HTD leverages Intrexon’s enabling technologies and broad expertise for the development of new, controllable DNA- and cell-based therapies that address unmet medical needs and improve human health. The goal is to facilitate new treatment modalities that provide breakthroughs in therapeutic efficacy and safety.
To this end, HTD utilizes Intrexon’s proprietary RheoSwitch Therapeutic System®
) technology, the first clinically-evaluated gene switch with in vivo
data showing the ability to control gene expression with a broad dynamic range. Intrexon’s switch platform provides a mechanism for titrating therapeutic effects on a patient-specific and predictable basis, as well as a safety switch to rapidly turn off gene-expression. Further, HTD focuses on producing therapeutic effectors in vivo
through a variety of gene delivery approaches including plasmids, exosomes, viral vectors, and genetically-engineered human cells, as well as probiotics to mediate targeted gene delivery and expression.
Key disease application areas for HTD include ophthalmic diseases, rare skin and connective tissue disorders, neuro-muscular diseases, metabolic and gastrointestinal disorders, autoimmune and autoinflammatory conditions, cardiac diseases, infertility, cancer, and neuropathic pain.