Patents by Inventor Amit Nathwani
Amit Nathwani has filed for patents to protect the following inventions. This listing includes patent applications that are pending as well as patents that have already been granted by the United States Patent and Trademark Office (USPTO).
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Patent number: 11723925Abstract: There is described a chimeric antigen receptor (CAR) which comprises an antigen binding domain which selectively binds to Receptor Tyrosine Kinase Like Orphan Receptor 1 (ROR1), and its use. Also described is a T cell comprising the CAR and its use in the treatment of cancer.Type: GrantFiled: July 5, 2018Date of Patent: August 15, 2023Assignee: UCL Business LTDInventors: Amit Nathwani, Satyen Gohil, Marco Della Peruta
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Publication number: 20230241180Abstract: There is provided a nucleic acid molecule comprising a nucleotide sequence encoding for a functional factor VIII protein, wherein the portion of the nucleotide sequence encoding for the B domain of the factor VIII protein is between 90 and 111 nucleotides in length and encodes for an amino acid sequence comprising a sequence having at least 85% identity to SEQ ID NO: 4 and which comprises six asparagine residues. Also provided is a functional factor VIII protein, a vector comprising the above nucleic acid molecule, a host cell, a transgenic animal, a method of treating haemophilia, e.g. haemophilia A, and a method for the preparation of a parvoviral gene delivery vector.Type: ApplicationFiled: August 3, 2022Publication date: August 3, 2023Inventors: Amit Nathwani, Jenny McIntosh, Edward Tuddenham, Andrew Davidoff
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Publication number: 20230220073Abstract: There is described Receptor Tyrosine Kinase Like Orphan Receptor 1 (ROR1) antibodies that specifically bind a ROR1 polypeptide, and their use. In particular, isolated monoclonal antibodies are described and their use in a number of applications, including in the detection, prevention and treatment of cancer.Type: ApplicationFiled: July 21, 2022Publication date: July 13, 2023Inventors: Amit Nathwani, Satyen Gohil, Marco Della Peruta
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Patent number: 11517631Abstract: The present invention relates to polynucleotides comprising a Factor IX nucleotide sequence, wherein the Factor IX nucleotide sequence comprises a coding sequence that encodes a Factor IX protein or fragment thereof and wherein a portion of the coding sequence is not wild type. The present invention further relates to viral particles comprising a recombinant genome comprising the polynucleotide of the invention, compositions comprising the polynucleotides or viral particles, and methods and uses of the polynucleotides, viral particles or compositions.Type: GrantFiled: April 30, 2019Date of Patent: December 6, 2022Assignee: UCL BUSINESS LTDInventors: Amit Nathwani, Jenny McIntosh
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Publication number: 20220339262Abstract: The invention provides improved adeno-associated virus (AAV) Factor VIII (FVIII) vectors, including AAV FVIII vectors that produce a functional Factor VIII polypeptide and AAV FVIII vectors with high expression activity.Type: ApplicationFiled: June 30, 2022Publication date: October 27, 2022Inventors: Peter Cameron Colosi, Amit Nathwani, Jenny McIntosh, Edward Tuddenham, Andrew Davidoff
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Publication number: 20220331409Abstract: The invention relates to a new, more potent, coagulation Factor IX (FIX) expression cassette for gene therapy of Haemophilia B (HB). Disclosed is a vector for expressing factor IX protein, the vector comprising a promoter, a nucleotide sequence encoding for a functional factor IX protein, and an intron sequence, wherein the intron sequence is positioned between exon 1 and exon 2 of the nucleotide sequence encoding for a functional factor IX protein, and wherein the intron sequence has at least 80% identity to the sequence of SEQ ID NO. 1 as disclosed herein.Type: ApplicationFiled: April 29, 2022Publication date: October 20, 2022Inventors: Amit NATHWANI, Jenny MCINTOSH, Nishil PATEL
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Patent number: 11466083Abstract: There is described Receptor Tyrosine Kinase Like Orphan Receptor 1 (ROR1) antibodies that specifically bind a ROR1 polypeptide, and their use. In particular, isolated monoclonal antibodies are described and their use in a number of applications, including in the detection, prevention and treatment of cancer.Type: GrantFiled: July 5, 2018Date of Patent: October 11, 2022Assignee: UCL Business LTDInventors: Amit Nathwani, Satyen Gohil, Marco Della Peruta
