The Mass General Brigham Gene and Cell Therapy Institute (GCTI) today announced it has launched the RNA Therapeutics Core, a first-of-its-kind, state-of-the-art facility and resource to advance the use of RNA technologies within and beyond the Mass General Brigham research ecosystem. This new Core is dedicated to accelerating the exploration of novel therapeutic targets to effectively translate RNA-based medicines into clinical practice by leveraging advanced RNA vectors and delivery systems.
Until now, a Core of this kind has not existed within an academic setting. With this launch, the RNA Therapeutics Core enables access to high-quality tools that are critical for researchers who are investigating novel gene and cell therapies. Available to both industry and Mass General Brigham investigators, the Core will provide stock circular RNAs (circRNAs) encoding reporter proteins as well as custom manufactured circRNAs containing almost any sequence of interest. Multiple different RNA vector options are available to support both coding and noncoding applications. The Core has also developed unique codon optimization algorithms that improve protein expression from circRNA beyond existing state of the art algorithms.
CircRNA is a type of single-stranded RNA, which gets its name because of its molecular shape (which forms a continuous loop, unlike linear RNA). CircRNA is found naturally in humans and can have specialized functions within the body. When adapted as a coding RNA technology, circRNA can yield higher levels of protein expression for an extended duration compared to modified linear mRNA, while costing significantly less than mRNA due to its manufacturing simplicity. The Core's synthetic circRNAs will primarily be used for gene expression and noncoding functions, similar to viruses, plasmids or messenger RNA – elements that are key for developing gene and cell therapies.
Gene and cell therapy research is paving the way for future, novel treatments for patients with a wide range of critical illnesses, and we believe the application of circular RNA will only help get us even further. We are thrilled to be opening our RNA Therapeutics Core to help advance preclinical research into first-in-human clinical trials – for the hundreds of our own MGB gene and cell researchers, but also industry and investigators at large."
Nathan Yozwiak PhD, Head of Research, Mass General Brigham GCTI
The Core was created by renowned RNA expert R. Alexander Wesselhoeft, PhD, Director of RNA Therapeutics Core at the Mass General Brigham GCTI. Wesselhoeft created a novel RNA circularization technology while at MIT. His work led to key publications that substantiated the field of synthetic circular RNA and enabled their use for investigative therapeutic applications, and later, co-founded Orna Therapeutics. Under his leadership, the RNA Therapeutics Core will serve to support researchers exploring next-generation RNA medicine platforms and therapeutic applications of circular RNA.
"The range of disease and research areas for which circular RNA can be beneficial is wide-ranging and diverse," said Wesselhoeft. "From cancer vaccines to transient CAR-T approaches, infectious disease vaccines, tissue regeneration and beyond, our Core is focused on producing high-quality circRNA to support academic research and biotechnological innovation. We have the capability to formulate circRNA into lipid nanoparticles and plan to offer good manufacturing practice grade materials in the future to support the entire spectrum of research and development, from initial discovery to clinical application."
The Mass General Brigham Gene and Cell Therapy Institute was established in 2022 to fuel the discovery and development of targeted, transformative treatments that have the potential to cure diseases or halt their progression. The Institute unites more than 500 researchers and clinicians dedicated to advancing gene and cell therapy for first-in-human clinical trials, and ultimately, life-saving treatments for patients.
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