FOUNDATION FIGHTING BLINDNESS INC
6925 OAKLAND MILLS ROAD 701, COLUMBIA, MD 21045 www.fightingblindness.org

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Organizations Filed Purposes: THE URGENT MISSION OF THE FOUNDATION FIGHTING BLINDNESS IS TO DRIVE THE RESEARCH THAT WILL PROVIDE PREVENTIONS, TREATMENTS AND CURES FOR PEOPLE AFFECTED BY RETINITIS PIGMENTOSA, AGE-RELATED MACULAR DEGENERATION, USHER SYNDROME AND THE ENTIRE SPECTRUM OF RETINAL DEGENERATIVE DISEASES. THE FOUNDATION IS A BEACON FOR THOSE AFFECTED BY THESE BLINDING DISEASES.

RESEARCH: THE FOUNDATION FIGHTING BLINDNESS, INC. (THE FOUNDATION) FUNDED RESEARCHERS ARE ACHIEVING REMARKABLE SUCCESS IN DISCOVERING, TESTING AND DELIVERING TO PATIENTS A WIDE RANGE OF PROMISING THERAPIES TO PREVENT, STOP THE PROGRESSION OF, AND CURE BLINDNESS DUE TO RETINAL DEGENERATIVE DISEASES. THE FOUNDATION HAS ENCUMBRANCES OF $41.9 MILLION, NOT INCLUDED ON OUR JUNE 30 FINANCIAL REPORT, FOR ACTIVE RESEARCH PROJECTS THAT HAVE FUTURE RESEARCH MILESTONES. OUR RESEARCH OVERSIGHT COMMITTEE'S SPENDING PLAN INCLUDES SPENDING AT AN AVERAGE OF $21 MILLION ANNUALLY TO SUPPORT FUTURE RESEARCH.AS OF THE END OF OUR FISCAL YEAR 2020, THE FOUNDATION'S RESEARCH GRANTS PROGRAM IS FUNDING 82 GRANTS BEING CONDUCTED BY MORE THAN 90 INVESTIGATORS AT 67 INSTITUTIONS, EYE HOSPITALS AND UNIVERSITIES IN THE U.S. AND AROUND THE WORLD. A SAMPLING OF THE RESEARCH PROJECTS FUNDED ARE LISTED BELOW.HOMOLOGY-INDEPENDENT GENOME EDITING FOR TREATMENT OF STARGARDT DISEASEIVANA TRAPANI, PHD, UNIVERSITA DEGLI STUDI DI NAPOLI "FEDERICO II" NAPLES, ITALYGENE THERAPY DEVELOPMENT FOR STARGARDT DISEASE IS CHALLENGING BECAUSE ABCA4, THE MUTATED GENE CAUSING STARGARDT DISEASE, IS TOO LARGE FOR MOST VIRAL DELIVERY SYSTEMS. DR. TRAPANI IS EVALUATING A HIGHLY EFFICIENT CRISPR/CAS9 GENE-EDITING TECHNOLOGY - HOMOLOGY INDEPENDENT TARGETED INSERTION (HITI) - TO ADDRESS MUTATIONS IN ABCA4. HER EARLY PROOF-OF-CONCEPT STUDIES ARE BEING PERFORMED IN A STARGARDT DISEASE MOUSE MODEL AND A THREE-DIMENSIONAL RETINAL ORGANOID IN A DISH.ADAR-BASED RNA EDITING AS A POTENTIAL THERAPY FOR INHERITED RETINAL DEGENERATIONSDROR SHARON, PHD, HADASSAH-HEBREW UNIVERSITY MEDICAL CENTERMANY EMERGING THERAPIES TARGET MUTATIONS IN OUR GENES - OUR DNA. DR. SHARON IS INVESTIGATING A NEW TOOL FOR EDITING RNA - THE GENETIC MESSAGES DERIVED FROM DNA WHICH ARE USED BY OUR CELLS TO PRODUCE PROTEINS. KNOWN AS "INTRINSIC