TW202438086A - 治療或預防脊髓性肌萎縮之方法 - Google Patents

治療或預防脊髓性肌萎縮之方法 Download PDF

Info

Publication number
TW202438086A
TW202438086A TW113114478A TW113114478A TW202438086A TW 202438086 A TW202438086 A TW 202438086A TW 113114478 A TW113114478 A TW 113114478A TW 113114478 A TW113114478 A TW 113114478A TW 202438086 A TW202438086 A TW 202438086A
Authority
TW
Taiwan
Prior art keywords
antisense compound
salt
loading dose
dose
administered
Prior art date
Application number
TW113114478A
Other languages
English (en)
Chinese (zh)
Inventor
坎尼斯史瓦普 洛芙迪
法傑茱蒂 拜
莉莉 伊斯特
威爾頓 Ｒ 法爾威爾
Original Assignee
美商百健Ｍａ公司
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=71995097&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=TW202438086(A) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by 美商百健Ｍａ公司 filed Critical 美商百健Ｍａ公司
Publication of TW202438086A publication Critical patent/TW202438086A/zh

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/1136—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 against growth factors, growth regulators, cytokines, lymphokines or hormones
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A61K9/0021—Intradermal administration, e.g. through microneedle arrays or needleless injectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • G01N33/6893—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids related to diseases not provided for elsewhere
    • G01N33/6896—Neurological disorders, e.g. Alzheimer's disease
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/34—Allele or polymorphism specific uses
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Organic Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Epidemiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Neurology (AREA)
  • Hematology (AREA)
  • Urology & Nephrology (AREA)
  • Immunology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurosurgery (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Dermatology (AREA)
  • General Physics & Mathematics (AREA)
  • Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Cell Biology (AREA)
TW113114478A 2019-07-19 2020-07-17 治療或預防脊髓性肌萎縮之方法 TW202438086A (zh)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962876360P 2019-07-19 2019-07-19
US62/876,360 2019-07-19

Publications (1)

Publication Number Publication Date
TW202438086A true TW202438086A (zh) 2024-10-01

Family

ID=71995097

Family Applications (2)

Application Number Title Priority Date Filing Date
TW113114478A TW202438086A (zh) 2019-07-19 2020-07-17 治療或預防脊髓性肌萎縮之方法
TW109124224A TWI841762B (zh) 2019-07-19 2020-07-17 治療或預防脊髓性肌萎縮之方法

Family Applications After (1)

Application Number Title Priority Date Filing Date
TW109124224A TWI841762B (zh) 2019-07-19 2020-07-17 治療或預防脊髓性肌萎縮之方法

Country Status (33)

Country Link
US (3) US20220325282A1 (https=)
EP (2) EP4335503A3 (https=)
JP (3) JP7279253B2 (https=)
KR (1) KR20220042113A (https=)
CN (1) CN114302727A (https=)
AR (1) AR119430A1 (https=)
AU (1) AU2020316997A1 (https=)
BR (1) BR112022000870A2 (https=)
CA (1) CA3149906A1 (https=)
CL (1) CL2021003602A1 (https=)
CO (1) CO2022001354A2 (https=)
CR (1) CR20220071A (https=)
DK (1) DK3999643T5 (https=)
ES (1) ES2966791T3 (https=)
FI (1) FI3999643T3 (https=)
HR (1) HRP20231515T1 (https=)
HU (1) HUE064071T2 (https=)
IL (1) IL289745A (https=)
JO (2) JOP20220007A1 (https=)
LT (1) LT3999643T (https=)
MA (1) MA62935B1 (https=)
MD (1) MD3999643T2 (https=)
MX (1) MX2022000441A (https=)
PE (1) PE20220941A1 (https=)
PH (1) PH12022550114A1 (https=)
PL (1) PL3999643T3 (https=)
PT (1) PT3999643T (https=)
RS (1) RS64879B1 (https=)
SI (1) SI3999643T1 (https=)
SM (1) SMT202300442T1 (https=)
TW (2) TW202438086A (https=)
UY (1) UY38790A (https=)
WO (1) WO2021016032A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3797780B1 (en) 2014-04-17 2022-09-14 Biogen MA Inc. Compositions and methods for modulation of smn2 splicing in a subject
US10436802B2 (en) 2014-09-12 2019-10-08 Biogen Ma Inc. Methods for treating spinal muscular atrophy
PT3999643T (pt) 2019-07-19 2023-12-11 Biogen Ma Inc Métodos de tratamento da atrofia muscular espinhal
WO2024138018A1 (en) * 2022-12-23 2024-06-27 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Treatment of gemin5-mediated disorders

