2021-11-15T16:38:51-06:00 2020-12-31 222027092 WITHUMSMITHBROWN PC 601 CALIFORNIA STREET SUITE 1800 SAN FRANCISCO CA 94108 990 2020-01-01 465727883 GRACE SCIENCE FOUNDATION GRAC 4152557666 1142 CRANE ST 4 MENLO PARK CA 94025 MATTHEW R WILSEY PRESIDENT 4152557666 2021-11-11 1 MATTHEW WILSEY 999009999 BRADFORD WEISERT P00044664 4154343744 2021-11-11 3 AAL1 IAL1 2020 2022-09-23 18:48:47Z MATTHEW R WILSEY 1142 CRANE ST 4 MENLO PARK CA 94025 1454180 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 1522789 1447474 0 0 38513 6706 0 0 1561302 1454180 709424 1373067 0 0 0 0 0 0 0 129971 79834 839395 1452901 721907 1279 2889615 2884506 2201 386 2887414 2884120 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 279832 279337 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. THESE MOUSE MODELS HAVE ALLOWED THEM 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 FOR TESTING CANDIDATE THERAPIES, INCLUDING GENE THERAPIES AND SMALL MOLECULE DRUGS. THE TEAM HAS ALSO FOUND THAT NGLY1 DEFICIENCY IN MICE TRIGGERS A SPECIFIC INNATE IMMUNE PATHWAY MEDIATED BY THE CGAS/STING PATHWAY. THEY HAVE BEEN USING GENETIC AND PHARMACOLOGICAL APPROACHES TO TEST WHETHER ACTIVATED STING SIGNALLING CONTRIBUTES TO THE PATHOLOGY ASSOCIATED WITH NGLY1 DEFICIENCY AND WHETHER BLOCKING THIS PATHWAY PROVIDES THERAPEUTIC BENEFIT. 375842 375347 STANFORD UNIVERSITY NGLY1 NATURAL HISTORY STUDY (MAURA RUZHNIKOV) - TEAM IS CONDUCTING AN NGLY1 NATURAL HISTORY STUDY TO DEFINE THE CLINICAL SPECTRUM OF THE DISEASE AND ITS PROGRESSION, AS WELL AS BIOMARKER ENDPOINTS FOR USE IN THERAPEUTIC TRIALS. THIS STUDY IS LED BY DR. MAURA RUZHNIKOV, MD, A CHILD NEUROLOGIST AND MEDICAL GENETICIST FOCUSED 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 GATHERED UP TO 2 YEARS OF CLINICAL DATA FROM SEVERAL PATIENTS. AS OF THE END OF 2020, THE STUDY HAS ENROLLED TWENTY-NINE INDIVIDUALS CONFIRMED TO HAVE NGLY1 DEFICIENCY AS EITHER ON-SITE OR REMOTE PARTICIPANTS. ULTIMATELY THE STUDY WILL FOLLOW THE PARTICIPANTS LONGITUDINALLY OVER FIVE YEARS. 516818 516323 CHARLES RIVER LABORATORIES - TEAM HAS IMPORTED AND ESTABLISHED A COLONY OF NGLY1 KNOCKOUT RATS FOR USE IN TESTING THERAPIES TO TREAT NGLY1 DEFICIENCY. A BREEDING COLONY HAS BEEN ESTABLISHED, MUTANT PHENOTYPES HAVE BEEN CHARACTERIZED, AND WORK IS UNDERWAY TO USE THESE MODELS TO TEST ADENO-ASSOCIATED VIRUS (AAV)-MEDIATED GENE DELIVERY FOR RESCUE OF DISEASE-ASSOCIATED PHENOTYPES. THIS WORK WILL SERVE AS THE FOUNDATION FOR AN INVESTIGATIONAL NEW DRUG (IND) APPLICAITON TO THE FDA FOR AN NGLY1 DEFICIENCY GENE THERAPY CLINICAL STUDY; THE FINDINGS COULD ALSO SUPPORT TRIALS FOR OTHER THERAPEUTIC AVENUES FOR TREATING NGLY1 DEFICIDENCY 83305 82811 SALK INSTITUTE FOR BIOLOICAL SCIENCES (GAGE LAB) - TEAM HAS CONTINUED THEIR DEVELOPMENT AND STUDY OF 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 FOR REVERSING THESE 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 HUMAN NGLY1 GENE INTO THE CELLS, AND HAS BEEN CHARACTERIZING THE COMPOSITION OF THE AGGREGATES FOUND IN NGLY1 DEFICIENT 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. 