Our Science

AAV-delivered gene therapies are among the most promising therapeutics for treating monogenic diseases and delivering gene editors. However, AAV is limited in its ability to deliver large genes due to its limited cargo capacity. StitchR nearly doubles AAV cargo capacity by delivering a large gene or editor split into two AAVs and then recombined at the RNA level to create the full-length RNA. This efficient process can restore therapeutic expression of larger protein to endogenous levels. In vivo data have shown the approach is able to reverse disease phenotypes in animal models. The StitchR platform approach to gene therapy for large genes represents a meaningful and breakthrough solution for patients suffering with diseases caused by large genes.

RNA has become a promising method for developing vaccines and therapeutics. Nevertheless, existing linear mRNA technologies necessitate the incorporation of 5’ caps and 3’ polyadenylation tails to the linear mRNA molecule, which is a complex, inefficient, and costly process. Furthermore, linear mRNAs are susceptible to rapid degradation by endonucleases, diminishing the efficacy of the therapy. Scriptr’s mRNA circularization application, known as CirculR, connects the ends of the mRNA automatically, thereby eliminating the need for caps and tails, as well as the extensive bioprocessing and purification steps required by other circularization techniques, thus potentially enhancing both the efficacy and safety of the desired therapeutic.