US 11,903,971 B2
Treatment of von Willebrand disease
Keith Andrew Moskowitz, Westfield, IN (US); Shan Xu, Rockville, MD (US); William Matthew Dickerson, Washington, DC (US); Amber Nicole Lee, Rockville, MD (US); Braden Carl Ishler, Gaithersburg, MD (US); and Daniel Allen Sheik, Rockville, MD (US)
Assigned to Cellphire, Inc., Rockville, MD (US)
Filed by Cellphire, Inc., Rockville, MD (US)
Filed on Feb. 3, 2021, as Appl. No. 17/166,490.
Claims priority of provisional application 63/065,337, filed on Aug. 13, 2020.
Claims priority of provisional application 62/980,850, filed on Feb. 24, 2020.
Claims priority of provisional application 62/969,942, filed on Feb. 4, 2020.
Prior Publication US 2021/0315935 A1, Oct. 14, 2021
Int. Cl. A61K 35/19 (2015.01); A61K 9/19 (2006.01); A61P 7/04 (2006.01); A61K 31/195 (2006.01); A61K 31/197 (2006.01); A61K 38/36 (2006.01); A61K 38/57 (2006.01); A61K 9/50 (2006.01); C12N 5/078 (2010.01)
CPC A61K 35/19 (2013.01) [A61K 9/19 (2013.01); A61K 9/5068 (2013.01); A61K 31/195 (2013.01); A61K 31/197 (2013.01); A61K 38/363 (2013.01); A61K 38/57 (2013.01); A61P 7/04 (2018.01); C12N 5/0644 (2013.01)] 20 Claims
 
1. A method of treating von Willebrand disease in a subject, the method comprising:
administering a therapeutically effective amount of freeze-dried platelet derivatives intravenously to the subject in need thereof, wherein the plasma of the subject is deficient in von Willebrand factor, and the therapeutically effective amount of freeze-dried platelet derivatives is an amount sufficient to form clots in plasma of the subject, wherein the therapeutically effective amount of freeze-dried platelet derivatives is at least 8.5×108 particles/kg of the subject, and wherein the surface expression of CD42b on the freeze-dried platelet derivatives is between 25% and 50% of the surface expression of CD42b on normal platelets, and wherein the freeze-dried platelet derivatives are loaded with an anti-fibrinolytic agent selected from ε-aminocaproic acid, aprotinin, aminomethylbenzoic acid, tranexamic acid, and fibrinogen.