KR20250021401A - 척수 근위축증을 치료하기 위한 마이오스타틴 저해제의 용도 - Google Patents

척수 근위축증을 치료하기 위한 마이오스타틴 저해제의 용도 Download PDF

Info

Publication number
KR20250021401A
KR20250021401A KR1020247040101A KR20247040101A KR20250021401A KR 20250021401 A KR20250021401 A KR 20250021401A KR 1020247040101 A KR1020247040101 A KR 1020247040101A KR 20247040101 A KR20247040101 A KR 20247040101A KR 20250021401 A KR20250021401 A KR 20250021401A
Authority
KR
South Korea
Prior art keywords
sma
apitegromab
patient
therapy
subject
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
KR1020247040101A
Other languages
English (en)
Korean (ko)
Inventor
조지 노미코스
구오첸 송
융 츠융
미카 티. 웹스터
나탈리 케르테츠
Original Assignee
스칼러 락, 인크.
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
Application filed by 스칼러 락, 인크. filed Critical 스칼러 락, 인크.
Publication of KR20250021401A publication Critical patent/KR20250021401A/ko
Pending legal-status Critical Current

Links

Images

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/22—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against growth factors ; against growth regulators
    • 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
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/519—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
    • 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
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/395—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
    • A61K39/39533—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals
    • A61K39/3955—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals against proteinaceous materials, e.g. enzymes, hormones, lymphokines
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING 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
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/34—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving hydrolase
    • 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
    • G01N33/70—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving creatine or creatinine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/505—Medicinal preparations containing antigens or antibodies comprising antibodies
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/54—Medicinal preparations containing antigens or antibodies characterised by the route of administration
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/545—Medicinal preparations containing antigens or antibodies characterised by the dose, timing or administration schedule
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/55—Medicinal preparations containing antigens or antibodies characterised by the host/recipient, e.g. newborn with maternal antibodies
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/20—Immunoglobulins specific features characterized by taxonomic origin
    • C07K2317/24—Immunoglobulins specific features characterized by taxonomic origin containing regions, domains or residues from different species, e.g. chimeric, humanized or veneered
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/70—Immunoglobulins specific features characterized by effect upon binding to a cell or to an antigen
    • C07K2317/76—Antagonist effect on antigen, e.g. neutralization or inhibition of binding
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination therapy
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00—Detection or diagnosis of diseases
    • G01N2800/28—Neurological disorders
    • G01N2800/2878—Muscular dystrophy
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00—Detection or diagnosis of diseases
    • G01N2800/52—Predicting or monitoring the response to treatment, e.g. for selection of therapy based on assay results in personalised medicine; Prognosis

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • Veterinary Medicine (AREA)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Immunology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Analytical Chemistry (AREA)
  • Urology & Nephrology (AREA)
  • Hematology (AREA)
  • Endocrinology (AREA)
  • Mycology (AREA)
  • Food Science & Technology (AREA)
  • General Physics & Mathematics (AREA)
  • Pathology (AREA)
KR1020247040101A 2022-05-04 2023-05-03 척수 근위축증을 치료하기 위한 마이오스타틴 저해제의 용도 Pending KR20250021401A (ko)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US202263364188P 2022-05-04 2022-05-04
US63/364,188 2022-05-04
US202263366447P 2022-06-15 2022-06-15
US63/366,447 2022-06-15
US202263366662P 2022-06-20 2022-06-20
US63/366,662 2022-06-20
US202263378987P 2022-10-10 2022-10-10
US63/378,987 2022-10-10
PCT/US2023/020843 WO2023215384A2 (en) 2022-05-04 2023-05-03 Use of myostatin inhibitor for treating spinal muscular atrophy

Publications (1)

Publication Number Publication Date
KR20250021401A true KR20250021401A (ko) 2025-02-12

Family

ID=86732326

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020247040101A Pending KR20250021401A (ko) 2022-05-04 2023-05-03 척수 근위축증을 치료하기 위한 마이오스타틴 저해제의 용도

Country Status (13)

