Gene therapy — NIH Funding Overview
Reviewed by Dr. Meng ZhaoLast reviewed June 9, 2026Data refreshed September 5, 2026Editorial standards
Gene therapy is a fast-growing NIH funding category covering AAV-based delivery, lentiviral and ex vivo approaches, CRISPR-based corrections, and emerging RNA-based therapies. Multiple ICs fund disease-specific applications, with NHGRI and NHLBI leading platform development.
Funding snapshot
Award data on this page reflects a snapshot of NIH RePORTER records last refreshed on September 5, 2026. For live numbers, use the interactive trends view.
Why this matters now
Multiple FDA approvals (Zolgensma, Hemgenix, Casgevy, Elevidys, others) have validated the field, but each new therapy reveals long-tail safety, manufacturing, and access challenges that drive NIH portfolio reorganization toward delivery, immunogenicity, and durability research.
How NIH funds this area
Mechanisms include R01, U01, U19, P01, and the Bridge to Artificial Intelligence (Bridge2AI) and Somatic Cell Genome Editing programs. Data below covers all NIH awards mentioning gene therapy in title, abstract, or terms.
How to use this funding brief
Use this page to decide whether vector design, delivery, editing, manufacturing, safety, or a disease application is the real funding question. Search the platform and disease terms independently before using recent award counts as evidence of field momentum.
Official source: NHGRI: How genome editing works
Search tactics
- Search "AAV" or "lentiviral" for vector-specific subsets.
- For ex vivo cell therapies, search "CAR-T" or "cell therapy".
- NHGRI and NHLBI lead platform-level grants; disease-specific work flows to disease ICs.
What the data shows
- Funding peaked in FY2025 at $833M. The FY2025 total of $833M is +45% versus FY2021.
- The number of awards rose about 4% in FY2025.
- About 80% of FY2026 dollars so far are non-Type-1 award actions, including competing renewals, supplements, non-competing continuations, and institutional changes. This is a descriptive record mix, not a measure of renewal activity alone.
- The average FY2025 award was $696K, and R01 was the most common mechanism in the recent window.
Editorial read
Gene-therapy funding grew roughly 45% from FY2021 to a record FY2025 — among the fastest growth in our tracked set — and the money is following delivery vehicles, manufacturing, and clinical translation rather than proof-of-concept constructs. The portfolio also shows one of the lower renewal shares in the current-year snapshot, a sign that new projects are still being added at a healthy clip rather than the base simply rolling over.
Yearly NIH Awards for Gene therapy
Counts and total funding per fiscal year from NIH RePORTER. Recent fiscal years may understate final totals because of reporting lag.
| Fiscal Year | Project Count | Total Funding | Avg Award |
|---|---|---|---|
| FY2021 | 1,030 | $575,499,072 | $558,737 |
| FY2022 | 1,043 | $648,268,274 | $621,542 |
| FY2023 | 1,082 | $711,722,540 | $657,784 |
| FY2024 | 1,155 | $704,029,165 | $609,549 |
| FY2025 | 1,197 | $832,937,237 | $695,854 |
| FY2026 | 848 | $587,218,752 | $692,475 |
Top NIH Institutes (last 90 days)
Which NIH institutes funded the most Gene therapy projects in the most recent 90-day window.
| Institute | Awards (90d) | Funding (90d) |
|---|---|---|
| NIH | 370 | $284,934,653 |
| FDA | 1 | $812,248 |
| VA | 7 | $0 |
Common Activity Codes (last 90 days)
Which grant mechanisms (R01, R21, U01, P30, etc.) appeared most often for Gene therapy in the recent period.
Most Active Institutions (last 90 days)
Universities and research organizations with the most Gene therapy awards in the most recent 90-day window.
- BOSTON CHILDREN'S HOSPITAL — 16 awards
- MASSACHUSETTS EYE AND EAR INFIRMARY — 10 awards
- CHILDREN'S HOSP OF PHILADELPHIA — 10 awards
- UNIVERSITY OF CALIFORNIA, SAN DIEGO — 8 awards
- MASSACHUSETTS GENERAL HOSPITAL — 8 awards
- UNIVERSITY OF FLORIDA — 8 awards
- UNIV OF MASSACHUSETTS MED SCH WORCESTER — 8 awards
- CINCINNATI CHILDRENS HOSP MED CTR — 7 awards
Recently Awarded Gene therapy Grants
Twelve most recent awards mentioning Gene therapy, drawn from NIH RePORTER. Click through to Find PIs for the full investigator search.
