Harnessing the Promise of Adult Stem Cells to Transform the Lives of Patients with Orphan 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 including certain hematologic malignancies, lysosomal storage disorders and muscular dystrophies. Adult stem cells play a key role in the growth, maintenance and repair of many tissues and organ systems in the body. Due to their natural ability to self-renew, and to regenerate and repair diseased or damaged tissue, adult stem cells hold considerable therapeutic promise. Our novel approaches utilize established pharmacologic modalities, including small molecules and therapeutic proteins, and target well-characterized biological mechanisms to enhance the therapeutic potential of adult stem cells. Based on our deep understanding of these key biological mechanisms that guide the fate of adult stem cells, we have built two product platforms aimed at the development of therapeutics for the treatment of orphan diseases.
Our HSC modulation platform focuses on the ex vivo pharmacologic optimization of hematopoietic stem cells, or HSCs, which are adult stem cells that regenerate all types of blood cells throughout a person’s lifespan. HSCs have been used for decades in a potentially curative or life-saving procedure called hematopoietic stem cell transplant, or HSCT. This procedure is most commonly performed in patients with hematologic malignancies to replace a diseased hematopoietic system with a healthy one, and has also been used to treat over 50 rare genetic disorders, many of which are life-threatening and lack alternative therapeutic options. While over one million HSCT procedures have been performed to date, we believe HSCs have not been pharmacologically optimized to improve patient outcomes. Fate is pioneering an innovative therapeutic modality, the ex vivo pharmacologic optimization of HSCs for use in allogeneic HSCT, with the intent of curing patients afflicted with blood-borne cancers and genetic disorders of the blood, the immune system and metabolism.
Our satellite stem cell modulation platform focuses on the in vivo pharmacologic activation of satellite stem cells, which are adult stem cells that regenerate muscle throughout a person’s lifespan. The regenerative capacity of satellite stem cells in skeletal muscle is exhausted both as we age and in degenerative conditions such as muscular dystrophies. We have identified Wnt7a as a natural promoter of satellite stem cells to drive muscle regeneration and are initially focused on developing Wnt7a protein analogs for the treatment of muscular dystrophies. Our Wnt7a protein analogs offer a unique therapeutic opportunity, selectively expanding the muscle satellite stem cell population and driving tissue growth and repair irrespective of disease etiology. We believe that our regenerative approach for treating muscular dystrophies holds significant therapeutic promise and is distinct from other approaches, which focus on preventing muscle degeneration.
Founded in 2007, Fate Therapeutics is headquartered in San Diego, CA.