Attach to Form 990 or Form 990-EZ.
Go to
www.irs.gov/Form990 for instructions and the latest information.
| (i) Name of supported organization | (ii) EIN | (iii) Type of organization (described on lines 1- 10 above (see instructions)) | (iv) Is the organization listed in your governing document? | (v) Amount of monetary support (see instructions) | (vi) Amount of other support (see instructions) | |
|---|---|---|---|---|---|---|
| Yes | No | |||||
|
Total |
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Calendar year
(or fiscal year beginning in)
![]() |
(a) 2017 | (b) 2018 | (c) 2019 | (d) 2020 | (e) 2021 | (f) Total | |
|---|---|---|---|---|---|---|---|
| 1 | Gifts, grants, contributions, and membership fees received. (Do not include any "unusual grant.") .. | 1,515,412 | 2,052,054 | 1,256,624 | 1,862,485 | 2,135,123 | 8,821,698 |
| 2 | Tax revenues levied for the organization's benefit and either paid to or expended on its behalf.... | 0 | |||||
| 3 | The value of services or facilities furnished by a governmental unit to the organization without charge.. | 0 | |||||
| 4 | Total. Add lines 1 through 3 | 1,515,412 | 2,052,054 | 1,256,624 | 1,862,485 | 2,135,123 | 8,821,698 |
| 5 | The portion of total contributions by each person (other than a governmental unit or publicly supported organization) included on line 1 that exceeds 2% of the amount shown on line 11, column (f) .. | 0 | |||||
| 6 | Public support. Subtract line 5 from line 4. | 8,821,698 | |||||
Calendar year
(or fiscal year beginning in)
![]() |
(a) 2017 | (b) 2018 | (c) 2019 | (d) 2020 | (e) 2021 | (f) Total | |
|---|---|---|---|---|---|---|---|
| 7 | Amounts from line 4.. | 1,515,412 | 2,052,054 | 1,256,624 | 1,862,485 | 2,135,123 | 8,821,698 |
| 8 | Gross income from interest, dividends, payments received on securities loans, rents, royalties and income from similar sources... | 2,943,703 | 31,628 | 127,390 | 233,157 | 186,864 | 3,522,742 |
| 9 | Net income from unrelated business activities, whether or not the business is regularly carried on.. | ||||||
| 10 | Other income. Do not include gain or loss from the sale of capital assets (Explain in Part VI.).. | 10,483 | 7,985 | 1,436 | 19,904 | ||
| 11 | Total support. Add lines 7 through 10 | 12,364,344 | |||||
Calendar year (or fiscal year beginning in) ![]() |
(a) 2017 | (b) 2018 | (c) 2019 | (d) 2020 | (e) 2021 | (f) Total | |
|---|---|---|---|---|---|---|---|
| 1 | Gifts, grants, contributions, and membership fees received. (Do not include any "unusual grants.") . | ||||||
| 2 | Gross receipts from admissions, merchandise sold or services performed, or facilities furnished in any activity that is related to the organization's tax-exempt purpose | ||||||
| 3 | Gross receipts from activities that are not an unrelated trade or business under section 513 ..... | ||||||
| 4 | Tax revenues levied for the organization's benefit and either paid to or expended on its behalf... | ||||||
| 5 | The value of services or facilities furnished by a governmental unit to the organization without charge | ||||||
| 6 | Total. Add lines 1 through 5 | ||||||
| 7a | Amounts included on lines 1, 2, and 3 received from disqualified persons | ||||||
| b | Amounts included on lines 2 and 3 received from other than disqualified persons that exceed the greater of $5,000 or 1% of the amount on line 13 for the year. | ||||||
| c | Add lines 7a and 7b.. | ||||||
| 8 | Public support. (Subtract line 7c from line 6.) | ||||||
Calendar year (or fiscal year beginning in) ![]() |
(a) 2017 | (b) 2018 | (c) 2019 | (d) 2020 | (e) 2021 | (f) Total | |
|---|---|---|---|---|---|---|---|
| 9 | Amounts from line 6... | ||||||
| 10a | Gross income from interest, dividends, payments received on securities loans, rents, royalties and income from similar sources.. | ||||||
| b | Unrelated business taxable income (less section 511 taxes) from businesses acquired after June 30, 1975. | ||||||
