IL303239A - Compositions and their uses for the treatment of Engelmann syndrome - Google Patents
Compositions and their uses for the treatment of Engelmann syndromeInfo
- Publication number
- IL303239A IL303239A IL303239A IL30323923A IL303239A IL 303239 A IL303239 A IL 303239A IL 303239 A IL303239 A IL 303239A IL 30323923 A IL30323923 A IL 30323923A IL 303239 A IL303239 A IL 303239A
- Authority
- IL
- Israel
- Prior art keywords
- ube3a
- aav
- isoform
- composition according
- seq
- Prior art date
Links
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Classifications
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- 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
- A61K48/005—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
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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- 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
- A61K48/0075—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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
-
- 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
- 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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/93—Ligases (6)
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Biomedical Technology (AREA)
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- Bioinformatics & Cheminformatics (AREA)
- Biochemistry (AREA)
- Epidemiology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Neurosurgery (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Neurology (AREA)
- Microbiology (AREA)
- General Engineering & Computer Science (AREA)
- Toxicology (AREA)
- Gastroenterology & Hepatology (AREA)
- Biophysics (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063119860P | 2020-12-01 | 2020-12-01 | |
| US202163179807P | 2021-04-26 | 2021-04-26 | |
| PCT/US2021/061346 WO2022119890A1 (en) | 2020-12-01 | 2021-12-01 | Compositions and uses thereof for treatment of angelman syndrome |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| IL303239A true IL303239A (en) | 2023-07-01 |
Family
ID=80123326
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL303239A IL303239A (en) | 2020-12-01 | 2021-12-01 | Compositions and their uses for the treatment of Engelmann syndrome |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US20230414785A1 (https=) |
| EP (1) | EP4255500A1 (https=) |
| JP (1) | JP2023551911A (https=) |
| KR (1) | KR20230128001A (https=) |
| AU (1) | AU2021392642A1 (https=) |
| CA (1) | CA3200192A1 (https=) |
| IL (1) | IL303239A (https=) |
| MX (1) | MX2023006445A (https=) |
| WO (1) | WO2022119890A1 (https=) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP4661916A1 (en) * | 2023-02-08 | 2025-12-17 | Ginkgo Bioworks, Inc. | Gene therapy for angelman syndrome |
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| MX359371B (es) | 2001-11-13 | 2018-09-25 | Univ Pennsylvania | Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo. |
| PT1453547T (pt) | 2001-12-17 | 2016-12-28 | Univ Pennsylvania | Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas |
| EP2345731B1 (en) | 2003-09-30 | 2015-10-21 | The Trustees of the University of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
| ES2525067T3 (es) | 2005-04-07 | 2014-12-17 | The Trustees Of The University Of Pennsylvania | Método de incremento de la función de un vector de AAV |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| EP3269395A1 (en) | 2008-11-07 | 2018-01-17 | Massachusetts Institute Of Technology | Aminoalcohol lipidoids and uses thereof |
| WO2011126808A2 (en) | 2010-03-29 | 2011-10-13 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| US9315825B2 (en) | 2010-03-29 | 2016-04-19 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| ES2829199T3 (es) | 2010-11-22 | 2021-05-31 | Amicus Therapeutics Inc | Nuevas secuencias de señalización para mejorar la expresión y segregación de proteínas de enzimas recombinantes y otras proteínas |
