公开(公告)号：US20240191255A1

公开(公告)日：2024-06-13

申请号：US18507263

申请日：2023-11-13

Applicant: Gene Bridges GMBH

Inventor： Adrian Francis Stewart ,  Jun Fu ,  Anja Ehrhardt ,  Eric Ehrke-Schulz ,  Wenli Zhang

IPC: C12N15/86 ,  C12N7/00 ,  C12N9/22 ,  C12N15/11

CPC classification number: C12N15/86 ,  C12N7/00 ,  C12N9/22 ,  C12N15/11 ,  C12N2310/20 ,  C12N2710/10021 ,  C12N2710/10043 ,  C12N2800/80

Abstract: The invention relates to adenoviral vectors, cells for use in generating adenoviral vectors, methods for generating adenoviral vectors, and therapeutic uses of adenoviral vectors in gene therapy, tumour therapy and as vaccines.

公开(公告)号：US12006521B2

公开(公告)日：2024-06-11

申请号：US17226264

申请日：2021-04-09

Applicant: MONSANTO TECHNOLOGY LLC

Inventor： Jaishree M. Chittoor ,  Ervin Nagy

IPC: C12N9/22 ,  C12N15/10 ,  C12N15/11 ,  C12N15/82

CPC classification number: C12N9/22 ,  C12N15/102 ,  C12N15/11 ,  C12N15/8241 ,  C12N2310/20 ,  C12N2800/80

Abstract: Provided herein are systems, methods, and compositions for the modification of target DNA sequences. More particularly, systems, methods, and compositions for editing genomic DNA in eukaryotic cells with a CRISPR-associated transposase are provided. Also provided are vectors and vector systems which encode one or more CRISPR-associated transposases, as well as methods for the design and use of such vectors. Also provided are methods for identifying and validating novel CRISPR-associated transposases.

公开(公告)号：US20240167061A1

公开(公告)日：2024-05-23

申请号：US18515028

申请日：2023-11-20

Applicant: Taipei Veterans General Hospital

Inventor： Shih-Hwa Chiou ,  Shih-Jie Chou ,  Yueh Chien ,  Yi-Ping Yang

IPC: C12N15/90 ,  C12N9/22 ,  C12N15/11 ,  C12N15/88

CPC classification number: C12N15/907 ,  C12N9/22 ,  C12N15/11 ,  C12N15/88 ,  A61K48/00 ,  C12N2310/20 ,  C12N2800/80

Abstract: The present invention is directed to an integrated conceptual strategy for a gene delivery system, using the combination of nanoparticles, CRISPR-Cas9, and the HITI strategy to deliver CRISPR-Cas9 and achieve effective genome editing; wherein the advanced nanoparticles to overcome the limited packaging size of AAV-based vehicles. Also provided is a promising therapeutic solution for the treatment of hereditary diseases via gene therapy

公开(公告)号：US20240167060A1

公开(公告)日：2024-05-23

申请号：US18491534

申请日：2023-10-20

Applicant: CRISPR Therapeutics AG

Inventor： Troy Dean CARLO ,  Roman Lvovitch BOGORAD

IPC: C12N15/90 ,  C12N9/22 ,  C12N15/11

CPC classification number: C12N15/907 ,  C12N9/22 ,  C12N15/11 ,  C12N2310/18 ,  C12N2310/20 ,  C12N2800/80

Abstract: The present disclosure provides donor polynucleotides, genome editing systems, methods, pharmaceutical compositions, and kits which correct or induce a mutation that causes Glycogen Storage Disease 1a in a genomic DNA molecule in a cell. In some embodiments the present disclosure provides donor polynucleotides comprising two strands capable of correcting a mutation that causes Glycogen Storage Disease 1a.

公开(公告)号：US20240156985A1

公开(公告)日：2024-05-16

申请号：US17985765

申请日：2022-11-11

Applicant: City University of Hong Kong

Inventor： Wenjun Xiong ,  Anh Duc Hoang ,  Baoshan Liao

IPC: A61K48/00 ,  A61K31/7105 ,  A61K38/17 ,  A61K38/46 ,  A61P27/02 ,  C12N9/22 ,  C12N15/11 ,  C12N15/86 ,  C12N15/90

CPC classification number: A61K48/005 ,  A61K31/7105 ,  A61K38/177 ,  A61K38/465 ,  A61P27/02 ,  C12N9/22 ,  C12N15/11 ,  C12N15/86 ,  C12N15/907 ,  C12N2310/20 ,  C12N2750/14143 ,  C12N2750/14152 ,  C12N2750/14171 ,  C12N2800/80

Abstract: Novel 5′ untranslated region (UTR)-targeting gene knock-in (KI) compositions and methods of use are disclosed. The gene KI compositions and methods exploit homology-independent targeted integration (HITI)-mediated insertion of a wild-type coding sequence (CDS) into the 5′ UTR upstream of a translation initiation element of a mutated variant of the wild-type gene. The 5′ UTR-targeting gene KI therapy compositions and methods provide safer and more efficient gene insertion compared to other gene therapy approaches.

