GRACE SCIENCE FOUNDATION: Form 990, tax year 2024

View the entire original 990 at ProPublica ↗

Organization
GRACE SCIENCE FOUNDATION (EIN 46-5727883)
Tax period
2024-01-01 to 2024-12-31
Filed
2025-10-16
IRS object ID
202512899349300541
Schema version
2024v5.2
Source
Download XML · Original 990 at ProPublica

Every field in the e-filed return. Faint text is the IRS element name, for citing.

Return header ReturnHeader

FieldValueIRS element (for citing)
Return2025-10-16T08:48:57-04:00ReturnTs
Tax Period End2024-12-31TaxPeriodEndDt

Preparer Firm PreparerFirmGrp

Preparer Firm Name PreparerFirmName

FieldValueIRS element (for citing)
Business Name Line1WITHUMSMITHBROWNPCBusinessNameLine1Txt

Preparer US Address PreparerUSAddress

FieldValueIRS element (for citing)
Address Line16101 BOLLINGER CANYON RD STE 400AddressLine1Txt
CitySAN RAMONCityNm
State AbbreviationCAStateAbbreviationCd
ZIP94583ZIPCd
FieldValueIRS element (for citing)
Return Type990ReturnTypeCd
Tax Period Begin2024-01-01TaxPeriodBeginDt

Filer Filer

FieldValueIRS element (for citing)
EIN465727883EIN

Business Name BusinessName

FieldValueIRS element (for citing)
Business Name Line1GRACE SCIENCE FOUNDATIONBusinessNameLine1Txt
FieldValueIRS element (for citing)
In Care Of% GRACE SCIENCE FOUNDATIONInCareOfNm
Business Name ControlGRACBusinessNameControlTxt
Phone6507464591PhoneNum

US Address USAddress

FieldValueIRS element (for citing)
Address Line1873 SANTA CRUZ AVENUE SUITE 200AddressLine1Txt
CityMENLO PARKCityNm
State AbbreviationCAStateAbbreviationCd
ZIP94025ZIPCd

Business Officer BusinessOfficerGrp

FieldValueIRS element (for citing)
PersonMATTHEW R WILSEYPersonNm
Person TitlePRESIDENTPersonTitleTxt
Phone6507464591PhoneNum
Signature2025-11-15SignatureDt
Discuss With Paid PreparerYes (true)DiscussWithPaidPreparerInd
FieldValueIRS element (for citing)
IRS Responsible Prty Info CurrYes (true)IRSResponsiblePrtyInfoCurrInd

Preparer Person PreparerPersonGrp

FieldValueIRS element (for citing)
Preparer PersonRICHARD RUVELSONPreparerPersonNm
PTINP00234075PTIN
Phone9252779100PhoneNum
Preparation2025-11-15PreparationDt
FieldValueIRS element (for citing)
Tax2024TaxYr
Build TS2025-03-06 01:10:19ZBuildTS

Form 990 IRS990

FieldValueIRS element (for citing)
Principal OfficerMATTHEW R WILSEYPrincipalOfficerNm

US Address USAddress

FieldValueIRS element (for citing)
Address Line1873 SANTA CRUZ AVENUE SUITE 2AddressLine1Txt
CityMENLO PARKCityNm
State AbbreviationCAStateAbbreviationCd
ZIP94025ZIPCd
FieldValueIRS element (for citing)
Gross Receipts$943,011GrossReceiptsAmt
Group Return For AffiliatesfalseGroupReturnForAffiliatesInd
Organization501c3Yes (X)Organization501c3Ind
Website Addresswww.gracescience.orgWebsiteAddressTxt
Type Of Organization CorpYes (X)TypeOfOrganizationCorpInd
Formation2014FormationYr
Legal Domicile StateCALegalDomicileStateCd
Activity Or MissionSEE PART III LINE 1ActivityOrMissionDesc
Voting Members Governing Body7VotingMembersGoverningBodyCnt
Voting Members Independent7VotingMembersIndependentCnt
Total Employee0TotalEmployeeCnt
Total Volunteers0TotalVolunteersCnt
Total Gross UBI$0TotalGrossUBIAmt
Net Unrelated Bus Txbl Incm$0NetUnrelatedBusTxblIncmAmt
PY Contributions Grants$783,225PYContributionsGrantsAmt
CY Contributions Grants$869,699CYContributionsGrantsAmt
PY Program Service Revenue$0PYProgramServiceRevenueAmt
CY Program Service Revenue$0CYProgramServiceRevenueAmt
PY Investment Income$43,401PYInvestmentIncomeAmt
CY Investment Income$73,312CYInvestmentIncomeAmt
PY Other Revenue$0PYOtherRevenueAmt
CY Other Revenue$0CYOtherRevenueAmt
PY Total Revenue$826,626PYTotalRevenueAmt
CY Total Revenue$943,011CYTotalRevenueAmt
PY Grants And Similar Paid$1,133,449PYGrantsAndSimilarPaidAmt
CY Grants And Similar Paid$1,342,330CYGrantsAndSimilarPaidAmt
PY Benefits Paid To Members$0PYBenefitsPaidToMembersAmt
CY Benefits Paid To Members$0CYBenefitsPaidToMembersAmt
PY Salaries Comp Emp Bnft Paid$0PYSalariesCompEmpBnftPaidAmt
CY Salaries Comp Emp Bnft Paid$0CYSalariesCompEmpBnftPaidAmt
PY Total Prof Fndrsng Expns$0PYTotalProfFndrsngExpnsAmt
CY Total Prof Fndrsng Expns$0CYTotalProfFndrsngExpnsAmt
CY Total Fundraising Expense$0CYTotalFundraisingExpenseAmt
PY Other Expenses$155,725PYOtherExpensesAmt
CY Other Expenses$177,056CYOtherExpensesAmt
PY Total Expenses$1,289,174PYTotalExpensesAmt
CY Total Expenses$1,519,386CYTotalExpensesAmt
PY Revenues Less Expenses-$462,548PYRevenuesLessExpensesAmt
CY Revenues Less Expenses-$576,375CYRevenuesLessExpensesAmt
Total Assets BOY$2,790,274TotalAssetsBOYAmt
Total Assets EOY$2,224,370TotalAssetsEOYAmt
Total Liabilities BOY$42,997TotalLiabilitiesBOYAmt
Total Liabilities EOY$1,004TotalLiabilitiesEOYAmt
Net Assets Or Fund Balances BOY$2,747,277NetAssetsOrFundBalancesBOYAmt
Net Assets Or Fund Balances EOY$2,223,366NetAssetsOrFundBalancesEOYAmt
Info In Schedule O Part IIIYes (X)InfoInScheduleOPartIIIInd
MissionTHE 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 RESESARCH FACILITIES WHICH ARE CONDUCTING MEDICAL RESEARCH IN VARIOUS FIELDS RELATING TO THE SEARCH FOR UNDERSTNADING AND TREATMENT OF THIS CONDITION, FOSTERING COLLABORATION BETWEEN ACADEMIA, RESEARCH INSTITUTIONS, HOSPITALS, PRIVATE COMPANIES, AND GOVERNMENTAL AGENCIES.MissionDesc
Significant New Program SrvcfalseSignificantNewProgramSrvcInd
Significant ChangefalseSignificantChangeInd
Expense$452,697ExpenseAmt
Grant$452,697GrantAmt
DescBaylor College of Medicine NGLY1 Natural History Study (Bernhard Suter) Team is conducting 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. Bernhard Suter, MD, a child neurologist and medical geneticist. The study is collecting longitudinal measures of movement concurrently with clinical and biomarker measures to define aspects of gait and movement that are most affected in the disorder, characterize progression of the disease, select clinical features for performing longitudinal assessments, and inform clinical endpoint development for therapeutic trials.Desc

