Gene therapy — NIH Funding Overview
Reviewed by Dr. Meng ZhaoLast reviewed June 9, 2026Data refreshed June 9, 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 June 9, 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 $834M. The FY2025 total of $834M is +45% versus FY2021.
- The number of awards rose about 4% in FY2025.
- About 83% of FY2026 dollars so far are renewals and continuations. Mid-year snapshots overweight renewals because non-competing continuations are issued early in the fiscal year, but the share still indicates how much of the portfolio is committed before new applications compete.
- The average FY2025 award was $697K, 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 | $704,008,175 | $609,531 |
| FY2025 | 1,198 | $834,426,211 | $696,516 |
| FY2026 | 453 | $288,190,676 | $636,183 |
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 | 256 | $184,761,746 |
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.
- DUKE UNIVERSITY — 12 awards
- UNIVERSITY OF PENNSYLVANIA — 10 awards
- JOHNS HOPKINS UNIVERSITY — 7 awards
- MEDICAL COLLEGE OF WISCONSIN — 7 awards
- STANFORD UNIVERSITY — 7 awards
- UNIVERSITY OF MICHIGAN AT ANN ARBOR — 7 awards
- OREGON HEALTH & SCIENCE UNIVERSITY — 6 awards
- UNIVERSITY OF CALIFORNIA, SAN FRANCISCO — 6 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.
Defining Barriers to Gene Therapy
5R01EY018213-18Stephen Tsang · COLUMBIA UNIVERSITY HEALTH SCIENCES, NY · $405,751 · awarded Jun 5, 2026 · R01PROJECT SUMMARY Of the retinal degenerative diseases that affect 9 million Americans, rod and cone photoreceptor dystrophies are arguably the most devastating. Gene therapy is a potential means to strengthen photoreceptor viability. However, the first human gene therapy trial for retinal degeneration found improved visual function but did not slow…
Diversity and function of TRIO isoforms during human synapse development
1R21NS148969-01Marc Forrest · NORTHWESTERN UNIVERSITY, IL · $240,000 · awarded Jun 5, 2026 · R21ABSTRACT Alternative splicing vastly expands the functional repertoire of genes, and is especially important during human synapse development. Human synapses have unique structures and compositions, but the mechanisms involved in their development remains elusive. Investigations into human-specific aspects of synaptic machinery have revealed a critical role…
A link between lipid-mediated signaling and inflammation during neurodegeneration
5R01NS132760-04Mariana Pehar · UNIVERSITY OF WISCONSIN-MADISON, WI · $534,356 · awarded Jun 5, 2026 · R01Abstract Neuroinflammation, oxidative stress, and mitochondrial dysfunction have been associated with the pathophysiology of Alzheimer’s disease (AD), either as a primary cause or as a secondary component of the pathogenic process. AD, the most common cause of dementia in the elderly, is characterized by the accumulation of intracellular tau neurofibrillary…
AAV vector-mediated gene therapy for cystinuria type B
1R01DK146261-01A1Hiroyuki Nakai · OREGON HEALTH & SCIENCE UNIVERSITY, OR · $785,283 · awarded Jun 4, 2026 · R01PROJECT SUMMARY Urinary stone disease (urolithiasis) is a prevalent and global health issue, affecting approximately 9% of the U.S. population and over 10% in many developed countries. Due to lifestyle changes and global warming, the prevalence of urolithiasis has been increasing. Cystinuria, characterized by an abnormal elevation of cystine concentration…
Autologous Non-viral CRISPR/Cas Homology-directed Repair for Artemis-deficient Severe Combined Immunodeficiency
5K08AI187714-02Matthew Kan · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO, CA · $200,016 · awarded Jun 4, 2026 · K08PROJECT SUMMARY / ABSTRACT This application for the Mentored Clinical Scientist Research Career Development Award (K08), sponsored by the National Institute of Allergy and Infectious Diseases, describes the five-year career development plan of Dr. Matthew Kan, a pediatric immunologist and early career physician-scientist in the Division of Pediatric…
