MX2021005843A - Composiciones y métodos para aumentar la hemoglobina fetal y tratar la anemia de células falciformes. - Google Patents

Composiciones y métodos para aumentar la hemoglobina fetal y tratar la anemia de células falciformes.

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Publication number
MX2021005843A
MX2021005843A MX2021005843A MX2021005843A MX2021005843A MX 2021005843 A MX2021005843 A MX 2021005843A MX 2021005843 A MX2021005843 A MX 2021005843A MX 2021005843 A MX2021005843 A MX 2021005843A MX 2021005843 A MX2021005843 A MX 2021005843A
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MX
Mexico
Prior art keywords
methods
compositions
sickle cell
cell disease
fetal hemoglobin
Prior art date
Application number
MX2021005843A
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English (en)
Inventor
Peter Rahl
Angela Cacace
Michael Cameron
Akshay Kakumanu
Original Assignee
Fulcrum Therapeutics Inc
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Publication date
Application filed by Fulcrum Therapeutics Inc filed Critical Fulcrum Therapeutics Inc
Publication of MX2021005843A publication Critical patent/MX2021005843A/es

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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P7/06—Antianaemics
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C—CHEMISTRY; METALLURGY
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]

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  • Health & Medical Sciences (AREA)
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  • Wood Science & Technology (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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Abstract

La presente invención se refiere a composiciones y métodos para aumentar los niveles de hemoglobina fetal (HbF) en las células. La presente invención se refiere además a métodos para tratar pacientes que padecen enfermedades de las células sanguíneas, incluyendo las asociadas con cantidades reducidas de hemoglobina funcional adulta (HbA), tales como la anemia de células falciformes y las ß-talasemias.
MX2021005843A 2018-11-20 2019-11-20 Composiciones y métodos para aumentar la hemoglobina fetal y tratar la anemia de células falciformes. MX2021005843A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862769796P 2018-11-20 2018-11-20
PCT/US2019/062461 WO2020106876A2 (en) 2018-11-20 2019-11-20 Compositions and methods for increasing fetal hemoglobin and treating sickle cell disease

Publications (1)

Publication Number Publication Date
MX2021005843A true MX2021005843A (es) 2021-10-01

Family

ID=68848495

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2021005843A MX2021005843A (es) 2018-11-20 2019-11-20 Composiciones y métodos para aumentar la hemoglobina fetal y tratar la anemia de células falciformes.

Country Status (7)

Country Link
US (1) US20220017908A1 (es)
EP (1) EP3883597A2 (es)
JP (1) JP2022513100A (es)
AU (1) AU2019384547A1 (es)
CA (1) CA3119781A1 (es)
MX (1) MX2021005843A (es)
WO (1) WO2020106876A2 (es)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG10202110259QA (en) 2017-10-05 2021-10-28 Fulcrum Therapeutics Inc Use of p38 inhibitors to reduce expression of dux4
CN114144230B (zh) 2019-03-15 2024-04-23 弗尔康医疗公司 作为eed和prc2调节剂的大环唑并吡啶衍生物
WO2023034506A1 (en) * 2021-09-01 2023-03-09 Flagship Pioneering Innovations Vi, Llc Methods and compositions for inducing fetal hemoglobin

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4522811A (en) 1982-07-08 1985-06-11 Syntex (U.S.A.) Inc. Serial injection of muramyldipeptides and liposomes enhances the anti-infective activity of muramyldipeptides
AU2012321106C1 (en) * 2011-11-03 2016-11-24 Takeda Pharmaceutical Company Limited Administration of NEDD8-activating enzyme inhibitor and hypomethylating agent
US11179379B2 (en) * 2017-01-30 2021-11-23 The Children's Hospital Of Philadelphia Compositions and methods for hemoglobin production
AU2018251687B2 (en) * 2017-04-10 2021-07-29 The Regents Of The University Of Michigan Covalent small molecule DCN1 inhibitors and therapeutic methods using the same

Also Published As

Publication number Publication date
WO2020106876A2 (en) 2020-05-28
EP3883597A2 (en) 2021-09-29
JP2022513100A (ja) 2022-02-07
AU2019384547A1 (en) 2021-06-03
WO2020106876A3 (en) 2020-07-23
US20220017908A1 (en) 2022-01-20
CA3119781A1 (en) 2020-05-28

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