ASC Therapeutics products

Genetic diseases are caused by defective genes. Gene replacement therapy aims to treat such diseases by introducing a corrective gene into a patient’s body; the corrective gene can be a normal copy of the defective gene, or a copy that is engineered to provide enhanced biological activity; our focus for gene replacement therapy starts by identifying gene variants with superior therapeutic potency and to achieve optimal and sustainable production of the functional protein once administered to the patient.

Genome editing technologies enables permanent modifications to the genome by harnessing the activities of certain enzymes (e.g. nucleases and integrase) and invoking DNA repair mechanisms at specific locations in the genome. As a therapeutic modality, genome editing can correct a defective gene, disrupt a deleterious gene, or target a therapeutic gene to a precise location in the genome for safe and optimal expression of the therapeutic protein. We are applying the optimized CRISPR-Cas9 and in-house-developed TARGATT™ gene editing technologies for therapeutic development, focusing on improving both the potency and specificity of editing.

We are developing gene and cell-based therapies. Gene therapy is addition, alteration or removal of genes within cells and biological tissues to treat diseases. A correcting genetic agent can be delivered directly to the blood stream in vivo, or to the affected tissue in situ, or the targeted cells can be modified ex vivo and injected back to the patients. Cell-based therapies involve injecting or implanting intact or genetically modified cells to the patients, where the cells themselves act as a curative agent.

Stem cells, including inducible pluripotent stem cells, mesenchymal stem cells (MSCs) and other primary pluripotent cells, hold the potential to treat a variety of diseases. We are currently focusing on developing an allogeneic decidua stromal cells (DSCs)-based platform to treat severe immune-mediated diseases. Our pre-clinical and clinical data suggest that the immunomodulatory activity of DSCs is superior to that of MSCs.