NanoGhost: A Stem Cell-Based Off-the-Shelf Nano-Delivery Platform for Targeted Drug and Gene Therapy

Effective and targeted delivery remains one of the major challenges in drug delivery and particularely in RNA and gene-based therapeutics. NanoGhost is an innovative nano-delivery platform derived from the whole membrane of mesenchymal stem cells (MSCs), designed to combine the intrinsic tumor-homing and immunomodulatory properties of stem cells with a scalable, off-the-shelf nano-vesicle formulation.NanoGhost vesicles preserve key membrane proteins from parental MSCs, enabling selective targeting of pathological tissues, including tumors and inflamed microenvironments. The platform is engineered to encapsulate a variety of small molecules peptides, RNA cargo (e.g., siRNA, mRNA, microRNA) as well as gene-editing tools (e.g., CRISPR-Cas systems), ensuring efficient intracellular delivery with minimal off-target effects.We demonstrate that NanoGhost achieves high stability, biocompatibility, and loading efficiency, while bypassing common limitations of viral vectors and synthetic nanoparticles as well as bypasing the blood brein berriers. Preclinical studies show successful delivery of theraputics leading to GBM inhibition as well as RNA payloads delivery to other tumor sites in vivo. Unpublished data demonstrate that NGs can envelop LNPs thus adding to LNPs the inhernt taegeting ability of MSC to tumors opening the door for a safer nontoxic high loading capacity to the new biohybrid particles.