44 - Medical, veterinary, hygienic and cosmetic services; agriculture, horticulture and forestry services
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Promoting collaboration within the scientific, research and
provider communities to achieve advances in the fields of
treatment, diagnosis and prophylaxis of cancers and
hematologic diseases, syndromes and disorders (Term
considered too vague by the International Bureau pursuant to
Rule 13 (2) (b) of the Regulations). Providing medical information in the field of the diagnosis
and treatment of cancers and hematologic malignancies to
consumers, and providing medical information in the field of
prevention, screening, diagnosis and treatment to
scientists, researchers and medical providers on cancers and
hematologic diseases, syndromes and disorders.
2.
OLIGONUCLEOTIDE COMPOSITIONS AND METHODS OF MAKING THE SAME
The present disclosure provides a solid phase method of making oligonucleotides via sequential coupling cycles including at least one coupling of a dinucleotide dimer subunit to a free 3′-terminal group of a growing chain. The oligonucleotides include at least two nucleoside subunits joined by a N3′→P5′ phosphoramidate linkage. The method may include the steps of (a) deprotecting the protected 3′ amino group of a terminal nucleoside attached to a solid phase support, said deprotecting forming a free 3′ amino group; (b) contacting the free 3′ amino group with a 3′-protected amino-dinucleotide-5′-phosphoramidite dimer in the presence of a nucleophilic catalyst to form an internucleoside N3′→P5′ phosphoramidite linkage; and (c) oxidizing (e.g., sulfurizing) the linkage. The compositions produced by the subject methods may include a reduced amount of one or more (N-x) oligonucleotide products. Also provided are pharmaceutical compositions including the subject oligonucleotide compositions.
C07H 21/00 - Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
C12P 19/34 - Polynucleotides, e.g. nucleic acids, oligoribonucleotides
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promoting public awareness of medical disorders and their treatment; promoting public awareness of treatment, diagnosis or prophylaxis of human diseases, disorders and conditions, namely, cancer, and diseases, disorders and conditions of the hematological systems providing a website featuring medical information; providing medical information
4.
Crystalline Solids of 3-Palmitoyl-Amido-1,2-Propanediol and 3-Palmitoyl-Amido-2-Hydroxy-1-Dimethoxytriphenylmethylether-Propane and Methods of Making and Using the Same
Aspects of the disclosure include crystalline solids of 3-palmitoyl-amido-1,2-propanediol and 3-palmitoyl-amido-2-hydroxy-1-dimethoxytriphenylmethylether-propane. Methods for preparing the crystalline solids of 3-palmitoyl-amido-1,2-propanediol and single crystals of 3-palmitoyl-amido-2-hydroxy-1-dimethoxytriphenylmethylether-propane are also provided. Methods for preparing a 3-palmitoyl-amido-2-hydroxy-1-dimethoxytriphenylmethylether-propane from a crystalline solid of 3-palmitoyl-amido-1,2-propanediol are also described.
C07C 233/18 - Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms having the nitrogen atom of at least one of the carboxamide groups bound to a carbon atom of a hydrocarbon radical substituted by singly-bound oxygen atoms with the substituted hydrocarbon radical bound to the nitrogen atom of the carboxamide group by an acyclic carbon atom having the carbon atom of the carboxamide group bound to a hydrogen atom or to a carbon atom of an acyclic saturated carbon skeleton
36 - Financial, insurance and real estate services
45 - Legal and security services; personal services for individuals.
Goods & Services
Financial administration of patient financial assistance
programs for eligible patients. Providing personal support services for patients with
medical conditions, namely, help with navigating access to a
pharmaceutical by providing information on health insurance
benefits, prior authorization, and information on health
insurance appeals.
Provided are telomerase inhibitor compounds. Some compounds include a lactone or lactam group that is covalently bonded to a phenyl ring, which is itself bonded to a pyrazole group. In other cases, a sulfonamide-containing moiety is covalently bonded to a phenyl ring, which is itself bonded to a pyrazole group. In some embodiments, the telomerase inhibitor compound has a vinyl sulfonamide group bonded to an amide moiety and an aromatic group. In other cases, the inhibitor compound has an isothiazolidine 1,1-dioxide core that is bonded to a phenyl group. Aspects of the invention also include pharmaceutical compositions comprising the subject telomerase inhibitor compounds, as well as methods of treating telomerase-related diseases or conditions.
C07D 405/10 - Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing two hetero rings linked by a carbon chain containing aromatic rings
A61K 31/4155 - 1,2-Diazoles not condensed and containing further heterocyclic rings
C07D 403/10 - Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group containing two hetero rings linked by a carbon chain containing aromatic rings
7.
Method for Identification of Sensitivity of a Patient to Telomerase Inhibition Therapy
The invention provides methods for determining the susceptibility of cancer patients to developing adverse reactions if treated with a telomerase inhibitor drug by measurement of telomere length in appropriate cells of the patient prior to initiation of the telomerase inhibitor treatment.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
C12Q 1/6883 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
G16B 30/00 - ICT specially adapted for sequence analysis involving nucleotides or amino acids
8.
Synthesis of Protected 3'-amino Nucleoside Monomers
Orthogonally protected 3′-amino nucleoside monomers and efficient methods for their synthesis are described. The methods employ selective protection of the 3′-amino group in the presence of the unprotected nucleoside base.
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(1) Financial administration of patient financial assistance programs for eligible patients.
(2) Providing personal support services for patients with medical conditions, namely, help with navigating access to a pharmaceutical by providing information on health insurance benefits, prior authorization, and information on health insurance appeals.
10.
METHODS OF TREATING MYELODYSPLASTIC SYNDROME AND MONITORING THE TREATMENT
Methods of monitoring therapeutic efficacy in a subject with myelodysplastic syndrome (MDS) are provided. Also provided is a method of identifying a subject with MDS for treatment with a telomerase inhibitor, and methods of treating MDS. The methods include administering to the subject a telomerase inhibitor and assessing variant allele frequency (VAF) for one or more of the following genes: SF3B1, TET2, DNMT3A, ASXL1, and CUX1 in a biological sample obtained from the subject after administration of the telomerase inhibitor. In some cases, a 25% or more reduction in VAF identifies a subject who has an increased likelihood of benefiting from treatment with a telomerase inhibitor. In some instances, the telomerase inhibitor is imetelstat or imetelstat sodium.
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
Provided are telomerase inhibitor compounds. Some compounds include a lactone or lactam group that is covalently bonded to a phenyl ring, which is itself bonded to a pyrazole group. In other cases, a sulfonamide-containing moiety is covalently bonded to a phenyl ring, which is itself bonded to a pyrazole group. In some embodiments, the telomerase inhibitor compound has a vinyl sulfonamide group bonded to an amide moiety and an aromatic group. In other cases, the inhibitor compound has an isothiazolidine 1,1-dioxide core that is bonded to a phenyl group. Aspects of the invention also include pharmaceutical compositions comprising the subject telomerase inhibitor compounds, as well as methods of treating telomerase-related diseases or conditions.
A61K 31/341 - Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having five-membered rings with one oxygen as the only ring hetero atom, e.g. isosorbide not condensed with another ring, e.g. ranitidine, furosemide, bufetolol, muscarine
A61K 31/4015 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil having oxo groups directly attached to the heterocyclic ring, e.g. piracetam, ethosuximide
The present invention relates to a process for preparing the telomerase inhibitor imetelstat using a 3 steps per cycle solid-phase support bound process comprising the steps of deprotection of the 3′-amino group of the support-bound oligonucleotide, coupling with a 5′-phosphoramidite, and sulfurization with an acyl disulfide, characterized by the absence of an additional capping step in each cycle that is used to prevent unreacted 3′-amino oligonucleotide groups from reacting during subsequent cycles. Imetelstat has formula below.
