A gene editing system for inserting a nucleic acid into a genomic site, comprising: (a) a CRISPR nucleases or an encoding nucleic acid; (b) a guide RNA (gRNA) or an encoding nucleic acid, the gRNA being specific to a target sequence in a genomic site of interest; and (c) a donor DNA template comprising (i) a transgene; (ii) a left homology arm upstream to the transgene and a right homology arm downstream to the transgene; and (iii) two copies of a target sequence, one being upstream to the left homology arm and the other being downstream to the right homology arm. The left homology arm and the right homology arm are homologous to the upstream and downstream sequences flanking the target sequence in the genomic site.
A composition for genetic editing of a hydroxyacid oxidase 1 (HAO1) gene, comprising: (a) a messenger RNA (mRNA) encoding a Cas12i2 polypeptide, (b) a guide RNA (gRNA) targeting the HAO1 gene; and (c) lipid excipients, which optionally form lipid nanoparticles. Also provided herein are methods for genetic modification of the HAO1 gene in host cells with the composition provided herein.
The disclosure describes novel systems, methods, and compositions for the manipulation of nucleic acids in a targeted fashion. The disclosure describes non-naturally occurring, engineered CRISPR systems, components, and methods for targeted modification of nucleic acids such as DNA. Each system includes one or more protein components and one or more nucleic acid components that together target nucleic acids.
A system for genetic editing of a stathmin 2 (STMN2) gene, comprising (i) a Cas12i2 polypeptide or a first nucleic acid encoding the Cas12i2 polypeptide, and (ii) an RNA guide or second nucleic acid encoding the RNA guide, wherein the RNA guide comprises a spacer sequence specific to a target sequence within an STMN2 gene. Also provided herein are methods for editing a STMN2 gene using the gene editing system disclosed herein and/or for treating diseases associated with the STMN2 gene.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
A gene editing system comprising (a) a fusion polypeptide comprising a CRISPR nuclease and a reverse transcriptase, or a nucleic acid encoding the fusion polypeptide, and (b) an RNA molecule comprising a guide RNA and a reverse transcription donor RNA, or a nucleic acid encoding the RNA molecule. Also provided herein are methods of using the gene editing system for modifying target genes of interest.
Engineered CRISPR nuclease polypeptides derived from a reference CRISPR nuclease set forth as SEQ ID NO: 1, wherein, relative to the reference nuclease, the engineered CRISPR nuclease polypeptide comprises (i) one or more mutations in the HNH nuclease domain or in the RuvC nuclease domain that reduce or eliminate the nuclease activity thereof; (ii) one or more arginine and/or lysine substitutions; and/or (iii) one or more mutations for reducing PAM recognition stringency.
A gene editing system comprising (a) a fusion polypeptide comprising a CRISPR nuclease and a reverse transcriptase, or a nucleic acid encoding the fusion polypeptide, and (b) an RNA molecule comprising a guide RNA and a reverse transcription donor RNA, or a nucleic acid encoding the RNA molecule. Also provided herein are methods of using the gene editing system for modifying target genes of interest.
The disclosure describes novel systems, methods, and compositions for the manipulation of nucleic acids in a targeted fashion. The disclosure describes non-naturally occurring, engineered CRISPR systems, components, and methods for targeted modification of DNA, RNA, and protein substrates. Each system includes one or more protein components and one or more nucleic acid components that together target DNA, RNA, or protein substrates.
The present disclosure relates to cells (e.g., T cells) modified by Cas12i, methods of modifying the cells, processes for characterizing the modified cells, compositions and formulations comprising the modified cells, and uses of the compositions and formulations comprising the modified cells.
The present invention relates to variant Cas12i3 polypeptides, methods of preparing the variant Cas12i3 polypeptides, processes for characterizing the variant Cas12i3 polypeptides, compositions, gene editing systems, and cells comprising the variant Cas12i3 polypeptides, and methods of using the variant Cas12i3 polypeptides. The invention further relates to complexes comprising the variant Cas12i3 polypeptides, methods of producing the complexes, processes for characterizing the complexes, cells comprising the complexes, and methods of using the complexes.
A nuclease polypeptide such as a CRISPR nuclease polypeptide derived from a reference nuclease, which can be Nuclease A, Nuclease K, or Nuclease M, the nuclease polypeptide comprising a RuvC nuclease domain and an HNH nuclease domain. Also provided herein are gene editing systems comprising such a nuclease polypeptide and gene editing methods using the gene editing system.
