Friedreichs Ataxia Research Alliance Fara

TO TREAT AND CURE FRIEDREICH'S ATAXIA (FA) BY ADVANCING RESEARCH, AWARENESS AND PARTNERSHIPS.

RESEARCH AND GRANT PROGRAMFARA DEPLOYS FINANCIAL RESOURCES TO ACCELERATE RESEARCH FOR FRIEDREICH'S ATAXIA (FA). IN 2021, FARA AWARDED OVER $6.5 MILLION IN RESEARCH FUNDING. THIS INCLUDED MORE THAN 37 ACTIVE RESEARCH PROJECTS THROUGH FARA'S INVESTIGATOR-INITIATED GRANT PROGRAM AS WELL AS SUPPORT FOR INSTITUTIONAL BASED RESEARCH AND FARA DIRECTED PROJECTS. FIFTEEN NEW GRANTS WERE FUNDED AFTER PEER REVIEW THROUGH FARA'S INVESTIGATOR-INITIATED GRANT PROGRAM AND TWENTY-TWO APPROVED IN PREVIOUS YEARS RECEIVED CONTINUED FUNDING. SIX OF THE NEW GRANTS WERE AWARDED TO INVESTIGATORS NEW TO FA RESEARCH, INCLUDING 4 JUNIOR INVESTIGATORS. RESEARCHERS IN 9 COUNTRIES ARE RECEIVING RESEARCH FUNDING FROM FARA. SEVERAL OF THESE GRANTS WERE CO-FUNDED WITH FARA'S FA ADVOCACY GROUP PARTNERS; THIS COLLABORATIVE APPROACH BOTH INCREASES THE NUMBER OF AVAILABLE RESEARCH DOLLARS AND HELPS TO MINIMIZE DUPLICATION OF EFFORT.FARA HAS IDENTIFIED OPPORTUNITIES TO FUND RESEARCH AT INSTITUTIONS WHERE THERE ARE MULTIPLE INVESTIGATORS WITH AN EXPERTISE AND COMMITMENT TO FA RESEARCH AND/OR OPPORTUNITY TO LEVERAGE TECHNOLOGIES, INNOVATION OR NEW GROWTH TO THE FA COMMUNITY. BY ESTABLISHING THESE INSTITUTIONAL FUNDING PROGRAMS, WE ARE ABLE TO PROMOTE COLLABORATION AND SYNERGY ACROSS BASIC, TRANSLATIONAL AND CLINICAL RESEARCH, PROVIDE A LONGER-TERM COMMITMENT FOR RESEARCH, ATTRACT NEW INVESTIGATORS, AND LEVERAGE THE INSTITUTIONS RESOURCES AND ENGAGEMENT. FARA PROVIDES INSTITUTIONAL BASED RESEARCH SUPPORT FOR THE FA CENTER OF EXCELLENCE AT PENN MEDICINE/ CHILDREN'S HOSPITAL OF PHILADELPHIA AND THE FA ACCELERATOR PROGRAM THE BROAD INSTITUTE. FARA DIRECTED PROJECTS ARE RESEARCH INITIATIVES IDENTIFIED AND LEAD BY FARA'S SCIENTIFIC ADVISORY BOARD AND STAFF. ALL OF THESE RESEARCH PROJECTS ARE HIGH PRIORITY AS THEY AIM TO PROVIDE RESEARCH RESOURCES, ADDRESS SPECIFIC GAPS IN KNOWLEDGE OR DEEPEN OUR UNDERSTANDING OF DISEASE MECHANISM, PATHOLOGY, AND PROGRESSION AND DISCOVERY OF INNOVATIVE APPROACHES TO THERAPY. FRIEDREICH'S ATAXIA CENTER OF EXCELLENCE, PHILADELPHIA, PATHE FA CENTER OF EXCELLENCE (COE) IS A TRANSLATIONAL RESEARCH AND CLINICAL CARE CENTER DEVOTED TO FRIEDREICH ATAXIA: EXPEDITING BASIC