Therapeutic Strategy to Treat Alzheimer's Disease by VGF Delivery into Brain
5R01AG083981-04Jagdish Singh · NORTH DAKOTA STATE UNIVERSITY, ND · $657,502 · awarded Jun 3, 2026 · R01SUMMARY/ABSTRACT: Alzheimer’s disease (AD) is a progressive neurodegenerative disease that has emerged as the most prevalent form of late-life dementia in humans, in which the formation and accumulation of hyperphosphorylated tau protein and amyloid-β (Aβ) are believed to play key roles in AD pathogenesis. Of note, the recent multiscale causal network…
Development AMHR2 agonists as ovarian modulators and contraceptives
1R01HD120555-01David Pepin · MASSACHUSETTS GENERAL HOSPITAL, MA · $730,972 · awarded Jun 3, 2026 · R01Project Summary/Abstract: Current hormonal contraceptives work by disrupting the hypothalamic-pituitary-gonadal axis and preventing follicle development only in its last stage leading to ovulation. All current hormonal contraceptives share unwanted side effects due to the wide tissue distribution of sex steroid receptors. In contrast, anti-Müllerian hormone…
Neuron-specific modulation of gene expression using systemically administered bispecific antibody-ASO conjugates
5R01NS138455-03Greg Thurber · UNIVERSITY OF MICHIGAN AT ANN ARBOR, MI · $599,383 · awarded Jun 3, 2026 · R01The ability to selectively modulate the expression of one or more neuronal genes is critical for defining pathways that govern normal brain physiology and diverse disease phenotypes. Anti-sense oligonucleotides (ASOs) are attractive agents for manipulating neuronal gene expression, especially given their clear advantages relative to gene therapy, including…
eCD4-Mediated Control of SIV Infection in the Brain
5R01NS131098-04Michael Farzan · BOSTON CHILDREN'S HOSPITAL, MA · $848,912 · awarded Jun 3, 2026 · R01PROJECT SUMMARY Combined antiretroviral therapy (ART) has revolutionized the treatment of HIV but ART regimens are not without drawbacks. Cost, the need for daily administration, side effects, and social stigma all contribute to reduced patient compliance. Moreover, despite treatment, some 15-55% of people living with HIV will develop some form of…
Advancing Spatial Metallomics/Metabolomics, Genome Editing, and Biocatalysis by Designing and Selecting DNAzymes, Aptamers, and Biosynthetic Models
2R35GM141931-07Yi Lu · UNIVERSITY OF TEXAS AT AUSTIN, TX · $682,295 · awarded Jun 3, 2026 · R35Project summary/Abstract The overall goal is to develop novel tools to advance spatial metallomics, spatial metabolomics, genome editing, and biocatalysis by designing and selecting DNAzymes, DNA aptamers, and biosynthetic models of heteronuclear metalloenzymes involved in multi-electron and multi-proton processes. In the first project, we plan to develop…
Transposable Element (TE) RNA regulation via small RNA pathways in aging cells and neurodegeneration.
5R01AG078930-05NELSON LAU · BOSTON UNIVERSITY MEDICAL CAMPUS, MA · $683,102 · awarded Jun 3, 2026 · R01Project Summary/Abstract Transposable elements (TEs) are prolific genetic parasites infiltrating >45% of the human genome and are major proportions of all animal genomes. TE activation during aging and disease affects the transcriptomes of neurons and alter animal activity. This hypothesis is attractive because all animal genomes harbor a major reservoir of…
Development of peptoid-based barcodes for in vivo high-throughput screening of targeted nucleic acid delivery
5R21EB036261-03Jayoung Kim · UNIVERSITY OF NORTH TEXAS HLTH SCI CTR, TX · $185,000 · awarded Jun 3, 2026 · R21PROJECT SUMMARY / ABSTRACT The proposed project aims to deliver a research tool that supports accurate and resource- efficient biodistribution studies involving a library of gene-encapsulated nanoparticles. The state-of-the-art technology to perform in vivo high-throughput screening of non-viral gene delivery vectors is DNA/RNA barcoding, where…
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