| c | Add lines 10a and 10b. | ||||||
| 11 | Net income from unrelated business activities not included on line 10b, whether or not the business is regularly carried on. | ||||||
| 12 | Other income. Do not include gain or loss from the sale of capital assets (Explain in Part VI.) .. | ||||||
| 13 | Total support. (Add lines 9, 10c, 11, and 12.).. | ||||||
| Section A - Adjusted Net Income | (A) Prior Year |
(B) Current Year (optional) |
||||
| 1 | Net short-term capital gain | 1 | ||||
| 2 | Recoveries of prior-year distributions | 2 | ||||
| 3 | Other gross income (see instructions) | 3 | ||||
| 4 | Add lines 1 through 3 | 4 | ||||
| 5 | Depreciation and depletion | 5 | ||||
| 6 | Portion of operating expenses paid or incurred for production or collection of gross income or for management, conservation, or maintenance of property held for production of income (see instructions) | 6 | ||||
| 7 | Other expenses (see instructions) | 7 | ||||
| 8 | Adjusted Net Income (subtract lines 5, 6 and 7 from line 4) | 8 | ||||
| Section B - Minimum Asset Amount | (A) Prior Year |
(B) Current Year (optional) |
||||
| 1 | Aggregate fair market value of all non-exempt-use assets (see instructions for short tax year or assets held for part of year): | 1 | ||||
| a | Average monthly value of securities | 1a | ||||
| b | Average monthly cash balances | 1b | ||||
| c | Fair market value of other non-exempt-use assets | 1c | ||||
| d | Total (add lines 1a, 1b, and 1c) | 1d | ||||
| e |
Discount claimed for blockage or other factors (explain in detail in Part VI): |
|||||
| 2 | Acquisition indebtedness applicable to non-exempt use assets | 2 | ||||
| 3 | Subtract line 2 from line 1d | 3 | ||||
| 4 | Cash deemed held for exempt use. Enter 0.015 of line 3 (for greater amount, see instructions). | 4 | ||||
| 5 | Net value of non-exempt-use assets (subtract line 4 from line 3) | 5 | ||||
| 6 | Multiply line 5 by 0.035 | 6 | ||||
| 7 | Recoveries of prior-year distributions | 7 | ||||
| 8 | Minimum Asset Amount (add line 7 to line 6) | 8 | ||||
| Section C - Distributable Amount | Current Year | |||||
| 1 | Adjusted net income for prior year (from Section A, line 8, Column A) | 1 | ||||
| 2 | Enter 85% of line 1 | 2 | ||||
| 3 | Minimum asset amount for prior year (from Section B, line 8, Column A) | 3 | ||||
| 4 | Enter greater of line 2 or line 3 | 4 | ||||
| 5 | Income tax imposed in prior year | 5 | ||||
| 6 | Distributable Amount. Subtract line 5 from line 4, unless subject to emergency temporary reduction (see instructions) | 6 | ||||
| Section D - Distributions | Current Year | |
|---|---|---|
| 1 Amounts paid to supported organizations to accomplish exempt purposes | 1 | |
|
2
Amounts paid to perform activity that directly furthers exempt purposes of supported organizations, in excess of income from activity |
2 | |
| 3 Administrative expenses paid to accomplish exempt purposes of supported organizations | 3 | |
| 4 Amounts paid to acquire exempt-use assets | 4 | |
| 5 Qualified set-aside amounts (prior IRS approval required - provide details in Part VI) | 5 | |
| 6 Other distributions (describe in Part VI). See instructions | 6 | |
| 7Total annual distributions. Add lines 1 through 6. | 7 | |
|
8
Distributions to attentive supported organizations to which the organization is responsive (provide details in Part VI). See instructions |
8 | |
| 9 Distributable amount for 2021 from Section C, line 6 | 9 | |
| 10 Line 8 amount divided by Line 9 amount | 10 | |
| Section E - Distribution Allocations (see instructions) |
(i) Excess Distributions |
(ii) Underdistributions Pre-2021 |
(iii) Distributable Amount for 2021 |
|
|---|---|---|---|---|
| 1 Distributable amount for 2021 from Section C, line 6 | ||||
|
2
Underdistributions, if any, for years prior to 2021 (reasonable cause required-- explain in Part VI). See instructions. |
