Uniquely Positioned at the Intersection of Stem Cell Science & Rare Diseases
Fate Therapeutics is a biopharmaceutical company focused on the discovery and development of adult stem cell modulators that have the potential to save or transform the lives of patients with rare life-threatening disorders. Ongoing research into stem cell science holds great promise for delivering cures and innovative therapies for rare diseases. While much of the investigation has been directed toward stem cell therapeutics derived from pluripotency (e.g., embryonic stem cells and induced pluripotent stem cells), Fate Therapeutics’ strategy for therapeutic intervention is highly-differentiated on three fronts: Fate is focused on the modulation of adult stem cells, its target adult stem cell populations are well-characterized, and its means of intervention have strong medical and/or biological precedent.
Fate is pioneering an innovative therapeutic modality, the ex vivo pharmacologic modulation of hematopoietic stem cells (HSCs) for use in allogeneic HSC transplant, with the intent of curing patients afflicted with blood-borne cancers and genetic disorders of the blood, the immune system and metabolism. HSCs (absent pharmacologic modulation) have been used with medical precedent in stem cell transplantation for over 40 years to treat patients with hematologic malignancies, and 70+ orphan and hyper-orphan diseases have been treated with curative intent using hematopoietic stem cell transplant. The Company’s first clinical candidate, ProHema, is a cord blood-derived cell therapy containing pharmacologically-modulated HSCs, and is currently being investigated in a randomized, controlled, Phase 2 multi-center study in adult patients with hematologic malignancies undergoing double umbilical cord blood transplant.
In addition, Fate is leveraging its understanding of adult stem cell biology to advance a regenerative platform of Wnt-based protein therapeutics for in vivo use. The Wnt family of naturally-occurring signaling molecules is known to play an important physiologic roles in development, stem cell fate and tissue regeneration. Fate has identified and is initially developing a Wnt7a-based protein therapeutic, which targets and expands the muscle satellite stem cell pool. Muscle satellite stem cells are the progenitor population of cells responsible for normal growth, maintenance and repair of skeletal muscle. The satellite cell-mediated regenerative response is depleted as a part of the aging process and in the cycle of muscle degeneration and repair in diseases such as the muscular dystrophies. Wnt7a represents a unique therapeutic opportunity, that can selectively expand the muscle satellite stem cell population and drive tissue growth and repair irrespective of disease etiology. Fate is developing Wnt7a analogs for the treatment of muscle degenerative disorders with an initial focus on muscular dystrophies.
Founded in 2007, Fate Therapeutics is headquartered in San Diego, CA, with a subsidiary in Ottawa, Canada.