Jorna Therapeutics is a biotechnology company focused on developing new medicines. The company was founded by scientists with backgrounds from institutions such as the Broad Institute and Harvard. Jorna Therapeutics is a spin-off from Quantum Sky and operates from Cambridge, Massachusetts. It aims to address challenging problems in the field of genetic and degenerative diseases.
Targeting RNA
Jorna Therapeutics focuses on RNA-targeted therapeutics, utilizing advanced RNA editing systems designed with artificial intelligence. Ribonucleic acid, or RNA, serves as a messenger molecule that carries instructions from DNA to guide the production of proteins, which are the building blocks and functional machinery of cells. Targeting RNA offers a promising avenue for drug development because it allows for interventions at a stage between the genetic blueprint (DNA) and the final protein product.
Jorna’s approach involves designing AI-guided editors that can introduce specific changes into target messenger RNA (mRNA) sequences. This technology can enable large fragment editing on the target mRNA, which means that patients with various mutations of the same gene could potentially be treated with a single, unified therapy. The company’s proprietary platform, powered by a quantum mechanics-based AI system known as SkyEngine, analyzes interatomic interactions within and between proteins and small molecules to identify and design sequences. This allows for the precise design of proteins necessary for highly efficient RNA editing, offering a novel method to correct or modify gene expression without permanently altering the DNA itself.
Therapeutic Focus Areas
Jorna Therapeutics is committed to addressing devastating diseases through its innovative RNA editing systems. The company’s technology is particularly suited for genetic and degenerative conditions, aiming to provide transformative treatments for patients who previously had limited or no therapeutic options. This includes conditions where the endogenous regulatory circuitry and modifications of genes need to be kept intact, as their technology produces desired products at native expression levels, unlike some gene replacement therapies.
The Significance of Jorna’s Approach
Jorna Therapeutics’ RNA-targeting approach represents a significant advancement in drug discovery and development. By focusing on RNA editing, the company offers a distinct alternative to traditional drug development methods that often target proteins or DNA directly. This allows for a unique level of precision in addressing disease mechanisms. The ability to introduce mutations into target RNA using multiple approaches, guided by AI, enables the development of therapies that can efficiently and safely modify previously inaccessible RNA targets.
This method holds the potential to address unmet medical needs, particularly for genetic and degenerative diseases where current treatments are limited. The company’s AI-guided editor design, combined with high-throughput systems, allows for the precise engineering of proteins to enhance RNA editing efficiency.