BR112019025732A2 - vetores de aav auto-reguladores para expressão segura de mecp2 na síndrome de rett - Google Patents

vetores de aav auto-reguladores para expressão segura de mecp2 na síndrome de rett Download PDF

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Publication number
BR112019025732A2
BR112019025732A2 BR112019025732-2A BR112019025732A BR112019025732A2 BR 112019025732 A2 BR112019025732 A2 BR 112019025732A2 BR 112019025732 A BR112019025732 A BR 112019025732A BR 112019025732 A2 BR112019025732 A2 BR 112019025732A2
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BR
Brazil
Prior art keywords
mir
hsa
mirna
nucleic acid
expression
Prior art date
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BR112019025732-2A
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English (en)
Portuguese (pt)
Inventor
Miguel Sena ESTEVES
Guangping Gao
Michael R. Green
Dan Wang
Tessa Mercedes SIMONE
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University Of Massachusetts
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Application filed by University Of Massachusetts filed Critical University Of Massachusetts
Publication of BR112019025732A2 publication Critical patent/BR112019025732A2/pt

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    • 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
    • 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
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/40—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation
    • C07K2319/41—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation containing a Myc-tag
    • 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]
    • C12N2310/141—MicroRNAs, miRNAs
    • 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
    • C12N2330/00—Production
    • C12N2330/10—Production naturally occurring
    • 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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/007—Vectors comprising a special translation-regulating system cell or tissue specific
    • 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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/10—Vectors comprising a special translation-regulating system regulates levels of translation

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Physics & Mathematics (AREA)
  • Toxicology (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
BR112019025732-2A 2017-06-06 2018-06-06 vetores de aav auto-reguladores para expressão segura de mecp2 na síndrome de rett BR112019025732A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762516060P 2017-06-06 2017-06-06
US62/516,060 2017-06-06
PCT/US2018/036200 WO2018226785A1 (en) 2017-06-06 2018-06-06 Self-regulating aav vectors for safe expression of mecp2 in rett syndrome

Publications (1)

Publication Number Publication Date
BR112019025732A2 true BR112019025732A2 (pt) 2020-06-30

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
BR112019025732-2A BR112019025732A2 (pt) 2017-06-06 2018-06-06 vetores de aav auto-reguladores para expressão segura de mecp2 na síndrome de rett

Country Status (15)

Country Link
US (2) US11680275B2 (enExample)
EP (1) EP3635122A4 (enExample)
JP (2) JP2020522269A (enExample)
KR (1) KR20200015701A (enExample)
CN (1) CN111065742A (enExample)
AU (1) AU2018281145A1 (enExample)
BR (1) BR112019025732A2 (enExample)
CA (1) CA3066623A1 (enExample)
CL (1) CL2019003582A1 (enExample)
CO (1) CO2019014840A2 (enExample)
EA (1) EA201992882A1 (enExample)
IL (1) IL271170A (enExample)
MX (1) MX2019014760A (enExample)
SG (1) SG11201911737PA (enExample)
WO (1) WO2018226785A1 (enExample)