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| 3 Excess distributions carryover, if any, to 2021: | ||||
| a From 2016....... | ||||
| b From 2017....... | ||||
| c From 2018....... | ||||
| d From 2019....... | ||||
| e From 2020....... | ||||
| fTotal of lines 3a through e | ||||
| g Applied to underdistributions of prior years | ||||
| h Applied to 2021 distributable amount | ||||
|
i
Carryover from 2016 not applied (see instructions) |
||||
| j Remainder. Subtract lines 3g, 3h, and 3i from line 3f. | ||||
| 4Distributions for 2021 from Section D, line 7: | ||||
| $ | ||||
| a Applied to underdistributions of prior years | ||||
| b Applied to 2021 distributable amount | ||||
| c Remainder. Subtract lines 4a and 4b from line 4. | ||||
|
5
Remaining underdistributions for years prior to 2021, if any. Subtract lines 3g and 4a from line 2. If the amount is greater than zero, explain in Part VI. See instructions. |
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|
6
Remaining underdistributions for 2021. Subtract lines 3h and 4b from line 1. If the amount is greater than zero, explain in Part VI. See instructions. |
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|
7 Excess distributions carryover to 2022. Add lines 3j and 4c. |
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| 8 Breakdown of line 7: | ||||
| a Excess from 2017..... | ||||
| b Excess from 2018..... | ||||
| c Excess from 2019..... | ||||
| d Excess from 2020..... | ||||
| e Excess from 2021..... | ||||
| Facts And Circumstances Test |
|---|
| Return Reference | Explanation |
|---|
| Software ID: | 21013475 |
| Software Version: | 2021v4.0 |
Attach to Form 990 or 990-EZ.
Go to www.irs.gov/Form990 for the latest information.
| Return Reference | Explanation |
|---|---|
| Form 990, Part VI, Line 2: Description of Business or Family Relationship of Officers, Directors, Et | Glenn ONeill and Cara ONeill MD FAAP are husband and wife. |
| Form 990, Part VI, Line 11b: Form 990 Review Process | All Board Members have reviewed the Form 990. |
| Form 990, Part VI, Line 12c: Explanation of Monitoring and Enforcement of Conflicts | Conflicts of interest are reviewed during Board Meetings |
| Form 990, Part VI, Line 15a: Compensation Review & Approval Process - CEO, Top Management | The Employment Committee is responsible for all hiring decisions, including compensation, benefits, job performance etc... The Board members make up the Employment Committee. The Committee reviews multiple 990's and other resources and comparable benchmarks to establish reasonable compensation for employees. |
| Form 990, Part VI, Line 15b: Compensation Review and Approval Process for Officers and Key Employees | The Employment Committee reviews multiple 990's and other resources and comparable benchmarks to establish reasonable compensation for employees. |
| Form 990, Part VI, Line 19: Other Organization Documents Publicly Available | The organization's governing documents, policies, and financial statements are available upon request. The Form 990 is uploaded on the Guidestar website. |
| Other Changes In Net Assets Or Fund Balances - Other Decreases | Accrual to Cash Adjustment = -$62982 |
| Other Changes In Net Assets Or Fund Balances - Other Increases | PPP Loan = $63451 |
| Antifungal immunity in patients with mucopolysaccharidosis | TITLE: Antifungal immunity in patients with mucopolysaccharidosisAwardee: Koret School of Veterinary Medicine - Hebrew University of JerusalemRehovot, IsraelAMOUNT IN 2021: $45,000.00Date: July 23, 2021 |
| Blood-based biomarkers for Sanfilippo syndrome progression and treatme | TITLE: Blood-based biomarkers for Sanfilippo syndrome progression and treatmentAwardee: The Regents of the University of CaliforniaSan Diego, CaliforniaAMOUNT IN 2021: $64,479.00Date: October 12, 2021 |
| COVID-19 related pivots to Distance formats | TITLE: COVID-19 related pivots to Distance formats: advocacy for neuronopathic mucopolysaccharidosis disordersAWARDEE: University of MinnesotaMinneapolis, MN AMOUNT IN 2021: $5,000.00EIN: 41-6007513DATE: January, 20, 2021. |
