PL3370705T3 - Dooponowe dostarczanie sekwencji kwasów nukleinowych do kodowania abcd1 w leczeniu adrenomieloneuropatii - Google Patents

Dooponowe dostarczanie sekwencji kwasów nukleinowych do kodowania abcd1 w leczeniu adrenomieloneuropatii

Info

Publication number
PL3370705T3
PL3370705T3 PL16862990.5T PL16862990T PL3370705T3 PL 3370705 T3 PL3370705 T3 PL 3370705T3 PL 16862990 T PL16862990 T PL 16862990T PL 3370705 T3 PL3370705 T3 PL 3370705T3
Authority
PL
Poland
Prior art keywords
adrenomyeloneuropathy
treatment
nucleic acid
acid sequences
sequences encoding
Prior art date
Application number
PL16862990.5T
Other languages
English (en)
Inventor
Casey A. MAGUIRE
Florian EICHLER
Original Assignee
The General Hospital Corporation
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 The General Hospital Corporation filed Critical The General Hospital Corporation
Publication of PL3370705T3 publication Critical patent/PL3370705T3/pl

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
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • C12N7/02—Recovery or purification
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • 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
    • A61K9/0002—Galenical forms characterised by the drug release technique; Application systems commanded by energy
    • A61K9/0004—Osmotic delivery systems; Sustained release driven by osmosis, thermal energy or gas
    • 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
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • 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
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • 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
    • 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
    • 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
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • 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
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151—Methods of production or purification of viral material
    • C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Epidemiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Virology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Immunology (AREA)
  • Cell Biology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Psychology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Toxicology (AREA)
PL16862990.5T 2015-11-05 2016-11-03 Dooponowe dostarczanie sekwencji kwasów nukleinowych do kodowania abcd1 w leczeniu adrenomieloneuropatii PL3370705T3 (pl)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562251208P 2015-11-05 2015-11-05
US201662300691P 2016-02-26 2016-02-26
PCT/US2016/060375 WO2017079467A1 (en) 2015-11-05 2016-11-03 Intrathecal delivery of nucleic acid sequences encoding abcd1 for treatment of adrenomyeloneuropathy

Publications (1)

Publication Number Publication Date
PL3370705T3 true PL3370705T3 (pl) 2022-08-01

Family

ID=58662371

Family Applications (1)

Application Number Title Priority Date Filing Date
PL16862990.5T PL3370705T3 (pl) 2015-11-05 2016-11-03 Dooponowe dostarczanie sekwencji kwasów nukleinowych do kodowania abcd1 w leczeniu adrenomieloneuropatii

Country Status (15)

Country Link
US (3) US20180320176A1 (pl)
EP (2) EP3370705B1 (pl)
JP (2) JP7037490B2 (pl)
KR (1) KR102839699B1 (pl)
CN (2) CN118267490A (pl)
AU (1) AU2016349381B2 (pl)
BR (1) BR112018008993A2 (pl)
CA (1) CA3003747A1 (pl)
ES (1) ES2916325T3 (pl)
IL (1) IL258904B (pl)
MX (1) MX2018005689A (pl)
PL (1) PL3370705T3 (pl)
RU (2) RU2739384C2 (pl)
SG (1) SG11201803353PA (pl)
WO (1) WO2017079467A1 (pl)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PL3370705T3 (pl) * 2015-11-05 2022-08-01 The General Hospital Corporation Dooponowe dostarczanie sekwencji kwasów nukleinowych do kodowania abcd1 w leczeniu adrenomieloneuropatii
KR20220128632A (ko) 2019-12-31 2022-09-21 스완바이오 테라퓨틱스 리미티드 개선된 aav-abcd1 구축물 및 부신백질이영양증 (ald) 및/또는 부신척수신경병증 (amn)의 치료 또는 예방을 위한 용도