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JP7106534B2 (ja) * 2016-11-17 2022-07-26 ネイションワイド チルドレンズ ホスピタル， インコーポレイテッド メチル－ｃｐｇ結合タンパク質２をコードする組換えアデノ随伴ウイルスの髄腔内送達
WO2018138371A1 (en) 2017-01-30 2018-08-02 Brainvectis Expression vector for cholesterol 24-hydrolase in therapy of polyglutamine repeat spinocerebellar ataxias
SG11202009451VA (en) 2018-04-03 2020-10-29 Stridebio Inc Antibody-evading virus vectors
JP7406677B2 (ja) 2018-04-03 2023-12-28 ギンコ バイオワークス インコーポレイテッド 抗体を回避するウイルスベクター
CA3094465A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
US12467046B2 (en) 2018-10-02 2025-11-11 Voyager Therapeutics, Inc. Redirection of tropism of AAV capsids
CA3133453A1 (en) 2019-03-21 2020-09-24 Daniel Mccoy Recombinant adeno-associated virus vectors
KR20220003553A (ko) * 2019-04-24 2022-01-10 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 레트 증후군의 치료에 유용한 조성물
EP3972631B1 (en) * 2019-05-21 2024-07-10 Institut National de la Santé et de la Recherche Médicale (INSERM) Expression vector for cholesterol 24-hydrolase in therapy of rett syndrome
TW202128736A (zh) 2019-10-17 2021-08-01 美商史崔德生物公司 用於治療ｃ型尼曼—匹克病之腺相關病毒載體
JP7702949B2 (ja) * 2019-12-05 2025-07-04 ザ ボード オブ リージェンツ オブ ザ ユニバーシティー オブ テキサス システム ヒトｍｅｃｐ２遺伝子を発現するように設計されたトランスジーンカセット
WO2021183433A1 (en) 2020-03-09 2021-09-16 University Of Massachusetts Gene replacement therapy for foxg1 syndrome
CN116096734A (zh) * 2020-05-13 2023-05-09 沃雅戈治疗公司 Aav衣壳的向性的重定向
IL299414A (en) * 2020-06-30 2023-02-01 Univ Court Univ Of Edinburgh Transgene expression system
CN116745409A (zh) * 2020-08-19 2023-09-12 萨雷普塔治疗公司 用于治疗雷特综合征的腺相关病毒载体
CN113717248B (zh) * 2020-09-30 2022-07-08 广州派真生物技术有限公司 腺相关病毒突变体及其应用
WO2022094078A1 (en) * 2020-10-28 2022-05-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
US20250001012A1 (en) 2021-11-02 2025-01-02 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
GB202201242D0 (en) * 2022-01-31 2022-03-16 Univ Edinburgh Recombinant optimized mecp2 cassettes and methods for treating rett syndrome and related disorders
AU2024241563A1 (en) * 2023-03-22 2025-10-09 Consiglio Nazionale Delle Ricerche Gene therapy
CN121175427A (zh) * 2023-05-15 2025-12-19 上海金珂博生物技术有限公司 一种Rett综合症的基因治疗
TW202513796A (zh) * 2023-06-09 2025-04-01 大陸商仁景（蘇州）生物科技有限公司 表達可調控的工程化ｒｎａ分子及其用途

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Also Published As

Publication number Publication date
CO2019014840A2 (es) 2020-01-17
IL271170A (en) 2020-01-30
EA201992882A1 (ru) 2020-05-25
US20200181646A1 (en) 2020-06-11
US11680275B2 (en) 2023-06-20
KR20200015701A (ko) 2020-02-12
EP3635122A1 (en) 2020-04-15
AU2018281145A1 (en) 2020-01-02
EP3635122A4 (en) 2021-03-31
JP2023164447A (ja) 2023-11-10
JP2020522269A (ja) 2020-07-30
CN111065742A (zh) 2020-04-24
WO2018226785A1 (en) 2018-12-13
MX2019014760A (es) 2020-08-03
CA3066623A1 (en) 2019-12-13
US20230416779A1 (en) 2023-12-28
SG11201911737PA (en) 2020-01-30
CL2019003582A1 (es) 2020-09-25

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Legal Events

Date Code Title Description
B350 Update of information on the portal [chapter 15.35 patent gazette]
B06W Patent application suspended after preliminary examination (for patents with searches from other patent authorities) chapter 6.23 patent gazette]
B08F Application dismissed because of non-payment of annual fees [chapter 8.6 patent gazette]

Free format text: REFERENTE A 7A ANUIDADE.

B08K Patent lapsed as no evidence of payment of the annual fee has been furnished to inpi [chapter 8.11 patent gazette]

Free format text: EM VIRTUDE DO ARQUIVAMENTO PUBLICADO NA RPI 2830 DE 01-04-2025 E CONSIDERANDO AUSENCIA DE MANIFESTACAO DENTRO DOS PRAZOS LEGAIS, INFORMO QUE CABE SER MANTIDO O ARQUIVAMENTO DO PEDIDO DE PATENTE, CONFORME O DISPOSTO NO ARTIGO 12, DA RESOLUCAO 113/2013.