Data / Research funding / Mucopolysaccharidoses (MPS)
Mucopolysaccharidoses (MPS): 47 NIH awards ($69.4M) to 28 organizations
47 NIH awards ($69.4M) in the NIH spending category Mucopolysaccharidoses (MPS), to 28 organizations, read 2026-11-03. NIH fiscal years 2021 to 2025.
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Awards
47 awards in the window.
| Award | Agency | Institute or division | Fiscal year | Amount in window | Start | End | Principal investigator | Source |
|---|---|---|---|---|---|---|---|---|
| Guide RNA manufacturing capabilities for platform in vivo base editing therapies for metabolic liver diseases | NIH | National Institute of Neurological Disorders and Stroke | 2023 to 2025 | $17,556,074 | August 1, 2023 | July 31, 2028 | PERANTEAU, WILLIAM H. | reporter.nih.gov |
| Evaluation of Clinical Outcome Assessment (COA) and Potential Biomarkers to Facilitate Interventional Trial for Mucopolysaccharidosis IIID Patients | NIH | National Institute of Neurological Disorders and Stroke | 2021 to 2024 | $3,519,934 | September 21, 2021 | August 31, 2025 | SINGAMSETTY, SRIKANTH | reporter.nih.gov |
| Development of Gene Replacement Therapy for Sanfilippo Syndrome Type C | NIH | National Institute of Neurological Disorders and Stroke | 2022 to 2023 | $2,990,505 | September 19, 2022 | August 31, 2025 | SINGAMSETTY, SRIKANTH | reporter.nih.gov |
| Non-invasive functional assessment and pathogenesis of Morquio A | NIH | Eunice Kennedy Shriver National Institute of Child Health & Human Development | 2021 to 2025 | $2,932,986 | May 1, 2021 | April 30, 2027 | TOMATSU, SHUNJI | reporter.nih.gov |
| Enhancement of Newborn Screening Diagnostic Paradigms to Improve the Efficacy of Treatment for Krabbe Disease, Pompe Disease, and Mucopolysaccharidosis Type 1 | NIH | Eunice Kennedy Shriver National Institute of Child Health & Human Development | 2022 to 2025 | $2,716,923 | March 18, 2022 | February 28, 2027 | LANGAN, THOMAS J | reporter.nih.gov |
| Phase 1 Study of In Utero Enzyme Replacement Therapy for the Treatment of Lysosomal Storage Diseases | NIH | Eunice Kennedy Shriver National Institute of Child Health & Human Development | 2022 to 2025 | $2,670,263 | September 20, 2022 | April 30, 2027 | MACKENZIE, TIPPI | reporter.nih.gov |
| NEWBORN SCREENING PILOT STUDIES FOR MUCOPOLYSACCHARIDOSIS TYPE II | NIH | Eunice Kennedy Shriver National Institute of Child Health & Human Development | 2021 to 2023 | $2,631,501 | September 28, 2021 | September 27, 2024 | RASPA, MELISSA | reporter.nih.gov |
| Real-Time Tracking of Gene Therapy by Bioactivated MR contrast Probes | NIH | National Institute of Neurological Disorders and Stroke | 2021 to 2024 | $2,508,603 | September 1, 2020 | May 31, 2026 | MEADE, THOMAS J | reporter.nih.gov |
| Development and Validation of Therapy for Mucopolysaccharidosis III | NIH | National Institute of Neurological Disorders and Stroke | 2022 | $2,435,870 | September 1, 2014 | June 30, 2025 | WOOD, JILL | reporter.nih.gov |
| Protein depleting pre-existing antibodies for viral gene therapy | NIH | National Institute of Allergy and Infectious Diseases | 2023 to 2025 | $2,276,317 | August 1, 2023 | July 31, 2026 | MCCARTY, DOUGLAS M | reporter.nih.gov |
| Identification of Potential Biomarkers and Clinical Tools for use in Mucopolysaccharidosis IIIC Patients. | NIH | National Institute of Neurological Disorders and Stroke | 2024 to 2025 | $1,944,180 | July 1, 2024 | June 30, 2027 | SINGAMSETTY, SRIKANTH | reporter.nih.gov |
| Advancing CNS drug delivery via epigenetic modulation | NIH | National Institute of Neurological Disorders and Stroke | 2023 to 2025 | $1,868,790 | January 18, 2023 | December 31, 2026 | PAN, DAO | reporter.nih.gov |
| Combination Gene Therapy for Treatment of Canine Mucopolysaccharidosis Type I | NIH | Eunice Kennedy Shriver National Institute of Child Health & Human Development | 2023 to 2025 | $1,867,960 | May 5, 2023 | February 29, 2028 | WANG, RAYMOND YU JEANG | reporter.nih.gov |