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3470072A1 (en) * 2005-06-23 2019-04-17 Biogen MA Inc. Compositions and methods for modulation of smn2 splicing
WO2008157753A1 (en) * 2007-06-21 2008-12-24 Sma Foundation Methods of treatment for spinal muscular atrophy
ES2699827T3 (es) 2009-06-17 2019-02-13 Biogen Ma Inc Composiciones y métodos para la modulación de corte y empalme de SMN2 en un sujeto
WO2011032109A1 (en) 2009-09-11 2011-03-17 Sma Foundation Biomarkers for spinal muscular atrophy
EP2714037B1 (en) * 2011-05-25 2016-07-13 Université Paris Descartes Erk inhibitors for use in treating spinal muscular atrophy
WO2014110291A1 (en) 2013-01-09 2014-07-17 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of smn2 splicing in a subject
US10436802B2 (en) 2014-09-12 2019-10-08 Biogen Ma Inc. Methods for treating spinal muscular atrophy
EP3484524B1 (en) * 2016-07-15 2022-11-09 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of smn2
TW201927241A (zh) 2017-12-21 2019-07-16 瑞士商赫孚孟拉羅股份公司 用於肌肉失能之數位生物標記
CN119679944A (zh) 2018-01-25 2025-03-25 渤健马萨诸塞州股份有限公司 治疗脊髓性肌萎缩症的方法
IT201800004359A1 (it) 2018-04-10 2019-10-10 IDENTIFICAZIONE DI miRNA MUSCOLARI COME BIOMARCATORI E TRATTAMENTO CO-ADIUVANTE PER L’ATROFIA MUSCOLARE SPINALE
US20210308281A1 (en) * 2018-08-15 2021-10-07 Biogen Ma Inc. Combination therapy for spinal muscular atrophy
PT3999643T (pt) 2019-07-19 2023-12-11 Biogen Ma Inc Métodos de tratamento da atrofia muscular espinhal

Also Published As

Publication number Publication date
US20250230440A1 (en) 2025-07-17
PE20220941A1 (es) 2022-05-31
TWI841762B (zh) 2024-05-11
ES2966791T3 (es) 2024-04-24
US20220325282A1 (en) 2022-10-13
EP4335503A3 (en) 2024-06-05
JOP20220007A1 (ar) 2023-01-30
HRP20231515T1 (hr) 2024-03-01
TW202116331A (zh) 2021-05-01
US20240318182A1 (en) 2024-09-26
FI3999643T3 (fi) 2023-11-30
BR112022000870A2 (pt) 2022-03-22
JOP20250103A1 (ar) 2025-05-05
KR20220042113A (ko) 2022-04-04
EP3999643B1 (en) 2023-11-22
DK3999643T5 (da) 2024-08-05
JP2025096486A (ja) 2025-06-26
IL289745A (en) 2022-03-01
LT3999643T (lt) 2023-12-11
CN114302727A (zh) 2022-04-08
SMT202300442T1 (it) 2024-01-10
AU2020316997A1 (en) 2022-03-10
CL2021003602A1 (es) 2022-10-14
MD3999643T2 (ro) 2024-02-29
AR119430A1 (es) 2021-12-15
DK3999643T3 (da) 2023-12-04
PT3999643T (pt) 2023-12-11
JP7279253B2 (ja) 2023-05-22
EP4335503A2 (en) 2024-03-13
HUE064071T2 (hu) 2024-02-28
JP2022540993A (ja) 2022-09-21
MA62935B1 (fr) 2023-11-30
CA3149906A1 (en) 2021-01-28
RS64879B1 (sr) 2023-12-29
UY38790A (es) 2021-01-29
PH12022550114A1 (en) 2023-04-17
PL3999643T3 (pl) 2024-02-19
SI3999643T1 (sl) 2024-01-31
CO2022001354A2 (es) 2022-03-18
WO2021016032A1 (en) 2021-01-28
JP2023100911A (ja) 2023-07-19
CR20220071A (es) 2022-04-22
MX2022000441A (es) 2022-03-25
JP7665678B2 (ja) 2025-04-21
EP3999643A1 (en) 2022-05-25

Similar Documents

Publication Publication Date Title
JP7665678B2 (ja) 脊髄性筋萎縮症を処置または予防する方法
Agra Almeida Quadros et al. Cryptic splicing of stathmin-2 and UNC13A mRNAs is a pathological hallmark of TDP-43-associated Alzheimer’s disease
CN106715695B (zh) 用于治疗和诊断的微rna和包含所述微rna的组合物
Valsecchi et al. miR-206 reduces the severity of motor neuron degeneration in the facial nuclei of the brainstem in a mouse model of SMA
Zarrouki et al. Partial Restoration of Brain Dystrophin and Behavioral Deficits by Exon Skipping in the Muscular Dystrophy X‐Linked (mdx) Mouse
CN113966396A (zh) 遗传性神经病和相关障碍的治疗和检测
Andreone et al. Durable suppression of seizures in a preclinical model of KCNT1 genetic epilepsy with divalent small interfering RNA
EP4294461A1 (en) Methods and compositions for reducing nucleic acid vector-induced toxicity in the inner ear
HK40108697A (en) Methods of treating or preventing spinal muscular atrophy
EA047168B1 (ru) Способы лечения или предотвращения спинальной мышечной атрофии
EA052680B1 (ru) Способы лечения или предотвращения спинальной мышечной атрофии
US20220002726A1 (en) Methods of safe administration of an irf5 antisense oligonucleotide
US20260022377A1 (en) Polynucleotide compositions and methods for treatment of neurodegenerative diseases
US20230175065A1 (en) Methods for treating inflammatory and autoimmune disorders
DeSisto Investigation of Heterogeneity and Stemlike Cell Drivers of Oncogenesis in Pediatric High Grade Glioma
WO2026065401A1 (zh) 一种用于调控SCN1A基因mRNA和蛋白表达的方法和化合物
HK40107707A (zh) 用於治疗阿尔茨海默病的组合物和方法
Rodriguez et al. A specific prelimbic-nucleus accumbens pathway controls resilience versus vulnerability to food addiction
EP4594491A2 (en) Antisense oligonucleotides for the treatment of canavan disease
AU2024352343A1 (en) Antisense oligonucleotides for the treatment of canavan disease
AU2023348346A1 (en) Antisense oligonucleotides for the treatment of canavan disease
CN122003502A (zh) 用于治疗卡纳万病的反义寡核苷酸