| RU2013154295A (ru) | 2011-06-08 | 2015-07-20 | Шир Хьюман Дженетик Терапис, Инк. | КОМПОЗИЦИИ ЛИПИДНЫХ НАНОЧАСТИЦ И СПОСОБЫ ДЛЯ ДОСТАВКИ мРНК |
| FR2977562B1 (fr) | 2011-07-06 | 2016-12-23 | Gaztransport Et Technigaz | Cuve etanche et thermiquement isolante integree dans une structure porteuse |
| US20150126589A1 (en) | 2012-06-08 | 2015-05-07 | Ethris Gmbh | Pulmonary Delivery of Messenger RNA |
| US9567369B2 (en) | 2012-08-03 | 2017-02-14 | A.T. Still University | Method of treating metastatic cancer |
| ES2739288T3 (es) | 2013-09-13 | 2020-01-30 | California Inst Of Techn | Recuperación selectiva |
| SG10202000537RA (en) | 2014-02-28 | 2020-03-30 | Univ Bologna Alma Mater Studiorum | Tatk-cdkl5 fusion proteins, compositions, formulations, and use thereof |
| EP3387138B1 (en) | 2015-12-11 | 2022-01-26 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav9 |
| WO2017100674A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav1 |
| EP3411484B1 (en) * | 2016-02-05 | 2023-10-04 | Emory University | Injection of single-stranded or self-complementary adeno-associated virus 9 into the cerebrospinal fluid |
| EP3475418A4 (en) | 2016-06-28 | 2020-03-04 | Alma Mater Studiorum -Universita' di Bologna | TATK-CDKL5 FUSION PROTEINS, COMPOSITIONS, FORMULATIONS AND THEIR USE |
| PT3589730T (pt) | 2017-02-28 | 2024-02-28 | Univ Pennsylvania | Vetor de vírus adenoassociado (aav) de clado f e suas utilizações |
| BR112019027692A2 (pt) | 2017-06-28 | 2020-11-24 | University Of South Florida | gene ube3a modificado para uma abordagem terapêutica genética para a síndrome de angelman |
| JP2021505135A (ja) | 2017-11-30 | 2021-02-18 | アミカス セラピューティックス インコーポレイテッド | Ｃｄｋｌ５発現変異体及びｃｄｋｌ５融合タンパク質 |
| BR112020021962A2 (pt) | 2018-04-30 | 2021-01-26 | Amicus Therapeutics, Inc. | construtos para terapia gênica e métodos de uso |
| KR20210040358A (ko) * | 2018-06-14 | 2021-04-13 | 오비드 테라퓨틱스 인크. | 안젤만 증후군의 치료에서 mir-92a 또는 mir-145의 사용 |
| WO2020132455A1 (en) * | 2018-12-21 | 2020-06-25 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| CN114206393A (zh) * | 2019-03-21 | 2022-03-18 | Ptc医疗公司 | 用于治疗天使综合征的载体和方法 |
| SG11202111375RA (en) * | 2019-04-29 | 2021-11-29 | Univ Pennsylvania | Novel aav capsids and compositions containing same |
| US12465655B2 (en) * | 2019-05-22 | 2025-11-11 | The University Of North Carolina At Chapel Hill | UBE3A genes and expression cassettes and their use |
| IT201900008877A1 (it) | 2019-06-13 | 2020-12-13 | Univ Bologna Alma Mater Studiorum | Nuovi costrutti per terapia genica |
| KR20220077921A (ko) | 2019-10-10 | 2022-06-09 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 변이체 igf2 작제물 |
| MX2022004812A (es) * | 2019-10-22 | 2023-02-23 | Applied Genetic Tech Corporation | Sistemas de virus adeno-asociados (aav) para el tratamiento de enfermedades o trastornos neurodegenerativos asociados a la progranulina. |
| US20230043046A1 (en) | 2019-10-30 | 2023-02-09 | Amicus Therapeutics, Inc. | Recombinant CDKL5 Proteins, Gene Therapy and Production Methods |
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2021
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- 2021-12-01 AU AU2021392642A patent/AU2021392642A1/en active Pending
- 2021-12-01 US US18/254,893 patent/US20230414785A1/en active Pending
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- 2021-12-01 CA CA3200192A patent/CA3200192A1/en active Pending
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| MX2023006445A (es) | 2023-08-10 |
| CA3200192A1 (en) | 2022-06-09 |
| WO2022119890A9 (en) | 2023-07-27 |
| WO2022119890A1 (en) | 2022-06-09 |
| EP4255500A1 (en) | 2023-10-11 |
| AU2021392642A1 (en) | 2023-06-22 |
| US20230414785A1 (en) | 2023-12-28 |
| JP2023551911A (ja) | 2023-12-13 |
| KR20230128001A (ko) | 2023-09-01 |
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