Prog Srvc Accom Acty2 ProgSrvcAccomActy2Grp

FieldValueIRS element (for citing)
Expense$352,945ExpenseAmt
Grant$352,945GrantAmt
DescPsychogenics Inc. Team has imported and is analyzing mouse and rat models of NGLY1 Deficiency for testing of candidate therapies. Team has conducted several studies using an NGLY1 knockout rat model to test phenotypic rescue using candidate small molecule therapies (both novel and approved drugs).Desc

Prog Srvc Accom Acty3 ProgSrvcAccomActy3Grp

FieldValueIRS element (for citing)
Expense$203,574ExpenseAmt
Grant$203,574GrantAmt
DescTHE UNIVERSITY OF TEXAS SOUTHWESTERN MEDICAL CENTER (YAN LAB) Team continues work 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 including neuronal loss, neuropathological changes, inflammation, sensory and motor defects, and disease associated biomarkers, all of which are also observed in NGLY1 Deficiency patients These animal models provide 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. Using genetic and pharmacological approaches, the team has uncovered three different targets that can be modulated to reduce disease associated phenotypes and improve survival.Desc

Prog Srvc Accom Acty Other ProgSrvcAccomActyOtherGrp (14 entries)

ExpenseExpenseAmtGrantGrantAmtDescDesc
$80,637$80,637The University of North Carolina (Hantman Lab)
$68,025$68,025Charles River Laboratories
$49,108$49,108Boston Children's Hospital (Yu Lab)
$27,695$27,695Hilltop Lab Animals Inc.
$24,241$24,241Albert Einstein College of Medicine (Kaushik Lab)
$23,869$23,869Purdue University (Rochet Lab)
$20,356$20,356Pharmaron, Inc
$19,321$19,321Universitry of Utah (Chow Lab)
$10,115$10,115Standford University
$9,747$9,747University of Houston
$19,000$0Bioagilytix Labs
$13,904$0Transnetyx
$10,481$0Wuxi Apptec
$5,369$0MedChem Express LLC
FieldValueIRS element (for citing)
Total Other Prog Srvc Expense$381,868TotalOtherProgSrvcExpenseAmt
Total Other Prog Srvc Grant$333,114TotalOtherProgSrvcGrantAmt
Total Program Service Expenses$1,391,084TotalProgramServiceExpensesAmt
Described In Section501c3Yes (true)DescribedInSection501c3Ind
Schedule B RequiredYes (true)ScheduleBRequiredInd
Political Campaign ActyfalsePoliticalCampaignActyInd
Lobbying ActivitiesfalseLobbyingActivitiesInd
Subject To Proxy TaxfalseSubjectToProxyTaxInd
Donor Advised FundfalseDonorAdvisedFundInd
Conservation EasementsfalseConservationEasementsInd
Collections Of ArtfalseCollectionsOfArtInd
Credit CounselingfalseCreditCounselingInd
Donor Rstr Or Quasi EndowmentsfalseDonorRstrOrQuasiEndowmentsInd
Report Land Building EquipmentfalseReportLandBuildingEquipmentInd
Report Investments Other SecfalseReportInvestmentsOtherSecInd
Report Program Related InvstfalseReportProgramRelatedInvstInd
Report Other AssetsfalseReportOtherAssetsInd
Report Other LiabilitiesfalseReportOtherLiabilitiesInd
Include FIN48 FootnotefalseIncludeFIN48FootnoteInd
Independent Audit Fincl StmtfalseIndependentAuditFinclStmtInd
Consolidated Audit Fincl StmtfalseConsolidatedAuditFinclStmtInd
School OperatingfalseSchoolOperatingInd
Foreign OfficefalseForeignOfficeInd
Foreign ActivitiesYes (true)ForeignActivitiesInd
More Than5000 K To OrgYes (true)MoreThan5000KToOrgInd
More Than5000 K To IndividualsfalseMoreThan5000KToIndividualsInd
Professional FundraisingfalseProfessionalFundraisingInd
Fundraising ActivitiesfalseFundraisingActivitiesInd
Gaming ActivitiesfalseGamingActivitiesInd
Operate HospitalfalseOperateHospitalInd
Grants To OrganizationsYes (true)GrantsToOrganizationsInd
Grants To IndividualsfalseGrantsToIndividualsInd
Schedule J RequiredfalseScheduleJRequiredInd
Tax Exempt BondsfalseTaxExemptBondsInd
Engaged In Excess Benefit TransfalseEngagedInExcessBenefitTransInd
PY Excess Benefit TransfalsePYExcessBenefitTransInd
Loan OutstandingfalseLoanOutstandingInd
Grant To Related PersonfalseGrantToRelatedPersonInd
Business Rln With Org MemfalseBusinessRlnWithOrgMemInd
Business Rln With Fam MemfalseBusinessRlnWithFamMemInd
Business Rln With35 Ctrl EntfalseBusinessRlnWith35CtrlEntInd
Deductible Non Cash ContrifalseDeductibleNonCashContriInd
Deductible Art ContributionfalseDeductibleArtContributionInd
Terminate OperationsfalseTerminateOperationsInd
Partial LiquidationfalsePartialLiquidationInd
Disregarded EntityfalseDisregardedEntityInd
Related EntityfalseRelatedEntityInd
Related Organization Ctrl EntfalseRelatedOrganizationCtrlEntInd
Activities Conducted PrtshpfalseActivitiesConductedPrtshpInd
Schedule O RequiredYes (true)ScheduleORequiredInd
IRP Document3IRPDocumentCnt
IRP Document W2 G0IRPDocumentW2GCnt
Backup Wthld ComplianceYes (true)BackupWthldComplianceInd
Employee0EmployeeCnt
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Foreign Financial AccountfalseForeignFinancialAccountInd
Prohibited Tax Shelter TransfalseProhibitedTaxShelterTransInd
Taxable Party NotificationfalseTaxablePartyNotificationInd
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Quid Pro Quo ContributionsfalseQuidProQuoContributionsInd
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Pay Premiums Prsnl Bnft CntrctfalsePayPremiumsPrsnlBnftCntrctInd
Form8899 FiledindfalseForm8899Filedind
Form1098 C FiledfalseForm1098CFiledInd
Indoor Tanning ServicesfalseIndoorTanningServicesInd
Subj To Tax Rmnrtn Ex Prcht PymtfalseSubjToTaxRmnrtnExPrchtPymtInd
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Info In Schedule O Part VIYes (X)InfoInScheduleOPartVIInd
Governing Body Voting Members7GoverningBodyVotingMembersCnt
Independent Voting Member7IndependentVotingMemberCnt
Family Or Business RlnYes (true)FamilyOrBusinessRlnInd
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Change To Org DocumentsfalseChangeToOrgDocumentsInd
Material Diversion Or MisusefalseMaterialDiversionOrMisuseInd
Members Or StockholdersfalseMembersOrStockholdersInd
Election Of Board MembersYes (true)ElectionOfBoardMembersInd
Decisions Subject To ApprovafalseDecisionsSubjectToApprovaInd
Minutes Of Governing BodyfalseMinutesOfGoverningBodyInd
Officer Mailing AddressfalseOfficerMailingAddressInd
Local ChaptersfalseLocalChaptersInd
Form990 Provided To Gvrn BodyfalseForm990ProvidedToGvrnBodyInd
Conflict Of Interest PolicyYes (true)ConflictOfInterestPolicyInd
Annual Disclosure Covered PrsnfalseAnnualDisclosureCoveredPrsnInd
Regular Monitoring EnfrcfalseRegularMonitoringEnfrcInd
Whistleblower PolicyfalseWhistleblowerPolicyInd
Document Retention PolicyfalseDocumentRetentionPolicyInd
Compensation Process CEOfalseCompensationProcessCEOInd
Compensation Process OtherfalseCompensationProcessOtherInd
Investment In Joint VenturefalseInvestmentInJointVentureInd
States Where Copy Of Return Is FldCAStatesWhereCopyOfReturnIsFldCd
Other WebsiteYes (X)OtherWebsiteInd
Upon RequestYes (X)UponRequestInd

