	US 12,290,532 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, Montgomery Village, MD (US); Braden Carl Ishler, Montgomery Village, MD (US); and Daniel Allen Sheik, West Lafayette, IN (US)
Assigned to Cellphire, Inc., Rockville, MD (US)
Filed by Cellphire, Inc., Rockville, MD (US)
Filed on Jan. 5, 2024, as Appl. No. 18/406,008.
Application 18/406,008 is a continuation of application No. 17/166,490, filed on Feb. 3, 2021, granted, now 11,903,971.
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 2024/0139252 A1, May 2, 2024
This patent is subject to a terminal disclaimer.
Int. Cl. A61K 35/19 (2015.01); A61K 9/19 (2006.01); A61K 9/50 (2006.01); A61K 31/195 (2006.01); A61K 31/197 (2006.01); A61K 38/36 (2006.01); A61K 38/57 (2006.01); A61P 7/04 (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:

rehydrating freeze-dried platelet derivatives in a freeze-dried platelet derivative composition to form a rehydrated platelet derivative composition comprising rehydrated platelet derivatives, one or more salts, a buffer, and a saccharide,

administering a therapeutically effective amount of the rehydrated platelet derivatives intravenously to the subject in need thereof,

wherein the plasma of the subject is deficient in von Willebrand factor, wherein the therapeutically effective amount of the rehydrated platelet derivatives is an amount sufficient to form clots in plasma of the subject,

wherein the therapeutically effective amount of the rehydrated platelet derivatives is at least 8.5×10⁸particles/kg of the subject,

wherein the surface expression of CD42b on the rehydrated platelet derivatives is between 25% and 50% of the surface expression of CD42b on normal platelets,

wherein 50% to 99% of the rehydrated platelet derivatives are in the diameter range of 0.3 μm to 5 μm,

wherein the rehydrated platelet derivatives have less than 10% crosslinking of platelet membranes via proteins and/or lipids present on the membranes,

wherein the rehydrated platelet derivatives have the property of forming clots in vitro in von Willebrand factor deficient plasma, and

wherein the rehydrated platelet derivatives have a reduced aggregation characteristic such that the rehydrated platelet derivatives exhibit less aggregation as compared to normal platelets under in vitro aggregation conditions consisting of buffered saline, citrated plasma and ristocetin, such that less than or equal to 2% aggregation is achieved for a sample from the rehydrated platelet derivative composition under the in vitro aggregation conditions.