Fate Therapeutics is the first biopharmaceutical company dedicated to discovering and developing pharmacologic modulators of stem cells.
Fate has an unmatched ability to precisely understand and rigorously assess stem cell biology for the identification of novel points of therapeutic intervention and for the development of first-in-class therapeutics. Fate has developed a powerful discovery engine, designed to elucidate pharmacologically-induced changes in cell fate in the context of human physiological conditions. To uncover unique biological insights affecting cell fate, Fate’s engine incorporates cutting-edge technologies, including multiplex gene sequencing technologies, bioinformatics, and induced pluripotent stem cell technologies. Fate employs its research technologies to discover stem cell modulator therapeutics that act via two distinct approaches, as explained below.
Ex Vivo Modulation
One approach that Fate employs to modulate stem cells is to pharmacologically ‘activate’ those cells ex vivo prior to administration of these enhanced cells into patients. Fate’s most advanced product, ProHema, which is currently undergoing clinical testing, is an example of such an approach. Fate utilizes a small molecule to pharmacologically modulate hematopoietic stem cells (HSC) to achieve rapid homing to the bone marrow niche, which improves patient outcomes by decreasing time to engraftment. Ex vivo pharmacologic modulation of stem cells confers several advantages:
- Built-in specificity – only the ex vivo cell population is pharmacologically modulated
- Precise pharmacologic exposure – ex vivo modulation enables precise control over the conditions of pharmacologic modulation (e.g., dose, duration and temperature) without the physiologic limitations and off-target effects of systemic exposure
- Achieve supra-physiologic modifications – selected cell properties, such as homing and angiogenesis, may be uniquely targeted and pharmacologically enhanced.
- Validation of pharmacologic effect – modulated cells may be exquisitely characterized to confirm pharmacologic effects prior to patient administration
In Vivo Modulation
Exquisite specificity is required when modulating cell fate in vivo. Using the natural specificity conferred by endogenous proteins as a guide, Fate has built a robust pipeline of novel recombinant human protein therapeutics, each of which acts in vivo and targets adult stem cells and their regenerative mechanisms. Fate’s drug development capabilities are well-suited to successfully productize each of these naturally-occurring, endogenous proteins, including the utilization of certain protein engineering techniques that maintain the biological specificity of the endogenous proteins while improving their pharmaceutical properties and manufacturing potential. Fate’s protein therapeutics pipeline consists of novel, first in class molecules for the treatment of metabolic, cardiovascular and muscle degenerative indications.
Fate is also engaged in the discovery of small molecule stem cell modulators that can be locally delivered into a defined tissue compartment to achieve precise in vivo modulation of a desired stem or progenitor cell type.