Books In Care Of Detail BooksInCareOfDetail

FieldValueIRS element (for citing)
PersonGRACE SCIENCE FOUNDATIONPersonNm
Phone6507464591PhoneNum

US Address USAddress

FieldValueIRS element (for citing)
Address Line1873 SANTA CRUZ AVENUE SUITE 200AddressLine1Txt
CityMENLO PARKCityNm
State AbbreviationCAStateAbbreviationCd
ZIP94025ZIPCd
FieldValueIRS element (for citing)
No Listed Persons CompensatedYes (X)NoListedPersonsCompensatedInd

Form990 Part VII Section A Form990PartVIISectionAGrp (7 entries)

TitleTitleTxtAverage Hours Per WeekAverageHoursPerWeekRtAverage Hours Per Week Rltd OrgAverageHoursPerWeekRltdOrgRtIndividual Trustee Or DirectorIndividualTrusteeOrDirectorIndOfficerOfficerIndReportable Comp From OrgReportableCompFromOrgAmtReportable Comp From Rltd OrgReportableCompFromRltdOrgAmtOther CompensationOtherCompensationAmt
Matthew Wilsey
President0.00.0Yes (X)Yes (X)$0$0$0
Egon Durban
Chief Financial Officer0.00.0Yes (X)Yes (X)$0$0$0
KEN DRAZAN
Secretary0.00.0Yes (X)Yes (X)$0$0$0
Pete Briger
Director0.00.0Yes (X)$0$0$0
Chelsea Clinton
Director0.00.0Yes (X)$0$0$0
J Taylor Crandall
Director0.00.0Yes (X)$0$0$0
Elle Stephens
Director0.00.0Yes (X)$0$0$0
FieldValueIRS element (for citing)
Total Reportable Comp From Org$0TotalReportableCompFromOrgAmt
Tot Reportable Comp Rltd Org$0TotReportableCompRltdOrgAmt
Total Other Compensation$0TotalOtherCompensationAmt
Indiv Rcvd Greater Than100 K0IndivRcvdGreaterThan100KCnt
Former Ofcr Employees ListedfalseFormerOfcrEmployeesListedInd
Total Comp Greater Than150 KfalseTotalCompGreaterThan150KInd
Compensation From Other SrcsfalseCompensationFromOtherSrcsInd
Cntrct Rcvd Greater Than100 K0CntrctRcvdGreaterThan100KCnt
All Other Contributions$869,699AllOtherContributionsAmt
Total Contributions$869,699TotalContributionsAmt
Total Program Service Revenue$0TotalProgramServiceRevenueAmt

Investment Income InvestmentIncomeGrp

FieldValueIRS element (for citing)
Total Revenue Column$73,312TotalRevenueColumnAmt
Related Or Exempt Func Income$0RelatedOrExemptFuncIncomeAmt
Unrelated Business Revenue$0UnrelatedBusinessRevenueAmt
Exclusion$73,312ExclusionAmt

Incm From Invest Bond Proceeds IncmFromInvestBondProceedsGrp

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Total Revenue Column$0TotalRevenueColumnAmt

Royalties Revenue RoyaltiesRevenueGrp

FieldValueIRS element (for citing)
Total Revenue Column$0TotalRevenueColumnAmt

Rental Income Or Loss RentalIncomeOrLossGrp

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Real$0RealAmt
Personal$0PersonalAmt