CRISPR nuclease polypeptides derived from a reference CRISPR nuclease set forth as SEQ ID NO: 1, SEQ ID NO: 49, or SEQ ID NO: 64, which share an amino acid sequence at least 90% identical to the reference CRISPR nuclease, and optionally comprises one or more mutations relative to the reference CRISPR nuclease. Also provided herein are gene editing systems comprising such CRISPR nuclease polypeptides and gene editing methods using such.
The present invention relates to variant Cas12i4 polypeptides, methods of producing the variant Cas12i4 polypeptides, processes for characterizing the variant Cas12i4 polypeptides, cells comprising the variant Cas12i4 polypeptides, and methods of using the variant Cas12i4 polypeptides. The invention further relates to complexes comprising a variant Cas12i4 polypeptide and an RNA guide, methods of producing the complexes, processes for characterizing the complexes, cells comprising the complexes, and methods of using the complexes.
The disclosure describes Cas12i2 fusion proteins, methods, and compositions for the manipulation of nucleic acids in a targeted fashion. The disclosure describes non-naturally occurring, engineered Cas12i2 fusion proteins, components, and methods for targeted modification of nucleic acids. Each system, includes one or more protein components and one or more nucleic acid components that together target nucleic acids.
A method for inhibiting aberrant splicing in a Stathmin-2 (STMN2) transcript, the method comprising: genetically editing a STMN2 gene in a cell to delete (a) one or more nucleotides in a 3′ splice site of intron 1, wherein the 3′ splice site is adjacent to exon 2a, (b) one or more nucleotides in a region of intron 1 that is adjacent to the 3′ splice site, or both (a) and (b), thereby inhibiting production of STMN2 transcripts including exon 2a and improving production of functional STMN2 transcripts in the cell. Also provided herein are gene editing systems for genetic modification of the STMN2 gene.
A method for inhibiting aberrant splicing in a Stathmin-2 (STMN2) transcript, the method comprising: genetically editing a STMN2 gene in a cell to delete (a) one or more nucleotides in a 3' splice site of intron 1, wherein the 3' splice site is adjacent to exon 2a, (b) one or more nucleotides in a region of intron 1 that is adjacent to the 3' splice site, or both (a) and (b), thereby inhibiting production of STMN2 transcripts including exon 2a and improving production of functional STMN2 transcripts in the cell. Also provided herein are gene editing systems for genetic modification of the STMN2 gene.
C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
A system for genetic editing of a transthyretin (TTR) gene, comprising (i) a Cas12i2 polypeptide or a first nucleic acid encoding the Cas12i2 polypeptide, and (ii) an RNA guide or a second nucleic acid encoding the RNA guide, wherein the RNA guide comprises a spacer sequence specific to a target sequence within an TTR gene. Also provided herein are methods for editing a TTR gene using the gene editing system disclosed herein and/or for treating diseases associated with the TTR gene.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
A gene editing system comprising: (a) a Type V CRISPR nuclease polypeptide or a first nucleic acid encoding the Type V CRISPR nuclease polypeptide; (b) an engineered guide RNA (gRNA) or a second nucleic acid encoding the gRNA, wherein the engineered gRNA comprises: (i) a spacer sequence specific to a target sequence within a genomic site of interest, and (ii) an engineered scaffold sequence, which is recognizable by the Type V CRISPR nuclease, wherein the engineered scaffold sequence comprises one or more mutations relative to the wild-type counterpart.
Provided herein are gene editing systems and/or compositions comprising RNA guides targeting HAO1 for use in genetic editing of the HAO1 gene. Also provide herein are methods of using the gene editing system for introducing edits to the HAO1 gene and/or for treatment of primary hyperoxaluria (PH), and processes for characterizing the gene editing system.
C12N 15/88 - Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using liposome vesicle
C12N 15/90 - Stable introduction of foreign DNA into chromosome
24.
REVERSE TRANSCRIPTASE-MEDIATED GENETIC EDITING OF TRANSTHYRETIN (TTR) AND USES THEREOF
A system for genetic editing of a transthyretin (TTR) gene, comprising (a) a Cas12i2 polypeptide or a first nucleic acid encoding the Cas12i2 polypeptide; (b) a reverse transcriptase (RT) polypeptide or a second nucleic acid encoding the RT polypeptide; (c) an RNA guide or a third nucleic acid encoding the RNA guide, and (d) a reverse transcription donor RNA (RT donor RNA) or a fourth nucleic acid encoding the RT donor RNA. Also provided herein are methods for editing a TTR gene using the gene editing system disclosed herein and/or for treating diseases associated with the TTR gene, for example, amyloidogenic transthyretin.
The present invention relates to variant polypeptides, methods of preparing the variant polypeptides, processes for characterizing the variant polypeptides, compositions and cells comprising the variant polypeptides, and methods of using the variant polypeptides. The invention further relates to complexes comprising the variant polypeptides, methods of producing the complexes, processes for characterizing the complexes, cells comprising the complexes, and methods of using the complexes.
