2020-11-16T14:13:21-06:00 2019-12-31 942682998 OUM & CO LLP 601 CALIFORNIA STREET SUITE 1800 SAN FRANCISCO CA 94108 990 2019-01-01 465727883 GRACE SCIENCE FOUNDATION GRAC 4152557666 1142 CRANE ST 4 MENLO PARK CA 94025 MATTHEW R WILSEY PRESIDENT 4152557666 2020-11-13 1 BRADFORD WEISERT P00044664 4154343744 73.158.186.70 2020-11-13 18:04:55 PS P DA39A3EE5E6B4B0D3255BFEF95601890AFD80709 40035C32AB58F3080D53A27EBA070D7A16F3DC6C 2019 2021-01-29 14:40:06Z X MATTHEW R WILSEY 1142 CRANE ST 4 MENLO PARK CA 94025 1565376 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 0 0 0 0 1187802 1522789 0 0 12186 38513 0 0 1199988 1561302 898851 709424 0 0 0 0 0 0 0 175213 129971 1074064 839395 125924 721907 2196374 2889615 0 2201 2196374 2887414 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 172296 167534 THE JACKSON LABORATORY (CAT LUTZ) - THE TEAM GENERATED A SPECIFIC MOUSE MODEL OF NGLY1 LOSS AND COMPLETED A DEEP PHENOTYPIC ANALYSIS OF THESE MICE TO CHARACTERIZE DISEASE RELEVANT PHENOTYPES AND MAP THEIR APPEARANCE OVER TIME. THESE STUDIES PROVIDED INSIGHTS INTO WHICH TISSUES ARE IMPORTANT IN DISEASE PATHOLOGY AND PROVIDE A MODEL FOR TESTING POTENTIAL THERAPIES TO REVERSE PATHOLOGIES ASSOCIATED WITH NGLY1 DEFICIENCY 128978 124216 SALK INSTITUTE FOR BIOLOICAL SCIENCES (GAGE LAB)- TEAM HAS BEEN DEVELOPING INDUCED PLURIPOTENT STEM CELLS (IPSCS) FROM NGLY1 DEFICIENT PATIENTS AS A MODEL SYSTEM TO CHARACTERIZE THE FUNCTIONAL CHANGES CAUSED BY NGLY1 LOSS AND TO TEST CANDIDATE THERAPIES TO REVERSE THOSE CHANGES. THEY OBSERVED THE PRESENCE OF PROTEIN AGGREGATES, MITOCHONDIAL DYSFUNCTION AND REDUCED SYNAPTIC ACTIVITY IN NEURONS DERIVED FROM PATIENT IPSCS COMPARED WITH CONTROLS. THE TEAM HAS VERIFIED THAT THESE PHENOTYPIC CHANGES ARE RESUCED BY REINTRODUCING A FUNCTIONAL NGLY1 GENE INTO THE CELLS, AND HAS BEEN CHARACTERIZING THE COMPOSITION OF THE AGGREGATES FOUND IN NGLY1 CELLS. THESE DISCOVERIES IDENTIFIED PHENOTYPES THAT CAN BE LEVERAGED FOR HIGH-THROUGHPUT DRUG SCREENS AND PROVIDE INSIGHT AS TO THE CELL TYPES AFFECTED BY THE DISEASE IN PATIENTS. 76502 71740 UNIVERSITY OF TEXAS SOUTHWESTERN (YAN LAB) - TEAM HAS BEEN GENERATING AND CHARACTERIZING SEVERAL MOUSE MODELS OF NGLY1 DEFICIENCY, INCLUDING MODELS IN WHICH THE GENE IS INACTIVATED IN CERTAIN TISSUES AND CONDITIONALLY AFTER BIRTH.THIS HAS ALLOWED THEM TO USE THESE MOUSE MODELS TO CHARACTERIZE NEUROLOGICAL PHENOTYPES, NEUROPATHOLOGICAL CHANGES, INFLAMMATION, SENSORY AND MOTOR DEFECTS, AND DISEASE ASSOCIATED BIOMARKERS. THESE ANIMAL MODELS PROVIDE INSIGHTS INTO THE SPECIFIC DEFECTS THAT RESULT FROM NGLY1 LOSS AND ESTABLISH A FOUNDATION TO TEST CANDIDATE THERAPIES INCLUDING GENE THERAPIES AND SMALL MOLECULE DRUGS. THE TEAM HAS ALSO FOUND THAT NGLY1 DEFICIENCY TRIGGERS A SPECIFIC INNATE IMMUNE PATHWAY MEDIATED BY THE CGAS/STING PATHWAY. THEY HAVE DEVELOPED TOOLS TO DETERMINE WHETHER ACTIVATED STING SIGNALLING CONTRIBUTES TO THE PATHOLOGY ASSOCIATED WITH NGLY1 DEFICIENCY AND WHETHER BLOCKING THIS PATHWAY PROVIDES THERAPEUTIC BENEFIT. 