BETASTEM THERAPUETICS, INC. — Department of Health and Human Services SBIR Phase I: N

Description

Autologous TGFB Modified CD Stem Cells for Repair of Diabetic Macular Edema and Macular Ischemia Retinal vascular diseases such as diabetic macular edema and macular ischemia remain a common cause of vision loss and blindness Diabetes can damage the small blood vessels in the retina causing them to leak and occlude resulting in vision loss Although treatments are available for aspects of diabetic ocular disease no therapy is available to treat the damaged retinal vasculature and ischemic retina Vision loss from retinal ischemia can be irreversible A subgroup of DR patients suffers from macular ischemia and currently there is also no effective therapy Research over the last decade has identified a class of bone marrow derived circulating cells CD stem cells which are capable of homing to vascular lesions and facilitating vascular repair However many diabetic patients have dysfunctional CD stem cells with no reparative potential In this SBIR Fast Track phase l ll proposal we use a novel strategy to correct dysfunctional diabetic CD cells by transiently modifying CD stem cells derived from patient blood that both restores perfusion to the ischemic retina and correct vessel leaking Experiments from our NIH funded studies show that this CD dysfunction can be corrected by transiently inhibiting endogenous transforming growth factor TGF within the patientandapos s own dysfunctional CD stem cells using antisense phosphorodiamidate morpholino oligomers TGF PMO to TGF Our proposed studies are focused according to guidance of the FDA In we completed a pre IND meeting with the FDA A major goal is to complete an IND in order to begin a first in man clinical trial Our concern in developing this autologous stem cell approach is the safety of the patient and the efficacy of the therapy We have proposed in vitro phase l CD studies and phase ll in vivo studies including Akimba mice and diabetic baboons testing safety and efficacy of our therapy The investigators of this application include a very experienced CEO a well known practicing research ophthalmologist an international known PMO expert and a CD stem cell biologist Project Narrative BetaStemandapos s goal is to develop an efficient safe clinical treatment for diabetic retinopathy DR using stem cells from the patientandapos s blood that we activate outside of the patient then are returned to the patientandapos s eye where they repair damaged capillary vessels Currently no effective treatment exists that will reverse DR which is marked by vision loss as a result of damaged retinal blood vessels and reduction of blood oxygen supply to the retina