	US 11,865,148 B2
	Methods of delivering transgenes to the eye
Suma Krishnan, Pittsburgh, PA (US)
Assigned to Krystal Biotech, Inc., Pittsburgh, PA (US)
Filed by Krystal Biotech, Inc., Pittsburgh, PA (US)
Filed on Nov. 30, 2022, as Appl. No. 18/060,515.
Application 18/060,515 is a continuation of application No. 17/529,161, filed on Nov. 17, 2021, abandoned.
Application 17/529,161 is a continuation of application No. 16/598,982, filed on Oct. 10, 2019, granted, now 11,185,564, issued on Nov. 30, 2021.
Application 16/598,982 is a continuation of application No. 16/177,153, filed on Oct. 31, 2018, granted, now 10,441,614, issued on Oct. 15, 2019.
Application 16/177,153 is a continuation of application No. 15/851,488, filed on Dec. 21, 2017, granted, now 10,155,016, issued on Dec. 18, 2018.
Application 15/851,488 is a continuation of application No. 15/393,151, filed on Dec. 28, 2016, granted, now 9,877,990, issued on Jan. 30, 2018.
Claims priority of provisional application 62/320,316, filed on Apr. 8, 2016.
Prior Publication US 2023/0149486 A1, May 18, 2023
This patent is subject to a terminal disclaimer.
Int. Cl. A61K 35/763 (2015.01); A61K 9/00 (2006.01); A61K 38/17 (2006.01); A61K 38/39 (2006.01); A61K 48/00 (2006.01); C07K 14/78 (2006.01); C12N 9/02 (2006.01); A61K 9/06 (2006.01); A61K 47/38 (2006.01)

CPC A61K 35/763 (2013.01) [A61K 9/0014 (2013.01); A61K 38/1748 (2013.01); A61K 38/39 (2013.01); A61K 48/005 (2013.01); C07K 14/78 (2013.01); C12N 9/0071 (2013.01); C12Y 114/11004 (2013.01); A61K 9/06 (2013.01); A61K 47/38 (2013.01); C12N 2710/16643 (2013.01)]

21 Claims

1. A method of delivering a human transgene to an eye of a subject, the method comprising administering to the eye of the subject a pharmaceutical composition comprising:

a) a replication-defective herpes simplex virus type-1 (HSV-1) comprising a recombinant HSV-1 genome, wherein the recombinant HSV-1 genome comprises one or more polynucleotides comprising the human transgene; and

b) a pharmaceutically acceptable carrier,

wherein the pharmaceutical composition is administered topically or via injection to the eye of the subject,

wherein the one or more polynucleotides comprising the human transgene are operably linked to a promoter suitable for transcription in a mammalian cell,

wherein the subject has a disorder or disease of the eye associated with vision loss,

wherein the recombinant HSV-1 genome comprises an inactivating mutation in one or both copies of the ICP4 HSV-1 gene, and

wherein the recombinant HSV-1 genome comprises an inactivating mutation in the ICP22 HSV-1 gene.