2017-05-15T13:49:46-05:00 2016-12-31 942682998 OUM & CO LLP 601 CALIFORNIA STREET SUITE 1800 SAN FRANCISCO CA 94108 990 2016-01-01 465727883 GRACE SCIENCE FOUNDATION GRAC 4152557666 235 KANSAS STREET SUITE 200 SAN FRANCISCO CA 94103 MATTHEW R WILSEY PRESIDENT 4152557666 2017-05-12 1 BRADFORD WEISERT P00044664 4154343744 38.122.223.90 2017-05-12 14:10:23 PD P DA39A3EE5E6B4B0D3255BFEF95601890AFD80709 96FC091396EF191060C675787B6AB743C83BF1A5 2016 2021-01-29 14:40:06Z MATTHEW R WILSEY 235 KANSAS STREET SUITE 200 SAN FRANCISCO CA 94103 600124 0 X WWW.GRACESCIENCE.ORG X 2014 CA THE MISSION OF THE FOUNDATION IS TO SUPPORT AND FUND THE DEVELOPMENT OF LIFE-SAVING TREATMENT FOR PATIENTS THAT ARE DEFICIENT IN THE N-GLYCANESE ENZYME AND THOSE SUFFERING FROM RELATED GENETIC DISEASES. 7 7 1 5 0 0 1560381 600124 0 0 0 0 0 0 1560381 600124 409865 612814 0 0 0 1247 0 0 0 148480 255746 558345 869807 1002036 -269683 1605814 1336131 0 0 1605814 1336131 X THE MISSION OF THE FOUNDATION IS TO SUPPORT AND FUND THE DEVELOPMENT OF LIFE-SAVING TREATMENT FOR PATIENTS THAT ARE DEFICIENT IN THE N-GLYCANESE ENZYME AND THOSE SUFFERING FROM RELATED GENETIC DISEASES. THE ACTIVITIES OF THE FOUNDATION WILL INVOLVE PROVIDING GRANT FUNDING TO HOSPITALS AND MEDICAL RESEARCH FACILITIES WHICH ARE CONDUCTING MEDICAL RESEARCH IN VARIOUS FIELDS RELATING TO THE SEARCH FOR UNDERSTANDING AND TREATMENT OF THIS CONDITION, AND FOSTERING COLLABORATION BETWEEN ACADEMIA, RESEARCH INSTITUTIONS, HOSPITALS, PRIVATE COMPANIES, AND GOVERNMENTAL AGENCIES. 0 0 140713 125416 GLADSTONE INSTITUTE - THE GOALS OF THE PROJECT WERE TO GENERATE IPSC LINES FROM A NGLY1 FAMILY WITH ISOGENIC CONTROLS AND TO FIND CELLULAR PHENOTYPES OF NGLY1 DEFICIENCY. OVER 2016, THE TEAM RAN INTO A CRITICAL ROADBLOCK REGARDING MAINTENANCE OF PATIENT-DERIVED IPSCS, PARTICULARLY REGARDING INSTABILITY OF CELLS OVER SUBSEQUENT PASSAGES. THE TEAM GENERATED NEW IPS CELL LINES FROM THE AFFECTED FAMILY AND 2ND PATIENT FAMILY TO OBTAIN STABLE PATIENT AND ISOGENIC CONTROL CELL LINES, AS WELL AS DETERMINE WHETHER INSTABILITY OF CELLS IS A PHENOTYPE OF NGLY1 DEFICIENCY. IN ADDITION, THEY GENERATED ISOGENIC CONTROL CELL LINES USING BOTH CRISPR/CA9 GENOME EDITING AND CRISPR IN WILD TYPE IPSCS TO ISOLATE PHENOTYPE OF DISEASE. FINALLY, THEY BEGAN OPTIMIZING HEPATIC DIFFERENTIATION AND LIVER BUD GENERATION. 