61762 57000 MASSACHUSETTS GENERAL HOSPITAL (RUVKUN LAB) - TEAM CONTINUED THEIR ANALYSIS OF THE GENETIC MODIFIERS OF PROTEASOME DYSFUNCTION THEY PREVIOUSLY IDENTIFIED IN PNG-1 / NGLY1 DEFICIENT C. ELEGANS. THIS LED TO THE IDENTIFICATION OF SEVERAL PATHWAYS WHOSE DISRUPTION APPEARS BENEFICIAL TO PROTEASOME DYSFUNCTION IN NGLY1 DEFICIENT ANIMALS, A FINDING THAT COULD TRANSLATE INTO A POTENTIAL THERAPY FOR THE DISEASE IN HUMANS. THEY ALSO DEMONSTRATED NGLY1 HAS A CRITICAL FUNCTION TO EDIT THE PROTEIN SEQUENCE OF ONE OF ITS KEY SUBSTRATES, THE TRANSCRIPTIONAL REGULATOR SKN-1A / NFE2L1, AND THEREBY REGULATE ITS FUNCTION TO MAINTAIN PROTEIN HOMEOSTASIS IN THE CELL. ADDITIONALLY, THE TEAM DEMONSTRATED THAT SKN-1A / NFE2L1 IS ACTIVATED BY MISFOLDED PROTEINS, A FINDING WITH POTENTIAL RELEVANCE TO NEURODEGENERATIVE DISEASES. COLLECTIVELY, THESE RESULTS LED TO TWO PUBLICATIONS IN INFLUENTIAL JOURNALS. 44497 39735 STANFORD UNIVERSITY (BERTOZZI LAB) - TEAM CONTINUED INVESTIGATIONS ON THE ROLE OF FERROPTOSIS AND OXIDATIVE STRESS IN THE PATHOLOGIES ASSOCIATED WITH NGLY1 DEFICIENCY. THESE FINDINGS TOUCH ON KNOWN PATHWAYS AND COULD THEREFORE INFORM POTENTIAL THERAPEUTIC STRATEGIES. ADDITIONALLY, THE TEAM HAS BEEN IDENTIFIYING AND STUDYING NGLY1 INTERACTORS AND POTENTIAL SUBSTRATES. THIS IS CRITICAL TO UNCOVERING PROTEINS INVOLVED IN NGLY1 FUNCTION AS WELL AS ADDITIONAL PROTEINS, LIKE NFE2L1, THAT NGLY1 MAY REGULATE THROUGH DEGLYCOSYLATION. SUCH PROTEINS COULD SERVE AS POTENTIAL TARGETS FOR A SMALL COMPOUND THERAPEUTIC AND COULD PROVIDE ESSENTIAL INSIGHT INTO NGLY1 BIOLOGY. 71818 67057 STANFORD UNIVERSITY NGLY1 NATURAL HISTORY STUDY (MAURA RUZHNIKOV) - TEAM INITIATED A NATURAL HISTORY STUDY TO DEFINE THE CLINICAL SPECTRUM OF THE DISEASE AND ITS PROGRESSION, AND DEFINE BIOMARKER ENDPOINTS FOR USE IN THERAPEUTIC TRIALS. THIS STUDY IS LED BY DR. MAURA RUZHNIKOV, MD, A CHILD NEUROLOGIST AND MEDICAL GENETICIST FOCUSING ON THE DIAGNOSIS AND MANAGEMENT OF RARE NEUROLOGIC DISORDERS, INCLUDING GENETIC EPILEPSY SYNDROMES, CHILDHOOD NEURODEGENERATIVE DISEASES AND UNDIAGNOSED SUSPECTED GENETIC CONDITIONS. TEAM HAS DEVELOPED AND RECEIVED APPROVAL FOR THE STUDY DESIGN, RECRUITED PARTICIPANTS, AND BEGUN GATHERING CLINICAL DATA IN THE FIRST PATIENT VISITS. ULTIMATELY, THE STUDY WILL ENROLL MORE THAN TWENTY-FIVE PATEINTS AND FOLLOW THEM LONGITUDINALLY OVER FIVE YEARS. 182142 182142 OTHER PROGRAM SERVICES ARE RELATED TO RESEARCH AND DEVELOPMENT TO UNDERSTAND AND CURE CARE DISEASES BEGINNING WITH NGLY1 DEFICIENCY. 