The present invention relates to a process for preparing the telomerase inhibitor imetelstat using a 3 steps per cycle solid-phase support bound process comprising the steps of deprotection of the 3′-amino group of the support-bound oligonucleotide, coupling with a 5′-phosphoramidite, and sulfurization with an acyl disulfide, characterized by the absence of an additional capping step in each cycle that is used to prevent unreacted 3′-amino oligonucleotide groups from reacting during subsequent cycles. Imetelstat has formula below.
C07H 1/00 - Processes for the preparation of sugar derivatives
C07H 21/04 - Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
44 - Medical, veterinary, hygienic and cosmetic services; agriculture, horticulture and forestry services
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(1) Promoting collaboration within the scientific, research and provider communities to achieve advances in the fields of treatment, diagnosis and prophylaxis of cancers and hematologic diseases, syndromes and disorders (Term considered too vague by the International Bureau pursuant to Rule 13 (2) (b) of the Regulations).
(2) Providing medical information in the field of the diagnosis and treatment of cancers and hematologic malignancies to consumers, and providing medical information in the field of prevention, screening, diagnosis and treatment to scientists, researchers and medical providers on cancers and hematologic diseases, syndromes and disorders.
14.
METHODS OF TREATING MYELODYSPLASTIC SYNDROME AND MONITORING THE TREATMENT
Methods of monitoring therapeutic efficacy in a subject with myelodysplastic syndrome (MDS) are provided. Also provided is a method of identifying a subject with MDS for treatment with a telomerase inhibitor, and methods of treating MDS. The methods include administering to the subject a telomerase inhibitor and assessing variant allele frequency (VAF) for one or more of the following genes: SF3B1, TET2, DNMT3A, ASXL1, and CUX1 in a biological sample obtained from the subject after administration of the telomerase inhibitor. In some cases, a 25% or more reduction in VAF identifies a subject who has an increased likelihood of benefiting from treatment with a telomerase inhibitor. In some instances, the telomerase inhibitor is imetelstat or imetelstat sodium.
C12Q 1/6883 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
A61K 31/7088 - Compounds having three or more nucleosides or nucleotides
A61P 7/00 - Drugs for disorders of the blood or the extracellular fluid
C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
The present invention relates to a process for preparing the telomerase inhibitor imetelstat using a 3 steps per cycle solid-phase support bound process comprising the steps of deprotection of the 3′-amino group of the support-bound oligonucleotide, coupling with a 5′-phosphoramidite, and sulfurization with an acyl disulfide, characterized by the absence of an additional capping step in each cycle that is used to prevent unreacted 3′-amino oligonucleotide groups from reacting during subsequent cycles. Imetelstat has formula below.
The present invention relates to a process for preparing the telomerase inhibitor imetelstat using a 3 steps per cycle solid-phase support bound process comprising the steps of deprotection of the 3′-amino group of the support-bound oligonucleotide, coupling with a 5′-phosphoramidite, and sulfurization with an acyl disulfide, characterized by the absence of an additional capping step in each cycle that is used to prevent unreacted 3′-amino oligonucleotide groups from reacting during subsequent cycles. Imetelstat has formula below.
C07H 1/00 - Processes for the preparation of sugar derivatives
C07H 21/04 - Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
16.
OLIGONUCLEOTIDE COMPOSITIONS AND METHODS OF MAKING THE SAME
The present disclosure provides a solid phase method of making oligonucleotides via sequential coupling cycles including at least one coupling of a dinucleotide dimer subunit to a free 3′-terminal group of a growing chain. The oligonucleotides include at least two nucleoside subunits joined by a N3′→P5′ phosphoramidate linkage. The method may include the steps of (a) deprotecting the protected 3′ amino group of a terminal nucleoside attached to a solid phase support, said deprotecting forming a free 3′ amino group; (b) contacting the free 3′ amino group with a 3′-protected amino-dinucleotide-5′-phosphoramidite dimer in the presence of a nucleophilic catalyst to form an internucleoside N3′→P5′ phosphoramidite linkage; and (c) oxidizing (e.g., sulfurizing) the linkage. The compositions produced by the subject methods may include a reduced amount of one or more (N-x) oligonucleotide products. Also provided are pharmaceutical compositions including the subject oligonucleotide compositions.
C07H 21/00 - Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
C12P 19/34 - Polynucleotides, e.g. nucleic acids, oligoribonucleotides
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Financial administration in the nature of patient financial assistance programs for eligible patients Providing patient advocate services for patients with medical conditions, namely, help with navigating access to a pharmaceutical by providing information on health insurance benefits, prior authorization, and information on health insurance appeals
44 - Medical, veterinary, hygienic and cosmetic services; agriculture, horticulture and forestry services
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Promoting collaboration within the scientific, research and provider communities to achieve advances in the fields of treatment, diagnosis and prophylaxis of cancers and hematologic diseases, syndromes and disorders. Providing medical information in the field of the diagnosis and treatment of cancers and hematologic malignancies to consumers, and providing medical information in the field of prevention, screening, diagnosis and treatment to scientists, researchers and medical providers on cancers and hematologic diseases, syndromes and disorders.
05 - Pharmaceutical, veterinary and sanitary products
Goods & Services
Pharmaceuticals for the treatment, diagnosis or prophylaxis
of human diseases, disorders and conditions, namely, cancer,
and diseases, disorders and conditions of the hematological
systems.
05 - Pharmaceutical, veterinary and sanitary products
Goods & Services
Pharmaceuticals for the treatment, diagnosis or prophylaxis
of human diseases, disorders and conditions, namely, cancer,
and diseases, disorders and conditions of the hematological
systems.
44 - Medical, veterinary, hygienic and cosmetic services; agriculture, horticulture and forestry services
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Promoting collaboration within the scientific, research and provider communities to achieve advances in the fields of treatment, diagnosis and prophylaxis of cancers and hematologic diseases, syndromes and disorders Providing medical information in the field of the diagnosis and treatment of cancers and hematologic malignancies to consumers, and providing medical information in the field of prevention, screening, diagnosis and treatment to scientists, researchers and medical providers on cancers and hematologic diseases, syndromes and disorders
05 - Pharmaceutical, veterinary and sanitary products
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(1) Pharmaceuticals for the treatment, diagnosis or prophylaxis of human diseases, disorders and conditions, namely, cancer, and diseases, disorders and conditions of the hematological systems.
05 - Pharmaceutical, veterinary and sanitary products
Goods & Services
(1) Pharmaceuticals for the treatment, diagnosis or prophylaxis of human diseases, disorders and conditions, namely, cancer, and diseases, disorders and conditions of the hematological systems.
24.
Amorphous solid succinylated 3-(fatty acid amido)-2-hydroxy-1- (protected hydroxy)-propane salts and methods of making the same
Aspects of the disclosure includes methods for preparing an amorphous solid composition of a fatty acid metal salt. In practicing the subject methods according to certain embodiments, a succinylated 3-(fatty acid amido)-2-hydroxy-1-(protected hydroxy)-propane organic salt is contacted with a metal base to produce a succinylated 3-(fatty acid amido)-2-hydroxy-1-(protected hydroxy)-propane metal salt; and the succinylated 3-(fatty acid amido)-2-hydroxy-1-(protected hydroxy)-propane metal salt is precipitated in a solvent to produce an amorphous solid succinylated 3-(fatty acid amido)-2-hydroxy-1-(protected hydroxy)-propane metal salt composition. An amorphous solid succinylated 3-(fatty acid amido)-2-hydroxy-1-(protected hydroxy)-propane lithium salt is also provided.
