Patent ID: 11865148
Assignee: KRYSTAL BIOTECH, INC.
Field: Pharmaceuticals (Chemistry)
Classification: CPC A  C | IPC A  C

Claim 0:
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.