2022-08-05T05:41:17-05:00 2021-12-31 871353108 EISNER ADVISORY GROUP LLC 130 NORTH 18TH STREET SUITE 3000 PHILADELPHIA PA 191032757 990 2021-01-01 522122720 FRIEDREICH'S ATAXIA RESEARCH ALLIANCE FRIE 4848796160 PO BOX 1537 SPRINGFIELD VA 22151 JENNIFER FARMER CHIEF EXECUTIVE OFFICER 4848796160 2022-08-03 1 JENNIFER FARMER 999009999 HELEN M MARTIN P01330899 2158818800 3 AAL1 IAL1 2021 2022-09-23 18:48:47Z JENNIFER FARMER 533 W UWCHLAN AVENUE DOWNINGTOWN PA 19335 9652238 0 X WWW.CUREFA.ORG X 1998 VA TO TREAT AND CURE FRIEDREICH'S ATAXIA (FA) BY ADVANCING RESEARCH, AWARENESS AND PARTNERSHIPS. 18 16 13 500 0 0 12628384 7683701 0 0 108792 306924 -193341 -272847 12543835 7717778 6348843 7398876 0 0 995045 1160225 0 0 193923 394814 552566 7738702 9111667 4805133 -1393889 9314067 7478784 608696 283508 8705371 7195276 X TO MARSHAL AND FOCUS THE RESOURCES AND RELATIONSHIPS NEEDED TO CURE FA BY RAISING FUNDS FOR RESEARCH, PROMOTING PUBLIC AWARENESS AND ALIGNING SCIENTISTS, PATIENTS, CLINICIANS, GOVERNMENT AGENCIES AND PHARMACEUTICAL COMPANIES DEDICATED TO CURING FA AND RELATED DISORDERS. 0 0 7072712 6519272 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 1087755 879604 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 338711 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. 177445 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. 177445 8676623 1 1 0 1 0 0 0 0 0 0 1 0 0 0 0 1 1 0 0 1 1 1 0 0 1 0 0 1 0 1 0 0 0 0 0 0 0 0 1 0 0 0 0 0 0 0 0 1 1 0 1 13 1 0 0 0 0 0 1 1 0 0 0 0 0 0 X 18 16 0 0 0 0 0 0 0 1 1 0 0 1 1 1 1 1 1 1 1 0 AL AK AZ AR CA CO CT FL GA IL KS KY ME MD MS MI MN MS MO NH NJ NM NY NC ND OH OK OR PA RI SC TN UT VA WA WV WI MA X X JENNIFER FARMER 4848796160 533 W UWCHLAN AVENUE DOWNINGTOWN PA 19335 BARBARA A TATE CHIEF SCIENTIFIC OFFICER 40.00 X 250000 0 6439 JENNIFER M FARMER CHIEF EXECUTIVE OFFICER 40.00 X 150000 0 5123 RONALD BARTEK PRESIDENT/DIRECTOR 40.00 X X 110000 0 3734 ELISABETTA SORAGNI DIRECTOR OF RESEARCH 40.00 X 103000 0 13380 RUTH ACTON TREASURER/DIR OF FINANCE ADMIN, HR 25.00 X X 43050 0 12185 PAUL AVERY CHAIRMAN/ DIRECTOR 10.00 X X 0 0 0 MARILYN E DOWNING SECRETARY/ DIRECTOR 10.00 X X 0 0 0 DR SANJAY BIDICHANDANI DIRECTOR 10.00 X 0 0 0 BRIGID BRENNAN DIRECTOR/ GENERAL COUNSEL 20.00 X 0 0 0 THOMAS BRENNINKMEIJER DIRECTOR 10.00 X 0 0 0 ALEX FIELDING DIRECTOR 10.00 X 0 0 0 JENNIFER GOOD DIRECTOR 10.00 X 0 0 0 THOMAS HAMILTON DIRECTOR 10.00 X 0 0 0 DR HOLLY HEDRICK DIRECTOR 10.00 X 0 0 0 DEREK G HENNECKE DIRECTOR 10.00 X 0 0 0 DR STEVE KLASKO DIRECTOR 