SCIENCE AND DRUG DISCOVERY FINDINGS TO NEW TREATMENTS AND DEDICATING RESOURCES TO CLINICAL RESEARCH AND CARE TO FURTHER UNDERSTAND THE DISEASE, INFORM DRUG DEVELOPMENT AND IMPROVE OUTCOMES FOR INDIVIDUALS LIVING WITH FA. THE CENTER WAS ESTABLISHED IN MARCH 2014, WITH A COMMITMENT TO PENN MEDICINE/ CHILDREN'S HOSPITAL OF PHILADELPHIA, PRESENTED BY FARA IN PARTNERSHIP WITH THE HAMILTON AND FINNERAN FAMILIES. FARA HAS MAINTAINED THIS FUNDING PARTNERSHIP WITH THE CUREFA FOUNDATION (ESTABLISHED BY THE HAMILTON AND FINNERAN FAMILIES) TO ADVANCE RESEARCH THROUGH THE COE. PROJECTS AND INVESTIGATORS WITH CONTINUED FUNDING INCLUDED DR. ROB WILSON- DRUG DISCOVERY, DR. DAVID LYNCH- TRANSLATIONAL AND CLINICAL NEUROSCIENCE RESEARCH, DR. IAN BLAIR- BIOMARKER DISCOVERY, DR. KIM LIN- CARDIAC RESEARCH, DR. SHANA MCCORMACK- METABOLISM AND ENDOCRINOLOGY, AND DR. JENNIFER PHILLIPS CREMINS- GENETIC MODELING.THE COE ESTABLISHED RESEARCH INFRASTRUCTURE CONSISTING OF FOUR CORE AREAS- NEUROLOGY, CARDIAC, BIOMARKER AND DRUG DISCOVERY. THE CENTER NOT ONLY SUPPORTS WORK WITHIN THESE DISCIPLINES, BUT ALSO FOSTERS EFFICIENT COLLABORATION AND SYNERGY ACROSS THEM. OVER THE PAST EIGHT YEARS, THE COE REACHED SEVERAL SIGNIFICANT RESEARCH MILESTONES WITH THE DISCOVERY OF POSSIBLE TREATMENT CANDIDATES, THE STUDY OF NEW BIOMARKERS TO MEASURE THE DISEASE, AND LAUNCH OF PROMISING NEUROLOGIC AND CARDIAC CLINICAL TRIALS AND STUDIES. FUNDING PROVIDED BY THE COE ENABLED EARLY RESEARCH FINDINGS WHICH HAS ALLOWED INVESTIGATORS TO APPLY FOR AND RECEIVE LARGER GRANTS FROM THE NATIONAL INSTITUTES OF HEALTH TO CONTINUE TO EXPAND THIS IMPORTANT WORK. FRIEDREICH'S ATAXIA ACCELERATOR AT THE BROAD INSTITUTE OF MIT AND HARVARDTHE FRIEDREICH'S ATAXIA ACCELERATOR (FAA) AT THE BROAD INSTITUTE OF MIT AND HARVARD WAS ESTABLISHED IN AUGUST 2020. FUNDED BY FARA, IN COLLABORATION WITH THE CUREFA FOUNDATION AND ENDFA, THE FAA IS A COLLABORATIVE, MULTI-DISCIPLINARY EFFORT AIMED AT GALVANIZING RESEARCH INTO FA AND SEEDING A GROWING COMMUNITY ACROSS BROAD, MIT, HARVARD AND AFFILIATED INSTITUTIONS COMMITTED TO TACKLING FA. THE LEADER OF THE ACCELERATOR, VAMSI MOOTHA, A HOWARD HUGHES MEDICAL INSTITUTE INVESTIGATOR, HAS SEVERAL LINES OF FA RESEARCH UNDERWAY INCLUDING DRUG DISCOVERY THROUGH IDENTIFICATION OF CHEMICAL AND GENETIC MODIFIERS OF FA, AND