| Development of gene therapy product for treating MPS IIIB | NIH | National Institute of Neurological Disorders and Stroke | 2024 to 2025 | $1,762,240 | September 1, 2024 | August 31, 2027 | MCCARTY, DOUGLAS M | reporter.nih.gov |
| Procession to IND of a capsid mutated AAV8 codon optimized NAGLU vector for treatment of Sanfilippo Syndrome type B (Supplement) | NIH | National Institute of Neurological Disorders and Stroke | 2024 to 2025 | $1,657,797 | September 29, 2025 | June 30, 2026 | HELDERMON, COY D | reporter.nih.gov |
| Engineered B Cells as a Universal Platform for the Treatment of Enzymopathies | NIH | National Institute of Allergy and Infectious Diseases | 2021 to 2024 | $1,549,167 | March 1, 2020 | February 28, 2025 | MORIARITY, BRANDEN S | reporter.nih.gov |
| Trans-BBB AAV9 gene therapy targeting CNS neuropathy of MPS I | NIH | National Institute of Neurological Disorders and Stroke | 2025 | $1,499,504 | September 16, 2025 | August 31, 2027 | MCCARTY, DOUGLAS M | reporter.nih.gov |
| Cellular Phenotypes of Genetic Variants in Mucopolysaccharidosis | NIH | National Institute of Neurological Disorders and Stroke | 2023 to 2025 | $1,338,069 | April 1, 2023 | March 31, 2028 | DICKSON, PATRICIA I | reporter.nih.gov |
| Identification of glycosaminoglycans for newborn screening and therapeutic monitoring of mucopolysaccharidoses | NIH | Eunice Kennedy Shriver National Institute of Child Health & Human Development | 2021 to 2022 | $1,331,791 | September 21, 2021 | August 31, 2024 | NG, RAINER | reporter.nih.gov |
| Pathogenic Mechanisms of Lysosomal Disease | NIH | National Institute of Neurological Disorders and Stroke | 2022 to 2025 | $1,221,237 | January 1, 2009 | June 30, 2027 | STEET, RICHARD | reporter.nih.gov |
| Enzyme Replacement Therapeutics for Rare Childhood Genetic Diseases: An ERT Delivery System that Mitigates Immune-sensitization | NIH | Eunice Kennedy Shriver National Institute of Child Health & Human Development | 2021 | $905,262 | July 6, 2016 | March 31, 2024 | CRAMER, CAROLE L. | reporter.nih.gov |
| Cardiovascular Structure and Function in the Mucopolysaccharidoses | NIH | National Heart, Lung, and Blood Institute | 2025 | $852,280 | July 15, 2025 | April 30, 2030 | WANG, RAYMOND YU JEANG | reporter.nih.gov |
| Feasibility and validation of an integrated newborn screening algorithm with targeted Next Generation Sequencing (tNGS) technology as part of a 2nd-tier test for Pompe and MPS I | NIH | Eunice Kennedy Shriver National Institute of Child Health & Human Development | 2021 | $793,462 | August 16, 2018 | December 31, 2022 | AMIN, VIREN R | reporter.nih.gov |
| Develop AAV9 gene replacement therapy for treating MPS I | NIH | National Institute of Neurological Disorders and Stroke | 2022 to 2023 | $702,684 | August 1, 2022 | January 31, 2025 | FU, HAIYAN | reporter.nih.gov |
| An integrated nano- and micro-indentation system for musculoskeletal tissue and biomaterials | NIH | Office of the Director, National Institutes of Health | 2023 | $691,040 | April 1, 2023 | March 31, 2025 | LIU, XIAOWEI SHERRY | reporter.nih.gov |
| Recombinant alpha-N-acetylglucosaminidase with improved mannose 6-phosphorylation for treatment of Sanfilippo B syndrome | NIH | National Institute of Neurological Disorders and Stroke | 2022 | $644,183 | December 1, 2019 | November 30, 2024 | DICKSON, PATRICIA I | reporter.nih.gov |
| Disease Severity Stratification in Multiple Sulfatase Deficiency | NIH | Eunice Kennedy Shriver National Institute of Child Health & Human Development | 2022 to 2023 | $490,070 | September 13, 2022 | August 31, 2025 | AHRENS-NICKLAS, REBECCA CLARE | reporter.nih.gov |
| Patient-Centric Clinical Outcome Measures for Multiple Sulfatase Deficiency (MSD) | NIH | National Center for Advancing Translational Sciences | 2025 | $489,500 | September 3, 2025 | September 2, 2027 | ADANG, LAURA ANN | reporter.nih.gov |
