Autologous Stem Cell Therapy for Diabetic Retinopathy

The complications of diabetes can be devastating. Of adults (29 million) with diabetes aged 40 years or older, 7.7 million (26.6%) had Diabetic Retinopathy (DR). Of these, 1.2 million (4.1%) had advanced to clinically significant macular edema and proliferative diabetic retinopathy—that can lead to severe vision loss (National Diabetes Statistics Report, 2014, CDC). The fundamental vision for BetaStem is to treat diabetic retinopathy by repairing and regenerating damaged retinal endothelial cells in the patient’s eye using each patient’s own stem cells. However, over time diabetic stem cells become unable to repair vascular endothelium due to the diabetic milieu. BetaStem has discovered a way to “correct” the diabetic stem cell dysfunction by targeting TGFß1 expressed within the patients circulating stem cell (“CD34+” stem cells). This procedure returns the cells to a functional state able to regulate and repair vascular endothelium in the patient’s eye.

BetaStem’s technology is derived from discoveries that CD34+ stem cells become activated gaining highly advantageous regenerative properties when treated with our proprietary antisense to block a major stem cell activation inhibitor, namely transforming growth factor-beta, (TGFβ1). The activated stem cells home into the damaged vascular regions of the retina, initiating a process of blood vessel regeneration and thereby preserving eye health.

BetaStem offers the first real solution for DR by preventing and repairing micro-capillary closure (ischemia). 

Demonstrated safe and effective in experimental animals
Investigational New Drug (IND) pathway agreed with FDA

First-in Man human clinical trials planned .
About BetaStem

Our fundamental vision is to correct the dysfunction of Diabetic stem cells from the patient allowing them to repair and regenerate damaged retinal endothelium damaged by Diabetic Retinopathy in a patient's eye. The treatment is based on stem cells taken from a patient's own blood or bone marrow "rehabilitated" through our Patented and proprietary process. The patients rehabilitated CD34+ stem cells regain a potent state giving rise to millions of daughter cells capable of bringing about endothelial repair when brought into contact with autologous injured tissue.

Products

BetaStem will sell reagent kits to hospitals that contain proprietary formulations of phosphorodiamidate morpholino oligomers (PMO) used to create rehabilitated stem cells from peripheral blood or bone marrow extracted from patients. Hospitals will extract the patientís cells, perform an overnight rehabilitation process, and a Retinal Ophthalmologists will inject the patients rehabilitated CD34+ stem cells into the affected eye.

The BetaStem Therapeutics will also train and certify practitioners to perform this treatment.

Company status

Demonstrated safe and effective in experimental animals

Investigational New Drug (IND) pathway agreed with FDA

First-in Man human clinical trials planned