HYPOXIA-INSPIRED RESEARCH BASED ON PRIOR FINDINGS THAT LIMITED OXYGEN IN CELLS, OR HYPOXIA, REJUVENATES CELLS THAT LACK FRATAXIN. GARY RUVKUN, A PROFESSOR OF GENETICS AT HARVARD MEDICAL SCHOOL AND MASSACHUSETTS GENERAL HOSPITAL, LEADS A TEAM USING SIMPLE MODEL SYSTEM TO TEST DIFFERENT MOLECULES FOR THEIR ABILITY TO RESCUE FRATAXIN-DEFICIENT CELLS. DAVID LIU, WHO IS ALSO A HOWARD HUGHES MEDICAL INSTITUTE INVESTIGATOR, HAS A TEAM APPLYING NEW GENE EDITING TECHNOLOGIES TO DIRECTLY CORRECT THE GENETIC CAUSES OF FRIEDREICH'S ATAXIA. IN 2021, THE SEIDMAN LAB JOINED THE FA ACCELERATOR. LED BY CHRISTINE AND JONATHAN SEIDMAN AT THE HARVARD MEDICAL SCHOOL AND BRIGHAM AND WOMEN'S HOSPITAL, THE SEIDMAN LAB WILL FOCUS ON RESEARCH TO UNDERSTAND THE MECHANISMS OF CARDIOMYOPATHY IN FA.FARA DIRECTED RESEARCHIN 2021, FARA FUNDED DIRECTED RESEARCH PROGRAMS INCLUDING: 1. MAINTAINING A REPOSITORY OF FA CELL LINES FOR RESEARCH HTTPS://SITES.UAB.EDU/THENAPIERALALAB/FRDA-CELL-LINE-REPOSITORY/ 2. TRACK-FA, A GLOBAL NEUROIMAGING CONSORTIUM ESTABLISHED IN 2020, IS A NATURAL HISTORY STUDY TO TRACK THE CHANGES IN THE BRAIN AND THE SPINAL CORD IN FA. PARTICIPATING CLINICAL SITES IN USA, BRAZIL, GERMANY, AND AUSTRALIA INITIATED ENROLLMENT IN 2021. THE GOAL IS TO ENROLL 200 INDIVIDUALS WITH FA AND 100 MATCHED CONTROLS AND CONDUCT NEUROIMAGING OF THE BRAIN AND SPINAL CORD, ALONG WITH CLINICAL OUTCOMES AND BLOOD BIOMARKERS 3. FOSTERING A COLLABORATIVE EFFORT BETWEEN UNIVERSITY OF OKLAHOMA, UNIVERSITY OF ALABAMA, AND UNIVERSITY OF PENNSYLVANIA TO STUDY THE PRECISE MECHANISM OF THIS GENE SILENCING IN DIFFERENT TISSUES AND MODELS OVER TIME. 4. ASSEMBLING GLOBAL CLINICAL EXPERTS TO LEAD A RE-EVALUATION AND UPDATE TO THE CLINICAL MANAGEMENT GUIDELINES IN FA, NEW GUIDELINES ARE EXPECTED TO BE PUBLISHED IN 2022. 5. INITIATING DEVELOPMENT AND VALIDATION OF A POTENCY ASSAY FOR USE IN THE DEVELOPMENT OF GENE AND PROTEIN REPLACEMENT THERAPIES. 6. LEADING WORK TO CREATE A NEW DISEASE MODEL FOR PRE-CLINICAL RESEARCH, AN FA RAT MODEL. 