Provided herein are methods for reducing neoplastic progenitor cell proliferation and alleviating symptoms associated in individuals diagnosed with or thought to have Essential Thrombocythemia (ET). Also provided herein are methods for using telomerase inhibitors for maintaining blood platelet counts at relatively normal ranges in the blood of individuals diagnosed with or suspected of having ET.
C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
B82Y 5/00 - Nanobiotechnology or nanomedicine, e.g. protein engineering or drug delivery
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
05 - Pharmaceutical, veterinary and sanitary products
Goods & Services
pharmaceuticals for the treatment, diagnosis or prophylaxis of human diseases, disorders and conditions, namely, cancer, and diseases, disorders and conditions of the hematological systems
05 - Pharmaceutical, veterinary and sanitary products
Goods & Services
pharmaceuticals for the treatment, diagnosis or prophylaxis of human diseases, disorders and conditions, namely, cancer, and diseases, disorders and conditions of the hematological systems
05 - Pharmaceutical, veterinary and sanitary products
Goods & Services
(1) Pharmaceuticals for the treatment, diagnosis or prophylaxis of human diseases, disorders and conditions, namely, cancer, and diseases, disorders and conditions of the hematological systems.
05 - Pharmaceutical, veterinary and sanitary products
Goods & Services
(1) Pharmaceuticals for the treatment, diagnosis or prophylaxis of human diseases, disorders and conditions, namely, cancer, and diseases, disorders and conditions of the hematological systems.
05 - Pharmaceutical, veterinary and sanitary products
Goods & Services
Pharmaceuticals for the treatment, diagnosis or prophylaxis
of human diseases, disorders and conditions, namely, cancer,
and diseases, disorders and conditions of the hematological
systems.
05 - Pharmaceutical, veterinary and sanitary products
Goods & Services
Pharmaceuticals for the treatment, diagnosis or prophylaxis
of human diseases, disorders and conditions, namely, cancer,
and diseases, disorders and conditions of the hematological
systems.
32.
Guanine Analogs as Telomerase Substrates and Telomere Length Affectors
This invention relates to compounds useful for inhibiting telomere elongation. More specifically, the invention provides nucleotide analogs that are incorporated into telomeres by telomerase thereby inhibiting elongation of telomeres. The compounds are use in treating cancer and other cell proliferative diseases.
C07F 9/6561 - Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing systems of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring or ring system, with or without other non-condensed hetero rings
C07D 473/18 - Heterocyclic compounds containing purine ring systems with oxygen, sulfur, or nitrogen atoms directly attached in positions 2 and 6 one oxygen and one nitrogen atom, e.g. guanine
33.
Oligonucleotide compositions and methods of making the same
The present disclosure provides a solid phase method of making oligonucleotides via sequential coupling cycles including at least one coupling of a dinucleotide dimer subunit to a free 3′-terminal group of a growing chain. The oligonucleotides include at least two nucleoside subunits joined by a N3′→P5′ phosphoramidate linkage. The method may include the steps of (a) deprotecting the protected 3′ amino group of a terminal nucleoside attached to a solid phase support, said deprotecting forming a free 3′ amino group; (b) contacting the free 3′ amino group with a 3′-protected amino-dinucleotide-5′-phosphoramidite dimer in the presence of a nucleophilic catalyst to form an internucleoside N3′→P5′ phosphoramidite linkage; and (c) oxidizing (e.g., sulfurizing) the linkage. The compositions produced by the subject methods may include a reduced amount of one or more (N−x) oligonucleotide products. Also provided are pharmaceutical compositions including the subject oligonucleotide compositions.
C07H 21/00 - Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
C12P 19/34 - Polynucleotides, e.g. nucleic acids, oligoribonucleotides
05 - Pharmaceutical, veterinary and sanitary products
Goods & Services
pharmaceuticals for the treatment, diagnosis or prophylaxis of human diseases, disorders and conditions, namely, cancer, and diseases, disorders and conditions of the hematological systems
05 - Pharmaceutical, veterinary and sanitary products
Goods & Services
pharmaceuticals for the treatment, diagnosis or prophylaxis of human diseases, disorders and conditions, namely, cancer, and diseases, disorders and conditions of the hematological systems
05 - Pharmaceutical, veterinary and sanitary products
35 - Advertising and business services
44 - Medical, veterinary, hygienic and cosmetic services; agriculture, horticulture and forestry services
Goods & Services
Therapeutic, diagnostic, and prophylactic preparations,
pharmaceutical products and agents for the treatment,
diagnosis or prophylaxis of human diseases, disorders and
conditions, namely, cancers and hematologic diseases,
syndromes and disorders. Promoting collaboration within the scientific, research and
provider communities to achieve advances in the fields of
treatment, diagnosis and prophylaxis of cancers and
hematologic diseases, syndromes and disorders. Providing medical information in the field of the diagnosis
and treatment of cancers and hematologic malignancies to
consumers, and providing medical information in the field of
prevention, screening, diagnosis and treatment to
scientists, researchers and medical providers on cancers and
hematologic diseases, syndromes and disorders.
05 - Pharmaceutical, veterinary and sanitary products
35 - Advertising and business services
44 - Medical, veterinary, hygienic and cosmetic services; agriculture, horticulture and forestry services
Goods & Services
Therapeutic, diagnostic, and prophylactic preparations,
pharmaceutical products and agents for the treatment,
diagnosis or prophylaxis of human diseases, disorders and
conditions, namely, cancers and hematologic diseases,
syndromes and disorders. Promoting collaboration within the scientific, research and
provider communities to achieve advances in the fields of
treatment, diagnosis and prophylaxis of cancers and
hematologic diseases, syndromes and disorders. Providing medical information in the field of the diagnosis
and treatment of cancers and hematologic malignancies to
consumers, and providing medical information in the field of
prevention, screening, diagnosis and treatment to
scientists, researchers and medical providers on cancers and
hematologic diseases, syndromes and disorders.
38.
SUBCUTANEOUS TELOMERASE INHIBITOR COMPOSITIONS AND METHODS FOR USING SAME
Aspects of the disclosure include telomerase inhibitor compositions formulated for subcutaneous administration. Compositions according to certain embodiments include a hyaluronidase enzyme and a telomerase inhibitor having an oligonucleotide and a lipid moiety linked to the 5′ and/or 3′ end of the oligonucleotide. Methods for subcutaneously administering the telomerase inhibitor compositions, such as in the treatment of a neoplasm are also described. Kits having or not having a subcutaneous injector are also provided.
The present invention relates to a combination treatment for hematological cancers. More specifically; a combination of a telomerase inhibitor and a Bcl-2 inhibitor are useful in treating hematological cancers, including AML. In certain embodiments, the telomerase inhibitor is imetelstat or imetelstat sodium and the Bcl-2 inhibitor is ABT-199.
A61K 31/7125 - Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
A61K 31/635 - Compounds containing para-N-benzene- sulfonyl-N-groups, e.g. sulfanilamide, p-nitrobenzenesulfonohydrazide having a heterocyclic ring, e.g. sulfadiazine
A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
A61K 31/496 - Non-condensed piperazines containing further heterocyclic rings, e.g. rifampin, thiothixene or sparfloxacin
05 - Pharmaceutical, veterinary and sanitary products
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(1) Therapeutic, diagnostic, and prophylactic preparations, pharmaceutical products and agents for the treatment, diagnosis or prophylaxis of human diseases, disorders and conditions, namely, cancers and hematologic diseases, syndromes and disorders. (1) Advertising services for promoting collaboration, interest and awareness among members of the scientific, research and healthcare provider communities to achieve advances in the fields of treatment, diagnosis and prophylaxis of cancers and hematologic diseases, syndromes and disorders.