119297 104000 LUCILE PACKARD FOUNDATION FOR CHILDREN'S HEALTH - PRIMARY GOAL FOR THIS PROJECT IS TO STUDY NGLY1 DEFICIENCY USING INDUCED PLURIPOTENT STEM CELLS (IPSC) AS A TOOL. IN THE PAST, THIS GROUP HAS DERIVED IPSC LINES FROM FIBROBLAST CELLS WITH DIFFERENT NGLY1 MUTATIONS. ONE OF OUR MAJOR GOALS IS TO BETTER UNDERSTAND THE EFFECT OF NGLY1 DEFICIENCY DURING DISEASE DEVELOPMENT, ESPECIALLY IN THE DELAYED NEURAL DEVELOPMENT. 2015 WAS FOCUSED ON EXPANDING THE IPSC LINES AND DIFFERENTIATING THEM INTO NEURAL PROGENITOR CELLS. 2016 WAS DOING DETAILED OMICS PROFILING ON THE CELLS LINES. THE TEAM HAS FOUND INTERESTING PATHWAYS AND DRUG TARGETS VIA THIS RESEARCH. THEY HAVE ALSO SEEN CONNECTIONS WITH OTHER DISEASES THAT COULD HELP MANY PEOPLE IN ADDITION TO SPEEDING UP A TREATMENT FOR NGLY1 DEFICIENCY. 115467 100170 THE JACKSON LABORATORY - USING CRISPR TECHNOLOGIES THE JACKSON LABORATORY HAS CREATED THREE MUTATIONS, TWO THAT REPRESENT THE ALLELES A NGLY1 PATIENT AS WELL AS AN OUT OF FRAME NULL MUTATION. THE OUT OF FRAME NULL MUTATION IS A BY-PRODUCT OF THE CREATING THE KNOCK IN MUTATION. WHILE A COLLABORATOR IN JAPAN HAS MADE AN ENGASE KNOCK OUT ALLELE AVAILABLE FROM A REPOSITORY, THE COST TO PROCURE AND SHIP MICE TO THE U.S. IS GREAT AND THE MTAS REQUIRED TO PROCURE THE MICE ARE TIME CONSUMING AS TECH TRANSFER OFFICES NEGOTIATE TERMS. FOR EASE OF USE TO THE SCIENTIFIC COMMUNITY, WE HAVE USED CRISPR TECHNOLOGY TO GENERATE AN ADDITIONAL ENGASE KO MICE. THE ADVANTAGES OF HAVING THE ADDITIONAL JAX ALLELE ARE THAT NO MTAS ARE REQUIRED FOR DISTRIBUTION AND IT IS ALSO ON A PURE B6 GENETIC BACKGROUND. FINALLY, THE TEAM HAS ALSO MADE A CONDITIONAL KNOCK OUT MODEL THAT SHOULD BE QUITE USEFUL TO THE BROADER RESEARCH COMMUNITY IN 2017. 89273 89273 TEXAS CHILDREN'S HOSPITAL - DETERMINED THE EFFECT OF THE R402 DELETION ON THE STABILITY AND FUNCTION OF NGLY1. THE TEAM ALSO DETERMINED THE MECHANISM UNDERLYING THE PNGL MUTANT PHENOTYPES IN FLIES. FINALLY, THE RESEARCHERS IDENTIFIED GENETIC MODIFIERS OF THE PNGL PHENOTYPES IN FLIES. 60000 60000 PERLSTEIN LAB PBC - DEVELOPED A DROSOPHILA-BASED HIGH-THROUGHPUT DRUG SCREENFOR NGLY1. THEY OVERCAME TWO PERSISTENT CHALLENGES THROUGHOUT THE OPTIMIZATION PROCESS, SO INPARALLEL THEY PILOTED YEAST-TO-FLY SCREENING APPROACH TO IDENTIFY NOVEL EVOLUTIONARILY CONSERVEDCHEMICAL MODIFIERS OF NGLY1 DEFICIENCY. THEY ALSO PERFORMED A SCREEN WITH SEVERAL THOUSAND BIOACTIVE COMPOUNDS INCLUDING THE SET OF ALL FDA APPROVED DRUGS. 