7. IDENTIFYING AND FUNDING RESEARCH TO UNDERSTAND THE THERAPEUTIC WINDOW (MINIMAL AMOUNT OF FRATAXIN NEEDED FOR A THERAPEUTIC BENEFIT AND THRESHOLD FOR MAXIMUM AMOUNTS OR TOXIC AMOUNTS OF FRATAXIN IN THE CELL).OF NOTE, FA SHARES SIMILAR SYMPTOMS AND DISEASE MECHANISMS WITH OTHER DISEASES, BOTH RARE AND COMMON. RESEARCH INTO FA CAN PROVIDE INSIGHTS AND ADVANCES IN OTHER DISEASES SUCH AS MITOCHONDRIAL DISEASES, MUSCULAR DYSTROPHIES, DIABETES, AND CARDIOMYOPATHY. A COMPLETE LIST OF 2021 FUNDED GRANTS CAN BE FOUND AT: CUREFA.ORG/GRANT-AWARDSA COMPLETE LIST OF 2021 PUBLICATIONS OF RESEARCH FUNDED BY FARA CAN BE FOUND AT: CUREFA.ORG/SCIENTIFIC-NEWS/FUNDED-RESEARCH

CLINICAL RESEARCH INFRASTRUCTUREIN ADDITION TO RESEARCH GRANTS, FARA FUNDS THE ONGOING DEVELOPMENT OF DOMAIN RESOURCES IN THE FORM OF VITAL CLINICAL RESEARCH INFRASTRUCTURE. CLINICAL RESEARCH INFRASTRUCTURE REFERS TO THE RESOURCES NEEDED TO FACILITATE ANY TYPE OF RESEARCH, INCLUDING CLINICAL TRIALS THAT INVOLVE PATIENTS. THESE RESOURCES CAN INCLUDE THINGS LIKE: FRIEDREICH'S ATAXIA GLOBAL PATIENT REGISTRY: THE FRIEDREICH'S ATAXIA GLOBAL PATIENT REGISTRY (FAGPR) IS THE ONLY WORLDWIDE REGISTRY OF FRIEDREICH'S ATAXIA PATIENTS. THE GOALS OF THE FAGPR ARE TO COLLECT INFORMATION ON ALL FA PATIENTS IN ONE REGISTRY, TO DEVELOP THE REGISTRY INTO A POWERFUL RESOURCE FOR RESEARCH, AND TO ENGAGE THE FA COMMUNITY IN STUDIES AIMED AT ADVANCING OUR KNOWLEDGE OF FA AND THE TREATMENTS BEING DEVELOPED. FARA PARTNERS WITH INTERNATIONAL PATIENT ADVOCACY ORGANIZATIONS THROUGH A GOVERNANCE BOARD TO ENSURE MULTI-STAKEHOLDER ENGAGEMENT AND OVERSIGHT OF THE FAGPR. MORE THAN 1000 INDIVIDUALS WITH FA ARE ENROLLED AND FAGPR WAS USED TO RECRUIT FOR SEVERAL CLINICAL TRIALS AND MANY CLINICAL RESEARCH STUDIES. TO LEARN MORE, VISIT CUREFA.ORG/REGISTRY. COLLABORATIVE CLINICAL RESEARCH NETWORK IN FA (CCRN IN FA): THE CCRN IS AN INTERNATIONAL NETWORK OF 13 CLINICAL RESEARCH CENTERS (UNITED STATES, CANADA, AUSTRALIA, NEW ZEALAND) THAT WORK TOGETHER TO ADVANCE TREATMENTS AND CLINICAL CARE FOR INDIVIDUALS WITH FRIEDREICH'S ATAXIA. HAVING SUCH A NETWORK MEANS THAT THERE ARE TRAINED PHYSICIANS AND RESEARCH COORDINATORS READY TO DO CLINICAL RESEARCH STUDIES AND TRIALS. ALSO, THIS NETWORK IS BACKED BY A DATA COORDINATION CENTER THAT FACILITATES ALL ASPECTS OF DATA COLLECTION, DATABASE MANAGEMENT, AND STATISTICAL ANALYSIS OF STUDY DATA. IN 2021, A NEW CLINIC SITE IN NEW DEHLI, INDIA WAS ADDED TO THE NETWORK. TO LEARN MORE AND REVIEW A LIST OF RESEARCH PUBLICATIONS, VISIT CUREFA.ORG/NETWORK.HTMLTHE CCRN LEADS A NATURAL HISTORY AND CLINICAL OUTCOME MEASURE STUDY, CALLED