Game-changing therapeutic proteins
Cytokines for cancer treatment
The activation of tumor-fighting immune cells has provided another option for cancer treatment, with some patients experiencing curative effects with several therapeutic modalities. However, the rate of responses leading to long-term remission and increased overall survival is still limited. Combination therapies involving immune-activating cytokines are showing great promise in pre-clinical and clinical studies, pointing the way to increasing therapeutic response rates. Many of these cytokines show pleiotropic effects and sub-optimal half-life in human circulation, which dramatically narrow the therapeutic window. Synthorx is developing several modified cytokines for immuno-oncology, with the most advanced project presently in IND-enabling studies.
Cytokines for autoimmune disease
Autoimmune and inflammatory conditions are driven by the loss of tolerance to self-antigens, with autoreactive T cells driving tissue destruction and production of pathogenic antibodies. These diseases affect about seven percent of the U.S. population and comprise chronic, debilitating conditions often leading to death. Current therapeutic agents mostly address symptomatology with very few effecting disease modification, but none are curative. Many treatments carry significant adverse events that affect quality of life and life expectancy. Synthorx is developing modified cytokines specifically tuned to propagate and enhance the activity of endogenous T regulatory cells. These cells play a critical role in the reversal of autoimmune responses and establishment of tolerance to self-antigens. This approach promotes tolerance without global immunosuppression, with the goal of safer treatments and curative potential.
Broad universe of opportunity
In addition to immune-oncology and autoimmune disorders, many proteins in clinical or pre-clinical development for a host of indications suffer from poor stability, rapid clearance, and/or a sub-optimal pharmacological profile. The Synthorx platform is well positioned to address these issues, in a way that allows for efficient exploration of SAR for efficacy and stability, in a production setup (E. coli) that facilitates a relatively low cost of goods for scale-up production. We are developing highly innovative methods for the in vivo incorporation of non-natural amino acids into therapeutic peptides.