Net Rental Income Or Loss NetRentalIncomeOrLossGrp

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Total Revenue Column$0TotalRevenueColumnAmt

Net Gain Or Loss Investments NetGainOrLossInvestmentsGrp

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Total Revenue Column$0TotalRevenueColumnAmt
FieldValueIRS element (for citing)
Fundraising Gross Income$0FundraisingGrossIncomeAmt
Fundraising Direct Expenses$0FundraisingDirectExpensesAmt

Net Incm From Fundraising Evt NetIncmFromFundraisingEvtGrp

FieldValueIRS element (for citing)
Total Revenue Column$0TotalRevenueColumnAmt
FieldValueIRS element (for citing)
Gaming Gross Income$0GamingGrossIncomeAmt
Gaming Direct Expenses$0GamingDirectExpensesAmt

Net Income From Gaming NetIncomeFromGamingGrp

FieldValueIRS element (for citing)
Total Revenue Column$0TotalRevenueColumnAmt
FieldValueIRS element (for citing)
Gross Sales Of Inventory$0GrossSalesOfInventoryAmt
Cost Of Goods Sold$0CostOfGoodsSoldAmt

Net Income Or Loss NetIncomeOrLossGrp

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Total Revenue Column$0TotalRevenueColumnAmt
FieldValueIRS element (for citing)
Other Revenue Total$0OtherRevenueTotalAmt

Total Revenue TotalRevenueGrp

FieldValueIRS element (for citing)
Total Revenue Column$943,011TotalRevenueColumnAmt
Related Or Exempt Func Income$0RelatedOrExemptFuncIncomeAmt
Unrelated Business Revenue$0UnrelatedBusinessRevenueAmt
Exclusion$73,312ExclusionAmt

Grants To Domestic Orgs GrantsToDomesticOrgsGrp

FieldValueIRS element (for citing)
Total$1,342,330TotalAmt
Program Services$1,342,330ProgramServicesAmt

Grants To Domestic Individuals GrantsToDomesticIndividualsGrp

FieldValueIRS element (for citing)
Total$0TotalAmt

Foreign Grants ForeignGrantsGrp

FieldValueIRS element (for citing)
Total$0TotalAmt

Benefits To Members BenefitsToMembersGrp

FieldValueIRS element (for citing)
Total$0TotalAmt

Comp Current Ofcr Directors CompCurrentOfcrDirectorsGrp

FieldValueIRS element (for citing)
Total$0TotalAmt

Comp Disqual Persons CompDisqualPersonsGrp

FieldValueIRS element (for citing)
Total$0TotalAmt

Other Salaries And Wages OtherSalariesAndWagesGrp

FieldValueIRS element (for citing)
Total$0TotalAmt

Pension Plan Contributions PensionPlanContributionsGrp

FieldValueIRS element (for citing)
Total$0TotalAmt

Other Employee Benefits OtherEmployeeBenefitsGrp

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Total$0TotalAmt

Payroll Taxes PayrollTaxesGrp

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Total$0TotalAmt

Fees For Services Management FeesForServicesManagementGrp

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Total$0TotalAmt

Fees For Services Legal FeesForServicesLegalGrp

FieldValueIRS element (for citing)
Total$779TotalAmt
Management And General$779ManagementAndGeneralAmt
Fundraising$0FundraisingAmt

Fees For Services Accounting FeesForServicesAccountingGrp

FieldValueIRS element (for citing)
Total$17,420TotalAmt
Management And General$17,420ManagementAndGeneralAmt

Fees For Services Lobbying FeesForServicesLobbyingGrp

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Total$0TotalAmt

Fees For Services Prof Fundraising FeesForServicesProfFundraising

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Total$0TotalAmt

Fees For Srvc Invst Mgmnt Fees FeesForSrvcInvstMgmntFeesGrp

FieldValueIRS element (for citing)
Total$0TotalAmt

Fees For Services Other FeesForServicesOtherGrp

FieldValueIRS element (for citing)
Total$99,200TotalAmt
Management And General$99,200ManagementAndGeneralAmt

Advertising AdvertisingGrp

FieldValueIRS element (for citing)
Total$5,000TotalAmt
Management And General$5,000ManagementAndGeneralAmt

Office Expenses OfficeExpensesGrp

FieldValueIRS element (for citing)
Total$2,717TotalAmt
Management And General$2,717ManagementAndGeneralAmt

Information Technology InformationTechnologyGrp

FieldValueIRS element (for citing)
Total$645TotalAmt
Management And General$645ManagementAndGeneralAmt

Royalties RoyaltiesGrp

FieldValueIRS element (for citing)
Total$0TotalAmt

Occupancy OccupancyGrp

FieldValueIRS element (for citing)
Total$0TotalAmt

Travel TravelGrp

FieldValueIRS element (for citing)
Total$559TotalAmt
Management And General$559ManagementAndGeneralAmt

Pymt Travel Entrtnmnt Pub Ofcl PymtTravelEntrtnmntPubOfclGrp

FieldValueIRS element (for citing)
Total$0TotalAmt

Conferences Meetings ConferencesMeetingsGrp

FieldValueIRS element (for citing)
Total$0TotalAmt

Interest InterestGrp

FieldValueIRS element (for citing)
Total$0TotalAmt

Payments To Affiliates PaymentsToAffiliatesGrp

FieldValueIRS element (for citing)
Total$0TotalAmt

Depreciation Depletion DepreciationDepletionGrp

FieldValueIRS element (for citing)
Total$0TotalAmt

Insurance InsuranceGrp

FieldValueIRS element (for citing)
Total$0TotalAmt

Other Expenses OtherExpensesGrp (3 entries)

DescDescTotalTotalAmtManagement And GeneralManagementAndGeneralAmtProgram ServicesProgramServicesAmt
SHIPPING FEES$1,782$1,782
TAX EXPENSE$200$200
PROGRAM EXPENSES$48,754$48,754

Total Functional Expenses TotalFunctionalExpensesGrp

FieldValueIRS element (for citing)
Total$1,519,386TotalAmt
Program Services$1,391,084ProgramServicesAmt
Management And General$128,302ManagementAndGeneralAmt
Fundraising$0FundraisingAmt

Cash Non Interest Bearing CashNonInterestBearingGrp

FieldValueIRS element (for citing)
BOY$352,830BOYAmt
EOY$142,773EOYAmt

Savings And Temp Cash Invst SavingsAndTempCashInvstGrp

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BOY$2,437,444BOYAmt
EOY$2,081,597EOYAmt

