IP Library Granted Patent US 10,155,016
Granted Patent B2
US 10,155,016 · App. 15/851,488 · Granted Dec 18, 2018

Compositions and methods for the treatment of wounds, disorders, and diseases of the skin

Inventors: Suma Krishnan (Belvedere, CA); Pooja Agarwal (Germantown, MD)
Assignee: Krystal Biotech, Inc.
A61K35/763A61K9/0014A61K38/1748A61K38/39A61K48/005C07K14/78C12N9/0071C12Y114/11004A61K9/06A61K47/38C12N2710/16643
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Quick Facts
Patent No.
US 10,155,016
App. No.
15/851,488
Granted
Dec 18, 2018
Kind
B2
Abstract

The present disclosure relates, in part, to pharmaceutical compositions comprising one or more polynucleotides suitable for enhancing, increasing, augmenting, and/or supplementing the levels of Collagen alpha-1 (VII) chain polypeptide and/or Lysyl hydroxylase 3 polypeptide and/or Keratin type I cytoskeletal 17 polypeptide in a subject. The present disclosure also relates, in part, to pharmaceutical compositions and methods of use for providing prophylactic, palliative, or therapeutic relief of a wound, disorder, or disease of the skin in a subject, including a subject having, or at risk of developing, one or more symptoms of epidermolysis bullosa.

Claims (29)

1. A pharmaceutical composition comprising:

a) a herpes simplex virus (HSV) comprising a recombinant herpes simplex virus genome, wherein the recombinant herpes simplex virus genome comprises one or more transgenes encoding a Collagen alpha-1 (VII) chain polypeptide; and

b) a pharmaceutically acceptable carrier;

wherein the recombinant herpes simplex virus genome comprises an inactivating mutation in one or both copies of the Infected Cell Protein (ICP) 4 herpes simplex virus gene.

2. The pharmaceutical composition of claim 1 , wherein the recombinant herpes simplex virus genome comprises the one or more transgenes encoding the Collagen alpha-1 (VII) chain polypeptide within one or both of the ICP4 viral gene loci.

3. The pharmaceutical composition of claim 1 , wherein the recombinant herpes simplex virus genome further comprises an inactivating mutation in another herpes simplex virus gene selected from the group consisting of ICP0, ICP22, ICP27, ICP47, thymidine kinase (tk), long unique region (UL)41, and UL55.

4. The pharmaceutical composition of claim 1 , wherein the Collagen alpha-1 (VII) chain polypeptide has at least 80% sequence identity to the sequence of SEQ ID NO: 2.

5. The pharmaceutical composition of claim 1 , wherein the pharmaceutically acceptable carrier is suitable for a route of administration selected from the group consisting of topical administration, transdermal administration, subcutaneous administration, intradermal administration, oral administration, sublingual administration, buccal administration, rectal administration, via inhalation, intravenous (IV) injection, intra-arterial injection, intramuscular injection, intracardiac injection, intraosseous injection, intraperitoneal injection, transmucosal administration, vaginal administration, intravitreal administration, intra-articular administration, peri-articular administration, local administration, and epicutaneous administration.

6. A pharmaceutical composition comprising:

a) a herpes simplex type 1 virus (HSV-1) comprising a recombinant herpes simplex virus genome, wherein the recombinant herpes simplex virus genome comprises one or more transgenes encoding a Collagen alpha-1 (VII) chain polypeptide; and

b) a pharmaceutically acceptable carrier;

wherein the HSV-1 has reduced cytotoxicity as compared to a wild-type herpes simplex type 1 virus.

7. The pharmaceutical composition of claim 6 , wherein the HSV-1 comprising the recombinant herpes simplex virus genome is replication-defective.

8. The pharmaceutical composition of claim 6 , wherein the HSV-1 comprising the recombinant herpes simplex virus genome is replication-competent.