| Optimizing AAV Vectors for Central Nervous System transduction | NIH | National Institute of Neurological Disorders and Stroke | 2021 | $477,909 | August 1, 2017 | November 30, 2023 | HELDERMON, COY D | reporter.nih.gov |
| Title: Development and Validation of Therapy for GM1 Gangliosidosis | NIH | National Institute of Neurological Disorders and Stroke | 2023 | $461,701 | September 15, 2023 | August 31, 2025 | BROWN, JILLIAN R | reporter.nih.gov |
| Microglial-Mediated Glycosaminoglycan Catabolism in MPS VII Neuropathology. | NIH | National Institute of Neurological Disorders and Stroke | 2025 | $453,033 | August 1, 2025 | July 31, 2027 | YELLAJOSHYULA, DHANANJAY | reporter.nih.gov |
| Gene Therapy for MPS IV A in a Novel Porcine Model of the Disease | NIH | National Institute of Arthritis and Musculoskeletal and Skin Diseases | 2024 | $384,999 | September 18, 2024 | August 31, 2025 | CAMPBELL, JARRYD MATTHEW | reporter.nih.gov |
| Gene Therapy of Corneal Dystrophy: Lysosomal Storage Diseases | NIH | National Eye Institute | 2021 | $379,618 | September 30, 2019 | June 30, 2023 | KAO, WINSTON W | reporter.nih.gov |
| Tolerability and Potency of Sequential and Repeated AAV Corneal Gene Therapy | NIH | National Eye Institute | 2022 | $367,624 | May 1, 2022 | April 30, 2023 | HIRSCH, MATTHEW LOUIS | reporter.nih.gov |
| Novel AAV gene therapy for treating CNS neuropathy of MPS IIIC | NIH | National Institute of Neurological Disorders and Stroke | 2025 | $349,909 | July 8, 2025 | April 30, 2027 | FU, HAIYAN | reporter.nih.gov |
| Pathogenesis and Treatment of Bone Disease in the Mucopolysaccharidoses | NIH | National Institute of Arthritis and Musculoskeletal and Skin Diseases | 2021 | $344,172 | August 1, 2017 | May 31, 2024 | SMITH, LACHLAN JAMES | reporter.nih.gov |
| Cell-based Gene Therapy for MPS IVA | NIH | National Institute of Arthritis and Musculoskeletal and Skin Diseases | 2024 | $306,872 | August 1, 2024 | July 31, 2026 | MOUNZIH, KHALID | reporter.nih.gov |
| Engineered Memory T cells as a Platform to Treat Enzymopathies | NIH | National Institute of Allergy and Infectious Diseases | 2025 | $304,838 | September 26, 2025 | August 31, 2026 | SHIRKEY-SON, NICOLE J. | reporter.nih.gov |
| Morquio A therapy integrating gene transfer with lectin-enhanced enzyme delivery to treat multisystemic clinical impairments of rare metabolic childhood diseases | NIH | Eunice Kennedy Shriver National Institute of Child Health & Human Development | 2023 | $295,923 | September 20, 2023 | August 31, 2025 | ACOSTA, WALTER | reporter.nih.gov |
| Development of gene therapy product for treating MPS IIIB | NIH | National Institute of Neurological Disorders and Stroke | 2021 | $284,425 | April 15, 2020 | March 31, 2023 | FU, HAIYAN | reporter.nih.gov |
| Novel diagnostic biomarker reference standards for newborn screening of Mucopolysaccharidoses type I and II. | NIH | Eunice Kennedy Shriver National Institute of Child Health & Human Development | 2023 | $272,872 | September 22, 2023 | January 31, 2025 | KHALEDI, HAMID | reporter.nih.gov |
| Correction of Mucopolysaccharidosis type 1: Targeting safe harbor loci using genome editing | NIH | National Institute of Neurological Disorders and Stroke | 2021 to 2022 | $250,236 | July 15, 2017 | February 15, 2023 | GOMEZ-OSPINA, NATALIA | reporter.nih.gov |
| Characterizing and treating peripheral nervous system dysfunction in Sanfilippo Syndrome | NIH | National Institute of Neurological Disorders and Stroke | 2025 | $226,500 | February 1, 2025 | January 31, 2027 | WEISS, RYAN JOSEPH | reporter.nih.gov |
| Cellular phenotype of mucopolysaccharidosis II for studies of genomic variants | NIH | National Institute of Neurological Disorders and Stroke | 2022 to 2023 | $157,500 | April 1, 2022 | March 31, 2024 | DICKSON, PATRICIA I | reporter.nih.gov |