Development of CRISPR/Cas9-based exon-skipping strategies for the treatment of USH-associated deafness
5R01DC019404-04SUSAN BLANTON · UNIVERSITY OF MIAMI SCHOOL OF MEDICINE, FL · $666,228 · awarded Aug 28, 2026 · R01Abstract: Usher syndrome (USH) is the most common form of inherited deaf-blindness, with a prevalence of 1/6.000. Inherited as an autosomal recessive trait, it affects about 15,000 people in the United States and is responsible for 6% of early childhood deafness. Usher syndrome is classified under three clinical subtypes (USH-1, -2 and -3) according to the…
Gene Therapy for Alpha 1-Antitrypsin Deficiency
5R33HL169190-03RONALD CRYSTAL · WEILL MEDICAL COLL OF CORNELL UNIV, NY · $2,451,275 · awarded Aug 28, 2026 · R33Abstract. Alpha 1-antitrypsin (AAT) deficiency, an autosomal recessive disorder, manifests in lung as early onset panacinar emphysema. AAT functions to inhibit neutrophil elastase (NE) and other neutrophil serine proteases. AAT is produced predominantly in liver and diffuses into the lung from the circulation. Low AAT levels in AAT deficiency are associated…
Gene Therapy for LCA5-Assocated Inherited Retinal Degeneration: Extension to Pediatric Cohorts
5R01FD008174-03Tomas Aleman · UNIVERSITY OF PENNSYLVANIA, PA · $812,248 · awarded Aug 28, 2026 · R01PROJECT SUMMARY / ABSTRACT Advances in genetics and genomics have raised hopes for the development of gene-based treatments for inherited retinal diseases (IRDs) which are single-gene defects that cause vision loss. Some of the greatest successes for gene-based treatments has occurred in the most severe subset of IRDs that fall into the rubric of Leber…
Fundamentals of Anti-Sickling Therapies
5R01HL179440-02FRANK FERRONE · DREXEL UNIVERSITY, PA · $482,463 · awarded Aug 27, 2026 · R01Sickle cell disease affects 100,000 Americans and millions world-wide: a mutation in the β-globin gene gives rise to a hemoglobin variant (sickle hemoglobin, or HbS) that is prone to self-assembly. The resulting protein fibers distort red blood cells and cause the disease’s pathology. Two gene therapies have recently been approved for treatment of this…
Inflammatory Biosignatures for the Identification of Sickle Cell Associated Pain Phenotypes (IBIS-CAP)
1R01HL183340-01A1Susanna Curtis · ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI, NY · $834,899 · awarded Aug 27, 2026 · R01Project Summary: SCD is characterized by a globin mutation that leads to hemolysis, vaso-occlusion, progressive organ injury, pain and death. Despite recent advances chronic pain remains a major cause of suffering and morbidity, with poorly understood biology and no targeted treatments. Previous attempts to generate biomarkers for chronic SCD pain failed,…
Cincinnati Cooperative Center of Excellence in Hematology
5U54DK126108-06YI ZHENG · CINCINNATI CHILDRENS HOSP MED CTR, OH · $700,000 · awarded Aug 27, 2026 · U54SUMMARY/Abstract: The long-term goal of the Cincinnati Cooperative Center of Excellence in Hematology (CCCEH) is to understand and correct, at the molecular level, hematological diseases of various lineages. To attain this goal, we must understand basic biological processes that affect behaviors of hematopoietic stem cells and their various differentiated…
Clinical and mechanistic studies defining optimal preparative approaches to infants with IL2RG/JAK3/RAG1/RAG2 SCID: a randomized trial of busulfan dosage
3U01AI184132-03S1JEFFERY AULETTA · NATIONAL MARROW DONOR PROGRAM, MN · $111,904 · awarded Aug 27, 2026 · U01Project Summary Severe combined immunodeficiency (SCID) is a group of genetic disorders that abrogate T cell development and function. Allogeneic hematopoietic cell transplantation (HCT) is the standard treatment for the disease and can be performed successfully in SCID patients without the high dose pre-HCT busulfan conditioning due to the unique capacity…
AAV gene therapy to restore fertility in mammalian Sertoli cell dysfunction models
6F31HD114406-04Georgia Rae Atkins · UNIVERSITY OF PITTSBURGH AT PITTSBURGH, PA · $50,114 · awarded Aug 27, 2026 · F31ABSTRACT: Infertility impacts 48 million couples worldwide and 50% of cases are due to a male factor. Non-obstructive azoospermia (NOA) is the absence of sperm due to spermatogenic failure. Some cases are due to known conditions (Klinefelter’s Syndrome, Y chromosome microdeletions, and hypogonadism) but many cases are idiopathic (unexplained). Recent…
PhD Training Program in Virology and Gene Therapy
5T32AI132165-09ROBERTO CATTANEO · MAYO CLINIC ROCHESTER, MN · $142,661 · awarded Aug 27, 2026 · T32Project Summary Unifying scientific principles in the life sciences continue to be discovered in viral systems. Gene therapy and oncolytic virotherapy depend on viral vectors. Awareness that a deep understanding of fundamental virology is necessary for the development of gene therapy led to the foundation of the Virology and Gene Therapy (VGT) track of Mayo…
Examining the impact of cobalamin C deficiency in human brain development
1K08NS146682-01A1Yue Huang · UNIVERSITY OF CALIFORNIA, SAN DIEGO, CA · $236,115 · awarded Aug 27, 2026 · K08PROJECT SUMMARY/ABSTRACT Inborn errors of metabolism (IEMs) are a group of genetic disorders resulting from defects in cellular metabolism. While individually rare, IEMs collectively affect approximately 1 in 1000 newborns, with 85% of them exhibiting neurodevelopmental symptoms. Cobalamin C deficiency, one of the most common IEMs, interrupts the…
Complement Factor H-based Therapeutic Strategies in Macular Degeneration
5I01RX000444-16Baerbel Rohrer · RALPH H JOHNSON VA MEDICAL CENTER, SC · awarded Aug 27, 2026 · I01Age-related macular degeneration (AMD) is a slowly progressing disease involving genetic abnormalities and environmental insults. It is the leading cause of blindness for older Americans; and as the population ages, the prevalence of AMD continues to grow. Since smoking increases AMD risk and there is a higher incidence of smoking in veterans,…
The Cnidarian Nematocyte as a Model of Vertebrate Hair Cells: Molecular targeting of the METC
1R21DC023990-01Eduardo Perozo · UNIVERSITY OF CHICAGO, IL · $451,000 · awarded Aug 26, 2026 · R21Abstract The mechanoelectrical transduction complex (METC) in the stereocilia of hair cells is responsible for converting sound waves into electrical signals that the brain can decode. This intricate system allows organisms to perceive mechanical stimuli such as sound, linear and angular acceleration. However, uncovering its structural dynamics and…
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