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1. WO2020112854 - METHODS OF TREATING MYELODYSPLASTIC SYNDROME

Publication Number WO/2020/112854
Publication Date 04.06.2020
International Application No. PCT/US2019/063372
International Filing Date 26.11.2019
IPC
C12Q 1/48 2006.01
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
1Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
48involving transferase
B82Y 5/00 2011.01
BPERFORMING OPERATIONS; TRANSPORTING
82NANOTECHNOLOGY
YSPECIFIC USES OR APPLICATIONS OF NANOSTRUCTURES; MEASUREMENT OR ANALYSIS OF NANOSTRUCTURES; MANUFACTURE  OR TREATMENT OF NANOSTRUCTURES
5Nanobiotechnology or nanomedicine, e.g. protein engineering or drug delivery
A61K 31/454 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
33Heterocyclic compounds
395having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
435having six-membered rings with one nitrogen as the only ring hetero atom
44Non-condensed pyridines; Hydrogenated derivatives thereof
445Non-condensed piperidines, e.g. piperocaine
4523containing further heterocyclic ring systems
454containing a five-membered ring with nitrogen as a ring hetero atom, e.g. pimozide, domperidone
A61K 47/54 2017.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
47Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
50the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
51the non-active ingredient being a modifying agent
54the modifying agent being an organic compound
CPC
A61K 31/4439
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
33Heterocyclic compounds
395having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
435having six-membered rings with one nitrogen as the only ring hetero atom
44Non condensed pyridines; Hydrogenated derivatives thereof
4427containing further heterocyclic ring systems
4439containing a five-membered ring with nitrogen as a ring hetero atom, e.g. omeprazole
A61K 31/706
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
70Carbohydrates; Sugars; Derivatives thereof
7042Compounds having saccharide radicals and heterocyclic rings
7052having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides
706containing six-membered rings with nitrogen as a ring hetero atom
A61K 31/7125
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
70Carbohydrates; Sugars; Derivatives thereof
7088Compounds having three or more nucleosides or nucleotides
7125Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
A61P 35/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
35Antineoplastic agents
G01N 2333/9128
GPHYSICS
01MEASURING; TESTING
NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
2333Assays involving biological materials from specific organisms or of a specific nature
90Enzymes; Proenzymes
91Transferases (2.)
912transferring phosphorus containing groups, e.g. kinases (2.7)
91205Phosphotransferases in general
91245Nucleotidyltransferases (2.7.7)
9125with a definite EC number (2.7.7.-)
9128RNA-directed DNA polymerases, e.g. RT (2.7.7.49)
G01N 2800/52
GPHYSICS
01MEASURING; TESTING
NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
2800Detection or diagnosis of diseases
52Predicting or monitoring the response to treatment; Prognosis
Applicants
  • GERON CORPORATION [US]/[US]
  • RIZO, Aleksandra
Inventors
  • RIZO, Aleksandra
  • BUSSOLARI, Jacqueline Cirillo
  • HUANG, Fei
Agents
  • NG, Rudy J.
Priority Data
62/772,86129.11.2018US
62/811,27127.02.2019US
62/860,55712.06.2019US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) METHODS OF TREATING MYELODYSPLASTIC SYNDROME
(FR) PROCÉDÉS DE TRAITEMENT DU SYNDROME MYÉLODYSPLASIQUE
Abstract
(EN)
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.
(FR)
Procédés de surveillance de l'efficacité d'une thérapie chez un sujet atteint de syndrome myélodysplasique (SMD). L'invention concerne également un procédé d'identification d'un sujet atteint d'un SMD afin de le traiter avec un inhibiteur de télomérase, et des procédés de traitement de SMD. Les procédés selon l'invention peuvent comprendre l'administration au sujet d'une quantité efficace d'un inhibiteur de télomérase et l'évaluation des niveaux d'expression de hTERT dans un échantillon biologique prélevé sur le sujet. Dans certains cas, une réduction de 50 % ou plus dans le niveau d'expression de hTERT identifie un sujet qui a une probabilité accrue de tirer bénéfice d'un traitement avec l'inhibiteur de télomérase. Le sujet peut être naif au traitement par HMA, lénalidomide, ou aux deux. Dans certains cas, le sujet traité est classé comme SMD à IPSS de risque faible ou intermédiaire 1 et/ou subit une rechute de SMD/SMD réfractaire à l'agent stimulant l'érythropoïèse (ASE). Dans certains cas, l'inhibiteur de télomérase est le sodium d'imetelstat.
Latest bibliographic data on file with the International Bureau