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LT3329002T - Transtiretino (ttr) irnr kompozicijos ir jų naudojimo būdai, skirti naudoti su ttr susijusių ligų gydymui arba prevencijai - Google Patents

Transtiretino (ttr) irnr kompozicijos ir jų naudojimo būdai, skirti naudoti su ttr susijusių ligų gydymui arba prevencijai

Info

Publication number
LT3329002T
LT3329002T LTEP16750567.6T LT16750567T LT3329002T LT 3329002 T LT3329002 T LT 3329002T LT 16750567 T LT16750567 T LT 16750567T LT 3329002 T LT3329002 T LT 3329002T
Authority
LT
Lithuania
Prior art keywords
ttr
transthyretin
treating
methods
associated diseases
Prior art date
Application number
LTEP16750567.6T
Other languages
English (en)
Inventor
Tracy Zimmermann
Amy Chan
Vasant Jadhav
Martin Maier
Kallanthottathil Rajeev
Original Assignee
Alnylam Pharmaceuticals, Inc.
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 Alnylam Pharmaceuticals, Inc. filed Critical Alnylam Pharmaceuticals, Inc.
Publication of LT3329002T publication Critical patent/LT3329002T/lt

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    • 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
    • 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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • 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
    • A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
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    • A61P5/14—Drugs for disorders of the endocrine system of the thyroid hormones, e.g. T3, T4
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    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
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    • 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
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    • C12N2310/35—Nature of the modification
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LTEP16750567.6T 2015-07-31 2016-07-28 Transtiretino (ttr) irnr kompozicijos ir jų naudojimo būdai, skirti naudoti su ttr susijusių ligų gydymui arba prevencijai LT3329002T (lt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562199563P 2015-07-31 2015-07-31
US201662287518P 2016-01-27 2016-01-27
PCT/US2016/044359 WO2017023660A1 (en) 2015-07-31 2016-07-28 TRANSTHYRETIN (TTR) iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING OR PREVENTING TTR-ASSOCIATED DISEASES

Publications (1)

Publication Number Publication Date
LT3329002T true LT3329002T (lt) 2021-01-11

Family

ID=56682264

Family Applications (2)

Application Number Title Priority Date Filing Date
LTEP16750567.6T LT3329002T (lt) 2015-07-31 2016-07-28 Transtiretino (ttr) irnr kompozicijos ir jų naudojimo būdai, skirti naudoti su ttr susijusių ligų gydymui arba prevencijai
LTPA2023505C LTC3329002I2 (lt) 2015-07-31 2023-02-08

Family Applications After (1)

Application Number Title Priority Date Filing Date
LTPA2023505C LTC3329002I2 (lt) 2015-07-31 2023-02-08

Country Status (35)

Country Link
US (5) US10208307B2 (lt)
EP (1) EP3329002B1 (lt)
JP (4) JP6896703B2 (lt)
KR (2) KR20240074895A (lt)
CN (2) CN116064515A (lt)
BR (1) BR112018000542B1 (lt)
CA (1) CA2994285A1 (lt)
CL (1) CL2018000198A1 (lt)
CY (1) CY1124545T1 (lt)
DK (1) DK3329002T3 (lt)
EA (1) EA201890394A1 (lt)
ES (1) ES2842300T3 (lt)
FI (1) FIC20230006I1 (lt)
FR (1) FR23C1008I2 (lt)
HK (1) HK1256683A1 (lt)
HR (1) HRP20202082T1 (lt)
HU (2) HUE051998T2 (lt)
IL (4) IL296476B2 (lt)
LT (2) LT3329002T (lt)
LU (1) LUC00297I2 (lt)
MA (1) MA43335B1 (lt)
MX (2) MX2018000981A (lt)
MY (1) MY195720A (lt)
NL (1) NL301216I2 (lt)
NO (1) NO2023007I1 (lt)
PL (1) PL3329002T3 (lt)
PT (1) PT3329002T (lt)
RS (1) RS61297B1 (lt)
SG (1) SG10201912341SA (lt)
SI (1) SI3329002T1 (lt)
SM (1) SMT202100020T1 (lt)
TW (4) TW202532647A (lt)
UA (1) UA126276C2 (lt)
WO (1) WO2017023660A1 (lt)
ZA (2) ZA201800571B (lt)