FACOMS. LONGITUDINAL DATA (USUALLY ABOUT 10 YEARS) ON INDIVIDUALS WITH A DISEASE THAT DESCRIBES AND QUANTIFIES THE PROGRESSION OF THE DISEASE ALONG WITH THE SYMPTOMS AND MANIFESTATIONS OF THE DISEASE. NATURAL HISTORY CAN SOMETIMES SERVE AS THE BASIS FROM WHICH MEASUREMENTS CAN BE MADE TO DETERMINE EFFECTS OF NEW TREATMENTS, DRUGS OR INTERVENTIONS.CLINICAL OUTCOME MEASURES OR FUNCTIONAL PERFORMANCE TESTS (E.G., TIMED PEGBOARD OR WALK TESTS, VISION, HEARING OR SPEECH TESTS) ARE ASSESSED TO QUANTIFY HOW MUCH CHANGE TAKES PLACE IN A SPECIFIC AMOUNT OF TIME AND ARE USED IN CLINICAL TRIALS TO MEASURE WHETHER A DRUG IS ALTERING THE COURSE OF THE DISEASE.BIOMARKERS: ANYTHING THAT CAN BE USED AS AN INDICATOR OF A PARTICULAR DISEASE STATE - USUALLY PROTEINS, ENZYMES, GENETIC VARIANTS, IMAGING (MRI, CT OR PET SCANS). THE CCRN AND FACOMS COLLECT BLOOD AND TISSUE SAMPLES FOR BIOMARKER DISCOVERY. BIOMARKERS CAN BE USED TO ASSESS RISK OF DISEASE, DIAGNOSIS, OR OUTCOMES. USE OF BIOMARKERS IN DRUG DEVELOPMENT IS ESSENTIAL BECAUSE BIOMARKERS CAN PROVIDE EVIDENCE OF BIOLOGICAL ACTIVITY, POTENTIALLY DEMONSTRATING THERAPEUTIC BENEFIT MORE QUICKLY THAN TRADITIONAL OUTCOME MEASURES. BIOREPOSITORY: A REPOSITORY OF STORED BIOLOGICAL MATERIALS SUCH AS BLOOD SAMPLES, DNA, ORGANS, AND TISSUES (SUCH AS SKIN, MUSCLE, HEART) THAT CAN BE USED FOR RESEARCH.THROUGH THE CCRN IN FA WE HAVE COLLECTED NATURAL HISTORY DATA (ONGOING) IN MORE THAN 1,100 INDIVIDUALS WITH FA, VALIDATED CLINICAL OUTCOME MEASURES AND THE FARS SCALE, STUDIED SPEECH, VISION AND HEARING, LAUNCHED BIOMARKER STUDIES, ESTABLISHED DNA AND RNA REPOSITORIES, AND PROVIDED MANY BLOOD SAMPLES TO RESEARCHERS AROUND THE WORLD. THE CCRN IN FA INVESTIGATORS HAVE BEEN INVOLVED IN MULTIPLE CLINICAL TRIALS INCLUDING A FEW THAT WERE DESIGNED AND CONDUCTED SOLELY THROUGH NETWORK SITES. CRITICAL PATH INSTITUTE DATA COLLABORATION PROJECTIN 2017, FARA INITIATED A PROJECT WITH CRITICAL PATH INSTITUTE'S (C-PATH) DATA COLLABORATION CENTER (DCC) TO DEVELOP AN AGGREGATED DATABASE OF CLINICAL DATA FOR FA. SIX DE-IDENTIFIED DATASETS FROM PREVIOUS CLINICAL TRIALS AND THE CCRN FACOMS DATASETS HAVE BEEN AGGREGATED INTO A SINGLE DATABASE (FA-ICD) IN A SCIENTIFICALLY RIGOROUS MANNER. AS OF DECEMBER 31, 2021, THIS DATA WAS SHARED WITH >20 RESEARCH ORGANIZATIONS (ACADEMIC AND INDUSTRY) TO