公开(公告)号：US20240150741A1

公开(公告)日：2024-05-09

申请号：US18507709

申请日：2023-11-13

Applicant: The General Hospital Corporation

Inventor： J. Keith Joung ,  Benjamin Kleinstiver

IPC: C12N9/22 ,  C12N15/10 ,  C12N15/11 ,  C12N15/63 ,  C12N15/90

CPC classification number: C12N9/22 ,  C12N15/102 ,  C12N15/111 ,  C12N15/63 ,  C12N15/90 ,  C12Y301/00 ,  A01K2227/40 ,  A61K38/00 ,  C07K2319/71 ,  C12N2310/20 ,  C12N2800/22 ,  C12N2800/80

Abstract: Engineered CRISPR-Cas9 nucleases with altered and improved PAM specificities and their use in genomic engineering, epigenomic engineering, and genome targeting.

公开(公告)号：US20240141386A1

公开(公告)日：2024-05-02

申请号：US18161806

申请日：2023-01-30

Applicant: EDITAS MEDICINE, INC.

Inventor： Morgan Lee MAEDER ,  Rina J. MEPANI ,  Michael STEFANIDAKIS

IPC: C12N15/90 ,  A61K9/00 ,  A61K35/761 ,  C12N9/22 ,  C12N15/11 ,  C12N15/113 ,  C12N15/86

CPC classification number: C12N15/907 ,  A61K9/0048 ,  A61K35/761 ,  C12N9/22 ,  C12N15/11 ,  C12N15/113 ,  C12N15/86 ,  C12N2310/20 ,  C12N2320/32 ,  C12N2330/50 ,  C12N2330/51 ,  C12N2750/14143 ,  C12N2800/80

Abstract: Nucleic acids and viral vectors, particularly adeno-associated virus (AAV) vectors are provided that encode Cas9 and paired guide RNAs. The nucleic acids and vectors, and compositions that comprise them, can be used in methods to treat subjects, to alter cells in subjects who may suffer from an inherited retinal dystrophy such as CEP290 associated disease or who may be in need of alteration of a cell or a cellular nucleic acid sequence associated with an inherited retinal dystrophy such as the CEP290 gene, and/or to treat inherited retinal dystrophies including CEP290 associated disease.

公开(公告)号：US20240141323A1

公开(公告)日：2024-05-02

申请号：US18526826

申请日：2023-12-01

Applicant: BLUEALLELE CORPORATION

Inventor： Nicholas J. BALTES

IPC: C12N15/10 ,  C12N15/90

CPC classification number: C12N15/102 ,  C12N15/907 ,  C12N2310/20 ,  C12N2800/80

Abstract: Methods and compositions for modifying the coding sequence of endogenous genes using rare-cutting endonucleases and transposases. The methods and compositions described herein can be used to modify the coding sequence of endogenous genes.

公开(公告)号：US20240139319A1

公开(公告)日：2024-05-02

申请号：US18274752

申请日：2022-02-01

Applicant: EPSILEN BIO S.R.L

Inventor： Angelo Leone Lombardo ,  Alice Reschigna ,  Tania Baccega

IPC: A61K39/00 ,  A61K31/7088 ,  A61K38/46 ,  C12N9/22 ,  C12N15/11 ,  C12N15/90

CPC classification number: A61K39/4631 ,  A61K31/7088 ,  A61K38/465 ,  A61K39/4611 ,  A61K39/4632 ,  C12N9/22 ,  C12N15/11 ,  C12N15/907 ,  C12N2310/20 ,  C12N2800/80

Abstract: An engineered transcriptional modulator (ETM) comprising: (a) at least one epigenetic effector domain; operably linked to (b) an endonuclease.

公开(公告)号：US20240131120A1

公开(公告)日：2024-04-25

申请号：US17969590

申请日：2022-10-18

Applicant: PAROLE LABORATORIES INC.

Inventor： Oleg NEPOTCHATYKH ,  Evguenia NEPOTCHATYKH ,  Olga NEPOTCHATYKH

IPC: A61K38/46 ,  C12N9/22 ,  C12N15/11 ,  C12N15/90

CPC classification number: A61K38/465 ,  C12N9/22 ,  C12N15/11 ,  C12N15/907 ,  C12N2310/20 ,  C12N2800/80

Abstract: Nanoparticles capable of delivering active pharmaceutical compounds (payload) into cells and methods of preparing same; wherein the payload is ionized and combined with a first polymer ion of an opposite charge resulting in the formation of an initial molecular assembly. The initial molecular assembly is then combined with a second polymer ion having an opposite charge and a different size than the first polymer ion, resulting in the formation of a secondary molecular assembly, wherein the payload becomes trapped between the first and the second polymer ions. The non-conjugated charged segments of the secondary molecular assembly are then repeatedly combined with additional polymer ions of alternating opposite charges to such segments charges and pre-determined size to extend and branch the molecular assembly resulting in the formation of a nanoparticle.