Beta Thalassemia — NIH Grants & Funding

MONDO:0019402

Funding Landscape

Beta Thalassemia has active NIH research funding with 20 funded grants totaling $25.3M.

20funded grants from NIH

Portfolio: $8.7MCumulative: $25.3M10 active10 completed27 PIs18 institutions

-38% YoY(FY2024 vs FY2025)

1 new grant started in the last 12 months.

By Research Theme

Basic Science

Clinical

Gene/Cell Therapy

Translational

Funding by Fiscal Year

NIH fiscal year (Oct–Sep)

$7.8M

FY22

$7.2M

FY23

$6.2M

FY24

$3.8M

FY25

$158K

FY26

By Funding Mechanism

R01

F30

F31

R56

K01

R21

Basic Science(13)

Basic ScienceZIACompleted

Repurposing an EU Therapeutic for Hemoglobinopathies

Haugabook, Sharie·NATIONAL CENTER FOR ADVANCING TRANSLATIONAL SCIENCES

$757,464 (FY2025)

NIH RePORTER

Basic ScienceR01Active

ULK-mediated autophagy of α-globin in ß-thalassemia

Weiss, Mitchell·ST. JUDE CHILDREN'S RESEARCH HOSPITAL·MEMPHIS, TN

$652,585 (FY2025)

NIH RePORTER

Basic ScienceR01Active

Skeletal heterogeneity of bone marrow stress responses.

Lucas, Daniel·CINCINNATI CHILDRENS HOSP MED CTR·CINCINNATI, OH

$503,478 (FY2025)

NIH RePORTER

Basic ScienceR01Active

MECHANISM OF ACTION OF ERYTHROFERRONE AND PATHOLOGICAL IMPLICATIONS

GANZ, TOMAS·UNIVERSITY OF CALIFORNIA LOS ANGELES·LOS ANGELES, CA

$458,477 (FY2024)

NIH RePORTER

Basic ScienceR01Active

Investigating PUM1 mediated post-transcriptional regulation of human hemoglobin switching and erythropoiesis

Gnanapragasam, Merlin·CLEVELAND STATE UNIVERSITY·CLEVELAND, OH

$401,908 (FY2025)

NIH RePORTER

Basic ScienceR56Active

ZIP Proteins and Iron Metabolism

Knutson, Mitchell·UNIVERSITY OF FLORIDA·GAINESVILLE, FL

$225,000 (FY2024)

NIH RePORTER

Basic ScienceK01Active

Defining the mechanisms of hemoglobin switching and genotoxicities associated with its manipulation

Doerfler, Phillip·VERSITI BLOOD HEALTH, INC.·MILWAUKEE, WI

$158,220 (FY2026)

NIH RePORTER

Basic ScienceF30Active

Investigating the mechanisms of polycomb group protein mediated fetal hemoglobin silencing

Kaminski, Paul·UNIVERSITY OF PENNSYLVANIA·PHILADELPHIA, PA

$54,538 (FY2025)

NIH RePORTER

Basic ScienceF30Active

Development of novel fetal hemoglobin inducers using targeted protein degradation

Zheng, Sijin·YALE UNIVERSITY·NEW HAVEN, CT

$54,242 (FY2025)

NIH RePORTER

Basic ScienceR01Completed

The Role of Erythroferrone in Regulating Bone Metabolism in Beta-Thalassemia

Ginzburg, Yelena et al.·ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI·NEW YORK, NY

$570,650 (FY2025)

NIH RePORTER

Translational(1)

TranslationalR33Completed

Development of a Clinical Hemoglobin Modulator

Faller, Douglas et al.·PHOENICIA BIOSCIENCES, INC.·WESTON, MA

$1,630,002 (FY2022)

NIH RePORTER

Clinical(3)

ClinicalR01Active

Pathological consequences of iron excess and hypoxia in β-thalassemia pregnancy

Collins, James et al.·UNIVERSITY OF FLORIDA·GAINESVILLE, FL

$657,000 (FY2025)

NIH RePORTER

ClinicalR01Completed

QSM to Guide Iron Chelating Therapy in Transfusional Iron Overload

Brittenham, Gary et al.·WEILL MEDICAL COLL OF CORNELL UNIV·NEW YORK, NY

$437,946 (FY2023)

NIH RePORTER

ClinicalR56Completed

Iron Pathobiology in β-thalassemia Pregnancy

Collins, James et al.·UNIVERSITY OF FLORIDA·GAINESVILLE, FL

$100,000 (FY2023)

NIH RePORTER

Gene/Cell Therapy(3)

Gene/Cell TherapyR01Completed

Multiplex base/prime editors for in vivo selection of modified HSPCs in hemoglobinopathy

KIEM, HANS-PETER·FRED HUTCHINSON CANCER CENTER·SEATTLE, WA

$728,713 (FY2024)

NIH RePORTER

Gene/Cell TherapyR01Completed

In Vivo Hematopoietic Stem Cell Gene Therapy of Beta-Thalassemia and Sickle Cell Disease

LIEBER, ANDRE·UNIVERSITY OF WASHINGTON·SEATTLE, WA

$644,784 (FY2024)

NIH RePORTER

Gene/Cell TherapyR21Completed

Precise in vivo gene editing of HSPC for the treatment of genetic hematologic diseases

Tong, Sheng·UNIVERSITY OF KENTUCKY·LEXINGTON, KY

$191,250 (FY2024)

NIH RePORTER

Adjacent Research(25 grants)

These grants mention Beta Thalassemia as part of broader research but are not primarily focused on this disease. They are not included in the funding totals above.