79745 79250 MASSACHUSETTS GENERAL HOSPITAL (RUVKUN LAB) - TEAM CONTINUED THEIR ANALYSIS OF THE GENETIC MODIFIERS OF PROTEASOME DYSFUNCTION THAT 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 GIVEN ITS ABILITY TO EDIT THE PROTEIN SEQUENCE OF ONE OF ITS KEY SUBSTRATES, THE TRANSCRIPTIONAL REGULATOR SKN-1A / NFE2L1, AND THEREBY REGULATE NFE2L1'S FUNCTION IN MAINTAINING 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 THREE PUBLICATIONS IN INFLUENTIAL JOURNALS. 21745 21250 THE NEW YORK STEM CELL FOUNDATION - TEAM IS DEVELOPING ADDITIONAL PATIENT-DERIVED INDUCED PLURIPOTENT STELL CELL (IPSC) LINES TO COMPLEMENT AND EXPAND HUMAN CELL MODELS OF NGLY1 DEFICIENCY. CRISPR-MEDIATED GENOME EDITING WILL BE USED TO PRODUCE ISOGENIC LINES FOR USE AS A CONTROL TO DETEMINE DISEASE-SPECIFIC CHARACTERISTICS. THESE LINES WILL SERVE AS ADDITIONAL MODELS FOR TESTING POTENTIAL THERAPIES TO REVERSE PATHOLOGIES ASSOCIATED WITH NGLY1 DEFICIENCY. 18750 18750 OTHER PROGRAM SERVICES ARE RELATED TO RESEARCH AND DEVELOMENT TO UNDERSTAND AND CURE DISEASES BEGINNING WITH NGLY1 DEFICIENCY 203545 202061 1376037 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 0 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 1447474 1447474 6706 6706 1454180 0 0 6706 1354317 1354317 18750 18750 5100 5100 53551 53551 11225 11225 2823 2823 147 147 955 955 OTHER FEES (BANK, CREDI 4502 4502 SHIPPING FEES 1387 1387 BRANDING/LOGO/VIDEO/WEB 113 113 FILING FEES 31 31 1452901 1376037 76864 0 2885541 2884506 4074 2889615 2884506 2201 386 2201 386 X 0 0 0 0 2887414 2884120 2887414 2884120 2889615 2884506 X 1279 -4573 X 0 0 0 X 600124 1677107 1157365 1522789 1447474 6404859 600124 1677107 1157365 1522789 1447474 6404859 2200958 4203901 6404859 0.65640 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 18750 CHECK 0 0 0 0 0 0 PART III ACCOUNTING METHOD: 1 STANFORD UNIVERSITY 3145 PORTER DRIVE PALO ALTO CA 94304 941156365 501(C)(3) 375347 NGLY1 WORK IN BERTOZZI/SNYDER/STEINMETZ LABS & NATURAL HISTORY PROTOCOL DEVELOPMENT MASSACHUSETTS GENERAL HOSPITAL 32 FRUIT ST BOSTON MA 02114 041564655 501(C)(3) 79250 NYGLY1 WORK IN RUKVUN LAB SALK INSTITUTE FOR BIOLOGICAL STUDIES 10010 N TORREY PINES ROAD LA JOLLA CA 92037 952160097 501(C)(3) 82810 NGLY1 WORK IN GAGE LAB UNIVERSITY OF TEXAS SOUTHWESTERN MEDICAL CENTER 5323 HARRY HINES BLVD DALLAS TX 75390 756002868 501(C)(6) 279337 NGLY1 WORK IN YAN LAB CHARLES RIVER LABORATORIES 251 BALLARDVALE ST WILMINGTON MA 01887 516323 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 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 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. FORM 990, PART XI, LINE 9: PRIOR PERIOD ADJUSTMENT -4,573.