360219 345934 737995 1 1 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 1 0 0 0 0 0 0 0 0 0 0 1 0 0 0 0 0 0 0 0 1 4 0 0 0 0 0 0 0 0 0 0 0 0 X 7 7 1 0 0 0 0 1 0 0 0 0 0 0 1 0 0 0 0 0 0 0 CA X X GRACE SCIENCE FOUNDATION 4152557666 1142 CRANE ST 4 MENLO PARK CA 94025 X MATTHEW WILSEY PRESIDENT 20.00 X X 0 0 0 EGON DURBAN CHIEF FINANCIAL OFFICER 1.00 X X 0 0 0 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 1522789 480354 1522789 20581 20581 22006 4074 17932 17932 17932 1561302 0 0 38513 653174 653174 56250 56250 13368 13368 4250 4250 68750 68750 7244 7244 1637 1637 26934 26934 1119 1119 955 955 SHIPPING FEES 2751 2751 BRANDING/LOGO/VIDEO/WEB 1424 1424 OTHER FEES (BANK, CREDI 1379 1379 FILING FEES 160 160 839395 737995 101400 0 2192300 2885541 4074 4074 2196374 2889615 2201 0 2201 X 0 0 0 0 2196374 2887414 2196374 2887414 2196374 2889615 721907 -30867 0 X 0 0 0 X 1560381 600124 1677107 1157365 1522789 6517766 1560381 600124 1677107 1157365 1522789 6517766 2463829 4053937 6517766 0.62200 X RESTRICTED RESTRICTED RESTRICTED RESTRICTED RESTRICTED RESTRICTED RESTRICTED RESTRICTED 0 0 0 0 0 0 0 0 0 CANADA NGYL1 WORK IN MOFFAT'S LABS AT UNIVERSITY OF TORONTO 56250 CHECK 0 0 0 0 0 0 PART III ACCOUNTING METHOD: 1 BIOIVT 123 FROST ST WESTBURY NY 11590 2083 COMPARING CEREBROSPINAL FLUID CONTROL SAMPLES STANFORD UNIVERSITY 3145 PORTER DRIVE PALO ALTO CA 94304 941156365 501(C)(3) 106792 NGLY1 WORK IN BERTOZZI/SNYDER/STEINMETZ LABS & NATURAL HISTORY PROTOCOL DEVELOPMENT MASSACHUSETTS GENERAL HOSPITAL 32 FRUIT ST BOSTON MA 02114 041564655 501(C)(3) 57000 NYGLY1 WORK IN RUKVUN LAB VIRGINIA COMMONWEALTH UNIVERSITY 1201 E MARSHALL ST 4-100 RICHMOND VA 23298 546001758 501(C)(3) 58000 NGLY1 WORK IN RADHAKRISHNAN LAB SALK INSTITUTE FOR BIOLOGICAL STUDIES 10010 N TORREY PINES ROAD LA JOLLA CA 92037 952160097 501(C)(3) 124216 NGLY1 WORK IN GAGE LAB THE JACKSON LABORATORY 600 MAIN STREET BAR HARBOR ME 04609 010211513 501(C)(3) 167533 NGLY1 WORK WITH CAT LUTZ UNIVERSITY OF MASSACHUSETTS MEDICAL SCHOOL 55 LAKE AVENUE NORTH WORCESTER MA 01655 043167352 501(C)(6) 30000 NGLY1 WORK IN GAO LAB UNIVERSITY OF TEXAS SOUTHWESTERN MEDICAL CENTER 5323 HARRY HINES BLVD DALLAS TX 75390 756002868 501(C)(6) 71740 NGLY1 WORK IN YAN LAB CHARLES RIVER LABORATORIES 251 BALLARDVALE ST WILMINGTON MA 01887 14560 NGLY1 RAT MODEL NEW YORK STEM CELL FOUNDATION 619 W 54TH STREET NEW YORK NY 10019 202905531 501(C)(3) 21250 NGLY1 STEM CELL LINES 8 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. X 3 480354 FAIR MARKET VALUE 0 0 0 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 8A THE GOVERNING BODY OF THE FOUNDATION DID NOT MEET DURING THE YEAR. 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 C, LINE 18 AVAILABLE UPON REQUEST. FORM 990, PART VI, SECTION C, LINE 19 AVAILABLE UPON REQUEST.