ADENOSINE DEAMINASE ACTING ON RNA, OR ADAR, THE TECHNOLOGY SPECIFICALLY ADDRESSES G-TO-A MUTATIONS. DR. SHARON BELIEVES THAT ABOUT 30 PERCENT OF THE MOST COMMONLY REPORTED IRD-CAUSING MUTATIONS CAN BE TARGETED FOR ADAR EDITING (MAINLY IN RELATIVELY LARGE GENES SUCH AS ABCA4 AND USH2A). HIS LAB IS EVALUATING THE TECHNOLOGY IN CELLS AND MOUSE MODELS OF RETINAL DISEASE.PRIME EDITING FOR USHER SYNDROME TYPE 2ABENCE GYORGY, MD, PHD, INSTITUTE OF MOLECULAR AND CLINICAL OPHTHALMOLOGY BASELDR. GYORGY IS DEVELOPING A GENE CORRECTION STRATEGY KNOWN AS PRIME EDITING, A NOVEL TECHNOLOGY THAT IS IN SOME WAYS SIMILAR TO GENE EDITING WITH CRISPR/CAS9, BUT POTENTIALLY MORE PRECISE AND EFFICIENT. INSTEAD OF CUTTING THE DOUBLE STRANDS OF DNA, IT NICKS THE DNA, WHICH MAY BE A PREFERRED APPROACH TO CORRECTING SINGLE-LETTER MUTATIONS. DR. GYORGY IS DEVELOPING A PRIME EDITING TREATMENT TO INSERT THE MISSING G NUCLEOTIDE INTO RETINAL CELLS WITH THE RELATIVELY COMMON USH2A MUTATION DEL2299G. IN ORDER TO FACILITATE TRANSLATION OF THIS CONCEPT TO THE CLINIC, HE WILL EVALUATE THE THERAPY IN HUMAN RETINAL EXPLANTS, HUMAN ENGINEERED RETINAL ORGANOIDS AND HUMANIZED MOUSE MODELS. IF THE APPROACH IS SUCCESSFUL, IT MAY BE APPLIED TO OTHER MUTATIONS AND IRDS.CREATION OF NEW MODELS OF INHERITED RETINAL DISEASE IN PIGSMAUREEN MCCALL, PHD, UNIVERSITY OF LOUISVILLETHOUGH RODENTS ARE OFTEN USED AS MODELS FOR INHERITED RETINAL DISEASES (IRDS), THEY ARE LIMITED IN RECAPITULATING HUMAN IRDS BECAUSE THEY DON'T HAVE CONES, THE RETINAL CELLS THAT ENABLE HUMANS TO PERCEIVE DETAILS AND COLORS AND SEE IN LIGHTED SETTINGS. PIGS DO HAVE CONES AND ARE EASY TO ENGINEER TO MODEL IRDS IN HUMANS. DR. MCCALL IS CREATING A SLOW DEGENERATION PIG MODEL OF RP (RHO-P23H) AS WELL AS A PIG MODEL OF STARGARDT DISEASE. THE MODELS WILL BE USEFUL FOR EVALUATING EMERGING THERAPIES IN PREPARATION FOR CLINICAL TRIALS.GENERATION AND CHARACTERIZATION OF A PIG MODEL OF RETINITIS PIGMENTOSA (EYS MUTATION)HEMANT KHANNA, PHD, UNIVERSITY OF MASSACHUSETTS MEDICAL SCHOOLMUTATIONS IN THE EYS GENE ARE THE SECOND MOST COMMON CAUSE OF AUTOSOMAL RECESSIVE RP IN HUMANS. HOWEVER, THE INTACT EYS GENE IS ABSENT AND/OR DISRUPTED IN MANY ANIMALS, INCLUDING MICE AND RATS. THE GENE IS PRESENT IN PIGS, SO DR. KHANNA AND HIS COLLEAGUES ARE DEVELOPING A PIG MODEL OF EYS-ASSOCIATED RP, WHICH WILL BE USEFUL IN UNDERSTANDING THE EFFECTS OF EYS MUTATIONS AND TESTING EMERGING EYS THERAPIES.