The present invention relates to variant polypeptides, methods of preparing the variant polypeptides, processes for characterizing the variant polypeptides, compositions and cells comprising the variant polypeptides, and methods of using the variant polypeptides. The invention further relates to complexes comprising the variant polypeptides, methods of producing the complexes, processes for characterizing the complexes, cells comprising the complexes, and methods of using the complexes.
A gene editing system comprising: (a) a Type V CRISPR nuclease polypeptide or a first nucleic acid encoding the Type V CRISPR nuclease polypeptide; (b) a reverse transcriptase (RT) polypeptide or a second nucleic acid encoding the RT polypeptide; (c) a guide RNA (gRNA) or a third nucleic acid encoding the gRNA, wherein the gRNA comprises one or more binding sites recognizable by the Type V CRISPR nuclease (CRISPR nuclease binding sites) and a spacer sequence specific to a target sequence within a genomic site of interest, the target sequence being adjacent to a protospacer adjacent motif (PAM); and (d) a reverse transcription donor RNA (RT donor RNA) or a fourth nucleic acid encoding the RT donor RNA, wherein the RT donor RNA comprises a primer binding site (PBS) and a template sequence.
The disclosure describes novel systems, methods, and compositions for the manipulation of nucleic acids in a targeted fashion. The disclosure describes non-naturally occurring, engineered CRISPR systems, components, and methods for targeted modification of nucleic acids. Each system includes one or more protein components and one or more nucleic acid components that together target nucleic acids.
The present invention relates to nucleases or nucleic acids encoding the nucleases, RNA guides or nucleic acids encoding the RNA guides, processes for characterizing the nucleases and/or RNA guides, compositions comprising the nucleases and/or RNA guides, and kits and/or methods for preparing and/or using the nucleases and/or RNA guides.
The present invention relates to variant polypeptides, methods of preparing the variant polypeptides, processes for characterizing the variant polypeptides, compositions and cells comprising the variant polypeptides, and methods of using the variant polypeptides. The invention further relates to complexes comprising the variant polypeptides, methods of producing the complexes, processes for characterizing the complexes, cells comprising the complexes, and methods of using the complexes.
The present invention relates to variant polypeptides, methods of preparing the variant polypeptides, processes for characterizing the variant polypeptides, gene editing systems and cells comprising the variant polypeptides, and methods of using the variant polypeptides. The invention further relates to complexes comprising the variant polypeptides, methods of producing the complexes, processes for characterizing the complexes, cells comprising the complexes, and methods of using the complexes.
The present invention relates to variant polypeptides, methods of preparing the variant polypeptides, processes for characterizing the variant polypeptides, compositions and cells comprising the variant polypeptides, and methods of using the variant polypeptides. The invention further relates to complexes comprising the variant polypeptides, methods of producing the complexes, processes for characterizing the complexes, cells comprising the complexes, and methods of using the complexes.
The present invention relates to compositions comprising RNA guides targeting TRAC, processes for characterizing the compositions, cells comprising the compositions, and methods of using the compositions.
The present invention relates nucleases, processes for characterizing the nucleases, compositions comprising the nucleases, and methods of using the nucleases.
The present invention relates to variant CRISPR nuclease polypeptides, methods of preparing the variant CRISPR nuclease polypeptides, processes for characterizing the variant CRISPR nuclease polypeptides, compositions and cells comprising the variant CRISPR nuclease polypeptides, and methods of using the variant CRISPR nuclease polypeptides. The invention further relates to complexes comprising a variant CRISPR nuclease polypeptide, methods of producing the complexes, processes for characterizing the complexes, cells comprising the complexes, and methods of using the complexes.
The present invention relates to variant Cas12i4 polypeptides, methods of producing the variant Cas12i4 polypeptides, processes for characterizing the variant Cas12i4 polypeptides, cells comprising the variant Cas12i4 polypeptides, and methods of using the variant Cas12i4 polypeptides. The invention further relates to complexes comprising a variant Cas12i4 polypeptide and an RNA guide, methods of producing the complexes, processes for characterizing the complexes, cells comprising the complexes, and methods of using the complexes.
The present invention relates to compositions comprising RNA guides targeting BCL11A, processes for characterizing the compositions, cells comprising the compositions, and methods of using the compositions.
The disclosure describes novel systems, methods, and compositions for the manipulation of nucleic acids in a targeted fashion. The disclosure describes non-naturally occurring, engineered Type III-E CRISPR-Cas systems, components, and methods for targeted modification of DNA, RNA, and protein substrates. Each system includes one or more protein components and one or more nucleic acid components that together target DNA, RNA, or protein substrates.