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BOY$0BOYAmt
EOY$0EOYAmt

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BOY$0BOYAmt
EOY$0EOYAmt

Receivables From Officers Etc ReceivablesFromOfficersEtcGrp

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BOY$0BOYAmt
EOY$0EOYAmt

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BOY$0BOYAmt
EOY$0EOYAmt

Oth Notes Loans Receivable Net OthNotesLoansReceivableNetGrp

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EOY$0EOYAmt

Inventories For Sale Or Use InventoriesForSaleOrUseGrp

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BOY$0BOYAmt
EOY$0EOYAmt

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BOY$0BOYAmt
EOY$0EOYAmt

Land Bldg Equip Basis Net LandBldgEquipBasisNetGrp

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BOY$0BOYAmt
EOY$0EOYAmt

Investments Pub Traded Sec InvestmentsPubTradedSecGrp

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BOY$0BOYAmt
EOY$0EOYAmt

Investments Other Securities InvestmentsOtherSecuritiesGrp

FieldValueIRS element (for citing)
BOY$0BOYAmt
EOY$0EOYAmt

Investments Program Related InvestmentsProgramRelatedGrp

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BOY$0BOYAmt
EOY$0EOYAmt

Intangible Assets IntangibleAssetsGrp

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BOY$0BOYAmt
EOY$0EOYAmt

Other Assets Total OtherAssetsTotalGrp

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BOY$0BOYAmt
EOY$0EOYAmt

Total Assets TotalAssetsGrp

FieldValueIRS element (for citing)
BOY$2,790,274BOYAmt
EOY$2,224,370EOYAmt

Accounts Payable Accr Expnss AccountsPayableAccrExpnssGrp

FieldValueIRS element (for citing)
BOY$42,997BOYAmt
EOY$1,004EOYAmt

Grants Payable GrantsPayableGrp

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BOY$0BOYAmt
EOY$0EOYAmt

Deferred Revenue DeferredRevenueGrp

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BOY$0BOYAmt
EOY$0EOYAmt

Tax Exempt Bond Liabilities TaxExemptBondLiabilitiesGrp

FieldValueIRS element (for citing)
BOY$0BOYAmt
EOY$0EOYAmt

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BOY$0BOYAmt
EOY$0EOYAmt

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BOY$0BOYAmt
EOY$0EOYAmt

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BOY$0BOYAmt
EOY$0EOYAmt

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BOY$0BOYAmt
EOY$0EOYAmt

Other Liabilities OtherLiabilitiesGrp

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BOY$0BOYAmt
EOY$0EOYAmt

Total Liabilities TotalLiabilitiesGrp

FieldValueIRS element (for citing)
BOY$42,997BOYAmt
EOY$1,004EOYAmt
FieldValueIRS element (for citing)
Org Does Not Follow FASB117Yes (X)OrgDoesNotFollowFASB117Ind

Cap Stk Tr Prin Current Funds CapStkTrPrinCurrentFundsGrp

FieldValueIRS element (for citing)
BOY$0BOYAmt
EOY$0EOYAmt

Pd In Cap Srpls Land Bldg Eqp Fund PdInCapSrplsLandBldgEqpFundGrp

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BOY$0BOYAmt
EOY$0EOYAmt

Rtn Earn Endowment Incm Oth Fnds RtnEarnEndowmentIncmOthFndsGrp

FieldValueIRS element (for citing)
BOY$0BOYAmt
EOY$0EOYAmt

Total Net Assets Fund Balance TotalNetAssetsFundBalanceGrp

FieldValueIRS element (for citing)
BOY$2,747,277BOYAmt
EOY$2,223,366EOYAmt

Tot Liab Net Assets Fund Balance TotLiabNetAssetsFundBalanceGrp

FieldValueIRS element (for citing)
BOY$2,790,274BOYAmt
EOY$2,224,370EOYAmt
FieldValueIRS element (for citing)
Reconcilation Revenue Expnss-$576,375ReconcilationRevenueExpnssAmt
Net Unrlzd Gains Losses Invst$52,464NetUnrlzdGainsLossesInvstAmt
Method Of Accounting CashYes (X)MethodOfAccountingCashInd
Accountant Compile Or ReviewfalseAccountantCompileOrReviewInd
FS AuditedfalseFSAuditedInd
Federal Grant Audit RequiredfalseFederalGrantAuditRequiredInd

Schedule A IRS990ScheduleA

FieldValueIRS element (for citing)
Public Organization170Yes (X)PublicOrganization170Ind

Gifts Grants Contri Rcvd170 GiftsGrantsContriRcvd170Grp

FieldValueIRS element (for citing)
Current Tax Year Minus4 Years$1,447,474CurrentTaxYearMinus4YearsAmt
Current Tax Year Minus3 Years$1,325,647CurrentTaxYearMinus3YearsAmt
Current Tax Year Minus2 Years$1,206,450CurrentTaxYearMinus2YearsAmt
Current Tax Year Minus1 Year$783,225CurrentTaxYearMinus1YearAmt
Current Tax Year$869,699CurrentTaxYearAmt
Total$5,632,495TotalAmt

Tax Rev Levied Orgnztnl Bnft170 TaxRevLeviedOrgnztnlBnft170Grp

FieldValueIRS element (for citing)
Total$0TotalAmt

Govt Furn Srvc Fclts Vl170 GovtFurnSrvcFcltsVl170Grp

FieldValueIRS element (for citing)
Total$0TotalAmt

Total Calendar Year170 TotalCalendarYear170Grp

FieldValueIRS element (for citing)
Current Tax Year Minus4 Years$1,447,474CurrentTaxYearMinus4YearsAmt
Current Tax Year Minus3 Years$1,325,647CurrentTaxYearMinus3YearsAmt
Current Tax Year Minus2 Years$1,206,450CurrentTaxYearMinus2YearsAmt
Current Tax Year Minus1 Year$783,225CurrentTaxYearMinus1YearAmt
Current Tax Year$869,699CurrentTaxYearAmt
Total$5,632,495TotalAmt
FieldValueIRS element (for citing)
Substantial Contributors Tot$2,543,020SubstantialContributorsTotAmt
Public Support Total170$3,089,475PublicSupportTotal170Amt