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KR101885383B1 (ko) 2009-07-06 2018-08-03 웨이브 라이프 사이언시스 리미티드 신규한 핵산 프로드러그 및 그의 사용 방법
KR20180026798A (ko) 2010-04-29 2018-03-13 아이오니스 파마수티컬즈, 인코포레이티드 트랜스티레틴 발현의 조절
US10428019B2 (en) 2010-09-24 2019-10-01 Wave Life Sciences Ltd. Chiral auxiliaries
EP2734208B1 (en) 2011-07-19 2017-03-01 Wave Life Sciences Ltd. Methods for the synthesis of functionalized nucleic acids
LT3366775T (lt) 2011-11-18 2022-08-10 Alnylam Pharmaceuticals, Inc. Modifikuoti rnri agentai
RU2678807C2 (ru) 2011-11-18 2019-02-01 Элнилэм Фармасьютикалз, Инк. СРЕДСТВА ДЛЯ РНКи, КОМПОЗИЦИИ И СПОСОБЫ ИХ ПРИМЕНЕНИЯ ДЛЯ ЛЕЧЕНИЯ ЗАБОЛЕВАНИЙ, АССОЦИИРОВАННЫХ С ТРАНСТИРЕТИНОМ (TTR)
CN104661664B (zh) 2012-07-13 2020-07-03 波涛生命科学有限公司 手性控制
PL2872485T3 (pl) 2012-07-13 2021-05-31 Wave Life Sciences Ltd. Asymetryczna grupa pomocnicza
WO2015108047A1 (ja) 2014-01-15 2015-07-23 株式会社新日本科学 免疫誘導活性を有するキラル核酸アジュバンド及び免疫誘導活性剤
JPWO2015108048A1 (ja) 2014-01-15 2017-03-23 株式会社新日本科学 抗腫瘍作用を有するキラル核酸アジュバンド及び抗腫瘍剤
EP4137572A1 (en) 2014-01-16 2023-02-22 Wave Life Sciences Ltd. Chiral design
JP6896703B2 (ja) 2015-07-31 2021-06-30 アルナイラム ファーマシューティカルズ， インコーポレイテッドＡｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． ＴＴＲ関連疾患を治療または予防するためのトランスサイレチン（ＴＴＲ）ｉＲＮＡ組成物およびその使用方法
JOP20210043A1 (ar) * 2015-10-01 2017-06-16 Arrowhead Pharmaceuticals Inc تراكيب وأساليب لتثبيط تعبير جيني للـ lpa
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US11806360B2 (en) 2017-09-19 2023-11-07 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating transthyretin (TTR) mediated amyloidosis
CA3083968C (en) * 2017-12-01 2024-04-23 Suzhou Ribo Life Science Co., Ltd. Double-stranded oligonucleotide, composition and conjugate comprising double-stranded oligonucleotide, preparation method thereof and use thereof
AU2019350765A1 (en) 2018-09-28 2021-05-27 Alnylam Pharmaceuticals, Inc. Transthyretin (TTR) iRNA compositions and methods of use thereof for treating or preventing TTR-associated ocular diseases
EP4022062A1 (en) 2019-08-30 2022-07-06 Alnylam Pharmaceuticals, Inc. Neurofilament light chain (nfl) as a biomarker for transthyretin amyloidosis polyneuropathy
EP4055165A1 (en) * 2019-11-06 2022-09-14 Alnylam Pharmaceuticals, Inc. Transthyretin (ttr) irna compositions and methods of use thereof for treating or preventing ttr-associated ocular diseases
KR20220152270A (ko) * 2020-03-06 2022-11-15 알닐람 파마슈티칼스 인코포레이티드 트랜스티레틴 (ttr)의 발현을 억제하기 위한 조성물 및 방법
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