50000 50000 STANFORD UNIVERSITY - PRIMARY GOALS ARE TO IDENTIFY AND INVESTIGATE IMPORTANT NGLY1 SUBSTRATES. IN ADDITION, THEY ARE GENERATING AND CHARACTERIZING ZEBRAFISH MODELS. 99253 83955 OTHER PROGRAM SERVICES ARE RELATED TO RESEARCH AND DEVELOPMENT TO UNDERSTAND AND CURE RARE DISEASES BEGINNING WITH NGLY1 DEFICIENCY. 298526 283228 674003 1 1 0 0 0 0 0 0 0 0 0 0 0 0 0 0 0 0 0 0 0 0 0 0 0 0 0 1 0 0 0 0 0 0 0 0 0 0 0 0 0 0 0 0 0 0 0 1 8 0 1 1 0 0 0 0 0 0 0 0 0 0 X 7 7 1 0 0 0 0 1 0 1 0 0 0 0 1 1 1 0 0 0 0 0 CA X GRACE SCIENCE FOUNDATION 4152557666 235 KANSAS STREET SUITE 200 SAN FRANCISCO CA 94103 X MATTHEW WILSEY PRESIDENT 40.00 X X 0 0 0 EGON DURBAN CHIEF FINANCIAL OFFICER 1.00 X X 0 0 0 DR KEN DRAZAN SECRETARY 1.00 X X 0 0 0 PETE BRIGER DIRECTOR 1.00 X 0 0 0 CHELSEA CLINTON DIRECTOR 1.00 X 0 0 0 J TAYLOR CRANDALL DIRECTOR 1.00 X 0 0 0 ELLE STEPHENS DIRECTOR 1.00 X 0 0 0 0 0 0 0 0 0 0 0 600124 600124 600124 0 0 0 612814 612814 974 974 273 273 27760 27760 5750 5750 77050 77050 62207 62207 9239 192 9047 17654 17265 389 47017 43727 3290 401 401 MISCELLANEOUS EXPENSES 7044 7044 PHOTOGRAPHY 875 875 OTHER FEES (BANK, CREDI 589 5 584 FILING FEES 160 160 869807 674003 195804 0 1605814 1336006 125 1605814 1336131 0 0 X 0 0 0 0 1605814 1336131 1605814 1336131 1605814 1336131 -269683 0 X 0 0 0 X 664490 1560381 600124 2824995 664490 1560381 600124 2824995 2824995 2824995 X PART II, LINE 13 GRACE SCIENCE FOUNDATION WAS FORMED ON MAY 19, 2014. 2016 IS THE FOUNDATION'S THIRD YEAR OF EXISTENCE. RESTRICTED RESTRICTED RESTRICTED RESTRICTED RESTRICTED RESTRICTED RESTRICTED RESTRICTED 1 GLADSTONE INSTITUTE 1650 OWENS ST PALO ALTO CA 94301 237203666 501(C)(3) 125416 STEM CELL GENERATION AND ANALYSIS LUCILE PACKARD FOUNDATION FOR CHILDREN'S HEALTH 400 HAMILTON AVE 340 PALO ALTO CA 94301 770440090 501(C)(3) 104000 STEM CELL GENERATION AND ANALYSIS THE JACKSON LABORATORY 600 MAIN STREET BAR HARBOR ME 04609 010211513 501(C)(3) 108170 MOUSE RESEARCH TEXAS CHILDREN'S HOSPITAL 6621 FANNIN STREET HOUSTON TX 77030 741100555 501(C)(3) 89273 FLY RESEARCH PERLSTEIN LAB PBC 6000 SHORELINE CT SUITE 204 S SAN FRANCISCO CA 94080 60000 FLY NATURAL HISTORY STUDY STANFORD UNIVERSITY 3145 PORTER DRIVE PALO ALTO CA 94304 941156365 501(C)(3) 50000 STUDYING NGLY1'S CONNECTION WITH TRANSCRIPTION FACTOR VIRGINIA COMMONWEALTH UNIVERSITY 