All-purpose HSCs

UCSD spins out GenStem to develop HSC therapies for genetic diseases

While most eyes are on hematopoietic stem cells for their ability to treat blood cancers, a UCSD group has built a body of work showing the immune cell precursors can treat diseases with no relation to the immune system, and has spun out GenStem Therapeutics Inc. to develop the technology.

The company was formed last year based on work from the lab of Stephanie Cherqui, an associate professor in the Department of Pediatrics at the University of California San Diego. Its funding is undisclosed.

In the last month, the researchers have signed a deal with gene and cell therapy company Avrobio Inc. to co-develop a program in cystinosis, and published a preclinical study in Science Translational Medicine showing the cells can be used to treat the neurological disorder Friedreich’s ataxia.

Bone marrow transplants have long been used in oncology, sickle cell disease and a handful of other indications in which immune cells are cancerous or defective. The procedure replaces the diseased cells with hematopoietic stem cells (HSCs) from a disease-free donor that differentiate into mature cells with the relevant myeloid or lymphoid phenotypes.

Cherqui told BioCentury that although it was unclear whether or

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