10.00 X 0 0 0 DR KATHY MATHEWS DIRECTOR 10.00 X 0 0 0 DR JAMES MCARTHUR DIRECTOR 10.00 X 0 0 0 TONY PLOHOROS DIRECTOR 10.00 X 0 0 0 PATRICK RITSCHEL DIRECTOR 10.00 X 0 0 0 DR JAMES R RUSCHE DIRECTOR 10.00 X 0 0 0 656050 0 40861 4 0 1 0 0 9288 3415421 212315 4046677 457781 7683701 47170 47170 1404133 1144379 259754 259754 259754 517234 3415421 790081 -272847 -272847 7717778 0 0 34077 5509037 5509037 1889839 1889839 324092 212690 65387 46015 678343 604783 51495 22065 21914 13569 1284 7061 67130 36401 5400 25329 68746 54635 7783 6328 18200 18200 207357 195461 8116 3780 10 10 25748 7169 4667 13912 37357 24905 6948 5504 79359 37806 41553 15950 11993 3957 SUBSCRIPTIONS & LICENSI 94983 81879 4559 8545 MISCELLANEOUS 32003 144 31859 FACILITIES & EQUIPMENT 15276 2887 12134 255 CREDIT CARD & BANK FEES 13569 60 189 13320 12754 5502 1269 5983 9111667 8676623 241121 193923 3850878 3334786 1807813 277880 259819 42086 94042 7135 7135 0 0 5137765 1976866 5458 5458 9314067 7478784 508696 261148 100000 22360 608696 283508 X 4124902 4608013 4580469 2587263 8705371 7195276 9314067 7478784 X -1393889 -100126 -17664 1584 X X 0 1 X 1 0 X 7518776 7662625 7146206 12628384 7808772 42764763 7518776 7662625 7146206 12628384 7808772 42764763 6952524 35812239 37283 42296 45345 38997 47170 211091 87760 87760 43063614 0.83160 0.71800 X RESTRICTED RESTRICTED RESTRICTED RESTRICTED RESTRICTED RESTRICTED RESTRICTED RESTRICTED 3570 16117 19687 9577658 9597345 629867 157467 0 0 481734 567238 564107 629867 2242946 3364419 8925 12259 9997 19687 50868 120434 141810 141027 157467 560738 841107 5326 7316 5966 3570 22178 7135 7135 0 0 X 8486069 -100126 96000 790081 785955 7700114 17664 17664 7717778 9996164 96000 790081 886081 9110083 1584 1584 9111667 PART X, LINE 2: THE INTERNAL REVENUE SERVICE HAS CLASSIFIED FARA AS EXEMPT FROM FEDERAL INCOME TAXES UNDER SECTION 501(C)(3) OF THE INTERNAL REVENUE CODE. ACCOUNTING FOR UNCERTAINTY IN INCOME TAXES CLARIFIES THE ACCOUNTING FOR UNCERTAINTY IN INCOME TAXES RECOGNIZED IN AN ENTERPRISE'S FINANCIAL STATEMENTS. MANAGEMENT HAS ANALYZED THE TAX POSITIONS TAKEN BY FARA, AND HAS CONCLUDED THAT AS OF DECEMBER 31, 2021, THERE ARE NO UNCERTAIN TAX POSITIONS TAKEN OR EXPECTED TO BE TAKEN THAT WOULD REQUIRE RECOGNITION OF A LIABILITY OR DISCLOSURE IN THE FINANCIAL STATEMENTS. FARA RECOGNIZES ACCRUED INTEREST AND PENALTIES ASSOCIATED WITH UNCERTAIN TAX POSITIONS, IF ANY. THERE WERE NO INCOME TAX RELATED INTEREST OR PENALTIES RECORDED FOR THE YEARS ENDED DECEMBER 31, 2021 AND 2020. PART XI, LINE 2D - OTHER ADJUSTMENTS: SPECIAL EVENTS EXPENSE 790,081. PART XII, LINE 2D - OTHER ADJUSTMENTS: SPECIAL EVENTS EXPENSE 790,081. PART XII, LINE 4B - OTHER ADJUSTMENTS: PRIOR YEAR GRANT REFUNDS NETTED AGAINST EXPENSE ON FINANCIAL STATEMENTS 1,584. 