The present invention relates to compositions comprising RNA guides targeting PDCD1, processes for characterizing the compositions, cells comprising the compositions, and methods of using the compositions.
The present invention relates to variant Cas12i2 polypeptides, methods of producing the variant Cas12i2 polypeptides, processes for characterizing the variant Cas12i2 polypeptides, cells comprising the variant Cas12i2 polypeptides, and methods of using the variant Cas12i2 polypeptides. The invention further relates to complexes comprising the variant Cas12i2 polypeptides, methods of producing the complexes, processes for characterizing the complexes, cells comprising the complexes, and methods of using the complexes.
The present invention relates to compositions comprising a Cas12i polypeptide, a deaminase polypeptide, and an RNA guide, processes for characterizing the compositions, cells comprising the compositions, Cas12i fusion proteins, Cas12i complexes, and methods of using the compositions.
The present invention relates to variant polypeptides, methods of preparing the variant polypeptides, processes for characterizing the variant polypeptides, compositions and cells comprising the variant polypeptides, and methods of using the variant polypeptides. The invention further relates to complexes comprising the variant polypeptides, methods of producing the complexes, processes for characterizing the complexes, cells comprising the complexes, and methods of using the complexes.
05 - Pharmaceutical, veterinary and sanitary products
42 - Scientific, technological and industrial services, research and design
Goods & Services
Pharmaceutical preparations and therapeutic pharmaceuticals
for the treatment of genetic diseases and disorders, genomic
diseases and disorders, and cancer; gene therapy products;
cell therapy products. Pharmaceutical research and development; research and
development in the fields of gene therapy and cell therapy;
manufacturing of pharmaceuticals, gene therapy products, and
cell therapy products for others.
The disclosure describes novel systems, methods, and compositions for the manipulation of nucleic acids in a targeted fashion. The disclosure describes non-naturally occurring, engineered CRISPR systems, components, and methods for targeted modification of nucleic acids such as DNA. Each system includes one or more protein components and one or more nucleic acid components that together target nucleic acids.
The present disclosure relates to variant polypeptides, methods of preparing the variant polypeptides, processes for characterizing the variant polypeptides, compositions and cells comprising the variant polypeptides, and methods of using the variant polypeptides. The disclosure further relates to complexes comprising the variant polypeptides, methods of producing the complexes, processes for characterizing the complexes, cells comprising the complexes, and methods of using the complexes.
The present disclosure relates to cells modified by a Cas12i polypeptide, methods of modifying the cells, processes for characterizing the modified cells, compositions and formulations comprising the modified cells, and uses of the compositions and formulations comprising the modified cells.
05 - Pharmaceutical, veterinary and sanitary products
42 - Scientific, technological and industrial services, research and design
Goods & Services
(1) Pharmaceutical preparations and therapeutic pharmaceuticals for the treatment of genetic diseases and disorders, genomic diseases and disorders, and cancer; gene therapy products; cell therapy products. (1) Pharmaceutical research and development; research and development in the fields of gene therapy and cell therapy; manufacturing of pharmaceuticals, gene therapy products, and cell therapy products for others.
48.
COMPOSITIONS COMPRISING AN RNA GUIDE TARGETING CD38 AND USES THEREOF
The present invention relates to compositions comprising RNA guides targeting CD38, processes for characterizing the compositions, cells comprising the compositions, and methods of using the compositions.
Provided herein are gene editing systems and/or compositions comprising RNA guides targeting HAO1 for use in genetic editing of the HAO1 gene. Also provide herein are methods of using the gene editing system for introducing edits to the HAO1 gene and/or for treatment of primary hyperoxaluria (PH), and processes for characterizing the gene editing system.
A61P 13/12 - Drugs for disorders of the urinary system of the kidneys
A61K 31/7105 - Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
A61K 38/16 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof
C12N 15/11 - DNA or RNA fragmentsModified forms thereof
Provided herein are gene editing systems and/or compositions comprising RNA guides targeting LDHA for use in genetic editing of the LDHA gene. Also provide herein are methods of using the gene editing system for introducing edits to the LDHA gene and/or for treatment of primary hyperoxaluria (PH), and processes for characterizing the gene editing system.
A61K 31/7105 - Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
A61K 47/54 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
A61P 13/12 - Drugs for disorders of the urinary system of the kidneys
C12N 9/04 - Oxidoreductases (1.), e.g. luciferase acting on CHOH groups as donors, e.g. glucose oxidase, lactate dehydrogenase (1.1)
C12N 15/10 - Processes for the isolation, preparation or purification of DNA or RNA
C12N 15/11 - DNA or RNA fragmentsModified forms thereof
The disclosure describes novel systems, methods, and compositions for the manipulation of nucleic acids in a targeted fashion. The disclosure describes non-naturally occurring, engineered CRISPR systems, components, and methods for targeted modification of nucleic acids. Each system includes one or more protein components and one or more nucleic acid components that together target nucleic acids.
