APTITUDE MEDICAL SYSTEMS INC — Department of Health and Human Services SBIR Phase I: NEI

Description

Abstract SignificanceProliferative vitreoretinopathyPVRis a blinding orphan indication that occurs inof patients receiving rhegmatogenous retinal reattachment surgeryand accounts forof all primary surgical failuresPVR is characterized by migration and proliferation of ectopic cells beneath or on the surface of the retinawhich then assemble into a membranecausing retinal traction and detachmentSince currently there is no effective therapeutics that reverse the cell proliferation processthe only treatment option for PVR is additional surgerieswhich often have very poor vision outcome due to retinal damage from recurrent detachmentrisking blindnessThere is an urgent unmet need for novel pharmacotherapies for PVRHypothesis and aimsExtensive research established PDGFs as leading candidates for treating PVRHoweverinhibiting multiple PDGF targets may be required to be effectiveAptamers are single stranded oligonucleotides that bind to molecular targets in a similar manner to monoclonal antibodiesmAbsbut possess unique advantages for treating ocular diseases such as PVRthanks to its small sizesuperior stabilityand lack of immunogenicityWe have already developed a fully modified aptamer for PDGF Band verified its potency and stability in vitro and bioactivity in vivo in two different animal modelsWe propose to developmodifiedhigh potency aptamers against PDGF ABandCto extend the key advantages of aptamers to this potential first in class treatment NarrativeProliferative vitreoretinopathyPVRis a blinding orphan indication that occurs inof patients receiving rhegmatogenous retinal reattachment surgeryand accounts forof all primary surgical failuresPVR is characterized by migration and proliferation of ectopic cells beneath or on the surface of the retinawhich then assemble into a membranecausing retinal traction and detachmentSince currently there is no effective therapeutics that reverse the cell proliferation processthe only treatment option for PVR is additional surgerieswhich often have very poor vision outcome due to retinal damage from recurrent detachmentrisking blindnessWe aim to develop next generation aptamers that effectively inhibit cell proliferation and membrane formationand serve as a potential first in class pharmacotherapy for PVR