公开(公告)号：US20240376467A1

公开(公告)日：2024-11-14

申请号：US18672385

申请日：2024-05-23

Applicant: Christiana Care Gene Editing Institute, Inc.

Inventor： Eric B. Kmiec ,  Pawel Bialk ,  Kelly H. Banas

IPC: C12N15/11 ,  A61K38/00 ,  A61K48/00 ,  A61P35/00 ,  C12N9/22 ,  C12N15/85

Abstract: The disclosure provides a guide RNA (gRNA) comprising a DNA-binding domain and a Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)-associated endonuclease protein-binding domain, wherein the DNA-binding domain is complementary to a target domain from a variant NRF2 gene found in a cancer cell but not in a non-cancerous cell. The disclosure also provides nucleic acid sequence encoding the gRNA. The disclosure further provides a method of treating cancer in a subject comprising administering to the subject a therapeutically effective amount of a pharmaceutical composition comprising a Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)-associated endonuclease and a guide RNA that is complementary to a target domain from a variant NRF2 gene in the subject. Methods of treating cancer comprising administering a pharmaceutical composition comprising: a DNA sequence encoding a guide RNA that is complementary to a target domain from a variant NRF2 gene in the subject; and a nucleic acid sequence encoding a Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)-associated endonuclease, are also provided.

公开(公告)号：US20230416731A1

公开(公告)日：2023-12-28

申请号：US17960210

申请日：2022-10-05

Applicant: Christiana Care Gene Editing Institute, Inc.

Inventor： Eric B. Kmiec ,  Pawel Bialk ,  Kelly H. Banas

IPC: C12N15/11 ,  A61P35/00 ,  A61K48/00 ,  C12N9/22 ,  C12N15/85

CPC classification number: C12N15/11 ,  A61P35/00 ,  A61K48/005 ,  C12N9/22 ,  C12N15/85 ,  C12N2310/20 ,  A61K38/00

Abstract: The disclosure provides a guide RNA (gRNA) comprising a DNA-binding domain and a Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)-associated endonuclease protein-binding domain, wherein the DNA-binding domain is complementary to a target domain from a variant NRF2 gene found in a cancer cell but not in a non-cancerous cell. The disclosure also provides nucleic acid sequence encoding the gRNA. The disclosure further provides a method of treating cancer in a subject comprising administering to the subject a therapeutically effective amount of a pharmaceutical composition comprising a Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)-associated endonuclease and a guide RNA that is complementary to a target domain from a variant NRF2 gene in the subject. Methods of treating cancer comprising administering a pharmaceutical composition comprising: a DNA sequence encoding a guide RNA that is complementary to a target domain from a variant NRF2 gene in the subject; and a nucleic acid sequence encoding a Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)-associated endonuclease, are also provided.

公开(公告)号：US20250018060A1

公开(公告)日：2025-01-16

申请号：US18711355

申请日：2022-11-18

Applicant: Christiana Care Gene Editing Institute, Inc.

Inventor： Eric B. Kmiec ,  Byung-chun Yoo ,  Steven Yang ,  Kelly H. Banas

IPC: A61K48/00 ,  A61K31/337 ,  A61K31/475 ,  A61K31/555 ,  A61K33/243 ,  A61P35/00 ,  C12N9/22 ,  C12N15/11 ,  C12N15/86

Abstract: The disclosure provides recombinant adenoviruses comprising a polynucleotide comprising: a first DNA sequence encoding a guide RNA (gRNA), wherein the gRNA comprises a DNA-binding domain and a Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)-associated endonuclease protein-binding domain, and the DNA-binding domain is complementary to a target sequence in a gene; and a first promoter that is operably linked to the DNA sequence. The disclosure also provides pharmaceutical compositions comprising the adenoviruses described herein. The disclosure further provides a method of treating cancer in a subject, the method comprising administering to the subject a therapeutically effective amount of the pharmaceutical compositions as described herein to the subject.

公开(公告)号：US12037581B2

公开(公告)日：2024-07-16

申请号：US17960210

申请日：2022-10-05

Applicant: Christiana Care Gene Editing Institute, Inc.