9. The pharmaceutical composition of claim 6 , wherein the recombinant herpes simplex virus genome comprises an inactivating mutation in a herpes simplex virus gene.

10. The pharmaceutical composition of claim 9 , wherein the herpes simplex virus gene is selected from the group consisting of ICP0, ICP4, ICP22, ICP27, ICP47, tk, UL41, and UL55.

11. The pharmaceutical composition of claim 6 ; wherein the Collagen alpha-1 (VII) chain polypeptide has at least 80% sequence identity to the sequence of SEQ ID NO: 2.

12. The pharmaceutical composition of claim 6 , wherein the pharmaceutically acceptable carrier is suitable for a route of administration selected from the group consisting of topical administration, transdermal administration, subcutaneous administration, intradermal administration, oral administration, sublingual administration, buccal administration, rectal administration, via inhalation, intravenous (IV) injection, intra-arterial injection; intramuscular injection, intracardiac injection, intraosseous injection, intraperitoneal injection, transmucosal administration, vaginal administration, intravitreal administration, intra-articular administration, peri-articular administration, local administration, and epicutaneous administration.

13. A pharmaceutical composition comprising:

a) a herpes simplex virus (HSV) comprising a recombinant herpes simplex virus genome, wherein the recombinant herpes simplex virus genome comprises one or more transgenes encoding a Collagen alpha-1 (VII) chain polypeptide; and

b) a pharmaceutically acceptable carrier;

wherein the Collagen alpha-1 (VII) chain polypeptide comprises an amino acid sequence having at least 90% identity to the sequence of SEQ ID NO: 2; and

wherein the HSV has reduced cytotoxicity as compared to a wild-type herpes simplex virus.

14. The pharmaceutical composition of claim 13 , wherein the Collagen alpha-1 (VII) chain polypeptide comprises an amino acid sequence having at least 95% identity to the sequence of SEQ ID NO: 2.

15. The pharmaceutical composition of claim 13 , wherein the HSV comprising the recombinant herpes simplex virus genome is replication-defective.

16. The pharmaceutical composition of claim 13 , wherein the HSV comprising the recombinant herpes simplex virus genome is replication-competent.

17. The pharmaceutical composition of claim 13 , wherein the recombinant herpes simplex virus genome comprises an inactivating mutation in a herpes simplex virus gene.

18. The pharmaceutical composition of claim 17 , wherein the herpes simplex virus gene is selected form the group consisting of ICP0, ICP4, ICP22, ICP27, ICP47, tk, UL41, and UL55.

19. The pharmaceutical composition of claim 13 , wherein the pharmaceutically acceptable carrier is suitable for a route of administration selected from the group consisting of topical administration, transdermal administration, subcutaneous administration, intradermal administration, oral administration, sublingual administration, buccal administration, rectal administration, via inhalation, intravenous (IV) injection, intra-arterial injection, intramuscular injection, intracardiac injection, intraosseous injection, intraperitoneal injection, transmucosal administration, vaginal administration, intravitreal administration, intra-articular administration, peri-articular administration, local administration, and epicutaneous administration.

Assignments (3)
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Jun 15, 2020
From: AGARWAL, POOJA; KRISHNAN, SUMA
To: KRYSTAL BIOTECH, INC.
Reel/Frame 052939/0775 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Jun 5, 2018
From: KRISHNAN, SUMA; AGARWAL, POOJA
To: KRYSTAL BIOTECH, LLC
Reel/Frame 045991/0861 →
CHANGE OF NAME Recorded Jun 5, 2018
From: KRYSTAL BIOTECH, LLC
To: KRYSTAL BIOTECH, INC.
Reel/Frame 046302/0516 →
Continuity (3)
Continuation 15393151 · Dec 28, 2016
Provisional Application 62320316 · Apr 8, 2016
Related Publication 20180169160A1 · Jun 21, 2018
Cited By (4)
US 12,364,775 US 12,522,636 US 12,582,684 US 12,655,447