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Patent number: 11419920Abstract: There is provided a nucleic acid molecule comprising a nucleotide sequence encoding a Factor VIII protein, wherein a B domain portion of the Factor VIII protein is encoded by a nucleotide sequence between 90 and 111 nucleotides in length and has an amino acid sequence that is at least 85% identical to SEQ ID NO: 4 which comprises six asparagine residues. Also provided is a Factor VIII protein, a vector comprising the above nucleic acid molecule, a host cell, a transgenic animal, a method of treating Haemophilia for example, Haemophilia A, and a method for the preparation of a parvoviral gene delivery vector.Type: GrantFiled: September 2, 2020Date of Patent: August 23, 2022Assignees: UCL BUSINESS LTD, ST. JUDE CHILDREN'S RESEARCH HOSPITALInventors: Amit Nathwani, Jenny McIntosh, Edward Tuddenham, Andrew Davidoff
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Patent number: 11406690Abstract: The present disclosure provides methods of generating multiplexed genetically modified animals, for example, porcine endogenous retrovirus (PERV)-inactivated pigs. The disclosure also provides methods of improving the birth rate of multiplexed genetically modified animals. In some embodiments, the present closure is concerned with the generation and utilization of porcine cells in which porcine endogenous retroviral (PERV) elements have been inactivated. In sonic embodiments, the PERV-free or PERV-reduced porcine cells are cloned to produce porcine embryos. In some embodiments, the PERV-free or PERV-reduced embryos may be grown into adult swine from which organs and/or tissues may be extracted and used for such purposes as xenotransplantation into non-porcine animals such as humans.Type: GrantFiled: September 30, 2019Date of Patent: August 9, 2022Assignees: BIOMARIN PHARMACEUTICAL INC., UCL BUSINESS LTD, ST. JUDE CHILDREN'S RESEARCH HOSPITALInventors: Peter Cameron Colosi, Amit Nathwani, Jenny McIntosh, Edward Tuddenham, Andrew Davidoff
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Publication number: 20220204621Abstract: There is described bispecific antibodies which selectively bind to Receptor Tyrosine Kinase Like Orphan Receptor 1 (ROR1) and the CD3 subunit of the T-Cell Receptor (TCR), their production and their use. Also described is the use of the bispecific antibodies in the treatment of cancer.Type: ApplicationFiled: March 18, 2022Publication date: June 30, 2022Inventors: Amit Nathwani, Satyen Gohil, Marco Della Peruta
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Patent number: 11344608Abstract: The invention relates to a new, more potent, coagulation Factor IX (FIX) expression cassette for gene therapy of Haemophilia B (HB). Disclosed is a vector for expressing factor IX protein, the vector comprising a promoter, a nucleotide sequence encoding for a functional factor IX protein, and an intron sequence, wherein the intron sequence is positioned between exon 1 and exon 2 of the nucleotide sequence encoding for a functional factor IX protein, and wherein the intron sequence has at least 80% identity to the sequence of SEQ ID NO. 1 as disclosed herein.Type: GrantFiled: September 6, 2019Date of Patent: May 31, 2022Assignee: UCL BUSINESS LTDInventors: Amit Nathwani, Jenny Mcintosh, Nishil Patel
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Publication number: 20220154159Abstract: The present invention relates to polynucleotides comprising a GBA nucleotide sequence that encodes a GCase protein or fragment thereof and wherein a portion of the coding sequence is not wild type. The present invention further relates to viral particles comprising a recombinant genome comprising the polynucleotide of the invention, compositions comprising the polynucleotides or viral particles, and methods and uses of the polynucleotides, viral particles or compositions.Type: ApplicationFiled: February 4, 2020Publication date: May 19, 2022Inventors: Amit NATHWANI, Jenny MCINTOSH, Romuald CORBAU, Azadeh KIA, Carlos MIRANDA
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Patent number: 11306142Abstract: There is described bispecific antibodies which selectively bind to Receptor Tyrosine Kinase Like Orphan Receptor 1 (ROR1) and the CD3 subunit of the T-Cell Receptor (TCR), their production and their use. Also described is the use of the bispecific antibodies in the treatment of cancer.Type: GrantFiled: July 5, 2018Date of Patent: April 19, 2022Assignee: UCL Business LTDInventors: Amit Nathwani, Satyen Gohil, Marco Della Peruta