A system for genetic editing of a stathmin 2 (STMN2) gene, comprising (i) a Cas12i2 polypeptide or a first nucleic acid encoding the Cas12i2 polypeptide, and (ii) an RNA guide or a second nucleic acid encoding the RNA guide, wherein the RNA guide comprises a spacer sequence specific to a target sequence within an STMN2 gene. Also provided herein are methods for editing a STMN2 gene using the gene editing system disclosed herein and/or for treating diseases associated with the STMN2 gene.
The present disclosure relates to gene editing systems and/or compositions comprising RNA guides targeting HAO1 and RNA guides targeting LDHA. Also provide herein are methods of using the gene editing system for introducing edits to the HAO1 gene, introducing edits to the LDHA gene, and/or for treatment of primary hyperoxaluria (PH), and processes for characterizing the gene editing system.
The present invention relates to compositions comprising a Cas12i2 polypeptide and an RNA guide, processes for characterizing the compositions, cells comprising the compositions, and methods of using the compositions.
The disclosure describes novel systems, methods, and compositions for the manipulation of nucleic acids in a targeted fashion. The disclosure describes non-naturally occurring, engineered CRISPR systems, components, and methods for targeted modification of nucleic acids such as DNA. Each system includes one or more protein components and one or more nucleic acid components that together target nucleic acids.
The present invention relates to nucleases or nucleic acids encoding the nucleases, RNA guides or nucleic acids encoding the RNA guides, processes for characterizing the nucleases and/or RNA guides, compositions comprising the nucleases and/or RNA guides, and kits and/or methods for preparing and/or using the nucleases and/or RNA guides.
The present invention relates to nucleases or nucleic acids encoding the nucleases, RNA guides or nucleic acids encoding the RNA guides, processes for characterizing the nucleases and/or RNA guides, compositions comprising the nucleases and/or RNA guides, and kits and/or methods for preparing and/or using the nucleases and/or RNA guides.
The present invention relates to nucleases or nucleic acids encoding the nucleases, RNA guides or nucleic acids encoding the RNA guides, processes for characterizing the nucleases and/or RNA guides, compositions comprising the nucleases and/or RNA guides, and kits and/or methods for preparing and/or using the nucleases and/or RNA guides.
The present invention relates to compositions comprising RNA guides targeting CIITA, processes for characterizing the compositions, cells comprising the compositions, and methods of using the compositions. The invention provides a composition comprising an RNA guide, wherein the RNA guide comprises (i) a spacer sequence that is substantially complementary to a target sequence within a CIITA gene and (ii) a direct repeat sequence; wherein the target sequence is adjacent to a protospacer adjacent motif (PAM) comprising the sequence 5 '-NTTN-3'.
The present disclosure relates to variant polypeptides, methods of preparing the variant polypeptides, processes for characterizing the variant polypeptides, compositions and cells comprising the variant polypeptides, and methods of using the variant polypeptides. The disclosure further relates to complexes comprising the variant polypeptides, methods of producing the complexes, processes for characterizing the complexes, cells comprising the complexes, and methods of using the complexes.
The present invention relates to compositions comprising a Cas12i polypeptide, a deaminase polypeptide, and an RNA guide, processes for characterizing the compositions, cells comprising the compositions, Cas12i fusion proteins, Cas12i complexes, and methods of using the compositions.
The present disclosure relates to cells (e.g., T cells) modified by Casl2i, methods of modifying the cells, processes for characterizing the modified cells, compositions and formulations comprising the modified cells, and uses of the compositions and formulations comprising the modified cells.
The present invention relates to gene editing systems comprising nucleases or nucleic acids encoding the nucleases and RNA guides or nucleic acids encoding the RNA guides, processes for characterizing the gene editing systems, and methods or preparing and/or using the gene editing systems.
The disclosure describes novel systems, methods, and compositions for the manipulation of nucleic acids in a targeted fashion. The disclosure describes non-naturally occurring, engineered CRISPR systems, components, and methods for targeted modification of nucleic acids. Each system includes one or more protein components and one or more nucleic acid components that together target nucleic acids.
A system for genetic editing of a stathmin 2 (STMN2) gene, comprising (i) a Casl2i2 polypeptide or a first nucleic acid encoding the Casl2i2 polypeptide, and (ii) an RNA guide or second nucleic acid encoding the RNA guide, wherein the RNA guide comprises a spacer sequence specific to a target sequence within an STMN2 gene. Also provided herein are methods for editing a STMN2 gene using the gene editing system disclosed herein and/or for treating diseases associated with the STMN2 gene.