FURTHER UNDERSTAND THE CLINICAL PHENOTYPE, NATURAL HISTORY, OUTCOME MEASURES AND TO ASSIST IN DESIGNING CLINICAL TRIALS. THE FA-ICD IS ALSO A PART OF THE RARE DISEASE CURES ACCELERATOR DATA AND ANALYTICS PLATFORM (COLLABORATIVE PLATFORM ESTABLISHED BY C-PATH, NORD AND FDA) WHERE THERE IS BETTER SEARCH AND ANALYTIC FUNCTIONS AND THE ABILITY TO LOOK AT FA DATA SET IN COMBINATION WITH DATA FROM OTHER DISEASES.ADDITIONAL INFORMATION ABOUT FARA'S PROGRAMS IN 2021 CAN BE ACCESSED VIA THE ANNUAL REPORT AT: CUREFA.ORG/FINANCIALS

EDUCATION, AWARENESS & OUTREACH PROGRAMSFRIEDREICH ATAXIA (FA) IS A RARE DISEASE; AFFECTING 1 IN 50,000 INDIVIDUALS. FARA IS DEDICATED TO ADVOCACY AND RAISING AWARENESS FOR FA. FARA HAS UTILIZED BOTH TRADITIONAL AND SOCIAL MEDIA STRATEGIES TO BRING GREATER AWARENESS TO FA IN THE GENERAL PUBLIC AND TO ENGAGE AND EDUCATE THE FA COMMUNITY. FOR EXAMPLE, FARA CONDUCTED A SOCIAL MEDIA CAMPAIGN THAT ENCOURAGED COMMUNITY PARTICIPATION LEADING UP TO RARE DISEASE DAY AND FA AWARENESS DAY. FARA AND THE NATIONAL ATAXIA FOUNDATION (NAF) ALSO PARTNERED ON SEVERAL ADVOCACY INITIATIVES RELEVANT TO THE ATAXIA COMMUNITY SUCH AS GETTING A RESOLUTION PASSED DECLARING SEPTEMBER 25, 2021 NATIONAL ATAXIA AWARENESS DAY (S.RES 339) AND HOSTING THE THIRD UNITED AGAINST ATAXIA HILL DAY. IN 2021, 240 PEOPLE REGISTERED FOR UNITED AGAINST ATAXIA HILL DAY, RESULTING IN 100 CONGRESSIONAL MEETINGS COVERING 44 STATES. THE PRIMARY ASK WAS TO ADD "HEREDITARY ATAXIA" TO THE CONGRESSIONALLY DIRECTED MEDICAL RESEARCH PROGRAM (CDMRP) AT THE DEPARTMENT OF DEFENSE, WHICH WOULD PROVIDE ADDITIONAL FUNDING FOR ATAXIA RESEARCHERS. FARA ALSO SUPPORTED THE ACT FOR ALS WHICH PROMISED TO BRING NEW FEDERAL RESOURCES TO ALL RARE DEGENERATIVE CONDITIONS AND WHICH WAS SIGNED INTO LAW ON DEC 23, 2021. (PUBLIC LAW NO: 118-79)THE FARA AMBASSADOR PROGRAM, WHICH WAS LAUNCHED IN 2011 WITH APPROXIMATELY 20 PARTICIPANTS, HAD A YEAR OF CONTINUED GROWTH AND ACTIVITY IN 2021. THE PROGRAM NOW INCLUDES MORE THAN 80 PARTICIPANTS. THE MISSION OF THE FARA AMBASSADORS IS TO BE POSITIVE, SUPPORTIVE, PEER REPRESENTATIVES FOR THE FA COMMUNITY; ACTIVELY RAISING AWARENESS AND FUNDS FOR FARA. THE FARA AMBASSADOR PROGRAM MEMBERS ORGANIZE SERVICE PROJECT TEAMS INCLUDING:1. A BLOG TEAM THAT FACILITATES WEEKLY MEET THE COMMUNITY INTERVIEWS WITH PEOPLE LIVING WITH FA AND A MONTHLY MEET THE RESEARCHER INTERVIEW. 