Other(25)

OtherZIACompleted

Nonmyeloablative haploidentical peripheral blood stem cell transplantation in congenital anemias

Fitzhugh, Courtney·NATIONAL HEART, LUNG, AND BLOOD INSTITUTE

$3,067,391 (FY2025)

NIH RePORTER

OtherR01Active

The Role of the MBD2-NuRD Complex in Gamma-Globin Gene Silencing

GINDER, GORDON et al.·VIRGINIA COMMONWEALTH UNIVERSITY·RICHMOND, VA

$2,965,140 (FY2025)

NIH RePORTER

OtherZIACompleted

Preclinical and Clinical Studies in Gene Therapy for Sickle Cell Disease

Tisdale, John·NATIONAL HEART, LUNG, AND BLOOD INSTITUTE

$1,457,612 (FY2025)

NIH RePORTER

OtherDP2Active

Generalizing genome editing for rare disease-causing variants

Newby, Gregory·JOHNS HOPKINS UNIVERSITY·BALTIMORE, MD

$1,416,375 (FY2025)

NIH RePORTER

OtherR44Active

Efficacy of Hypoxic Red Blood Cells Processed with the Hemanext ONE® System in Patients with Sickle Cell Anemia

Andemariam, Biree et al.·NEW HEALTH SCIENCES, INC.·Lexington, MA

$1,017,232 (FY2025)

NIH RePORTER

OtherR01Active

Chemotherapy-free cure of hemoglobin disorders through base editing

Bauer, Daniel et al.·BOSTON CHILDREN'S HOSPITAL·BOSTON, MA

$766,048 (FY2025)

NIH RePORTER

OtherR01Active

Targeting ZNF410 for HbF reactivation

Bauer, Daniel·BOSTON CHILDREN'S HOSPITAL·BOSTON, MA

$672,403 (FY2025)

NIH RePORTER

OtherR01Active

Regulation of Hemoglobin Switching

Blobel, Gerd·CHILDREN'S HOSP OF PHILADELPHIA·PHILADELPHIA, PA

$649,089 (FY2026)

NIH RePORTER

OtherR01Active

Systematic Genetic Dissection of Human Erythropoiesis

Sankaran, Vijay·BOSTON CHILDREN'S HOSPITAL·BOSTON, MA

$645,733 (FY2025)

NIH RePORTER

OtherR01Active

Ferroptosis in the Heart: Iron Calcium Crosstalk and Compartmentalization

Gwathmey, Judith et al.·RUTGERS BIOMEDICAL AND HEALTH SCIENCES·Newark, NJ

$589,278 (FY2025)

NIH RePORTER

Top Funded Institutions

PHOENICIA BIOSCIENCES, INC.$1.6M

MA1 grantFaller, Douglas, Kuo, Kevin +2

UNIVERSITY OF FLORIDA$982K

FL3 grantsCollins, James, Nemeth, Elizabeta +1

NATIONAL CENTER FOR ADVANCING TRANSLATIONAL SCIENCES$757K

1 grantHaugabook, Sharie

FRED HUTCHINSON CANCER CENTER$729K

WA1 grantKIEM, HANS-PETER

ST. JUDE CHILDREN'S RESEARCH HOSPITAL$653K

TN1 grantWeiss, Mitchell

UNIVERSITY OF WASHINGTON$645K

WA1 grantLIEBER, ANDRE

ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI$571K

NY1 grantGinzburg, Yelena, Yuen, Tony +1

CINCINNATI CHILDRENS HOSP MED CTR$503K

OH1 grantLucas, Daniel

UNIVERSITY OF CALIFORNIA LOS ANGELES$458K

CA1 grantGANZ, TOMAS

BOSTON CHILDREN'S HOSPITAL$443K

MA1 grantORKIN, STUART

Top Principal Investigators

Faller, Douglas$1.6M

PHOENICIA BIOSCIENCES, INC.·1 grant

Translational

Kuo, Kevin$1.6M

PHOENICIA BIOSCIENCES, INC.·1 grant

Translational

PERRINE, SUSAN$1.6M

PHOENICIA BIOSCIENCES, INC.·1 grant

Translational

SINGER, SYLVIA$1.6M

PHOENICIA BIOSCIENCES, INC.·1 grant

Translational

Haugabook, Sharie$757K

NATIONAL CENTER FOR ADVANCING TRANSLATIONAL SCIENCES·1 grant

Basic Science

Collins, James$757K

UNIVERSITY OF FLORIDA·2 grants

Clinical

Nemeth, Elizabeta$757K

UNIVERSITY OF FLORIDA·2 grants

Clinical

KIEM, HANS-PETER$729K

FRED HUTCHINSON CANCER CENTER·1 grant

Gene/Cell Therapy

Weiss, Mitchell$653K

ST. JUDE CHILDREN'S RESEARCH HOSPITAL·1 grant

Basic Science

LIEBER, ANDRE$645K

UNIVERSITY OF WASHINGTON·1 grant

Gene/Cell Therapy

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