800 E LEIGH ST STE 3200 BX RICHMOND VA 23298 546001758 501(C)(3) 37955 STUDYING NGLY1'S CONNECTION WITH TRANSCRIPTION FACTOR AMERICAN FOUNDATION FOR THE UNIVERSITY OF BRITISH COLUMBIA 1718 M ST NW WASHINGTON DC 20036 521559117 501(C)(3) 23000 READ THROUGH DRUG DISCOVERY RESEARCH RETROPHIN INC 12255 EL CAMINO REAL SUITE 250 SAN DIEGO CA 92130 274842691 15000 ASSAY DEVELOPMENT & SMALL MOLECULE DEVELOPMENT 7 2 PART I, LINE 2: * BEFORE FUNDING A PRINCIPAL INVESTIGATOR, THE FOUNDATION REQUIRES A DETAILED PROPOSAL WITH OBJECTIVES AND MILESTONES * THE PROPOSALS ARE REVIEWED BY THE FOUNDATION AND SCIENTIFIC ADVISORY BOARD * THE PROPOSALS ARE OFTEN REVISED SEVERAL TIMES TO GET THE CORRECT FIT * ONCE FUNDED, EACH PRINCIPAL INVESTIGATOR SUBMITS A WRITTEN SUMMARY EVERY QUARTER * THIS UPDATE IS TWO TO THREE PAGES IN LENGTH * THE REPORTS DISCUSS PROGRESS, HIGHLIGHT ROADBLOCKS AND IDENTIFY WHERE THEY NEED HELP * THE FOUNDATION STEPS-IN WHERE APPROPRIATE TO MAKE SURE OBJECTIVES AND MILESTONES ARE MET * IN ADDITION TO QUARTERLY REPORTS, THE FOUNDATION HOLDS A MONTHLY CONFERENCE CALL WHERE TWO TO THREE PRINCIPAL INVESTIGATORS PRESENT THEIR LATEST WORK * BEYOND THE ABOVE TWO INITIATIVES, THE FOUNDATION HOLDS ONE TO TWO IN-PERSON MEETINGS PER YEAR * DR. KEVIN LEE (CHIEF SCIENTIFIC OFFICER), DR. SELINA DWIGHT (PROGRAM MANAGER), AND MATT WILSEY(PRESIDENT) CONTACT RESEARCHERS AND PRINCIPAL INVESTIGATORS ON A REGULAR BASIS VIA EMAIL, PHONE, OR ONE ON ONE MEETINGS THE PRESIDENT ENSURES THAT EVERY DOLLAR IS USED TO SUPPORT THE GRACE SCIENCE FOUNDATION'S MISSION. FORM 990, PART VI, SECTION A, LINE 2 THE PRESIDENT OF THE FOUNDATION HAS IMMATERIAL OWNERSHIP INTEREST, IN THE INVESTMENTS OF FORTRESS AND SILVER LAKE, WHICH ARE MANAGED BY TWO OF THE DIRECTORS. FORM 990, PART VI, SECTION A, LINE 7A THE PRESIDENT OF THE FOUNDATION HAS AUTHORITY TO ELECT ONE OR MORE MEMBERS OF THE GOVERNING BODY. FORM 990, PART VI, SECTION A, LINE 8B THE FOUNDATION DOES NOT HAVE ANY COMMITTEES. FORM 990, PART VI, SECTION B, LINE 11B PRIOR TO FILING FROM 990, THE FOUNDATION'S PRESIDENT WILL REVIEW THE FORM. FORM 990, PART VI, SECTION B, LINE 12C NEW BOARD MEMBERS ARE REQUIRED TO COMPLETE A CONFLICTS OF INTEREST POLICY AND SELF REPORT ANY CHANGES THAT MAY PRESENT A CONFLICT. FORM 990, PART VI, SECTION C, LINE 18 AVAILABLE UPON REQUEST. FORM 990, PART VI, SECTION C, LINE 19 AVAILABLE UPON REQUEST.