Gross Investment Income170 GrossInvestmentIncome170Grp

FieldValueIRS element (for citing)
Current Tax Year Minus4 Years$6,706CurrentTaxYearMinus4YearsAmt
Current Tax Year Minus3 Years$1,466CurrentTaxYearMinus3YearsAmt
Current Tax Year Minus2 Years$29,778CurrentTaxYearMinus2YearsAmt
Current Tax Year Minus1 Year$43,401CurrentTaxYearMinus1YearAmt
Current Tax Year$73,312CurrentTaxYearAmt
Total$154,663TotalAmt

Unrelated Business Net Incm170 UnrelatedBusinessNetIncm170Grp

FieldValueIRS element (for citing)
Total$0TotalAmt

Other Income170 OtherIncome170Grp

FieldValueIRS element (for citing)
Total$0TotalAmt
FieldValueIRS element (for citing)
Total Support$5,787,158TotalSupportAmt
Public Support CY1700.53385PublicSupportCY170Pct
Public Support PY1700.60408PublicSupportPY170Pct
Thirty Thr Pct Suprt Tests CY170Yes (X)ThirtyThrPctSuprtTestsCY170Ind

Schedule B IRS990ScheduleB

Contributor Information ContributorInformationGrp

FieldValueIRS element (for citing)
ContributorRESTRICTEDContributorNum

Contributor Business Name ContributorBusinessName

FieldValueIRS element (for citing)
Business Name Line1RESTRICTEDBusinessNameLine1

Contributor US Address ContributorUSAddress

FieldValueIRS element (for citing)
Address Line1RESTRICTEDAddressLine1
Address Line2RESTRICTEDAddressLine2
CityRESTRICTEDCity
StateRESTRICTEDState
ZIP CodeRESTRICTEDZIPCode
FieldValueIRS element (for citing)
Total ContributionsRESTRICTEDTotalContributionsAmt

Schedule F IRS990ScheduleF

FieldValueIRS element (for citing)
Grant Records MaintainedYes (true)GrantRecordsMaintainedInd

Account Activities Outside US AccountActivitiesOutsideUSGrp

FieldValueIRS element (for citing)
RegionEast Asia and the PacificRegionTxt
Offices0OfficesCnt
Employee0EmployeeCnt
Type Of Activities ConductedProgram ServicesTypeOfActivitiesConductedTxt
Specific Services ProvidedRESEARCHSpecificServicesProvidedTxt
Region Total Expenditures$10,481RegionTotalExpendituresAmt
FieldValueIRS element (for citing)
Subtotal Offices0SubtotalOfficesCnt
Subtotal Employees0SubtotalEmployeesCnt
Total Office0TotalOfficeCnt
Total Employee0TotalEmployeeCnt
Subtotal Spent$10,481SubtotalSpentAmt
Total Spent$10,481TotalSpentAmt
Transfer To Foreign CorpfalseTransferToForeignCorpInd
Interest In Foreign TrustfalseInterestInForeignTrustInd
Foreign Corp OwnershipfalseForeignCorpOwnershipInd
Passive Foreign Investmest CofalsePassiveForeignInvestmestCoInd
Foreign PartnershipfalseForeignPartnershipInd
Boycott CountriesfalseBoycottCountriesInd

Schedule I IRS990ScheduleI

FieldValueIRS element (for citing)
Grant Records MaintainedYes (true)GrantRecordsMaintainedInd

Recipient Table RecipientTable (13 entries)

Address Line1USAddress/AddressLine1TxtCityUSAddress/CityNmState AbbreviationUSAddress/StateAbbreviationCdZIPUSAddress/ZIPCdRecipient EINRecipientEINIRC SectionIRCSectionDescCash GrantCashGrantAmtPurpose Of GrantPurposeOfGrantTxt
CHARLES RIVER LABORATORIES
251 BALLARDVALE STREETWILMINGTONMA01887061397316501(c)(6)$68,025NGLY1
THE UNIVERSITY OF TEXAS SOUTHWESTERN MEDICAL CENTE
5323 HARRY HINES BLVDDALLASTX75390756002868GOVERNMENT$203,574NGLY1 (YAN LAB)
ALBERT EINSTEIN COLLEGE OF MEDICINE
1300 MORRIS PARK AVENUEBRONXNY10461830621846501(c)(3)$24,241NGLY1 (KAUSHIK LAB)
HILLTOP LAB ANIMALS INC
131 HILLTOP DRSCOTTDALEPA15683251153656$27,695NGLY1
BAYLOR COLLEGE OF MEDICINE
ONE BAYLOR PLAZA BCM 200HOUSTONTX77030741613278501(c)(3)$452,697NGLY1 (JAFAR-NEJAD LAB)
PURDUE UNIVERSITY
610 PURDUE MALLWEST LAFAYETTEIN47907366002041501(c)(3)$23,869NGLY1
UNIVERSITY OF HOUSTON
4800 CALHOUN ROADHOUSTONTX77204746001399115$9,747NGLY1
PSYCHOGENICS INC
215 College RdPARAMUSNJ07652141989159$352,945NGLY1
STANFORD UNIVERSITY
3145 PORTER DRIVEPALO ALTOCA94304941156365501(c)(3)$10,115NGLY1
UNC CHAPEL HILL
250 E Franklin StChapel HillNC27514566001393501(c)(3)$80,637NGLY1
Boston Children's Hospital
300 LONGWOOD AVENUEBOSTONMA02115042774441501(c)(3)$49,108NGLY1
PHARMARON
201 EAST JEFFERSON STREET SUITE 30LOUISVILLEKY40202208419781$20,356NGLY1
University of Utah
201 S PRESIDENTS CIR RM 406SALT LAKE CITYUT84112876000525115$19,321NGLY1
FieldValueIRS element (for citing)
Total501c3 Org8Total501c3OrgCnt
Total Other Org5TotalOtherOrgCnt

Supplemental Information Detail SupplementalInformationDetail (2 entries)

Form And Line ReferenceFormAndLineReferenceDescExplanationExplanationTxt
Part I Description of Procedure for Monitoring Use of Funds* 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.
PART II:THE FOUNDATION REQUESTS AND OBTAINS TO THE BEST OF THEIR ABILITY THE FEIN'S FROM ORGANIZATIONS IT PROVIDES GRANT FUNDING.