The present invention relates to variant Cas12i3 polypeptides, methods of preparing the variant Cas12i3 polypeptides, processes for characterizing the variant Cas12i3 polypeptides, compositions, gene editing systems, and cells comprising the variant Cas12i3 polypeptides, and methods of using the variant Cas12i3 polypeptides. The invention further relates to complexes comprising the variant Cas12i3 polypeptides, methods of producing the complexes, processes for characterizing the complexes, cells comprising the complexes, and methods of using the complexes.
A system for genetic editing of a polypyrimidine tract binding protein 1 (PTBP1) gene, comprising (i) a Cas12i2 polypeptide or a first nucleic acid encoding the Cas12i2 polypeptide, and (ii) an RNA guide or a second nucleic acid encoding the RNA guide, wherein the RNA guide comprises a spacer sequence specific to a target sequence within an PTBP1 gene. Also provided herein are methods for editing a PTBP1 gene using the gene editing system disclosed herein and/or for treating diseases associated with the PTBP1 gene.
The present invention relates to genes coding for nucleases, processes for characterizing the nucleases, cells comprising the nucleases, and methods of using the nucleases.
05 - Pharmaceutical, veterinary and sanitary products
40 - Treatment of materials; recycling, air and water treatment,
42 - Scientific, technological and industrial services, research and design
Goods & Services
Pharmaceutical preparations and therapeutic pharmaceuticals
for the treatment of genetic diseases and disorders, genomic
diseases and disorders, and cancer; gene therapy products;
cell therapy products. Manufacturing of pharmaceuticals, gene therapy products, and
cell therapy products for others. Pharmaceutical research and development; research and
development in the fields of gene therapy and cell therapy.
71.
GENE EDITING SYSTEMS COMPRISING AN RNA GUIDE TARGETING HYDROXYACID OXIDASE 1 (HAO1) AND USES THEREOF
Provided herein are gene editing systems and/or compositions comprising RNA guides targeting HAO1 for use in genetic editing of the HAO1 gene. Also provide herein are methods of using the gene editing system for introducing edits to the HAO1 gene and/or for treatment of primary hyperoxaluria (PH), and processes for characterizing the gene editing system.
A61K 31/7105 - Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
A61P 13/12 - Drugs for disorders of the urinary system of the kidneys
72.
GENE EDITING SYSTEMS COMPRISING AN RNA GUIDE TARGETING LACTATE DEHYDROGENASE A (LDHA) AND USES THEREOF
Provided herein are gene editing systems and/or compositions comprising RNA guides targeting LDHA for use in genetic editing of the LDHA gene. Also provide herein are methods of using the gene editing system for introducing edits to the LDHA gene and/or for treatment of primary hyperoxaluria (PH), and processes for characterizing the gene editing system.
C12N 15/90 - Stable introduction of foreign DNA into chromosome
A61P 13/12 - Drugs for disorders of the urinary system of the kidneys
A61K 31/7105 - Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
The present invention relates to gene editing systems comprising nucleases or nucleic acids encoding the nucleases and RNA guides or nucleic acids encoding the RNA guides, processes for characterizing the gene editing systems, and methods or preparing and/or using the gene editing systems.
A gene editing system comprising: (a) a Type V CRISPR nuclease polypeptide or a first nucleic acid encoding the Type V CRISPR nuclease polypeptide; (b) a reverse transcriptase (RT) polypeptide or a second nucleic acid encoding the RT polypeptide; (c) a guide RNA (gRNA) or a third nucleic acid encoding the gRNA, wherein the gRNA comprises one or more binding sites recognizable by the Type V CRISPR nuclease (CRISPR nuclease binding sites) and a spacer sequence specific to a target sequence within a genomic site of interest, the target sequence being adjacent to a protospacer adjacent motif (PAM); and (d) a reverse transcription donor RNA (RT donor RNA) or a fourth nucleic acid encoding the RT donor RNA, wherein the RT donor RNA comprises a primer binding site (PBS) and a template sequence.