Family Cites Families (33)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US1975A (en) 1841-02-12 Manner of constructing corn-shellers
US4458066A (en) 1980-02-29 1984-07-03 University Patents, Inc. Process for preparing polynucleotides
US5609885A (en) 1992-09-15 1997-03-11 Alza Corporation Osmotic membrane and delivery device
US5869039A (en) * 1993-10-15 1999-02-09 Institut National De La Sante Et De La Recherche Medicale X-linked adrenoleukodystrophy gene and corresponding protein
US6156331A (en) 1996-02-02 2000-12-05 Alza Corporation Sustained delivery of an active agent using an implantable system
US6261584B1 (en) 1996-02-02 2001-07-17 Alza Corporation Sustained delivery of an active agent using an implantable system
US6395292B2 (en) 1996-02-02 2002-05-28 Alza Corporation Sustained delivery of an active agent using an implantable system
US6132420A (en) 1996-02-02 2000-10-17 Alza Corporation Osmotic delivery system and method for enhancing start-up and performance of osmotic delivery systems
PL189137B1 (pl) 1996-02-02 2005-06-30 Alza Corp Implantowane urządzenie do dostarczania substancji aktywnej
US6713300B1 (en) * 1997-02-27 2004-03-30 University Of Utah Research Foundation Nucleic acid and amino acid sequences for ATP-binding cassette transporter and methods of screening for agents that modify ATP-binding cassette transporter
ZA981610B (en) 1997-03-24 1999-08-26 Alza Corp Self adjustable exit port.
MY125849A (en) 1997-07-25 2006-08-30 Alza Corp Osmotic delivery system, osmotic delivery system semipermeable body assembly, and method for controlling delivery rate of beneficial agents from osmotic delivery systems
WO1999032095A1 (en) 1997-12-22 1999-07-01 Alza Corporation Rate controlling membranes for controlled drug delivery devices
ES2217613T3 (es) 1997-12-29 2004-11-01 Alza Corporation Sistema de liberacion osmotica con mecanismo de retencion de tapon de membrana.
JP4494629B2 (ja) 1997-12-30 2010-06-30 インターシア セラピューティクス，インコーポレイティド メンブレンプラグを備えた有益な作用剤の供給システム
ATE234603T1 (de) 1998-12-31 2003-04-15 Alza Corp Osmotisches verabreichungsystem mit raumsparenden kolben
US6225525B1 (en) * 1999-10-13 2001-05-01 Ortho-Mcneil Pharmaceutical, Inc. ATP-binding cassette transporter (ABC1) modified transgenic mice
DE60023361T2 (de) 1999-12-21 2006-04-27 Alza Corp., Mountain View Ventil für osmotische vorrichtungen
US7163688B2 (en) 2001-06-22 2007-01-16 Alza Corporation Osmotic implant with membrane and membrane retention means
WO2003004660A1 (en) 2001-07-03 2003-01-16 Institut National De La Sante Et De La Recherche Medicale (Inserm) Methods of administering vectors to synaptically connected neurons
US6939556B2 (en) 2002-06-26 2005-09-06 Alza Corporation Minimally compliant, volume efficient piston for osmotic drug delivery systems
US7014636B2 (en) 2002-11-21 2006-03-21 Alza Corporation Osmotic delivery device having a two-way valve and a dynamically self-adjusting flow channel
CA2520610A1 (en) 2003-03-31 2004-10-21 Alza Corporation Osmotic pump with means for dissipating internal pressure
US20050175701A1 (en) 2004-02-10 2005-08-11 Alza Corporation Capillary moderator for osmotic delivery system
WO2007140416A2 (en) 2006-05-30 2007-12-06 Intarcia Therapeutics, Inc. Two-piece, internal-channel osmotic delivery system flow modulator
PT2359808E (pt) 2006-08-09 2013-08-28 Intarcia Therapeutics Inc Sistemas de entrega osmótica e montagens de pistão
EP2510087A2 (en) * 2009-12-11 2012-10-17 The Brigham and Women's Hospital, Inc. Pathogen restriction factors
WO2011106376A2 (en) * 2010-02-23 2011-09-01 The General Hospital Corporation Use of microvesicles in the treatment of medical conditions
TR201819015T4 (tr) * 2011-06-10 2019-01-21 Bluebird Bio Inc Adrenolökodi̇strofi̇ ve adrenomi̇yelonöropati̇ i̇çi̇n gen terapi̇ vektörleri̇
EP2834259A4 (en) * 2012-04-02 2016-08-24 Moderna Therapeutics Inc MODIFIED POLYNUCLEOTIDES
CA2912678C (en) * 2013-05-15 2023-10-10 Regents Of The University Of Minnesota Adeno-associated virus mediated gene transfer to the central nervous system
GB201401707D0 (en) * 2014-01-31 2014-03-19 Sec Dep For Health The Adeno-associated viral vectors
PL3370705T3 (pl) * 2015-11-05 2022-08-01 The General Hospital Corporation Dooponowe dostarczanie sekwencji kwasów nukleinowych do kodowania abcd1 w leczeniu adrenomieloneuropatii