(2) Providing medical information in the field of the diagnosis and treatment of cancers and hematologic malignancies to consumers, and providing medical information in the field of prevention, screening, diagnosis and treatment to scientists, researchers and medical providers on cancers and hematologic diseases, syndromes and disorders.
05 - Pharmaceutical, veterinary and sanitary products
35 - Advertising and business services
44 - Medical, veterinary, hygienic and cosmetic services; agriculture, horticulture and forestry services
Goods & Services
(1) Therapeutic, diagnostic, and prophylactic preparations, pharmaceutical products and agents for the treatment, diagnosis or prophylaxis of human diseases, disorders and conditions, namely, cancers and hematologic diseases, syndromes and disorders. (1) Advertising services for promoting collaboration, interest and awareness among members of the scientific, research and healthcare provider communities to achieve advances in the fields of treatment, diagnosis and prophylaxis of cancers and hematologic diseases, syndromes and disorders.
(2) Providing medical information in the field of the diagnosis and treatment of cancers and hematologic malignancies to consumers, and providing medical information in the field of prevention, screening, diagnosis and treatment to scientists, researchers and medical providers on cancers and hematologic diseases, syndromes and disorders.
42.
SUBCUTANEOUS TELOMERASE INHIBITOR COMPOSITIONS AND METHODS FOR USING SAME
Aspects of the disclosure include telomerase inhibitor compositions formulated for subcutaneous administration. Compositions according to certain embodiments include a hyaluronidase enzyme and a telomerase inhibitor having an oligonucleotide and a lipid moiety linked to the 5' and/or 3' end of the oligonucleotide. Methods for subcutaneously administering the telomerase inhibitor compositions, such as in the treatment of a neoplasm are also described. Kits having or not having a subcutaneous injector are also provided.
A61K 31/7088 - Compounds having three or more nucleosides or nucleotides
C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
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
A61K 31/7125 - Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
A61K 38/27 - Growth hormone [GH], i.e. somatotropin
A61K 38/47 - Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
Aspects of the disclosure include telomerase inhibitor compositions formulated for subcutaneous administration. Compositions according to certain embodiments include a hyaluronidase enzyme and a telomerase inhibitor having an oligonucleotide and a lipid moiety linked to the 5' and/or 3' end of the oligonucleotide. Methods for subcutaneously administering the telomerase inhibitor compositions, such as in the treatment of a neoplasm are also described. Kits having or not having a subcutaneous injector are also provided.
C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
C12N 9/24 - Hydrolases (3.) acting on glycosyl compounds (3.2)
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
A61K 31/7125 - Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
A61K 31/7088 - Compounds having three or more nucleosides or nucleotides
A61K 38/27 - Growth hormone [GH], i.e. somatotropin
A61K 38/47 - Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
05 - Pharmaceutical, veterinary and sanitary products
35 - Advertising and business services
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Therapeutic, diagnostic, and prophylactic preparations, pharmaceutical products and agents for the treatment, diagnosis or prophylaxis of human diseases, disorders and conditions, namely, cancers and hematologic diseases, syndromes and disorders Promoting collaboration within the scientific, research and provider communities to achieve advances in the fields of treatment, diagnosis and prophylaxis of cancers and hematologic diseases, syndromes and disorders Providing medical information in the field of the diagnosis and treatment of cancers and hematologic malignancies to consumers, and providing medical information in the field of prevention, screening, diagnosis and treatment to scientists, researchers and medical providers on cancers and hematologic diseases, syndromes and disorders
05 - Pharmaceutical, veterinary and sanitary products
35 - Advertising and business services
44 - Medical, veterinary, hygienic and cosmetic services; agriculture, horticulture and forestry services
Goods & Services
Therapeutic, diagnostic, and prophylactic preparations, pharmaceutical products and agents for the treatment, diagnosis or prophylaxis of human diseases, disorders and conditions, namely, cancers and hematologic diseases, syndromes and disorders Promoting collaboration within the scientific, research and provider communities to achieve advances in the fields of treatment, diagnosis and prophylaxis of cancers and hematologic diseases, syndromes and disorders Providing medical information in the field of the diagnosis and treatment of cancers and hematologic malignancies to consumers, and providing medical information in the field of prevention, screening, diagnosis and treatment to scientists, researchers and medical providers on cancers and hematologic diseases, syndromes and disorders
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(1) Pharmaceuticals for the treatment, diagnosis or prophylaxis of human diseases, disorders and conditions, namely, cancer, and diseases, disorders and conditions of the hematological systems
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Goods & Services
(1) Pharmaceuticals for the treatment, diagnosis or prophylaxis of human diseases, disorders and conditions, namely, cancer, and diseases, disorders and conditions of the hematological systems
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(1) Pharmaceuticals for the treatment, diagnosis or prophylaxis of human diseases, disorders and conditions, namely, cancer, and diseases, disorders and conditions of the hematological systems
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(1) Pharmaceuticals for the treatment, diagnosis or prophylaxis of human diseases, disorders and conditions, namely, cancer, and diseases, disorders and conditions of the hematological systems
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(1) Pharmaceuticals for the treatment, diagnosis or prophylaxis of human diseases, disorders and conditions, namely, cancer, and diseases, disorders and conditions of the hematological systems
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(1) Pharmaceuticals for the treatment, diagnosis or prophylaxis of human diseases, disorders and conditions, namely, cancer, and diseases, disorders and conditions of the hematological systems.
58.
Crystalline solids of 3-palmitoyl-amido-1,2-propanediol and 3-palmitoyl-amido-2-hydroxy-1-dimethoxytriphenylmethylether-propane and methods of making and using the same
Aspects of the disclosure include crystalline solids of 3-palmitoyl-amido-1,2-propanediol and 3-palmitoyl-amido-2-hydroxy-1-dimethoxytriphenylmethylether-propane. Methods for preparing the crystalline solids of 3-palmitoyl-amido-1,2-propanediol and single crystals of 3-palmitoyl-amido-2-hydroxy-1-dimethoxytriphenylmethylether-propane are also provided. Methods for preparing a 3-palmitoyl-amido-2-hydroxy-1-dimethoxytriphenylmethylether-propane from a crystalline solid of 3-palmitoyl-amido-1,2-propanediol are also described.
C07C 233/18 - Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms having the nitrogen atom of at least one of the carboxamide groups bound to a carbon atom of a hydrocarbon radical substituted by singly-bound oxygen atoms with the substituted hydrocarbon radical bound to the nitrogen atom of the carboxamide group by an acyclic carbon atom having the carbon atom of the carboxamide group bound to a hydrogen atom or to a carbon atom of an acyclic saturated carbon skeleton
Aspects of the disclosure includes methods for preparing an amorphous solid composition of a fatty acid metal salt. In practicing the subject methods according to certain embodiments, a succinylated 3-(fatty acid amido)-2-hydroxy-1-(protected hydroxy)-propane organic salt is contacted with a metal base to produce a succinylated 3-(fatty acid amido)-2-hydroxy-1-(protected hydroxy)-propane metal salt; and the succinylated 3-(fatty acid amido)-2-hydroxy-1-(protected hydroxy)-propane metal salt is precipitated in a solvent to produce an amorphous solid succinylated 3-(fatty acid amido)-2-hydroxy-1-(protected hydroxy)-propane metal salt composition. An amorphous solid succinylated 3-(fatty acid amido)-2-hydroxy-1-(protected hydroxy)-propane lithium salt is also provided.
