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Targeting PGC-1a for the treatment of sickle cell disease

Award Number: R01HL155451
ORGANIZATION: NATIONAL HEART, LUNG, & BLOOD INSTITUTE
OPDIV: NIH
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)
PERIOD OF PERFORMANCE START DATE: 07/01/2022
PERIOD OF PERFORMANCE END DATE: 05/31/2026

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Targeting PGC-1a for the treatment of sickle cell disease - ABSTRACT Sickle cell disease (SCD) and ?-thalassemia are genetic disorders affecting the structure or production of hemoglobin. They are growing global health burden afflicting millions around the world. Sufficient fetal hemoglobin (HbF) induction can reduce morbidity and mortality in SCD. The same is true for β-thalassemia. The standard current therapy for HbF induction in SCD hydroxyurea (HU) which in adults, does not usually ameliorate sufficiently either sickle vaso-occlusion or hemolysis. Therefore, the search for more effective and safer small molecule HbF inducers is a key unmet need in the management of SCD. We recently discovered that the peroxisome proliferator activated receptor gamma coactivator-1 (PGC-1α) is involved in the regulation of the globin genes. Upregulation of PGC-1α by lentivirus infection or by a small molecule compound ZLN005 in human primary erythroid progenitor CD34+ cells induced HbF expression and increased the percent of F-cells without affecting cell proliferation and differentiation. Activation of PGC-1α by ZLN005 might provide a new path into hemoglobin regulation with a promising therapeutic benefit in SCD. We intend to test for in vivo therapeutic effects of ZLN005 in sickle cell mice. The findings from these preclinical studies could provide initial proof of principle for developing safer, more beneficial small molecule therapeutics for SCD treatment and lead to early- phase clinical trials.


Issue Date FY	Funding FY	Legal Entity Name	Legal Entity Address	Legal Entity City	Legal Entity State	Legal Entity Zip Code	Legal Entity COUNTY	Legal Entity COUNTRY	Assistance Listing	Award Code	Budget Year	Action Date	Action Type	Action Amount

Issue Date FY: 2025 ( Subtotal = $0 )
2025	2024	BOSTON MEDICAL CENTER CORPORATION	1 BOSTON MEDICAL CTR PL	BOSTON	MA	02118	SUFFOLK	USA	Blood Diseases and Resources Research	000	3	5/22/2025	NON-COMPETING CONTINUATION	$0
														Subtotal = $0

Issue Date FY: 2024 ( Subtotal = $350,000 )
2024	2024	BOSTON MEDICAL CENTER CORPORATION	1 BOSTON MEDICAL CTR PL	BOSTON	MA	02118	SUFFOLK	USA	Blood Diseases and Resources Research	000	3	5/10/2024	NON-COMPETING CONTINUATION	$350,000
														Subtotal = $350,000

Issue Date FY: 2023 ( Subtotal = $350,000 )
2023	2023	BOSTON MEDICAL CENTER CORPORATION	1 BOSTON MEDICAL CTR PL STE 1	BOSTON	MA	02118	SUFFOLK	USA	Blood Diseases and Resources Research	000	2	5/5/2023	NON-COMPETING CONTINUATION	$350,000
														Subtotal = $350,000

Issue Date FY: 2022 ( Subtotal = $350,000 )
2022	2022	BOSTON MEDICAL CENTER CORPORATION	1 BOSTON MEDICAL CTR PL STE 1	BOSTON	MA	02118	SUFFOLK	USA	Blood Diseases and Resources Research	000	1	6/8/2022	NEW	$350,000
														Subtotal = $350,000

Grand Total All Awards = $1,050,000

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Targeting PGC-1a for the treatment of sickle cell disease

Award Number: R01HL155451

ORGANIZATION: NATIONAL HEART, LUNG, & BLOOD INSTITUTE

OPDIV: NIH

AWARD CLASS: DISCRETIONARY

AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)

PERIOD OF PERFORMANCE START DATE: 07/01/2022

PERIOD OF PERFORMANCE END DATE: 05/31/2026

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