| Exploring novel photobiomodulation parameters in an animal model of Sa | TITLE: Exploring novel photobiomodulation parameters in an animal model of Sanfilippo syndromeAwardee: University of Sydney and Flinders UniversitySydney, AustraliaAMOUNT IN 2021: $27,667.00Date: August 23, 2021 |
| Facilitate & Enhance the CNS entry of AAV Vectors to treat MPSIIIA & B | TITLE: Facilitate and enhance the CNS entry of systemically delivered AAV vectors for treating MPSIIIA and MPSIIIB in mice.Awardee: Office of Sponsored Research at the University of North CarolinaChapel Hill, NCAMOUNT IN 2021: $46,700.00EIN: 56-6001393Date: November 8, 2021 |
| Gene Therapy JLK-247 for regulatory preclinical safety studies for Pho | TITLE: Gene Therapy JLK-247 for regulatory preclinical safety studies for Phoenix Nest for MPS IIICAWARDEE: Phoenix NestBrooklyn, NY AMOUNT IN 2021: $125,000.00DATE: March 1, 2021. |
| Grant Detail | The following grants were provided: |
| In Vivo testing of FDA-approved drug combination for MPSIII Treatment | TITLE: In Vivo testing of FDA-approved drug combination for MPSIII TreatmentAwardee: The Telethon Institute of Genetics and Medicine (TIGEM)Pozzuoli, ItalyAMOUNT IN 2021: $22,000.00Date: November 9, 2021 |
| NDST1 inhibitors as target for substrate reduction therapy | TITLE: NDST1 inhibitors as target for substrate reduction therapyAwardee: The Hospital for Sick ChildrebToronto, ONAMOUNT IN 2021: $44,656.00Date: March & October 2021 |
| Neuroprotective peptides for treatment of Sanfilippo disease | TITLE: Neuroprotective peptides for treatment of Sanfilippo diseaseAwardee: Phoenix Nest Inc.Brookyln, NY AMOUNT IN 2021: $51,750.00Date: October 18, 2021 |
| Novel Immune Deficient MPSIIIA Mouse Model Maintenance | TITLE: Novel Immune Deficient MPSIIIA Mouse Model MaintenanceAwardee: The Regents of the University of CaliforniaSan Diego, CaliforniaAmount in 2021: $15,015.00Date: October 12, 2021. |
| Open-Label Pilot Clinical Trial Study the effects of Anakinra in Mucop | TITLE: Open-Label Pilot Clinical Trial Study the effects of Anakinra in Mucopolysaccharidosis IIIAWARDEE: The Lundquist InstituteTorrance, CAAMOUNT IN 2021: $222,462.50EIN: 95-2138184Date: October 22, 2021. |
| Screen Plus Newborn Screening | TITLE: Screen Plus Newborn ScreeningAWARDEE: Albert Einstein College of MedicineBronx, NY 10461AMOUNT IN 2021: $125,000.00EIN: 83-0621846DATE: February & October 2021 |
| Strengthening the rationale for the use of the "molecular tweezer" CLR | TITLE: Strengthening the rationale for the use of the "molecular tweezer" CLR01 in the treatment of Sanfilippo syndromeAWARDEE: Ceinge BiotechNaples, ItalyAMOUNT IN 2021: $112,500.00DATE: March & December 2021 |
| Targeting amyloid agrregation as a new therapeutic approach to treat t | TITLE: Targeting amyloid agrregation as a new therapeutic approach to treat the CNS in Sanfilippo SyndromeAwardee: TIGEMPozzuoli, ItalyAMOUNT IN 2021: $41,250.00DATE: February 8, 2021 |
| Targeting Heparan Sulfate Proteoglycans as a Novel Therapeutic Strateg | TITLE: Targeting Heparan Sulfate Proteoglycans as a Novel Therapeutic Strategy for Sanfilippo diseasesAwardee: University of Naples Federico IINaples, ItalyAMOUNT IN 2021: $42,000.00Date: July 12, 2021 |
| The role of heparan sulfate and dopamine as disease' modifiers in Muco | TITLE: The role of heparan sulfate and dopamine as disease' modifiers in Mucopolysacchariodosis type IIIAAwardee: TIGEMPozzuoli, Italy AMOUNT IN 2021: $40,098.75Date: November 3, 2021 |
| Transplant MPSIII Human Neural Stem Cells | TITLE: Transplant MPSIII human neural stem cells engineered to overexpress and secrete SGSHAwardee: ReMotor TherapeuticsDoral, FLAMOUNT IN 2021: 933,000.00Date: Payments made in March & November 2021. |
| Software ID: | 21013475 |
| Software Version: | 2021v4.0 |