1 EUROPE 0 1 GRANTS TO RECIPIENTS LOCATED IN REGION RESEARCH AND GRANT PROGRAM 1227516 EAST ASIA AND THE PACIFIC 0 0 GRANTS TO RECIPIENTS LOCATED IN REGION RESEARCH AND GRANT PROGRAM 605388 SOUTH AMERICA 0 0 GRANTS TO RECIPIENTS LOCATED IN REGION RESEARCH AND GRANT PROGRAM 46935 CANADA 0 0 GRANTS TO RECIPIENTS LOCATED IN REGION RESEARCH AND GRANT PROGRAM 10000 0 1 0 0 0 1 1889839 0 1889839 EAST ASIA AND THE PACIFIC MEDICAL RESEARCH 85645 WIRE 0 EUROPE MEDICAL RESEARCH 82590 WIRE 0 EUROPE MEDICAL RESEARCH 150000 WIRE 0 EAST ASIA AND THE PACIFIC MEDICAL RESEARCH 265990 WIRE 0 EUROPE MEDICAL RESEARCH 236250 WIRE 0 EUROPE MEDICAL RESEARCH 148887 WIRE 0 EAST ASIA AND THE PACIFIC MEDICAL RESEARCH 52253 WIRE 0 EAST ASIA AND THE PACIFIC MEDICAL RESEARCH 10500 WIRE 0 EAST ASIA AND THE PACIFIC MEDICAL RESEARCH 150000 WIRE 0 EUROPE MEDICAL RESEARCH 82901 WIRE 0 SOUTH AMERICA - ARGENTINA, BOLIVIA, BRAZIL, CHILE, COLUMBIA, ECUADOR, MEDICAL RESEARCH 46935 WIRE 0 NORTH AMERICA - CANADA, MEXICO (NOT UNITED STATES) MEDICAL RESEARCH 10000 WIRE 0 EUROPE MEDICAL RESEARCH 35000 WIRE 0 EUROPE MEDICAL RESEARCH 59732 WIRE 0 EUROPE MEDICAL RESEARCH 99742 WIRE 0 EUROPE MEDICAL RESEARCH 125000 WIRE 0 EUROPE MEDICAL RESEARCH 73236 WIRE 0 EAST ASIA AND THE PACIFIC MEDICAL RESEARCH 41000 WIRE 0 EUROPE MEDICAL RESEARCH 134178 WIRE 0 17 2 0 0 0 0 0 0 PART I, LINE 2: GRANTS ARE REVIEWED BY INDEPENDENT SCIENTIFIC ADVISORS AND APPROVED BY BOARD. ONCE GRANTS ARE AWARDED THE ORGANIZATION REQUIRES REGULAR RESEARCH AND FINANCIAL REPORTS FROM THE INSTITUTIONS. PART III ACCOUNTING METHOD: FARA ENERGY BALL 1740051 1384581 355470 111416 8500 240548 RIDEATAXIA PHILA 612394 597169 15225 60802 12 1580210 1433671 146539 368815 3932655 3415421 517234 111416 8500 670165 790081 -272847 FELICIA DEROSA 533 W UWCHLAN AVENUE DOWNINGTOWN PA 19335 1 CHILDREN'S HOSPITAL OF PHILADELPHIA 34TH ST CIVIC CENTER BLVD PHILADELPHIA PA 19104 231352166 501(C)(3) 556704 0 MEDICAL RESEARCH CHILDREN'S HOSPITAL OF PHILADELPHIA FOUNDATION 34TH ST CIVIC CENTER BLVD PHILADELPHIA PA 19104 232237932 501(C)(3) 425000 0 MEDICAL RESEARCH OHIO STATE UNIVERSITY 333 W 10TH AVE COLUMBUS OH 43210 316025986 501(C)(3) 5300 0 MEDICAL RESEARCH THE REGENTS OF THE UNIVERSITY OF CALIFORNIA LOS ANGELES 10920 WILSHIRE