Therapeutic Applications

Fate Therapeutics is developing small molecule and biologic drug candidates to modulate stem cell pathways to stimulate a desired outcome, such as regenerating tissue or blocking cancer growth. To accelerate therapeutic development in stem cell medicines, Fate Therapeutics is exploring both novel and human-experienced small molecules and biologics to develop lead drug candidates.

OPPORTUNITY FOR INNOVATIVE MEDICINES

  • Fate’s scientific founders have demonstrated the potential of guiding cell fate to restore health and treat disease. The Company is exploring indications in hematology, metastatic cancer, traumatic injury and degenerative diseases.

Hematopoietic Stem Cell Support

Intensive chemotherapy, radiation, and/or immunotherapy are often used to treat patients with hematologic malignancies, such as leukemia and lymphoma, who have not been cured with conventional treatment. These high dose regimens designed to kill the cancer cells will also often destroy the patient’s normal blood and immune systems in the process.  Therefore, hematopoietic reconstitution through the administration of hematopoietic stem cells (HSCs) is necessary to restore normal bone marrow function. Also the immune cells generated by the HSCs, in some cases, play a role in eradicating cancer cells.  Possible sources of HSCs include bone marrow, peripheral blood or cord blood.  The entire procedure is often referred to as hematopoietic stem cell support.

Fate Therapeutics is developing a small molecule drug candidate, FT1050, that has shown promise in improving engraftment of administered HSCs. While Fate’s drug candidate can potentially be used on stem cells from any source, Fate is initially interested in the use of cord blood because:

  • Cord blood is less immunogenic, which means it is less likely to be rejected by the patient’s immune system.
  • Cord blood is more readily available, which reduces medical costs and procedures necessary with obtaining stem cells from bone marrow or a patient’s or donor’s blood.
  • Cord blood has been used in hematopoietic stem cell support since 1988.

The Company has initiated a Phase 1b clinical trial for FT1050 in hematopoietic stem cell support in May 2009. For more information, please see the press release.

Regenerative Therapeutics

Adult stem cells are found naturally in tissues or organs and primarily maintain and repair the tissue in which they are found. Adult stem cells are difficult to isolate and grow in the lab, but research has shown that they can renew themselves and can differentiate to yield the major specialized cell types of the tissue or organ. Some populations of adult stem cells remain quiescent (non-dividing) in areas of the body called “niches” until they are activated by disease or tissue injury. However, in certain instances, the activation is not optimized to promote tissue regeneration. The Company is currently evaluating a number of drug candidates in specific therapeutic applications that currently have limited to no therapeutic options.

Oncology

At the cellular level, cancer is a disease of misregulation of growth and differentiation wherein genes which regulate these pathways are hijacked and create abnormal tissues called tumors. Onco-differentiators (also known as differentiation therapy) are based on the concept that cancer cells can be forced into a more differentiated, less tumorigenic state. This approach may create a new class of therapeutics that have less toxicity than conventional cancer treatments and may also be able to enhance the effectiveness of current chemotherapies. Fate Therapeutics is currently evaluating the potential of several drug candidates as onco-differentiators.