Schedule O IRS990ScheduleO

Supplemental Information Detail SupplementalInformationDetail (22 entries)

Form And Line ReferenceFormAndLineReferenceDescExplanationExplanationTxt
FORM 990, PART III, LINE 1: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.
FORM 990, PART III, LINE 4D, OTHER PROGRAM SERVICESBaylor College of Medicine NGLY1 Natural History Study (Bernhard Suter)Team is conducting 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. Bernhard Suter, MD, a child neurologist and medical geneticist. The study is collecting longitudinal measures of movement concurrently with clinical and biomarker measures to define aspects of gait and movement that are most affected in the disorder, characterize progression of the disease, select clinical features for performing longitudinal assessments, and inform clinical endpoint development for therapeutic trials. EXPENSES $452,697. INCLUDING GRANTS OF $452,697. REVENUE $0.
FORM 990, PART III, LINE 4D, OTHER PROGRAM SERVICESPsychogenics Inc. Team has imported and is analyzing mouse and rat models of NGLY1 Deficiency for testing of candidate therapies. Team has conducted several studies using an NGLY1 knockout rat model to test phenotypic rescue using candidate small molecule therapies (both novel and approved drugs). EXPENSES $352,945. INCLUDING GRANTS OF $352,945. REVENUE $0.
FORM 990, PART III, LINE 4D, OTHER PROGRAM SERVICESThe University of Texas Southwestern Medical Center (Yan Lab) Team continues work 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 including neuronal loss, neuropathological changes, inflammation, sensory and motor defects, and disease associated biomarkers, all of which are also observed in NGLY1 Deficiency patients These animal models provide 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. Using genetic and pharmacological approaches, the team has uncovered three different targets that can be modulated to reduce disease associated phenotypes and improve survival. EXPENSES $203,574. INCLUDING GRANTS OF $203,574. REVENUE $0.
FORM 990, PART III, LINE 4D, OTHER PROGRAM SERVICESThe University of North Carolina (Hantman Lab) Team has utilized a mouse model on NGLY1 Deficiency to analyze directed movement behavior and identify the affected brain circuits responsible for the movement phenotypes charcateristic of NGLY1 Deficiency. The studies involve training wild type and Ngly1-deficient mice to perform a "reach-grab" task, comparing the task performance and movement dynamics between typical and affected animals, and recording from specific brain circuits while the animals perform the task. Identified changes will help to better understand the neural basis of the NGLY1 Deficiency movement disorder, define the neural circuits that must be targeted to treat the disease, and establish a platform to test potential therapies. EXPENSES $80,637. INCLUDING GRANTS OF $80,637. REVENUE $0.
FORM 990, PART III, LINE 4D, OTHER PROGRAM SERVICESCharles River Laboratories Team has imported and is maintaining a colony of Ngly1 knockout rats for use in evaluating therapies to treat NGLY1 Deficiency. This knockout model has been a key foundation to test adeno-associated virus (AAV)-mediated gene delivery for rescue of disease-associated phenotypes and has supported an investigational new drug (IND) application to the FDA for an NGLY1 Deficiency gene therapy clinical study. Team has also conducted a study to monitor clinical phenotypes and collect biological samples in homozygous mutant and heterozygous (carrier) NGLY1 knockout rats across the lifespan from birth to eighteen months of age, and has continued to produce Ngly1 KO animals for use in in vivo drug testing studies. EXPENSES $68,025. INCLUDING GRANTS OF $68,025. REVENUE $0.
FORM 990, PART III, LINE 4D, OTHER PROGRAM SERVICESBoston Children's Hospital (Yu Lab) TEAM HAS CONCEIVED AND TESTED METHODS TO USE DESIGNER ANTISENSE OLIGONUCLEOTIDES (ASOS) TO INHIBIT POTENTIAL TARGETS FOR NGLY1 THERAPY DEVELOPMENT. THIS RESEARCH HAS DEVELOPED CELLULAR MODELS TO MEASURE INHIBITION OF A SPECIFIC GENE TARGET WHOSE INACTIVATION HAS BEEN SHOWN TO R ESCUE NGLY1 LOSS. PILOT SCREENS WERE CONDUCTED AND OPTIMIZED ASOS HAVEBEEN IDENTIFIED. TEAM IS RESEARCHING METHODS TO ASSESS RESCUE OF NGLY1 DEFICIENCY PHENOTYPES UPON ASO TREATMENT. EXPENSES $49,108. INCLUDING GRANTS OF $49,108. REVENUE $0.
FORM 990, PART III, LINE 4D, OTHER PROGRAM SERVICESHILLTOP LAB ANIMALS, INC. TEAM HAS IMPORTED AND ESTABLISHED A COLONY OF NGLY1 KNOCKOUT MICE FOR USE IN TESTING THERAPIES TO TREAT NGLY1 DEFICIENCY. A BREEDING COLONY HAS BEEN ESTABLISHED, AND MUTANT PHENOTYPES HAVE BEEN CHARACTERIZED. THIS MODEL SYSTEM HAS BEEN USED TO TEST DRUG CANDIDATES FOR THEIR ABILITY TO RESCUE NGLY1 DEFICIENCY-ASSOCIATED NEUROLOGICAL SYMPTOMS AND FOR GENETIC STUDIES TO TEST WHETHER BLOCKING OTHER PATHWAYS (INCLUDING FBXO6) HAS POTENTIAL TO TREAT NGLY1 DEFICIENCY. EXPENSES $27,695. INCLUDING GRANTS OF $27,695. REVENUE $0.