2. A CARD WRITING TEAM THAT WRITES PERSONALIZED CARDS TO SEND TO THE VARIOUS STAKEHOLDERS AND COMMUNITY MEMBERS THROUGHOUT THE YEAR TO SAY THANK YOU OR TO OFFER ENCOURAGEMENT. 3. MONTHLY PEER ZOOM HANGOUT GROUPS FOR THE GREATER FA COMMUNITY TO ATTEND AND CONNECT WITH ONE ANOTHER ONLINE (ONE IS GEARED TO ADULTS, AND ONE IS FOR TEENS WITH FA). 4. A SOCIAL MEDIA TEAM THAT GENERATES ENGAGING CONTENT IN SUPPORT OF FARA INITIATIVES AND CAMPAIGNS. 5. A SPEAKING TEAM WHERE PARTICIPANTS PRACTICE TELLING THEIR STORY LIVING WITH FA ON BEHALF OF THE ORGANIZATION.AMBASSADORS AND OTHER COMMUNITY REPRESENTATIVES CONNECTED WITH MANY OF FARA'S ACADEMIC AND INDUSTRY PARTNERS BY SERVING ON PATIENT PANELS AT 15 EVENTS. AMBASSADORS ALSO PARTICIPATED IN EDUCATION PROGRAMS TARGETING MEDICAL PROFESSIONALS, E.G., SPEAKING TO TRAINEES IN GENETIC COUNSELING PROGRAMS AND MEDICAL STUDENTS. FOR MORE INFORMATION VISIT CUREFA.ORG/AMBASSADORS.GROUP FARA HOSTED SEVERAL EDUCATION AND ENGAGEMENT INITIATIVES IN 2021. EARLY IN THE YEAR, FARA LAUNCHED A MONTHLY VIDEO SERIES TO EDUCATE FA FAMILIES AND SUPPORTERS ON THE FARA GRANT PROGRAM. EACH EPISODE EXPLAINS A TYPE OF RESEARCH FUNDED BY FARA IN A 5-MINUTE ANIMATED VIDEO, NARRATED BY AN FA COMMUNITY MEMBER. FARA RELEASED NINE MINUTES OF SCIENCE VIDEOS BY THE END OF 2021. FARA ALSO OFFERED THE FLASH TALKS SERIES IN MAY WHICH ALLOWED YOUNG INVESTIGATORS TO EXPLAIN THEIR RESEARCH IN LAYMAN TERMS AND TO ANSWER QUESTIONS IN A VIRTUAL FORMAT. IN SEPTEMBER, SELF-PACED EDUCATION FOR GENE THERAPY MODULES WERE RELEASED. BACKGROUND CONCEPTS, INFORMED CONSENT, AND FACILITATED DECISION-MAKING WERE COVERED IN A WHITEBOARD VIDEO AND IN ONLINE MODULES FOR SELF-DIRECTED LEARNING. THROUGHOUT THE YEAR INFORMATIONAL WEBINARS WERE PRESENTED ON THE FA TREATMENT PIPELINE AND CLINICAL RESEARCH STUDIES.

WORKSHOPS, SYMPOSIA, & CONFERENCESFARA HOSTED A HYBRID IN-PERSON AND VIRTUAL SINGLE-DAY EDUCATIONAL SYMPOSIUM FOR >200 PATIENTS AND CAREGIVERS IN KING OF PRUSSIA, PA (OCTOBER 10, 2021). THIS SYMPOSIUM PROVIDED AN OPPORTUNITY TO EDUCATE THE PATIENT/FAMILY COMMUNITY ON RESEARCH ADVANCES, PROGRESS ON CLINICAL TRIALS AND WAS A UNIQUE FORUM FOR PATIENTS AND RESEARCHERS TO ENGAGE AND LEARN FROM EACH OTHER'S EXPERIENCES AND PERSPECTIVES.