Aspects of the disclosure include methods for treating a myeloproliferative neoplasm. Methods according to certain embodiments include co-administering to a subject a Janus kinase (JAK) inhibitor and a telomerase inhibitor comprising an oligonucleotide and a lipid moiety linked to the 5' and/or 3' end of the oligonucleotide. Methods for inducing apoptosis of a myeloproliferative neoplasm cell by contacting the cell with an amount of a JAK inhibitor and a telomerase inhibitor sufficient to induce apoptosis are also described. Compositions having a JAK inhibitor and a telomerase inhibitor for practicing the subject methods are also provided.
A61K 31/7088 - Compounds having three or more nucleosides or nucleotides
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
A61K 31/7125 - Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
Aspects of the disclosure include methods for treating a myeloproliferative neoplasm. Methods according to certain embodiments include co-administering to a subject a Janus kinase (JAK) inhibitor and a telomerase inhibitor comprising an oligonucleotide and a lipid moiety linked to the 5' and/or 3' end of the oligonucleotide. Methods for inducing apoptosis of a myeloproliferative neoplasm cell by contacting the cell with an amount of a JAK inhibitor and a telomerase inhibitor sufficient to induce apoptosis are also described. Compositions having a JAK inhibitor and a telomerase inhibitor for practicing the subject methods are also provided.
A61K 31/7088 - Compounds having three or more nucleosides or nucleotides
A61K 31/7125 - Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
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
Aspects of the disclosure includes methods for preparing an amorphous solid composition of a fatty acid metal salt. In practicing the subject methods according to certain embodiments, a succinylated 3-(fatty acid amido)-2-hydroxy-1-(protected hydroxy)-propane organic salt is contacted with a metal base to produce a succinylated 3-(fatty acid amido)-2-hydroxy-1-(protected hydroxy)-propane metal salt; and the succinylated 3-(fatty acid amido)-2-hydroxy-1-(protected hydroxy)-propane metal salt is precipitated in a solvent to produce an amorphous solid succinylated 3-(fatty acid amido)-2-hydroxy-1-(protected hydroxy)-propane metal salt composition. An amorphous solid succinylated 3-(fatty acid amido)-2-hydroxy-1-(protected hydroxy)-propane lithium salt is also provided.
C07C 233/18 - Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms having the nitrogen atom of at least one of the carboxamide groups bound to a carbon atom of a hydrocarbon radical substituted by singly-bound oxygen atoms with the substituted hydrocarbon radical bound to the nitrogen atom of the carboxamide group by an acyclic carbon atom having the carbon atom of the carboxamide group bound to a hydrogen atom or to a carbon atom of an acyclic saturated carbon skeleton
Aspects of the disclosure includes methods for preparing an amorphous solid composition of a fatty acid metal salt. In practicing the subject methods according to certain embodiments, a succinylated 3-(fatty acid amido)-2-hydroxy-1-(protected hydroxy)-propane organic salt is contacted with a metal base to produce a succinylated 3-(fatty acid amido)-2-hydroxy-1-(protected hydroxy)-propane metal salt; and the succinylated 3-(fatty acid amido)-2-hydroxy-1-(protected hydroxy)-propane metal salt is precipitated in a solvent to produce an amorphous solid succinylated 3-(fatty acid amido)-2-hydroxy-1-(protected hydroxy)-propane metal salt composition. An amorphous solid succinylated 3-(fatty acid amido)-2-hydroxy-1-(protected hydroxy)-propane lithium salt is also provided.
C12Q 1/68 - Measuring or testing processes involving enzymes, nucleic acids or microorganismsCompositions thereforProcesses of preparing such compositions involving nucleic acids
64.
CRYSTALLINE SOLIDS OF 3-PALMITOYL-AMIDO-1,2-PROPANEDIOL AND 3-PALMITOYL-AMIDO-2-HYDROXY-1-DIMETHOXYTRIPHENYLMETHYLETHER-PROPANE AND METHODS OF MAKING AND USING THE SAME
Aspects of the disclosure include crystalline solids of 3-palmitoyl-amido-1,2- propanediol and 3-palmitoyl-amido-2-hydroxy-1-dimethoxytriphenylmethylether-propane. Methods for preparing the crystalline solids of 3-palmitoyl-amido-1,2- propanediol and single crystals of 3-palmitoyl-amido-2-hydroxy-1-dimethoxytriphenylmethylether-propane are also provided. Methods for preparing a 3-palmitoyl-amido-2-hydroxy-1-dimethoxytriphenylmethyletherpropane from a crystalline solid of 3-palmitoyl-amido-1,2- propanediol are also described.
A61K 31/164 - Amides, e.g. hydroxamic acids of a carboxylic acid with an aminoalcohol, e.g. ceramides
C07C 233/16 - Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms having the nitrogen atom of at least one of the carboxamide groups bound to a carbon atom of a hydrocarbon radical substituted by singly-bound oxygen atoms
C07C 233/18 - Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms having the nitrogen atom of at least one of the carboxamide groups bound to a carbon atom of a hydrocarbon radical substituted by singly-bound oxygen atoms with the substituted hydrocarbon radical bound to the nitrogen atom of the carboxamide group by an acyclic carbon atom having the carbon atom of the carboxamide group bound to a hydrogen atom or to a carbon atom of an acyclic saturated carbon skeleton
A61K 31/7125 - Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
CRYSTALLINE SOLIDS OF 3-PALMITOYL-AMIDO-1,2-PROPANEDIOL AND 3-PALMITOYL-AMIDO-2-HYDROXY-1-DIMETHOXYTRIPHENYLMETHYLETHER-PROPANE AND METHODS OF MAKING AND USING THE SAME
Aspects of the disclosure include crystalline solids of 3-palmitoyl-amido-1,2- propanediol and 3-palmitoyl-amido-2-hydroxy-1-dimethoxytriphenylmethylether-propane. Methods for preparing the crystalline solids of 3-palmitoyl-amido-1,2- propanediol and single crystals of 3-palmitoyl-amido-2-hydroxy-1-dimethoxytriphenylmethylether-propane are also provided. Methods for preparing a 3-palmitoyl-amido-2-hydroxy-1-dimethoxytriphenylmethyletherpropane from a crystalline solid of 3-palmitoyl-amido-1,2- propanediol are also described.
A61K 31/164 - Amides, e.g. hydroxamic acids of a carboxylic acid with an aminoalcohol, e.g. ceramides
C07C 233/16 - Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms having the nitrogen atom of at least one of the carboxamide groups bound to a carbon atom of a hydrocarbon radical substituted by singly-bound oxygen atoms
C07C 233/18 - Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms having the nitrogen atom of at least one of the carboxamide groups bound to a carbon atom of a hydrocarbon radical substituted by singly-bound oxygen atoms with the substituted hydrocarbon radical bound to the nitrogen atom of the carboxamide group by an acyclic carbon atom having the carbon atom of the carboxamide group bound to a hydrogen atom or to a carbon atom of an acyclic saturated carbon skeleton
05 - Pharmaceutical, veterinary and sanitary products
Goods & Services
Pharmaceuticals for the treatment, diagnosis or prophylaxis of human diseases, disorders and conditions, namely, cancer, and diseases, disorders and conditions of the hematological systems
05 - Pharmaceutical, veterinary and sanitary products
Goods & Services
pharmaceuticals for the treatment, diagnosis or prophylaxis of human diseases, disorders and conditions, namely, cancer, and diseases, disorders and conditions of the hematological systems
05 - Pharmaceutical, veterinary and sanitary products
Goods & Services
pharmaceuticals for the treatment, diagnosis or prophylaxis of human diseases, disorders and conditions, namely, cancer, and diseases, disorders and conditions of the hematological systems
05 - Pharmaceutical, veterinary and sanitary products
Goods & Services
pharmaceuticals for the treatment, diagnosis or prophylaxis of human diseases, disorders and conditions, namely, cancer, and diseases, disorders and conditions of the hematological systems
The present invention relates to a process for preparing the telomerase inhibitor imetelstat using a 3 steps per cycle phase support bound process comprising the steps of deprotection of the 3′-amino group of the support-bound oligonucleotide, coupling with a 5′-phosphoramidite, and sulfurization with an acyl disulfide, characterized by the absence of an additional capping step in each cycle that is used to prevent unreacted 3′-amino oligonucleotide groups from reacting during subsequent cycles. Imetelstat has formula below.