FORM 990, PART III, LINE 4D, OTHER PROGRAM SERVICESALBERT EINSTEIN COLLEGE OF MEDICINE (KAUSHIK LAB) TEAM IS TESTING THE HYPOTHESIS THAT DEFECTS IN NGLY1 FUNCTION LEADS TO THE IMPAIRMENT OF CELLULAR PROTEOSTASIS PATHWAYS THAT RECOGNIZE AND CLEAR MISFOLDED AND AGGREGATED PROTEINS AND DEFECTIVE ORGANELLES. USING NGLY1 KNOCKOUT RAT AND MOUSE TISSUES, NGLY1 MUTANT CELL LINES, AND NGLY1 DEFICIENCY PATIENT FIBROBLASTS, THE TEAM HAS OBSERVED SIGNIFICANT IMPAIRMENT IN MACRO- AND CHAPERONE MEDIATED-AUTOPHAGY AND DEFECTS IN LYSOSOMAL BIOGENESIS. THESE RESULTS HIGHLIGHT THE POTENTIAL OF DRUGS THAT TARGET THE AUTOPHAGY-LYSOSOME PATHWAY TO REVERSE CELLULAR DEFECTS ASSOCIATED WITH NGLY1 DEFICIENCY. THE TEAM HAS BEGUN EVALUATING DRUG-LIKE CHEMICAL MODULATORS OF THE AUTOPHAGY-LYSOSOMAL SYSTEM FOR THEIR ABILITY TO REDUCE CELLULAR PROTEOTOXICITY IN NGLY1 DEFICIENCY MODELS. EXPENSES $24,241. INCLUDING GRANTS OF $24,241. REVENUE $0.
FORM 990, PART III, LINE 4D, OTHER PROGRAM SERVICESPURDUE UNIVERSITY (ROCHET LAB) TEAM IS DEVELOPING AND EMPLOYING CELLULAR MODELS TO TEST WHETHER NGLY1 AND ITS SUBSTRATE NFE2L1 HAVE A ROLE IN MODULATING CELLULAR STRESS AND PREVENTING OR CLEARING PROTEIN AGGREGATES IN THE BRAIN. THESE STUDIES ARE TESTING WHETHER LOSS OR DYSFUNCTION OF THE NGLY1-NFE2L1 PATHWAY MAY SENSITIZE NERVE CELLS TO OXIDATIVE OR PROTEIN MISFOLDING STRESS, LEADING TO NEURONAL DAMAGE AND NEURODEGENERATION. IN ADDITION, THE TEAM IS TESTING THE IDEA THAT ENHANCING NGLY1-NFE2L1 ACTIVITY CAN PREVENT OR SLOW NEURONAL LOSS USING CELLULAR MODELS OF PARKINSON'S DISEASE, A NEURODEGENERATIVE DISORDER ASSOCIATED WITH OXIDATIVE STRESS AND PROTEIN AGGREGATION. THESE STUDIES AIM TO UNDERSTAND THE CAUSE OF NEUROTOXICITY IN RARE AND COMMON NEURODEGENERATIVE DISORDERS, EXPLORE MECHANISMS SHARED BETWEEN NGLY1 DEFICIENCY AND PARKINSON'S DISEASE, AND PROVIDE INSIGHTS FOR THE DEVELOPMENT OF DISEASE-MODIFYING NEUROPROTECTIVE THERAPIES. EXPENSES $23,869. INCLUDING GRANTS OF $23,869. REVENUE $0.
FORM 990, PART III, LINE 4D, OTHER PROGRAM SERVICESPHARMARON, INC TEAM HAS CONDUCTED PHARMACOLOGY STUDIES TO SUPPORT IN VIVO TESTING OF SMALL MOLECULE THERAPIES FOR NGLY1 DEFICIENCY. EXPENSES $20,356. INCLUDING GRANTS OF $20,356. REVENUE $0.
FORM 990, PART III, LINE 4D, OTHER PROGRAM SERVICESUNIVERSITRY OF UTAH (CHOW LAB) TEAM HAS DEVELOPED AND IMPORTED MODEL ORGANISM AND HUMAN CELLULAR MODELS OF NGLY1 DEFICIENCY, CHARACTERIZED THEIR ABNORMALITIES AND BEGUN TO USE THESE TO INVESTIGATE CANDIDATE THERAPEUTIC TARGETS OF THIS RARE DISEASE. IN PARTICULAR THE TEAM IS TESTING PREVIOUSLY IDENTIFIED DRUG HITS AND INHIBITORS OF GLYCOGEN SYNTHASE 3-BETA (GSK3B) IN CELLULAR MODELS OF NGLY1 DEFICIENCY, AND IN FLY AND MOUSE MODELS OF NGLY1 DEFICIENCY. EXPENSES $19,321. INCLUDING GRANTS OF $19,321. REVENUE $0.
FORM 990, PART III, LINE 4D, OTHER PROGRAM SERVICESBIOAGILYTIX LABS - vendor has conducted biomarker and immune profiling of NGLY1 Deficiency patients in support of NGLY1 Deficiency natural history studies. EXPENSES $19,000. INCLUDING GRANTS OF $0. REVENUE $0. TRANSNETYX - vendor has provided genotyping services to support animal breeding and functional studies at Hilltop Lab Animals and Psychogenics, Inc. EXPENSES $13,904. INCLUDING GRANTS OF $0. REVENUE $0. WUXI APPTEC - vendor has synthesized and characterized small molecule drug candidates for in vivo studies. EXPENSES $10,481. INCLUDING GRANTS OF $0. REVENUE $0.
FORM 990, PART III, LINE 4D, OTHER PROGRAM SERVICESSTANFORD UNIVERSITY DIXON AND BERTOZZI LABORATORIES) TEAM HAS CHARACTERIZED THE ROLE OF FERROPTOSIS, A FORM OF CELL DEATH ASSOCIATED WITH OXIDATIVE STRESS, IN THE PATHOLOGIES ASSOCIATED WITH NGLY1 DEFICIENCY. THIS STUDY HAS ESTABLISHED THAT LOSS OF NGLY1 OR ITS SUBSTRATE NFE2L1 SENSITIZES CELLS TO FERROPTOSIS, AND THIS FERROPTOSIS SENSITIVITY CAN BE REVERSED BY EXPRESSION OF AN ACTIVATED FORM OF NFE2L1 MIMICKING THAT GENERATED BY NGLY1-CATALYZED PROTEIN MODIFICATION. THESE STUDIES MAY HELP EXPLAIN THE CAUSES OF CELL DYSFUNCTION AND LOSS IN NGLY1 DEFICIENCY AND SUGGEST POTENTIAL TARGETS FOR CANDIDATE THERAPY DEVELOPMENT. EXPENSES $10,115. INCLUDING GRANTS OF $10,115. REVENUE $0.
FORM 990, PART III, LINE 4D, OTHER PROGRAM SERVICESUNIVERSITY OF HOUSTON - Team has conducted a pilot movement disorder study to systematically and quantitatively assess movement disorder characteristics of individual patients with NGLY1 Deficiency. EXPENSES $9,747. INCLUDING GRANTS OF $9,747. REVENUE $0. MEDCHEM EXPRESS LLC - vendor has provided small molecule compounds for in vivo and in vitro studies. EXPENSES $5,369. INCLUDING GRANTS OF $0. REVENUE $0.
FORM 990, PART VI, SECTION A, LINE 2:THE PRESIDENT OF THE FOUNDATION HAS IMMATERIAL OWNERSHIP INTEREST, IN THE INVESTMENTS OF FORTRESS AND SILVERLAKE, 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 FORM 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.

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