05 - Pharmaceutical, veterinary and sanitary products
42 - Scientific, technological and industrial services, research and design
Goods & Services
Pharmaceutical preparations and therapeutic pharmaceuticals for the treatment of genetic diseases and disorders, genomic diseases and disorders, and cancer; gene therapy products, namely, genetically engineered DNA molecules and gene delivery pharmaceuticals for use in the treatment of genetic diseases and disorders, genomic diseases and disorders, and cancer; cell therapy products, namely, cells for medical and clinical use Pharmaceutical research and development; research and development in the fields of gene therapy and cell therapy
05 - Pharmaceutical, veterinary and sanitary products
42 - Scientific, technological and industrial services, research and design
Goods & Services
Pharmaceutical preparations and therapeutic pharmaceuticals for the treatment of genetic diseases and disorders, genomic diseases and disorders, and cancer; gene therapy products, namely, genetically engineered DNA molecules and gene delivery pharmaceuticals for use in the treatment of genetic diseases and disorders, genomic diseases and disorders, and cancer; cell therapy products, namely, cells for medical and clinical use Pharmaceutical research and development; research and development in the fields of gene therapy and cell therapy
The disclosure describes novel systems, methods, and compositions for the manipulation of nucleic acids in a targeted fashion. The disclosure describes non-naturally occurring, engineered CRISPR systems, components, and methods for targeted modification of nucleic acids. Each system includes one or more protein components and one or more nucleic acid components that together target nucleic acids.
A system for genetic editing of a transthyretin (TTR) gene, comprising (i) a Cas12i2 polypeptide or a first nucleic acid encoding the Cas12i2 polypeptide, and (ii) an RNA guide or a second nucleic acid encoding the RNA guide, wherein the RNA guide comprises a spacer sequence specific to a target sequence within an TTR gene. Also provided herein are methods for editing a TTR gene using the gene editing system disclosed herein and/or for treating diseases associated with the TTR gene.
A61K 31/7105 - Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
C12N 15/90 - Stable introduction of foreign DNA into chromosome
80.
GENE EDITING SYSTEMS COMPRISING AN RNA GUIDE TARGETING TRANSTHYRETIN (TTR) AND USES THEREOF
A system for genetic editing of a transthyretin (TTR) gene, comprising (i) a Cas12i2 polypeptide or a first nucleic acid encoding the Cas12i2 polypeptide, and (ii) an RNA guide or a second nucleic acid encoding the RNA guide, wherein the RNA guide comprises a spacer sequence specific to a target sequence within an TTR gene. Also provided herein are methods for editing a TTR gene using the gene editing system disclosed herein and/or for treating diseases associated with the TTR gene.
Provided herein are gene editing systems and/or compositions comprising RNA guides targeting HAO1 for use in genetic editing of the HAO1 gene. Also provide herein are methods of using the gene editing system for introducing edits to the HAO1 gene and/or for treatment of primary hyperoxaluria (PH), and processes for characterizing the gene editing system.
Provided herein are gene editing systems and/or compositions comprising RNA guides targeting LDHA for use in genetic editing of the LDHA gene. Also provide herein are methods of using the gene editing system for introducing edits to the LDHA gene and/or for treatment of primary hyperoxaluria (PH), and processes for characterizing the gene editing system.
A gene editing system comprising : (a) a Type V CRISPR nuclease polypeptide or a first nucleic acid encoding the Type V CRISPR nuclease polypeptide; (b) a reverse transcriptase (RT) polypeptide or a second nucleic acid encoding the RT polypeptide; (c) a guide RNA (gRNA) or a third nucleic acid encoding the gRNA, wherein the gRNA comprises one or more binding sites recognizable by the Type V CRISPR nuclease (CRISPR nuclease binding sites) and a spacer sequence specific to a target sequence within a genomic site of interest, the target sequence being adjacent to a protospacer adjacent motif (PAM); and (d) a reverse transcription donor RNA (RT donor RNA) or a fourth nucleic acid encoding the RT donor RNA, wherein the RT donor RNA comprises a primer binding site (PBS) and a template sequence.
The disclosure describes novel systems, methods, and compositions for the manipulation of nucleic acids in a targeted fashion. The disclosure describes non-naturally occurring, engineered CRISPR systems, components, and methods for targeted modification of nucleic acids. Each system includes one or more protein components and one or more nucleic acid components that together target nucleic acids.
The disclosure describes novel systems, methods, and compositions for the manipulation of nucleic acids in a targeted fashion. The disclosure describes non-naturally occurring, engineered CRISPR-Cas systems, components, and methods for targeted modification of nucleic acids such as DNA. Each system includes one or more protein components and one or more nucleic acid components that together target nucleic acids.