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Publication number: 20220016263Abstract: There is described a nucleic acid molecule comprising a nucleotide sequence encoding for a functional ?-galactosidase A protein wherein the nucleotide sequence has at least 85% identity to the sequence of SEQ ID NO. 1. Also described is a vector, host cell or transgenic animal comprising the nucleic acid molecule; and a pharmaceutical composition comprising the nucleic acid molecule or the vector. Further, the use of the nucleic acid molecule in a method of treating Fabry disease is described.Type: ApplicationFiled: July 27, 2021Publication date: January 20, 2022Inventors: Amit Nathwani, Deepak Raj
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Publication number: 20210395714Abstract: The present invention relates to modified Factor IX polypeptides comprising a mutation at a position corresponding to position 347 of wild type immature (precursor) Factor IX, polynucleotides encoding the polypeptides, and treatments utilising the polypeptides or polynucleotides.Type: ApplicationFiled: October 30, 2019Publication date: December 23, 2021Inventors: Amit NATHWANI, Nishil PATEL, Keith GOMEZ
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Publication number: 20210309985Abstract: The present invention relates to polynucleotides comprising a Factor IX nucleotide sequence, wherein the Factor IX nucleotide sequence comprises a coding sequence that encodes a Factor IX protein or fragment thereof and wherein a portion of the coding sequence is not wild type. The present invention further relates to viral particles comprising a recombinant genome comprising the polynucleotide of the invention, compositions comprising the polynucleotides or viral particles, and methods and uses of the polynucleotides, viral particles or compositions.Type: ApplicationFiled: August 20, 2019Publication date: October 7, 2021Inventors: Amit NATHWANI, Jenny MCINTOSH
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Patent number: 11103596Abstract: There is described a nucleic acid molecule comprising a nucleotide sequence encoding for a functional ?-galactosidase A protein wherein the nucleotide sequence has at least 85% identity to the sequence of SEQ ID NO. 1. Also described is a vector, host cell or transgenic animal comprising the nucleic acid molecule; and a pharmaceutical composition comprising the nucleic acid molecule or the vector. Further, the use of the nucleic acid molecule in a method of treating Fabry disease is described.Type: GrantFiled: May 10, 2016Date of Patent: August 31, 2021Assignee: UCL BUSINESS PLCInventors: Amit Nathwani, Deepak Raj
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Patent number: 11077208Abstract: There is described a new gene therapy approach for treating Wilson's disease in which a nucleic acid molecule is used which comprises a nucleotide sequence encoding for a functional ATP7B protein wherein the nucleotide sequence has at least 85% identity to the sequence of SEQ ID NO: 1. Also described are vectors comprising the nucleotide sequence and methods and uses thereof.Type: GrantFiled: December 19, 2016Date of Patent: August 3, 2021Assignee: UCL Business LtdInventors: Amit Nathwani, Deepak Raj
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Publication number: 20210000978Abstract: An optimized coding sequence of human blood clotting factor eight (VIII) and a promoter may be used in vectors, such as rAAV, for introduction of factor VIII, and/or other blood clotting factors and transgenes. Exemplary of these factors and transgenes are alpha-1-antitrypsin, as well as those involved in the coagulation cascade, hepatocyte biology, lysosomal storage, urea cycle disorders, and lipid storage diseases. Cells, vectors, proteins, and glycoproteins produced by cells transformed by the vectors and sequence, may be used in treatment.Type: ApplicationFiled: June 10, 2020Publication date: January 7, 2021Inventors: Amit Nathwani, Natalie Ward, Adrian Thrasher, Edward Tuddenham, John McVey, John Gray, Andrew Davidoff
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Publication number: 20200405759Abstract: There is described a chimeric antigen receptor (CAR) which comprises an antigen binding domain which selectively binds to Receptor Tyrosine Kinase Like Orphan Receptor 1 (ROR1), and its use. Also described is a T cell comprising the CAR and its use in the treatment of cancer.Type: ApplicationFiled: July 5, 2018Publication date: December 31, 2020Inventors: Amit Nathwani, Satyen Gohil, Marco Della Peruta