Biotechnology companies join forces in developing treatment for X-SCID

Jasper Therapeutics, Inc., a biotechnology company focused on blood stem cell therapies, and Graphite Bio, Inc., a biotechnology company focused on gene editing therapies to treat or cure serious diseases, announced a research and clinical collaboration for a treatment for X-SCID.

X-SCID, which stands for X-linked severe combined immunodeficiency, is a genetic disorder that interferes with the normal development of the immune system, leaving infants vulnerable to infections that most people can easily fight off. One treatment for X-SCID involves a blood stem cell transplant, in which the patient’s defective stem cells are wiped out with chemotherapy or radiation to make room for normal blood stem cells to take their place. Unfortunately, the problem with chemotherapy or radiation in young infants is that it can lead to lifelong effects such as neurological impairment, growth delays, infertility, and risk of cancer.

Fortunately, Jasper Therapeutics has developed JSP191, a non-toxic alternative to chemotherapy and radiation. It is an antibody that works by targeting and removing the defective blood forming stem cells. The approach has previously been used in a CIRM-funded clinical trial ($20M award) for X-SCID.

Graphite Bio has developed GPH201, the first-in-human investigational blood stem cell treatment that will be evaluated as a potential cure for patients suffering from X-SCID. GPH201 is generated using precise and efficient gene editing technology, It works by directly replacing a defective gene that causes problems with the immune system. The hope is that GPH201 will ultimately lead to the production of fully functional, healthy immune cells.

The ultimate goal of this collaboration is to use JSP191 as the non-toxic alternative to chemotherapy in patients in order to remove their defective blood stem cells. After that, the gene editing blood stem cell technology developed by Graphite Bio can be introduced to patients in order to treat X-SCID. The two companies have agreed to collaborate on research, and potentially a clinical study, evaluating JSP191 as the non-toxic conditioning agent for GPH201.

In a press release, Josh Lehrer, M.Phil., M.D., chief executive officer at Graphite Bio, expressed excitement about the collaboration between the two companies.

“This collaboration with Jasper demonstrates our shared commitment to pioneering novel therapeutic approaches with the potential to significantly improve the treatment experiences of individuals with devastating conditions who stand to benefit from gene replacement therapies, initially for patients with XSCID. GPH201 harnesses our targeted gene integration platform to precisely target the defective gene that causes XSCID and replace it with a normal copy.”

In the same press release, Bill Lis, executive chairman and CEO of Jasper Therapeutics, also expressed optimism in regards to the two companies teaming up.

“Our collaboration with Graphite Bio is an exciting opportunity to further advance the field of curative gene correction by combining a targeted gene integration platform with our first-in-class targeted CD117 antibody, JSP191, that has already demonstrated preliminary clinical efficacy and safety as a conditioning agent in X-SCID patients and those with blood cancers undergoing allogeneic hematopoietic stem cell transplant.”

Graphite Bio is also developing gene editing technology to help treat sickle cell disease.  It is currently supported by a CIRM  late stage preclinical grant ($4.8M award). Th goal is to complete the final preclinical studies, which will allow Graphite Bio to start clinical studies of the sickle cell disease gene therapy in sickle cell patients in 2021.

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