TS-000738 — In all living cells, extracellular vesicles (EVs) are naturally produced. Drs. Kevin Cassady and Uksha Saini have developed a streamlined approach to utilize these EVs to deliver biologics and clinical drug treatments that permit repeated treatments without the possibility of limiting therapeutic delivery due to the natural immune response. This method produces an immune inert drug delivery system that, unless the EVs are designed to encode immunostimulatory molecules, should not invoke an immune response. Our preliminary studies show that we can load extracellular vesicles (EVs) with a therapeutic payload of our choice. Retroviruses co­opt and use much of the same pathways involved in cellular EV production for virus exit of the infected cell. We therefore hypothesized that Lentiviruses (a non-replicating retrovirus commonly used in the lab for gene expression) would provide a modifiable platform to generate therapeutic gene products that that would be preferentially transported and packaged into cellular EVs. This platform could then be modified and developed as a potential therapeutic delivery platform.

TS-000194 — Investigators at Nationwide Children's Hospital have developed a novel splicing corrective treatment to modulate the splicing of p53-modifier Murine Double Minute 2 (MDM2) in cancer.

TS-000193 — SMA currently has no treatment and is the result of an mRNA splicing defect which we have found can be corrected with heat shock. We propose using increased temperature, small molecule activators of heat shock response, or other mechanisms for inducing the heat shock response for treatment of SMA.

TS-000113 — Technology Overview Researchers at the Research Institute have developed a stably transfected cell line that expresses wild type survival motor neuron gene-2 (SMN2) that can be used as a drug discovery tool aimed at gene splicing correction. SMN2 is a potential therapeutic target for Proximal Spinal…