C07H 1/00 - Processes for the preparation of sugar derivatives
C07H 21/04 - Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
71.
Methods of polynucleotide preparation using multivalent cation salt compositions
Aspects of the disclosure include methods for the preparation of a polynucleotide. In some embodiments, the method includes contacting a first polynucleotide composition including: a polynucleotide having a sequence of 7 or more nucleoside subunits and at least two of the nucleoside subunits are joined by a N3′→P5′ thiophosphoramidate inter-subunit linkage; and non-target synthetic products and reagents; with a multivalent cation salt to precipitate a polynucleotide salt including at least one multivalent cation counterion; and separating the polynucleotide salt from the contacted first polynucleotide composition to produce a second polynucleotide composition including the polynucleotide salt. In certain embodiments, the method further includes contacting the polynucleotide salt with a reverse phase chromatography support; and eluting from the chromatography support a third polynucleotide composition including the polynucleotide. Also provided are compositions including a salt of the polynucleotide including at least one multivalent cation counterion.
This invention relates to compounds useful for inhibiting telomere elongation. More specifically, the invention provides nucleotide analogs that are incorporated into telomeres by telomerase thereby inhibiting elongation of telomeres. The compounds are use in treating cancer and other cell proliferative diseases.
C07F 9/6561 - Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing systems of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring or ring system, with or without other non-condensed hetero rings
C07D 473/18 - Heterocyclic compounds containing purine ring systems with oxygen, sulfur, or nitrogen atoms directly attached in positions 2 and 6 one oxygen and one nitrogen atom, e.g. guanine
Methods of monitoring therapeutic efficacy in a subject with MDS are provided. Also provided is a method of identifying a subject with myelodysplastic syndrome (MDS) for treatment with a telomerase inhibitor, and methods of treating MDS. The subject methods can include administering to the subject an effective amount of a telomerase inhibitor and assessing the hTERT expression levels in a biological sample obtained from the subject. In some cases, a 50% or greater reduction in hTERT expression level identifies a subject who has an increased likelihood of benefiting from treatment with the telomerase inhibitor. The subject can be naive to treatment with a HMA, lenalidomide, or both. In some cases, the subject is classified as having low or intermediate-1 IPSS risk MDS and/or MDS relapsed/refractory to Erythropoiesis-Stimulating Agent (ESA). In some instances, the telomerase inhibitor is imetelstat sodium.
C12Q 1/48 - Measuring or testing processes involving enzymes, nucleic acids or microorganismsCompositions thereforProcesses of preparing such compositions involving transferase
B82Y 5/00 - Nanobiotechnology or nanomedicine, e.g. protein engineering or drug delivery
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
A61K 31/454 - Non-condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems containing a five-membered ring with nitrogen as a ring hetero atom, e.g. pimozide, domperidone
Methods of monitoring therapeutic efficacy in a subject with MDS are provided. Also provided is a method of identifying a subject with myelodysplastic syndrome (MDS) for treatment with a telomerase inhibitor, and methods of treating MDS. The subject methods can include administering to the subject an effective amount of a telomerase inhibitor and assessing the hTERT expression levels in a biological sample obtained from the subject. In some cases, a 50% or greater reduction in hTERT expression level identifies a subject who has an increased likelihood of benefiting from treatment with the telomerase inhibitor. The subject can be naive to treatment with a HMA, lenalidomide, or both. In some cases, the subject is classified as having low or intermediate-1 IPSS risk MDS and/or MDS relapsed/refractory to Erythropoiesis-Stimulating Agent (ESA). In some instances, the telomerase inhibitor is imetelstat sodium.
A61K 31/7125 - Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
A61K 31/4439 - Non-condensed pyridinesHydrogenated derivatives thereof containing further heterocyclic ring systems containing a five-membered ring with nitrogen as a ring hetero atom, e.g. omeprazole
A61K 31/706 - Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom
Methods of monitoring therapeutic efficacy in a subject with MDS are provided. Also provided is a method of identifying a subject with myelodysplastic syndrome (MDS) for treatment with a telomerase inhibitor, and methods of treating MDS. The subject methods can include administering to the subject an effective amount of a telomerase inhibitor and assessing the hTERT expression levels in a biological sample obtained from the subject. In some cases, a 50% or greater reduction in hTERT expression level identifies a subject who has an increased likelihood of benefiting from treatment with the telomerase inhibitor. The subject can be naive to treatment with a HMA, lenalidomide, or both. In some cases, the subject is classified as having low or intermediate-1 IPSS risk MDS and/or MDS relapsed/refractory to Erythropoiesis-Stimulating Agent (ESA). In some instances, the telomerase inhibitor is imetelstat sodium.
C12Q 1/48 - Measuring or testing processes involving enzymes, nucleic acids or microorganismsCompositions thereforProcesses of preparing such compositions involving transferase
B82Y 5/00 - Nanobiotechnology or nanomedicine, e.g. protein engineering or drug delivery
A61K 31/454 - Non-condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems containing a five-membered ring with nitrogen as a ring hetero atom, e.g. pimozide, domperidone
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
76.
METHODS OF IDENTIFYING PATIENTS LIKELY TO BENEFIT FROM TREATMENT WITH A TELOMERASE INHIBITOR
This disclosure provides methods of identifying or selecting a patient most likely to benefit from treatment with a telomerase inhibitor, such as e.g. imetelstat, by testing a patient for: a lack of a mutation in each of JAK2, CALR, and MPL; and/or a high-molecular risk (HMR), based on the presence of a mutation in at least one of the following genes: ASXL1, EZH2, SRSF2, and IDH1/2. The patient may be suffering from myelofibrosis. The disclosure also provides methods of treating myelofibrosis, which include identifying such patients.
C12Q 1/6886 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material for cancer
G16H 50/30 - ICT specially adapted for medical diagnosis, medical simulation or medical data miningICT specially adapted for detecting, monitoring or modelling epidemics or pandemics for calculating health indicesICT specially adapted for medical diagnosis, medical simulation or medical data miningICT specially adapted for detecting, monitoring or modelling epidemics or pandemics for individual health risk assessment
G16B 40/10 - Signal processing, e.g. from mass spectrometry [MS] or from PCR
C12Q 1/6827 - Hybridisation assays for detection of mutation or polymorphism
A61K 9/00 - Medicinal preparations characterised by special physical form
G16H 20/10 - ICT specially adapted for therapies or health-improving plans, e.g. for handling prescriptions, for steering therapy or for monitoring patient compliance relating to drugs or medications, e.g. for ensuring correct administration to patients
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
77.
