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Autologous TGFB Modified CD Stem Cells for Repair of Diabetic Macular Edema and Macular Ischemia

Award Information
Agency: Department of Health and Human Services
Branch: National Institutes of Health
Contract: 1R44EY028070-01
Agency Tracking Number: R44EY028070
Amount: $461,417.00
Phase: Phase I
Program: SBIR
Solicitation Topic Code: N
Solicitation Number: PA16-302
Solicitation Year: 2016
Award Year: 2017
Award Start Date (Proposal Award Date): 2017-09-30
Award End Date (Contract End Date): 2019-01-29
Small Business Information
Sausalito, CA 94965-2348
United States
DUNS: 784204864
HUBZone Owned: No
Woman Owned: No
Socially and Economically Disadvantaged: No
Principal Investigator
 (206) 427-0350
Business Contact
Phone: (415) 913-7595
Research Institution

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

* Information listed above is at the time of submission. *

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