Patient‐derived hematopoietic stem cells (HSC) from blood, bone marrow or cord blood represent a proven clinical strategy for treatment of a wide range of important human diseases. BetaStem has developed proprietary methods and reagents that dramatically improve the ability of a patient’s own stem cells to regenerate damaged blood vessels or regenerate bone marrow after chemotherapy. Applications for these stem cell products are broad and include unmet medical needs such as the repair of damaged blood vessels in diabetic retinopathy (a major cause of vision loss and peripheral vascular disease), repair of heart infarcts, and the dramatic acceleration of bone marrow stem cell engraftment in chemotherapy/transplantation settings. BetaStem has developed an efficient pre-clinical development strategy that blends leading academic health care centers, substantial manufacturing and clinical outsourcing, with the goal of filing our first IND during this summer (2012).

Our therapeutic approach is straightforward: 1) collect blood or bone marrow from the patient, 2) isolate their hematopoietic stem cells (~4 hours), 3) activate the stem cells (~4 hours) 4) introduce the activated stem cells back to the patient which then mediate repair and regeneration of vasculature or bone marrow. This approach is powerful not only because of the regenerative repair of vessels, but because it utilizes the patient’s own stem cells which do not cause an immune rejection response. Activation of the stem cells increases the efficacy of vessel repair and bone marrow engraftment significantly (Diabetes. 2010 Aug; 59(8):2010-19)

We are developing an efficient, safe clinical treatment for vessel repair using the patient's own blood stem cells or bone marrow stem cells that have been activated by our patented methods. The diabetic environment has been shown to markedly inhibit and reduce the number of the patient’s own hematopoietic stem cells ability to repair damaged blood vessels (stem cells from healthy individuals routinely repair vasculature in health). Patient stem cells are treated outside the body with drugs that transiently blocks important regulators of stem cell activation (TGFβ family of genes) found within the stem cells. In diabetes, the stem cells are over overly-suppressed by TGFβ family members and are unable to proliferate and home to damaged vessels. Stem cell activation overcomes this defect.

Our pre-clinical studies have closely defined the cellular mechanisms for more efficient vascular repair as well as optimizing dosage and delivery. We have shown that blood derived diabetic stem cells can repair damaged retinal vascular beds in two different human-mouse retinal models similar to healthy stem cells using our activation approach. This stem cell therapy has the potential to provide a targeted and highly specific approach to repair the damaged capillary bed of the retina. Since our treatment strategies utilize autologous (patient’s own) stem cells treated transiently ex vivo, we expect the long term safety of this process to be excellent

Valuation of BetaStem will continue to rise driven primarily through non-dilutive public grant funds. Current national interests are dedicating funds to advance stem cell therapies. BetaStem will thus continue to benefit from existing public infrastructures such as collaborations with University researchers, therefore requiring less capital to achieve its research goals. BetaStem is currently seeking funds from private investors for business development activity as well as continuing to submit research grants for additional pre=clinical studies. Application of our technology is broad based, and therefore we expect subsequent patents to be used in multiple disease verticals.

Our therapeutic model will consist of a central BetaStem facility where patient samples will be received (blood, bone marrow, cord blood), the hematopoietic stem cells will be rapidly isolated and treated with morpholino antisense overnight and the activated stem cells shipped to the patient's physician for administration. The cost of the entire procedure, including the patented morpholino antisense stem cell treatment, will be billed to the patient. Alternatively, we are in the process of developing a process such that the entire procedure could be done onsite in the hospital.

• Have received four NIH Grants in four years, including a prestigious NIH Challenge Grant (~$2.5 million)
• Patents filed (Three issued patents, one patent application in progress)
• Manuscripts submitted: 3 including description of a new target gene
• Will continue to obtain apply for public funding through NIH-SBIR (Small business Grants)
• Secured manufacturing contract for GMP grade morpholino antisense
• Concerted efforts to obtain IND by summer 2012.
• Advance pre-clinical dosing and efficacy studies using diabetic retinopathy and cardiovascular disease models in progress.
• Secure Required Licensing Agreement Rights for stem cell manufacturing
• Obtain initial private funding through series A round
• Complete pre-clinical and business development team

Stephen Bartelmez PhD | President, Chief Scientific Officer & Founder

Alan Moore PhD | Chief Executive Officer

Valerie Stepps, MSc | Senior Scientist, Flow Cytometrist

Casey Haynes MSc | Scientist

Major Collaborators:

• Maria Grant MD, Full Professor, University of Florida including three postdoctoral Scientists
• Pat Iversen, PhD, AVI-BioPharma, Seattle, Washington
• Fred Dorey, JD, Special Counsel, Cooley LLP
• Anton Gueth, GUETH CONSULTING LLC
• Timothy H. Van Dyke , JD, Patent Attorney, Beusse Wolter Sanks Mora & Maire LLP

BetaStem Therapeutics Inc
Stem Cell Therapy for Diabetes and Cancer
300 Brannan Street; Suite 407
San Francisco, CA 94107

415.913.7595 (lab)
206.427.0350 (office)