ASC Therapeutics is focused on developing innovative gene and cell therapies aimed at curing intractable and rare diseases, particularly blood disorders. The company utilizes its proprietary gene editing platform, TARGATT™, alongside advanced CRISPR/Cas9 technologies, to facilitate precise genetic modifications. Their therapeutic pipeline includes several preclinical and clinical programs, notably for hemophilia A and B, as well as β-thalassemia. The gene therapy approach involves delivering corrected genetic material directly to patients, either through in vivo methods or by modifying cells ex vivo before reintroducing them into the patient. This strategy aims to restore normal function in affected tissues, thereby providing potential cures for conditions that currently lack effective treatments.

In addition to gene therapy, ASC Therapeutics is also advancing cell-based therapies, which involve the injection of intact or genetically modified cells that act as therapeutic agents. The company employs state-of-the-art gene delivery systems, including recombinant Adeno-Associated Viruses and lipid nanoparticles, to enhance the efficacy of their treatments. Their commitment to collaboration with leading academic and industry partners further strengthens their research and development efforts, ensuring that they remain at the forefront of biopharmaceutical innovation. Through these technologies, ASC Therapeutics aims to revolutionize the treatment landscape for patients suffering from challenging diseases, ultimately improving their quality of life.

United States

ASC Therapeutics focuses on the development of curative therapies for rare diseases. Our gene, CRISPR and cell therapies were developed at Applied StemCell, a life sciences company for over 12 years, including proprietary gene editing technologies. Our development pipeline includes several preclinical stage projects focusing on monogenic blood disorders that exhibit a high genetic penetrance and conditions requiring immunomodulatory interventions. Contact us for partnership opportunities

ASC Therapeutics

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