METHODS OF IDENTIFYING PATIENTS LIKELY TO BENEFIT FROM TREATMENT WITH A TELOMERASE INHIBITOR
This disclosure provides methods of identifying or selecting a patient most likely to benefit from treatment with a telomerase inhibitor, such as e.g. imetelstat, by testing a patient for: a lack of a mutation in each of JAK2, CALR, and MPL; and/ or a high-molecular risk (HMR), based on the presence of a mutation in at least one of the following genes: ASXL1, EZH2, SRSF2, and IDH1/2. The patient may be suffering from myelofibrosis. The disclosure also provides methods of treating myelofibrosis, which include identifying such patients.
e.g.JAK2CALRMPLASXL1EZH2SRSF2IDH1/2IDH1/2. The patient may be suffering from myelofibrosis. The disclosure also provides methods of treating myelofibrosis, which include identifying such patients.
05 - Pharmaceutical, veterinary and sanitary products
35 - Advertising and business services
42 - Scientific, technological and industrial services, research and design
Goods & Services
Therapeutic, diagnostic, and prophylactic preparations, pharmaceutical products and agents for the treatment, diagnosis or prophylaxis of human diseases, disorders and conditions, namely, cancers and hematologic diseases, syndromes and disorders Promoting collaboration within the scientific, research and provider communities to achieve advances in the fields of treatment, diagnosis and prophylaxis of cancers and hematologic diseases, syndromes and disorders Providing medical information in the field of the diagnosis and treatment of cancers and hematologic malignancies to consumers, and providing medical information in the field of prevention, screening, diagnosis and treatment to scientists, researchers and medical providers on cancers and hematologic diseases, syndromes and disorders
80.
Guanine analogs as telomerase substrates and telomere length affectors
This invention relates to compounds useful for inhibiting telomere elongation. More specifically, the invention provides nucleotide analogs that are incorporated into telomeres by telomerase thereby inhibiting elongation of telomeres. The compounds are use in treating cancer and other cell proliferative diseases.
C07D 473/18 - Heterocyclic compounds containing purine ring systems with oxygen, sulfur, or nitrogen atoms directly attached in positions 2 and 6 one oxygen and one nitrogen atom, e.g. guanine
C07F 9/6561 - Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing systems of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring or ring system, with or without other non-condensed hetero rings
This disclosure provides methods of treating a myelodysplastic syndrome (MDS) in a subject that is naive to treatment with an agent selected from a hypomethylating agent (HMA) and lenalidomide, or both. The method includes administering to the subject an effective amount of a telomerase inhibitor, such as e.g. imetelstat or imetelstat sodium. In some cases, the subject treated is classified as low or intermediate-1 IPSS risk MDS and/or have MDS relapsed/refractory to Erythropoiesis-Stimulating Agent (ESA).
This disclosure provides methods of treating a myelodysplastic syndrome (MDS) in a subject that is naive to treatment with an agent selected from a hypomethylating agent (HMA) and lenalidomide, or both. The method includes administering to the subject an effective amount of a telomerase inhibitor, such as e.g. imetelstat or imetelstat sodium. In some cases, the subject treated is classified as low or intermediate-1 IPSS risk MDS and/or have MDS relapsed/refractory to Erythropoiesis-Stimulating Agent (ESA).
A61K 31/7125 - Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
A61K 9/00 - Medicinal preparations characterised by special physical form
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 7/00 - Drugs for disorders of the blood or the extracellular fluid
C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
83.
Synthesis of protected 3′-amino nucleoside monomers
Orthogonally protected 3′-amino nucleoside monomers and efficient methods for their synthesis are described. The methods employ selective protection of the 3′-amino group in the presence of the unprotected nucleoside base.
The present invention relates to a process for preparing the telomerase inhibitor imetelstat using a 3 steps per cycle solid- phase support bound process comprising the steps of deprotection of the 3'-amino group of the support-bound oligonucleotide, coupling with a 5'-phosphoramidite, and sulfurization with an acyl disulfide, characterized by the absence of an additional capping step in each cycle that is used to prevent unreacted 3'-amino oligonucleotide groups from reacting during subsequent cycles. Imetelstat has formula below.
C07H 21/04 - Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
C12N 15/11 - DNA or RNA fragmentsModified forms thereof
The present invention relates to a process for preparing the telomerase inhibitor imetelstat using a 3 steps per cycle solid- phase support bound process comprising the steps of deprotection of the 3'-amino group of the support-bound oligonucleotide, coupling with a 5'- phosphoramidite, and sulfurization with an acyl disulfide, characterized by the absence of an additional capping step in each cycle that is used to prevent unreacted 3'-amino oligonucleotide groups from reacting during subsequent cycles. lmetelstat has formula below.
C07H 21/04 - Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
C12N 15/11 - DNA or RNA fragmentsModified forms thereof
The present disclosure relates to RNA amidates and thioamidates useful for RNA interference applications. The RNA amidates and thioamidates contain at least one internucleoside linkage chosen from ribo-N3′→P5′ phosphoramidate (NP) and ribo-N3′→P5′ thiophosphoramidate (NPS) linkages, and optionally further containing at least one covalently conjugated lipid moiety. Compositions comprising the amidates and thioamidates are disclosed, as are methods for their use in modulating gene expression.
C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
C12N 15/11 - DNA or RNA fragmentsModified forms thereof
C07H 21/00 - Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
87.
COMBINATION OF IMETELSTAT AND ABT-199 FOR USE IN TREATING HEMATOLOGICAL CANCERS
The present invention relates to a combination treatment for hematological cancers. More specifically; a combination of a telomerase inhibitor and a Bcl-2 inhibitor are useful in treating hematological cancers, including AML. In certain embodiments, the telomerase inhibitor is imetelstat or imetelstat sodium and the Bcl-2 inhibitor is ABT-199.
A61K 31/635 - Compounds containing para-N-benzene- sulfonyl-N-groups, e.g. sulfanilamide, p-nitrobenzenesulfonohydrazide having a heterocyclic ring, e.g. sulfadiazine
A61K 31/7125 - Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
The present invention relates to a combination treatment for hematological cancers. More specifically; a combination of a telomerase inhibitor and a Bcl-2 inhibitor are useful in treating hematological cancers, including AML. In certain embodiments, the telomerase inhibitor is imetelstat or imetelstat sodium and the Bcl-2 inhibitor is ABT-199.
A61K 31/7125 - Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
A61K 31/496 - Non-condensed piperazines containing further heterocyclic rings, e.g. rifampin, thiothixene or sparfloxacin
A61K 31/635 - Compounds containing para-N-benzene- sulfonyl-N-groups, e.g. sulfanilamide, p-nitrobenzenesulfonohydrazide having a heterocyclic ring, e.g. sulfadiazine
A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
89.
COMBINATION OF IMETELSTAT AND ABT-199 FOR USE IN TREATING HEMATOLOGICAL CANCERS
The present invention relates to a combination treatment for hematological cancers. More specifically; a combination of a telomerase inhibitor and a Bc1-2 inhibitor are useful in treating hematological cancers, including AML. In certain embodiments, the telomerase inhibitor is imetelstat or imetelstat sodium and the Bc1-2 inhibitor is ABT-199.
The present invention relates to a combination treatment for hematological cancers. More specifically; a combination of a telomerase inhibitor and a Bcl-2 inhibitor are useful in treating hematological cancers, including AML. In certain embodiments, the telomerase inhibitor is imetelstat or imetelstat sodium and the Bcl-2 inhibitor is ABT-199.