Country Link
US (1) US20250326827A1 (enExample)
EP (1) EP4518970B1 (enExample)
JP (1) JP2025514978A (enExample)
KR (1) KR20250021401A (enExample)
CN (1) CN119451696A (enExample)
AU (1) AU2023265846A1 (enExample)
CA (1) CA3251441A1 (enExample)
DK (1) DK4518970T3 (enExample)
FI (1) FI4518970T3 (enExample)
IL (1) IL316463A (enExample)
LT (1) LT4518970T (enExample)
MX (1) MX2024013313A (enExample)
WO (1) WO2023215384A2 (enExample)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PT3922645T (pt) 2015-09-15 2025-05-29 Scholar Rock Inc ¿anticorpos anti-pró-miostatina/miostatina latente e suas utilizações
AU2023413166A1 (en) 2022-12-22 2025-07-03 Scholar Rock, Inc. Selective and potent inhibitory antibodies of myostatin activation
WO2026076383A1 (en) * 2024-10-06 2026-04-09 Scholar Rock, Inc. Use of myostatin inhibitor for treating spinal muscular atrophy

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20040044063A1 (en) 2002-05-31 2004-03-04 Brent Stockwell SMA therapy and cell based assay for identifying therapies
WO2008127722A1 (en) 2007-04-12 2008-10-23 Paratek Pharmaceuticals, Inc. Methods for treating spinal muscular atrophy using tetracycline compounds
NZ730607A (en) 2014-12-19 2022-07-01 Chugai Pharmaceutical Co Ltd Anti-myostatin antibodies, polypeptides containing variant fc regions, and methods of use
PT3922645T (pt) 2015-09-15 2025-05-29 Scholar Rock Inc ¿anticorpos anti-pró-miostatina/miostatina latente e suas utilizações
KR20210082548A (ko) * 2016-06-13 2021-07-05 스칼러 락, 인크. 미오스타틴 억제제의 용도 및 조합 요법
PL4232151T3 (pl) * 2020-10-26 2026-02-23 Scholar Rock, Inc. Zastosowanie przeciwciała przeciwko pro/latentnej miostatynie w leczeniu rdzeniowego zaniku mięśni
BR112023015177A2 (pt) 2021-01-29 2023-11-14 Biocad Joint Stock Co Ácido nucleico compreendendo smn1 e mir-23a para tratamento da atrofia muscular espinhal, cassete de expressão, vetor de expressão, vírus recombinante, composição farmacêutica, uso e método relacionados

Also Published As

Publication number Publication date
WO2023215384A2 (en) 2023-11-09
AU2023265846A1 (en) 2024-12-12
IL316463A (en) 2024-12-01
EP4518970A2 (en) 2025-03-12
CA3251441A1 (en) 2023-11-09
JP2025514978A (ja) 2025-05-13
MX2024013313A (es) 2025-02-10
LT4518970T (lt) 2026-04-27
CN119451696A (zh) 2025-02-14
WO2023215384A3 (en) 2023-12-07
EP4518970B1 (en) 2026-03-25
US20250326827A1 (en) 2025-10-23
DK4518970T3 (da) 2026-04-07
FI4518970T3 (fi) 2026-05-08

Similar Documents

Publication Publication Date Title
EP4218804B1 (en) Use of myostatin inhibitors and combination therapies
US20250326827A1 (en) Use of myostatin inhibitor for treating spinal muscular atrophy
US11918551B2 (en) Methods of treating seizure disorders and Prader-Willi syndrome
EP4232151B1 (en) Use of anti-pro/latent myostatin antibody for treating spinal muscular atrophy
US20240358704A1 (en) Combined administration
WO2026076383A1 (en) Use of myostatin inhibitor for treating spinal muscular atrophy
HK40100646A (en) Use of anti-pro/latent myostatin antibody for treating spinal muscular atrophy
HK40100646B (en) Use of anti-pro/latent myostatin antibody for treating spinal muscular atrophy
EA051098B1 (ru) Применение антитела к промиостатину/латентному миостатину для лечения спинальной мышечной атрофии
HK40043422A (en) Use of myostatin inhibitors and combination therapies
HK40097101A (en) Use of myostatin inhibitors and combination therapies
HK1260302A1 (en) Use of myostatin inhibitors and combination therapies
HK1260302B (en) Use of myostatin inhibitors and combination therapies

Legal Events

Date Code Title Description
PA0105 International application

St.27 status event code: A-0-1-A10-A15-nap-PA0105

PG1501 Laying open of application

St.27 status event code: A-1-1-Q10-Q12-nap-PG1501

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-NAP-X000 (AS PROVIDED BY THE NATIONAL OFFICE)

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000