Enhanced nucleic acid therapeutics: synthetic sequence elements for boosting protein expression in genetic medicines - nucleic acid-based medicines have demonstrated remarkable potential in preventing…

The National Center for Advancing Translational Sciences awarded Primrose Bio, Inc. $951,630 on July 8, 2026, under CFDA 93.350 to develop synthetic noncoding sequence elements that enhance protein expression in nucleic acid therapeutics.

Primrose Bio will utilize an ultra-high-throughput platform for discovering and optimizing noncoding sequence elements—including 5' untranslated regions and internal ribosome entry sites—to create a panel of synthetic sequences that boost efficacy in genetic medicines while reducing manufacturing costs and dosage requirements. The company will conduct this work in collaboration with an established mRNA medicines company and partner with SoCal Biosciences for testing. The resulting sequence elements will be designed for integration into nucleic acid therapeutics targeting applications including cancer therapeutics, with commercial pathways established through existing and new industry partners.

Performance takes place in San Diego, California, with a period of performance through May 31, 2028. This Project Grant supports NCATS's mission to accelerate the translational science pipeline by removing bottlenecks in the development and testing of diagnostics and therapeutics.

Enhanced nucleic acid therapeutics: synthetic sequence elements for boosting protein expression in genetic medicines - nucleic acid-based medicines have demonstrated remarkable potential in preventing and treating a wide range of diseases, as exemplified by their crucial role in the covid-19 pandemic response. However, several challenges remain, including inherent instability, manufacturing complexities, and the need for efficient delivery mechanisms. The development of new nucleic acidbased medicines offers potential solutions to these issues by reducing dosage requirements, lowering manufacturing costs, and improving efficacy. Noncoding sequence elements such as 5' untranslated regions and internal ribosome entry sites required for protein expression from these medicines offer promising targets to improve efficacy, lower manufacturing costs, and pave the way for the next generation of genetic medicines. In collaboration with an established mrna medicines company, primrose BIO has created a novel ultra-high-throughput platform for non-coding sequence element discovery and evolution. This platform has higher throughput and flexibility than other non-coding sequence discovery systems described to date and has unlimited potential to discover highly efficient synthetic non-coding sequence elements that stimulate protein expression. In this project, primrose BIO will utilize this platform to create a panel of optimized synthetic 5' noncoding sequence elements for enhancing protein expression that can be seamlessly integrated into nucleic acid therapeutics. These sequences elements will be tested in partnership with socal biosciences. The sequence elements developed in this program will find direct commercial outlets through existing and new partners developing disruptive therapies, such as cancer therapeutics.