| Assessment of cervical spinal pathology in patients with MPS I, II and VI | NIH | National Institute of Neurological Disorders and Stroke | 2024 | $155,000 | July 1, 2024 | June 30, 2026 | LABOUNEK, RENE | reporter.nih.gov |
| Pulmonary endothelium targeted adenoviral gene therapy for the correction of mucopolysaccharidosis type I | NIH | National Heart, Lung, and Blood Institute | 2023 to 2024 | $49,618 | May 1, 2023 | September 30, 2024 | HURT, SARAH | reporter.nih.gov |
| Third Consensus Meeting on Neurocognitive and Functional Endpoints in MPS Clinical Studies | NIH | National Institute of Neurological Disorders and Stroke | 2025 | $15,000 | August 1, 2025 | July 31, 2026 | ADAMS, HEATHER | reporter.nih.gov |
Organizations
| Organization | Place | NIH awards | NIH amount | NSF awards | NSF amount |
|---|---|---|---|---|---|
| Children's Hosp of Philadelphia | Philadelphia, PA | 3 | $18.5 million | 0 | $0 |
| Phoenix Nest, Inc. | Brooklyn, NY | 4 | $10.9 million | 0 | $0 |
| Neurogt, Inc. | Chapel Hill, NC | 6 | $6.9 million | 0 | $0 |
| Nemours Children's Hospital, Delaware | Wilmington, DE | 1 | $2.9 million | 0 | $0 |
| Children's Hospital of Orange County | Orange, CA | 2 | $2.7 million | 0 | $0 |
| SUNY at Buffalo | Amherst, NY | 1 | $2.7 million | 0 | $0 |
| University of California, San Francisco | San Francisco, CA | 1 | $2.7 million | 0 | $0 |
| Research Triangle Institute | Research Triangle Park, NC | 1 | $2.6 million | 0 | $0 |
| Northwestern University | Chicago, IL | 1 | $2.5 million | 0 | $0 |
| Washington University | Saint Louis, MO | 4 | $2.2 million | 0 | $0 |
| University of Florida | Gainesville, FL | 2 | $2.1 million | 0 | $0 |
| Baebies, Inc. | Durham, NC | 2 | $2.1 million | 0 | $0 |
| Cincinnati Childrens Hosp Med CTR | Cincinnati, OH | 1 | $1.9 million | 0 | $0 |
| University of Minnesota | Minneapolis, MN | 2 | $1.7 million | 0 | $0 |
| Greenwood Genetic Center | Greenwood, SC | 1 | $1.2 million | 0 | $0 |
| Biostrategies, LC | State University, AR | 2 | $1.2 million | 0 | $0 |
| University of Pennsylvania | Philadelphia, PA | 2 | $1 million | 0 | $0 |
| Tega Therapeutics, Inc. | La Jolla, CA | 1 | $461,701 | 0 | $0 |
| Case Western Reserve University | Cleveland, OH | 1 | $453,033 | 0 | $0 |
| Recombinetics, Inc. | Eagan, MN | 1 | $384,999 | 0 | $0 |
| University of Cincinnati | Cincinnati, OH | 1 | $379,618 | 0 | $0 |
| Rainbio, Inc. | Raleigh, NC | 1 | $367,624 | 0 | $0 |
| Mandalmed, Inc. | San Francisco, CA | 1 | $306,872 | 0 | $0 |
| Kommodo Holdings Corp. | Shoreview, MN | 1 | $304,838 | 0 | $0 |
| Gelbchem, LLC | Seattle, WA | 1 | $272,872 | 0 | $0 |
| Stanford University | Stanford, CA | 1 | $250,236 | 0 | $0 |
| University of Georgia Research Foundation Inc | Athens, GA | 1 | $226,500 | 0 | $0 |
| University of Rochester | Rochester, NY | 1 | $15,000 | 0 | $0 |
Funding by fiscal year
| Fiscal year | NIH awards | NIH amount | NSF awards | NSF amount |
|---|---|---|---|---|
| 2025 | 20 | $18,506,186 | 0 | $0 |
| 2024 | 19 | $14,522,982 | 0 | $0 |
| 2023 | 22 | $17,567,967 | 0 | $0 |
| 2022 | 16 | $10,901,902 | 0 | $0 |
| 2021 | 13 | $7,886,904 | 0 | $0 |
Open federal funding opportunities
From Grants.gov: notices that funded awards in this table, and open notices on the same agency and assistance listing.
Updated November 3, 2026. Read from NIH RePORTER ExPORTER and NSF Award Search. Published by NIH and NSF as U.S. government data. NIH awards are fiscal years 2021 to 2025 (NIH files published to 2026-07-09); NSF awards are fiscal years 2022 to 2026 (NSF files published to 2026-11-02). NIH amounts are the total cost of each fiscal year in the window; NSF amounts are the funds obligated in the window. Organizations are matched on the UEI. Open opportunities come from Grants.gov and match on agency and assistance listing number, and on the notice number NIH prints on an award. Free to reuse with credit to SpecStream under CC BY 4.0. Spot a wrong value? Write to [email protected] with the link to this page.