C12N 15/11 - DNA or RNA fragmentsModified forms thereof
C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
05 - Pharmaceutical, veterinary and sanitary products
40 - Treatment of materials; recycling, air and water treatment,
42 - Scientific, technological and industrial services, research and design
Goods & Services
(1) Pharmaceutical preparations and therapeutic pharmaceuticals for the treatment of genetic diseases and disorders, genomic diseases and disorders, and cancer; gene therapy products, namely, pharmaceutical preparations for use in gene therapy and biological preparations made from living tissues for use in gene therapy; cell therapy products, namely, pharmaceutical preparations for use in gene therapy and biological preparations made from living tissues for use in gene therapy. (1) Manufacturing of pharmaceuticals, gene therapy products, namely, pharmaceutical preparations for use in gene therapy and biological preparations made from living tissues for use in gene therapy, and cell therapy products, namely, pharmaceutical preparations for use in cell therapy and biological preparations made from living tissues for use in cell therapy for others.
(2) Pharmaceutical research and development; research and development in the fields of gene therapy and cell therapy.
The disclosure describes novel systems, methods, and compositions for the manipulation of nucleic acids in a targeted fashion. The disclosure describes non-naturally occurring, engineered CRISPR systems, components, and methods for targeted modification of nucleic acids such as DNA. Each system includes one or more protein components and one or more nucleic acid components that together target nucleic acids.
The disclosure describes novel systems, methods, and compositions for the manipulation of nucleic acids in a targeted fashion. The disclosure describes non-naturally occurring, engineered CRISPR-Cas systems, components, and methods for targeted modification of nucleic acids such as DNA. Each system includes one or more protein components and one or more nucleic acid components that together target nucleic acids.
C12N 15/11 - DNA or RNA fragmentsModified forms thereof
C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
89.
COMPOSITIONS COMPRISING A VARIANT POLYPEPTIDE AND USES THEREOF
The present invention relates to variant polypeptides, methods of preparing the variant polypeptides, processes for characterizing the variant polypeptides, compositions and cells comprising the variant polypeptides, and methods of using the variant polypeptides. The invention further relates to complexes comprising the variant polypeptides, methods of producing the complexes, processes for characterizing the complexes, cells comprising the complexes, and methods of using the complexes.
The present invention relates to variant polypeptides, methods of preparing the variant polypeptides, processes for characterizing the variant polypeptides, compositions and cells comprising the variant polypeptides, and methods of using the variant polypeptides. The invention further relates to complexes comprising the variant polypeptides, methods of producing the complexes, processes for characterizing the complexes, cells comprising the complexes, and methods of using the complexes.
The disclosure describes novel systems, methods, and compositions for the manipulation of nucleic acids in a targeted fashion. The disclosure describes non-naturally occurring, engineered CRISPR systems, components, and methods for targeted modification of nucleic acids. Each system includes one or more protein components and one or more nucleic acid components that together target nucleic acids.
The disclosure describes novel systems, methods, and compositions for the manipulation of nucleic acids in a targeted fashion. The disclosure describes non-naturally occurring, engineered CRISPR systems, components, and methods for targeted modification of nucleic acids. Each system includes one or more protein components and one or more nucleic acid components that together target nucleic acids.
The present invention relates to variant Cas12i4 polypeptides, methods of producing the variant Cas12i4 polypeptides, processes for characterizing the variant Cas12i4 polypeptides, cells comprising the variant Cas12i4 polypeptides, and methods of using the variant Cas12i4 polypeptides. The invention further relates to complexes comprising a variant Cas12i4 polypeptide and an RNA guide, methods of producing the complexes, processes for characterizing the complexes, cells comprising the complexes, and methods of using the complexes.
The present invention relates to nucleases or nucleic acids encoding the nucleases, RNA guides or nucleic acids encoding the RNA guides, processes for characterizing the nucleases and/or RNA guides, compositions comprising the nucleases and/or RNA guides, and kits and/or methods for preparing and/or using the nucleases and/or RNA guides.
The disclosure describes Casl2i2 fusion proteins, methods, and compositions for the manipulation of nucleic acids in a targeted fashion. The disclosure describes non-naturally occurring, engineered Casl2i2 fusion proteins, components, and methods for targeted modification of nucleic acids. Each system, includes one or more protein components and one or more nucleic acid components that together target nucleic acids.
The present invention relates to variant CRISPR nuclease polypeptides, methods of preparing the variant CRISPR nuclease polypeptides, processes for characterizing the variant CRISPR nuclease polypeptides, compositions and cells comprising the variant CRISPR nuclease polypeptides, and methods of using the variant CRISPR nuclease polypeptides. The invention further relates to complexes comprising a variant CRISPR nuclease polypeptide, methods of producing the complexes, processes for characterizing the complexes, cells comprising the complexes, and methods of using the complexes.
The present invention relates to compositions comprising RNA guides targeting B2M, processes for characterizing the compositions, cells comprising the compositions, and methods of using the compositions.
The present invention relates to compositions comprising RNA guides targeting PDCD1, processes for characterizing the compositions, cells comprising the compositions, and methods of using the compositions.
