December 30, 2017

Pipeline

Our completed pipeline milestones include the effective translation of integrated mouse technology platforms and preclinical product lines to humans. This includes lead mAb development for human regulatory B10 cell depletion in vivo and the development of human regulatory B10 cell manufacturing technologies. Both product lines are advancing to manufacturing, clinical application and commercialization.

Our near-term milestone is humanization of our human B10 cell depleting mAbs prior to lead drug selection and manufacturing for IND approval and clinical trials in oncology.

Within our B cell expansion program, current milestones include the optimization and efficient implementation of scaled-up human B cell manufacturing for phase I clinical trials for two orphan diseases, severe combined immunodeficiency (SCID) post-transplant and cGVHD, following IND approval.

 

More about our therapeutic indications

Severe Combined Immunodeficiency (SCID)

According to the NIH, severe combined immunodeficiency (SCID) is a term applied to a group of inherited disorders characterized by genetic defects in both T and B cell responses, hence the term "combined.” SCID makes those affected highly susceptible to life-threatening infections by viruses, bacteria and fungi. Because children with SCID experience multiple infections, they fail to grow and gain weight as expected, and those with untreated SCID rarely live past the age of two.

SCID is a rare disease that is estimated to affect perhaps 40 to 100 babies born in the U.S. each year. However, researchers have no clear idea of how many babies are not diagnosed and die of SCID-related infections each year. The actual number of cases could be higher.

While rare, SCID may be best known from news coverage and a movie in the 1980s about David, the “Boy in the Bubble”, who was born without a working immune system.

Chronic Graft Versus Host Disease (cGVHD)

Chronic GVHD (cGVHD) is a life-threatening condition that can occur in patients after they receive a stem cell transplant from blood or bone marrow, called hematopoietic stem cell transplantation (HSCT), to treat certain blood or bone marrow cancers. The condition is estimated to occur in 30 to 70 percent of all patients who receive HSCT.

cGVHD happens when cells from the transplant attack healthy cells in a patient’s tissues. The exact cause of cGVHD is not known.

Symptoms of cGVHD can occur in the skin, eyes, mouth, gut, liver and lungs. Skin rash and/or mouth sores are among the most common initial signs of the disease. Unlike acute GVHD, cGVHD can cause damage in the glands that produce tears in the eyes and saliva in the mouth resulting in dry eyes or a dry mouth. Patients may have mouth ulcers causing pain while eating, skin rashes, or liver inflammation. Chronic GVHD can also cause many other problems, such as formation of scar tissue in the skin (cutaneous sclerosis) and joints and damage to air passages in the lungs.