PUBLIC HEALTH EDUCATION:DURING FISCAL YEAR 2020, THROUGH OUR CHATLOS FOUNDATION PUBLIC EDUCATION PROGRAM, THE FOUNDATION AIMED TO CONTINUE TO PROVIDE EDUCATION AND SUPPORT TO PEOPLE AFFECTED BY RETINAL DEGENERATIVE DISEASES. THE FOUNDATION ALSO FIELDS A TEAM OF PROFESSIONALS FOCUSED ON EDUCATING OPHTHALMOLOGISTS AND OPTOMETRISTS ON THE LATEST ADVANCEMENTS WITH THE FOUNDATIONS RESOURCES AND RESEARCH ON THE INHERITED RETINAL DISEASE SPACE. TO KEEP OUR CONSTITUENTS AND PROFESSIONALS INFORMED, THE FOUNDATION PUBLISHES EXTENSIVE CONTENT VIA A WEBSITE AND SOCIAL MEDIA/EMAIL CHANNELS; DISTRIBUTES A NEWSLETTER ONLINE AND IN PRINT; HOLDS QUARTERLY INSIGHTS FORUM CALLS TO INFORM THE COMMUNITY ABOUT SCIENTIFIC PROGRESS. THE CONTENT PRODUCED INCLUDES THE LATEST DEVELOPMENTS IN RESEARCH AND CLINICAL TRIALS, AND PERSONAL STORIES FROM MEMBERS OF THE FIGHTING BLINDNESS COMMUNITY. ADDITIONALLY, IN MAY THE FOUNDATION LAUNCHED A NEW QUARTERLY CONTINUING MEDICAL EDUCATION PROGRAM FOCUSED ON PROVIDING TIMELY CONTENT TO EYECARE PROFESSIONALS.DESPITE THE PANDEMIC, THE FOUNDATION HELD A VIRTUAL NATIONAL VISIONS CONFERENCE. MORE THAN 24 DISTINGUISHED PANELISTS PRESENTED A TOTAL OF 15 SCIENTIFIC AND LIFESTYLE EDUCATION SESSIONS WITH APPROXIMATELY 1,600 PARTICIPATING IN THE SESSIONS. CHAPTERS BRING THE FOUNDATION'S MESSAGE AND MISSION TO LIFE. IN FY20 THE FOUNDATION SUPPORTED OVER 40 VOLUNTEER-LED CHAPTERS IN 22 STATES. THE CHAPTERS HELD 46 SPEAKER PRESENTATIONS, SOCIALS, AND OTHER OUTREACH ACTIVITIES WHICH NEARLY 1,400 PEOPLE ATTENDED, AND CO-HOSTED FOUR VISION SEMINARS WITH THE FOUNDATION WHICH NEARLY 500 PEOPLE ATTENDED.

MY RETINA TRACKER:THE FOUNDATION MAINTAINS A RESEARCH DATABASE OF PEOPLE AND FAMILIES AFFECTED BY RARE INHERITED RETINAL DEGENERATIVE DISEASES. THE REGISTRY IS DESIGNED TO SHARE DE-IDENTIFIED INFORMATION WITHIN THE IRD RESEARCH AND CLINICAL COMMUNITIES ABOUT PEOPLE WITH AN INHERITED RETINAL DISEASE TO HELP ACCELERATE THE DISCOVERY OF TREATMENTS AND CURES. DURING FISCAL YEAR 2020 APPROXIMATELY 2,300 ADDITIONAL PATIENTS WERE ADDED TO THE REGISTRY, BRINGING TOTAL REGISTERED INDIVIDUALS UP TO 14,929.

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