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Holger Russ, Ph.D.
Generation of functional, patient specific thymi for cell therapy
One of the challenges following bone marrow transplants for cancer therapy is often lethal complications including bacterial pneumonia and graft versus host disease. Although reconstitution of blood forming cells is rapid following transplantation, T cell reconstitution is very slow and incomplete due to the lack of a functional thymus in the patient. To resolve this, Dr. Russ and his team are generating functional, patient specific thymi for co-transplantation into bone marrow transplant recipients. Dr. Russ’s approach can also be used to treat patients with a congenital absence of a thymus. The Grubstake
Award will allow them to transfer the technology to the Gates Biomanufacturing Facility for manufacturing scale up and to prepare for pre-IND filing with the FDA.
Ram Nagaraj, Ph.D.
Therapeutic peptide for neuroprotection in acute closed angle glaucoma
Glaucoma is the leading cause of irreversible blindness, with an estimated 80 million people worldwide affected by 2020. Retinal ganglion cell death (RGC), a hallmark of the disease, leads to degeneration of the optic nerve, resulting in visual field loss. RGC death correlates with increased intraocular pressure (IOP) due to defects in the drainage of fluid from the anterior chamber of the eye. In acute angle closure glaucoma, there is a sudden rapid rise in IOP with damage that requires immediate medical attention, typically surgery. Despite immediate care, RGC continue to die and cause a permanent reduction in vision
within three months. Dr. Nagaraj and his team are developing a peptide-based therapy designed to prevent the rapid loss of RGC from acute angle closure glaucoma. The Grubstake Award will allow them to assess treatment schedules to assess whether additional formulation work is required.
Raj Kumar, Ph.D.
Production of Efficacious Recombinant hypo-glycosylated FSH Glycoform for Clinical Applications
Artificial reproductive technology (a $1.5 billion market) may be used when women do not produce sufficient Follicle-Stimulating Hormone (FSH) for optimal ovarian follicle growth and estrogen production to support pregnancy. Currently, the response rate to standard FSH is very poor. Dr. Kumar and his team have discovered that reason appears to be the glycosylation pattern (location sugars are added to the protein) of the FSH. Dr. Kumar has shown that FSH21, expressed in women with normal reproductive cycles, is
also more biologically active in animal models. He plans to use funds from the Gates Grubstake Award to begin the process of developing the tools needed for manufacturing FSH21 for clinical testing.
34 Gates Center for Regenerative Medicine