A61K 31/635 - Compounds containing para-N-benzene- sulfonyl-N-groups, e.g. sulfanilamide, p-nitrobenzenesulfonohydrazide having a heterocyclic ring, e.g. sulfadiazine
A61K 31/7125 - Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
The present disclosure provides a solid phase method of making oligonucleotides via sequential coupling cycles including at least one coupling of a dinucleotide dimer subunit to a free 3′-terminal group of a growing chain. The oligonucleotides include at least two nucleoside subunits joined by a N3′→P5′ phosphoramidate linkage. The method may include the steps of (a) deprotecting the protected 3′ amino group of a terminal nucleoside attached to a solid phase support, said deprotecting forming a free 3′ amino group; (b) contacting the free 3′ amino group with a 3′-protected amino-dinucleotide-5′-phosphoramidite dimer in the presence of a nucleophilic catalyst to form an internucleoside N3′→P5′ phosphoramidite linkage; and (c) oxidizing (e.g., sulfurizing) the linkage. The compositions produced by the subject methods may include a reduced amount of one or more (N−x) oligonucleotide products. Also provided are pharmaceutical compositions including the subject oligonucleotide compositions.
C07H 21/00 - Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
C12P 19/34 - Polynucleotides, e.g. nucleic acids, oligoribonucleotides
92.
Oligonucleotide compositions and methods of making the same
The present disclosure provides a solid phase method of making oligonucleotides via sequential coupling cycles including at least one coupling of a dinucleotide dimer subunit to a free 3′-terminal group of a growing chain. The oligonucleotides include at least two nucleoside subunits joined by a N3′→P5′ phosphoramidate linkage. The method may include the steps of (a) deprotecting the protected 3′ amino group of a terminal nucleoside attached to a solid phase support, said deprotecting forming a free 3′ amino group; (b) contacting the free 3′ amino group with a 3′-protected amino-dinucleotide-5′-phosphoramidite dimer in the presence of a nucleophilic catalyst to form an internucleoside N3′→P5′ phosphoramidite linkage; and (c) oxidizing (e.g., sulfurizing) the linkage. The compositions produced by the subject methods may include a reduced amount of one or more (N−x) oligonucleotide products. Also provided are pharmaceutical compositions including the subject oligonucleotide compositions.
C07H 21/00 - Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
C12P 19/34 - Polynucleotides, e.g. nucleic acids, oligoribonucleotides
93.
Phosphorodiamidate backbone linkage for oligonucleotides
This invention relates to antisense oligonucleotides comprising at least one N3′→P5′ phosphorodiamidate linkage (NPN) in the backbone, useful for modulating gene expression involved in the pathogenesis of a disease. Compounds useful as building blocks of said antisense oligonucleotides and methods of preparing building block compounds including NPN linkages are provided.
C07H 1/00 - Processes for the preparation of sugar derivatives
C07H 19/10 - Pyrimidine radicals with the saccharide radical being esterified by phosphoric or polyphosphoric acids
C07H 19/20 - Purine radicals with the saccharide radical being esterified by phosphoric or polyphosphoric acids
C07H 21/00 - Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
94.
Method for identification of sensitivity of a patient to telomerase inhibition therapy
The invention provides methods for determining the susceptibility of cancer patients to developing adverse reactions if treated with a telomerase inhibitor drug by measurement of telomere length in appropriate cells of the patient prior to initiation of the telomerase inhibitor treatment.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
C07H 21/04 - Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
C12Q 1/6883 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
95.
Guanine analogs as telomerase substrates and telomere length affectors
This invention relates to compounds useful for inhibiting telomere elongation. More specifically, the invention provides nucleotide analogs that are incorporated into telomeres by telomerase thereby inhibiting elongation of telomeres. The compounds are use in treating cancer and other cell proliferative diseases.
C07F 9/6561 - Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing systems of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring or ring system, with or without other non-condensed hetero rings
C07D 473/18 - Heterocyclic compounds containing purine ring systems with oxygen, sulfur, or nitrogen atoms directly attached in positions 2 and 6 one oxygen and one nitrogen atom, e.g. guanine
05 - Pharmaceutical, veterinary and sanitary products
42 - Scientific, technological and industrial services, research and design
Goods & Services
Therapeutic, diagnostic, and prophylactic preparations,
pharmaceutical products and agents for the treatment,
diagnosis or prophylaxis of human diseases, disorders and
conditions, namely, cancer, diseases, disorders and
conditions of the hematological systems. Scientific research and development of preparations, agents
and biotechnology for the treatment, diagnosis or
prophylaxis of human diseases, disorders and conditions,
namely cancer, diabetes, arthritis, osteoporosis, bone
fractures and wound healing, and diseases, disorders and
conditions of the cardiovascular, hematological, neuronal
and nervous systems; scientific research and development of
cosmetics, sundries and nutraceuticals; providing
information about medical and scientific research to
scientists, physicians and other health care providers.
97.
Synthesis of protected 3′-amino nucleoside monomers
Orthogonally protected 3′-amino nucleoside monomers and efficient methods for their synthesis are described. The methods employ selective protection of the 3′-amino group in the presence of the unprotected nucleoside base.
Aspects of the disclosure include methods for the preparation of a polynucleotide. In some embodiments, the method includes contacting a first polynucleotide composition including: a polynucleotide having a sequence of 7 or more nucleoside subunits and at least two of the nucleoside subunits are joined by a N3′→P5′ thiophosphoramidate inter-subunit linkage; and non-target synthetic products and reagents; with a multivalent cation salt to precipitate a polynucleotide salt including at least one multivalent cation counterion; and separating the polynucleotide salt from the contacted first polynucleotide composition to produce a second polynucleotide composition including the polynucleotide salt. In certain embodiments, the method further includes contacting the polynucleotide salt with a reverse phase chromatography support; and eluting from the chromatography support a third polynucleotide composition including the polynucleotide. Also provided are compositions including a salt of the polynucleotide including at least one multivalent cation counterion.
Aspects of the disclosure include methods for the preparation of a polynucleotide. In some embodiments, the method includes contacting a first polynucleotide composition including: a polynucleotide having a sequence of 7 or more nucleoside subunits and at least two of the nucleoside subunits are joined by a N3'?P5' thiophosphoramidate inter-subunit linkage; and non-target synthetic products and reagents; with a multivalent cation salt to precipitate a polynucleotide salt including at least one multivalent cation counterion; and separating the polynucleotide salt from the contacted first polynucleotide composition to produce a second polynucleotide composition including the polynucleotide salt. In certain embodiments, the method further includes contacting the polynucleotide salt with a reverse phase chromatography support; and eluting from the chromatography support a third polynucleotide composition including the polynucleotide. Also provided are compositions including a salt of the polynucleotide including at least one multivalent cation counterion.
A61K 31/7125 - Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
C12N 15/115 - Aptamers, i.e. nucleic acids binding a target molecule specifically and with high affinity without hybridising therewith
C07H 21/00 - Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
C12N 15/10 - Processes for the isolation, preparation or purification of DNA or RNA
C12N 15/11 - DNA or RNA fragmentsModified forms thereof
Aspects of the disclosure include methods for the preparation of a polynucleotide. In some embodiments, the method includes contacting a first polynucleotide composition including: a polynucleotide having a sequence of 7 or more nucleoside subunits and at least two of the nucleoside subunits are joined by a N3'→P5' thiophosphoramidate inter-subunit linkage; and non-target synthetic products and reagents; with a multivalent cation salt to precipitate a polynucleotide salt including at least one multivalent cation counterion; and separating the polynucleotide salt from the contacted first polynucleotide composition to produce a second polynucleotide composition including the polynucleotide salt. In certain embodiments, the method further includes contacting the polynucleotide salt with a reverse phase chromatography support; and eluting from the chromatography support a third polynucleotide composition including the polynucleotide. Also provided are compositions including a salt of the polynucleotide including at least one multivalent cation counterion.
C07H 21/00 - Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
C12P 19/34 - Polynucleotides, e.g. nucleic acids, oligoribonucleotides
C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