Inventor： Eric B. Kmiec ,  Pawel Bialk ,  Kelly H. Banas

IPC: C12N15/11 ,  A61K31/7088 ,  A61K38/00 ,  A61K38/46 ,  A61K48/00 ,  A61P35/00 ,  C12N9/22 ,  C12N15/113 ,  C12N15/85 ,  C12N15/90

CPC classification number: C12N15/11 ,  A61K48/005 ,  A61P35/00 ,  C12N9/22 ,  C12N15/85 ,  A61K38/00 ,  C12N2310/20 ,  C12N2800/80

Abstract: The disclosure provides a guide RNA (gRNA) comprising a DNA-binding domain and a Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)-associated endonuclease protein-binding domain, wherein the DNA-binding domain is complementary to a target domain from a variant NRF2 gene found in a cancer cell but not in a non-cancerous cell. The disclosure also provides nucleic acid sequence encoding the gRNA. The disclosure further provides a method of treating cancer in a subject comprising administering to the subject a therapeutically effective amount of a pharmaceutical composition comprising a Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)-associated endonuclease and a guide RNA that is complementary to a target domain from a variant NRF2 gene in the subject. Methods of treating cancer comprising administering a pharmaceutical composition comprising: a DNA sequence encoding a guide RNA that is complementary to a target domain from a variant NRF2 gene in the subject; and a nucleic acid sequence encoding a Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)-associated endonuclease, are also provided.

公开(公告)号：US20250001010A1

公开(公告)日：2025-01-02

申请号：US18760944

申请日：2024-07-01

Applicant: Christiana Care Gene Editing Institute, Inc.

Inventor： Eric B. Kmiec ,  Sophia Masciarelli ,  Brett Sansbury ,  Kelly H. Banas

IPC: A61K48/00 ,  A61K45/06 ,  A61P35/00 ,  C12N9/22 ,  C12N15/11

Abstract: The disclosure provides a guide RNA (gRNA) comprising a DNA-binding domain and a Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)-associated endonuclease protein-binding domain, wherein the DNA-binding domain is complementary to a target domain from an NRAS gene. The disclosure also provides nucleic acid sequence encoding the gRNA. The disclosure further provides a method of treating cancer in a subject comprising administering to the subject a therapeutically effective amount of a pharmaceutical composition comprising a CRISPR-associated endonuclease and a guide RNA that is complementary to a target domain from an NRAS gene in the subject. Methods of treating cancer comprising administering a pharmaceutical composition comprising: a nucleic acid sequence encoding a guide RNA that is complementary to a target domain from an NRAS gene in the subject; and a nucleic acid sequence encoding a CRISPR-associated endonuclease, are also provided.

公开(公告)号：US12037582B2

公开(公告)日：2024-07-16

申请号：US17960224

申请日：2022-10-05

Applicant: Christiana Care Gene Editing Institute, Inc.

Inventor： Eric B. Kmiec ,  Pawel Bialk ,  Kelly H. Banas

IPC: C12N15/11 ,  A61K31/7088 ,  A61K38/00 ,  A61K38/46 ,  A61K48/00 ,  A61P35/00 ,  C12N9/22 ,  C12N15/113 ,  C12N15/85 ,  C12N15/90

CPC classification number: C12N15/11 ,  A61K48/005 ,  A61P35/00 ,  C12N9/22 ,  C12N15/85 ,  A61K38/00 ,  C12N2310/20 ,  C12N2800/80

Abstract: The disclosure provides a guide RNA (gRNA) comprising a DNA-binding domain and a Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)-associated endonuclease protein-binding domain, wherein the DNA-binding domain is complementary to a target domain from a variant NRF2 gene found in a cancer cell but not in a non-cancerous cell. The disclosure also provides nucleic acid sequence encoding the gRNA. The disclosure further provides a method of treating cancer in a subject comprising administering to the subject a therapeutically effective amount of a pharmaceutical composition comprising a Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)-associated endonuclease and a guide RNA that is complementary to a target domain from a variant NRF2 gene in the subject. Methods of treating cancer comprising administering a pharmaceutical composition comprising: a DNA sequence encoding a guide RNA that is complementary to a target domain from a variant NRF2 gene in the subject; and a nucleic acid sequence encoding a Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)-associated endonuclease, are also provided.