.
To prevent the immune system from attacking the regenerated beta cells, WiNK is leveraging checkpoint inhibitors, which are commonly used in cancer immunotherapy. These inhibitors modulate the immune response, preventing T cells from destroying the beta cells. The targeted delivery of checkpoint inhibitors via RNA aptamers ensures that only autoreactive T cells are affected, minimizing systemic immunosuppression.
Clinical Trial Outlook
WiNK Therapeutics is currently developing a combination molecule that incorporates an RNA aptamer for beta cell targeting, along with molecules that promote regeneration and immune protection. The company is planning preclinical validation studies to seek FDA approval for a Phase 1 clinical trial in diabetes within the next two years. According to Dr. Serafini, this therapy could potentially treat diabetes with no off-target efficacy.
Expanding the Technology
While the primary focus is on diabetes, WiNK Therapeutics is also exploring the potential of its aptamer technology in cancer therapy. The approach involves targeting myeloid cells within tumors to deliver anti-cancer agents, potentially offering a new strategy for treating various cancer types. However, the cancer research is currently on hold as the company prioritizes the development of its diabetes therapy.