Also Published As

Publication number Publication date
US20230365967A1 (en) 2023-11-16
RU2018120484A (ru) 2019-12-05
CN108472264A (zh) 2018-08-31
JP7037490B2 (ja) 2022-03-16
US20180320176A1 (en) 2018-11-08
IL258904B (en) 2022-09-01
EP3370705B1 (en) 2022-03-09
AU2016349381A1 (en) 2018-06-21
SG11201803353PA (en) 2018-05-30
EP3370705A1 (en) 2018-09-12
EP3370705A4 (en) 2019-05-15
BR112018008993A2 (en) 2018-10-30
US20190225967A1 (en) 2019-07-25
AU2016349381B2 (en) 2023-02-23
IL258904A (en) 2018-06-28
KR20180066252A (ko) 2018-06-18
MX2018005689A (es) 2018-08-01
RU2020141860A (ru) 2021-04-01
EP4066862A1 (en) 2022-10-05
JP2022064910A (ja) 2022-04-26
WO2017079467A1 (en) 2017-05-11
CN118267490A (zh) 2024-07-02
ES2916325T3 (es) 2022-06-30
CA3003747A1 (en) 2017-05-11
JP2018537124A (ja) 2018-12-20
RU2739384C2 (ru) 2020-12-23
US10519445B2 (en) 2019-12-31
RU2018120484A3 (pl) 2020-03-11
KR102839699B1 (ko) 2025-07-31

Similar Documents

Publication Publication Date Title
IL284651B (en) 3' utr sequences for RNA stabilization
PT3167059T (pt) Estabilização de sequências de adn codificando sequências de poli(a)
SI3118311T1 (sl) Protismiselna nukleinska kislina
GB201701673D0 (en) Methods of well treatment
IL252873A0 (en) The l-tartrate salt of pridopidine
IL249456A0 (en) Methods for preparing analogues of modified nucleotides
IL252296A0 (en) Methods for treating multiple sclerosis
IL253461A0 (en) Hydrogels for the release of therapeutic compounds
IL257908A (en) Methods for the treatment of epilepsy
IL256207B (en) Methods for treating multiple sclerosis
SG11201701462SA (en) Inject insert for epi chamber
PT3215132T (pt) Amantadina para melhorar a marcha em esclerose múltipla
HUE050681T2 (hu) AAV-alapú génterápia szklerózis multiplexre
GB201412578D0 (en) Treatment of neurological diseases
IL253023A0 (en) Methods using antisense oligonucleotides for smad7
PT3142641T (pt) Formulações para o tratamento de hipertiroidismo
GB201404697D0 (en) Attachment of hoof blocks
IL258904A (en) Intrathecal administration of abcd1-encoding nucleic acid sequences for the treatment of adrenomyeloneurial disease
GB201517565D0 (en) Treatment of genetic diseases