BLVD 5TH FLOOR LOS ANGELES CA 90024 956006143 501(C)(3) 45550 0 MEDICAL RESEARCH UNIVERSITY OF FLORIDA DEPT OF NEUROLOGY L3-100 MCKNIGHT BRAIN INSTITUTE NEWELL DRIVE GAINESVILLE FL 32611 596002052 501(C)(3) 168440 0 MEDICAL RESEARCH UNIVERSITY OF ROCHESTER 515 HYLAN BUILDING RC BOX 270140 ROCHESTER NY 14627 160743209 501(C)(3) 333963 0 MEDICAL RESEARCH ALBANY RESEARCH INSTITUTE 113 HOLLAND AVE ALBANY NY 12208 141716021 501(C)(3) 109745 0 MEDICAL RESEARCH TRUSTREES OF THE UNIVERSITY OF PENNSYLVANIA 3451 WALNUT ST RM 310 PHILADELPHIA PA 19104 231352685 501(C)(3) 412551 0 MEDICAL RESEARCH UNIVERSITY OF ALABAMA - BIRMINGHAM 1720 2ND AVE S BIRMINGHAM AL 35294 636005396 501(C)(3) 150000 0 MEDICAL RESEARCH NYU SCHOOL OF MEDICINE 550 1ST AVENUE NEW YORK NY 10016 135562309 501(C)(3) 25000 0 MEDICAL RESEARCH WEILL MEDICAL COLLEGE OF CORNELL UNIVERSITY 1300 YORK AVENUE C-118 NEW YORK NY 10065 131623978 501(C)(3) 150000 0 MEDICAL RESEARCH UNIVERSITY OF OKLAHOMA HEALTH SCIENCES CENTER 865 RESEARCH PARKWAY SUITE 450 OKLAHOMA CITY OK 73126 731563627 501(C)(3) 57000 0 MEDICAL RESEARCH STANFORD UNIVERSITY 2770 SAND HILL ROAD MENLO PARK CA 94025 941156365 501(C)(3) 142643 0 MEDICAL RESEARCH THE BROAD INSTITUTE 415 MAIN STREET CAMBRIDGE MA 02142 263428781 501(C)(3) 1591000 0 MEDICAL RESEARCH UNIVERSITY OF CALIFORNIA DAVIS 1850 RESEARCH PARK DRIVE DAVIS CA 95618 946036494 501(C)(3) 225000 0 MEDICAL RESEARCH UNIVERSITY OF PITTSBURGH 116 ATWOOD STREET SUITE 201 PITTSBURGH PA 15213 250965591 501(C)(3) 225000 0 MEDICAL RESEARCH BURKE NEUROLOGICAL INSTITUTE 785 MAMARONECK AVENUE WHITE PLAINS NY 10605 133434924 501(C)(3) 75000 0 MEDICAL RESEARCH CYAGEN BIOMODELS LLC 2255 MARTIN AVE STE E SANTA CLARA CA 95050 20455 0 MEDICAL RESEARCH MASSACHUSETTS GENERAL HOSPITAL 55 FRUIT STREET BOSTON MA 02114 041564655 501(C)(3) 71875 0 MEDICAL RESEARCH NATIONAL ATAXIA FOUNDATION 600 HIGHWAY 169 SOUTH SUITE 1725 MINNEAPOLIS MN 55455 410832903 501(C)(3) 24750 0 MEDICAL RESEARCH THE REGENTS OF THE UNIVERSITY OF CALIFORNIA SAN DIEGO 9500 GILMAN DRIVE LA JOLLA CA 92093 956006144 501(C)(3) 125000 0 MEDICAL RESEARCH THE REGENTS OF THE UNIVERSITY OF MINNESOTA 200 SE OAK ST 600 MINNEAPOLIS MN 55455 416007513 501(C)(3) 234361 0 MEDICAL RESEARCH THOMAS JEFFERSON UNIVERSITY 1020 WALNUT STREET PHILADELPHIA PA 19107 232829095 501(C)(3) 150000 0 MEDICAL RESEARCH UNIVERSITY OF SOUTH FLORIDA FOUNDATION 4202 EAST FOWLER AVE TAMPA FL 33620 590879015 501(C)(3) 100000 0 MEDICAL RESEARCH THE TRUSTEES OF COLUMBIA UNIVERSITY IN THE CITY OF