	US 11,707,481 B2
	Compositions and methods of treating Fanconi Anemia
Alan D'Andrea, Winchester, MA (US); Kalindi Parmar, Arlington, MA (US); and Haojian Zhang, Shrewsbury, MA (US)
Assigned to Dana-Farber Cancer Institute, Inc., Boston, MA (US)
Appl. No. 15/553,905
Filed by DANA-FARBER CANCER INSTITUTE, INC., Boston, MA (US)
PCT Filed Feb. 26, 2016, PCT No. PCT/US2016/019625 § 371(c)(1), (2) Date Aug. 25, 2017, PCT Pub. No. WO2016/138300, PCT Pub. Date Sep. 1, 2016.
Claims priority of provisional application 62/120,593, filed on Feb. 25, 2015.
Prior Publication US 2018/0071330 A1, Mar. 15, 2018
This patent is subject to a terminal disclaimer.
Int. Cl. A61K 31/7088 (2006.01); A61K 45/06 (2006.01); C12N 15/113 (2010.01)

CPC A61K 31/7088 (2013.01) [A61K 45/06 (2013.01); C12N 15/1135 (2013.01); C12N 2310/10 (2013.01); C12N 2310/14 (2013.01); C12N 2310/20 (2017.05); C12N 2310/531 (2013.01)]

10 Claims

1. A method of treating or delaying the onset of bone marrow failure in a patient having Fanconi Anemia (FA) comprising administering to the patient a therapeutically effective amount of a compound that inhibits the expression or activity of transforming growth factor beta (TGFβ), wherein the compound is 1) an antibody that is specific for TGFβ or TGFβR1 or 2) a nucleic acid that is a short hairpin RNA (shRNA), a small interfering RNA (siRNA), or a single guide RNA (sgRNA) specific for SMAD3.