Dr. Frank Park and Andrew Wilber Honored with Orphan Dream Award for MiNiRoLu Gene Therapy Innovation

Biomedical researchers Dr. Frank Park and Dr. Andrew Wilber have been honored with the ninth annual Orphan Dream Award. This prestigious recognition celebrates their pioneering work on MiNiRoLu, a next-generation gene therapy platform specifically engineered to treat sickle cell anemia (SCA) and thalassemia. By focusing on these hereditary blood disorders, the researchers are addressing a significant gap in global healthcare through innovative molecular engineering.

The recent award ceremony served as a platform to highlight the duo's significant contributions to medical science. In a unique tribute, their names were featured in the closing credits of the film "Resurrection" as a salute to the 2025 award recipients. This recognition underscores the rapid advancements occurring within the gene therapy sector throughout 2026, particularly concerning treatments for inherited hematological conditions that have long lacked effective cures.

Dr. Frank Park, an associate professor at the University of Tennessee Health Science Center (UTHSC) since July 2016, collaborated with Dr. Andrew Wilber, an associate professor and director at the Southern Illinois University School of Medicine, to develop MiNiRoLu. This innovative therapy is designed to be a more effective, safer, and more affordable alternative to current treatments. Patrick Girondi, the founder of San Rocco Therapeutics (SRT)—a company formerly known as Errant Gene Therapeutics since 1993—lauded their achievements, stating that their work has "significantly enhanced the efficiency, safety, and economic viability" of SRT’s gene therapy framework.

According to Dr. Park, as cited in a UTHSC report dated January 9, 2026, the development of MiNiRoLu is a breakthrough that lowers treatment costs while simultaneously boosting therapeutic success rates. This advancement is vital on a global scale, as sickle cell anemia affects more than 20 million people worldwide. Notably, approximately 80 percent of these cases are concentrated in Sub-Saharan Africa. While the U.S. Food and Drug Administration (FDA) approved two gene therapy products for SCA in 2023, the need for more accessible options remains urgent for these underserved populations.

Existing treatments approved in 2023, such as Casgevy and Lyfgenia, have encountered significant hurdles, including high market prices and complex implementation. Furthermore, these therapies often rely on chemotherapy agents like busulfan, which carries severe side effects such as infertility. MiNiRoLu is specifically designed to mitigate these risks and improve patient outcomes by refining the delivery process. Dr. Wilber’s ongoing research at the Southern Illinois University School of Medicine utilizes cutting-edge techniques to explore globin gene regulation and the differentiation of human erythroid cells.

The presentation of the ninth Orphan Dream Award emphasizes the critical nature of sustained research into orphan diseases. The collaborative efforts of Park and Wilber, representing institutions like UTHSC and the SIU School of Medicine, illustrate the powerful synergy between academic research and the biotechnology industry. Their shared mission to create accessible, high-tech medical solutions represents a cornerstone of modern precision medicine, offering new hope to patients suffering from rare and neglected conditions across the globe.