NEW YORK 154 HAVEN AVENUE SECOND FLOOR NEW YORK NY 10032 135598093 501(C)(3) 75000 0 MEDICAL RESEARCH 24 1 PART I, LINE 2: GRANTS ARE REVIEWED BY INDEPENDENT SCIENTIFIC ADVISORS AND APPROVED BY BOARD. ONCE GRANTS ARE AWARDED THE ORGANIZATION REQUIRES REGULAR RESEARCH AND FINANCIAL REPORTS FROM THE INSTITUTIONS. X X 0 0 0 0 0 0 0 0 0 BARBARA A TATE CHIEF SCIENTIFIC OFFICER 250000 0 0 0 0 0 5850 0 589 0 256439 0 0 0 JENNIFER M FARMER CHIEF EXECUTIVE OFFICER 150000 0 0 0 0 0 4500 0 623 0 155123 0 0 0 X 10 457781 HI-LOW DATE OF GIFT 0 1 0 PART I, COLUMN (B): THE ORGANIZATION RECOGNIZES IN COLUMN B EACH UNIQUE NON-CASH CONTRIBUTION RECEIVED FROM A DONOR. FORM 990, PART VI, SECTION B, LINE 11B COPIES OF 990 ARE DISTRIBUTED TO BOARD MEMBERS FOR REVIEW AND APPROVAL PRIOR TO FILING. FORM 990, PART VI, SECTION B, LINE 12C ALL NEW AND EXISTING BOARD MEMBERS ARE REQUIRED TO ANNUALLY REVIEW AND SIGN A CONFLICT OF INTEREST POLICY AND REPORT DISCLOSURES. FORM 990, PART VI, SECTION B, LINE 15 EVALUATION AND COMPENSATION COMMITTEE PERFORMS EMPLOYEE EVALUATIONS AND DETERMINES SALARY INCREASES ON A YEARLY BASIS FOR ALL EMPLOYEES. COMMITTEE EXAMINES BENCHMARK DATA IN DETERMINING SALARIES FOR PRESIDENT, CEO, CSO AND DIRECTOR FINANCE ADMINISTRATION. FORM 990, PART VI, SECTION C, LINE 19 FORM 990 IS AVAILABLE ON THE ORGANIZATION'S WEBSITE AS WELL AS GUIDESTAR.ORG AND CHARITYNAVIGATOR.ORG FINANCIAL STATEMENTS ARE AVAILABLE ON THE ORGANIZATION'S WEBSITE. THE GOVERNING DOCUMENTS, CONFLICT OF INTEREST POLICY AND FORM 1023 ARE AVAILABLE UPON REQUEST. FORM 990, PART XI, LINE 9: PRIOR YEAR GRANT REFUNDS NETTED AGAINST EXPENSE ON FINANCIAL STATEMENTS 1,584. FORM 990, PART XII, LINE 2C FARA'S AUDIT COMMITTEE CONSISTS OF THE FINANCE COMMITTEE, CEO AND ONE AT-LARGE BOARD MEMBER. EACH YEAR THE AUDIT COMMITTEE SEEKS THE SERVICES OF AN OUTSIDE ACCOUNTING FIRM AND CONTRACTS FOR A FULL AUDIT, PREPARATION OF FINANCIAL STATEMENTS AND FILING OF THE 990. THE AUDIT COMMITTEE IS RESPONSIBLE FOR REVIEWING RECOMMENDATIONS FROM THE AUDIT AND PROPOSING NEW POLICIES AND PROCEDURES AS NECESSARY. THE AUDIT COMMITTEE ALSO PARTICIPATES IN DETAILED REVIEW OF FINANCIAL STATEMENTS AND 990 PRIOR TO SHARING WITH THE FULL BOARD FOR A VOTE. ALL 990 INFORMATION IS VERIFIED FOR ACCURACY AND COMPLETENESS BY FARA'S DIRECTOR OF FINANCE, FARA'S VP OF FUNDRAISING AND COMMUNICATIONS AND FARA'S CEO. THE VERIFIED DRAFT IS REVIEWED BY THE FARA FINANCE COMMITTEE AND SUBMITTED TO THE FULL FARA BOARD OF DIRECTORS FOR REVIEW AND APPROVAL PRIOR TO FILING.