EP4712948A2 - Geneditierungssysteme und zusammensetzungen zur behandlung von hämoglobinopathien und verfahren zur verwendung davon - Google Patents
Geneditierungssysteme und zusammensetzungen zur behandlung von hämoglobinopathien und verfahren zur verwendung davonInfo
- Publication number
- EP4712948A2 EP4712948A2 EP24808122.6A EP24808122A EP4712948A2 EP 4712948 A2 EP4712948 A2 EP 4712948A2 EP 24808122 A EP24808122 A EP 24808122A EP 4712948 A2 EP4712948 A2 EP 4712948A2
- Authority
- EP
- European Patent Office
- Prior art keywords
- mol
- certain embodiments
- optionally substituted
- lipid nanoparticle
- lipid
- 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
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
-
- 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
- A61K9/51—Nanocapsules; Nanoparticles
- A61K9/5107—Excipients; Inactive ingredients
- A61K9/5123—Organic compounds, e.g. fats, sugars
-
- 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
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
-
- 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
- C12N2320/32—Special delivery means, e.g. tissue-specific
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Molecular Biology (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Biochemistry (AREA)
- General Health & Medical Sciences (AREA)
- Biophysics (AREA)
- Medicinal Preparation (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202363502846P | 2023-05-17 | 2023-05-17 | |
| US202363584014P | 2023-09-20 | 2023-09-20 | |
| US202363593764P | 2023-10-27 | 2023-10-27 | |
| US202463557134P | 2024-02-23 | 2024-02-23 | |
| US202463570053P | 2024-03-26 | 2024-03-26 | |
| US202463638004P | 2024-04-24 | 2024-04-24 | |
| PCT/US2024/029749 WO2024238828A2 (en) | 2023-05-17 | 2024-05-16 | Gene editing systems and compositions for treatment of hemoglobinopathies and methods of using the same |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| EP4712948A2 true EP4712948A2 (de) | 2026-03-25 |
Family
ID=93520226
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| EP24808122.6A Pending EP4712948A2 (de) | 2023-05-17 | 2024-05-16 | Geneditierungssysteme und zusammensetzungen zur behandlung von hämoglobinopathien und verfahren zur verwendung davon |
Country Status (3)
| Country | Link |
|---|---|
| EP (1) | EP4712948A2 (de) |
| AU (1) | AU2024272060A1 (de) |
| WO (1) | WO2024238828A2 (de) |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN119410609B (zh) * | 2023-11-24 | 2025-10-14 | 山东舜丰生物科技有限公司 | 一种突变的Cas蛋白及其应用 |
| WO2025155753A2 (en) * | 2024-01-17 | 2025-07-24 | Renagade Therapeutics Management Inc. | Improved gene editing system, guides, and methods |
| WO2026158676A1 (zh) * | 2025-01-26 | 2026-07-30 | 深圳深信生物科技有限公司 | 氨基脂质化合物及其应用 |
Family Cites Families (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3749768A1 (de) * | 2018-02-05 | 2020-12-16 | Vertex Pharmaceuticals Incorporated | Materialien und verfahren zur behandlung von hämoglobinopathien |
| IL277234B2 (en) * | 2018-03-14 | 2026-03-01 | Editas Medicine Inc | Systems and methods for treating hemoglobin diseases |
| WO2022251665A1 (en) * | 2021-05-28 | 2022-12-01 | Renagade Therapeutics Management Inc. | Lipid nanoparticles and methods of use thereof |
-
2024
- 2024-05-16 AU AU2024272060A patent/AU2024272060A1/en active Pending
- 2024-05-16 WO PCT/US2024/029749 patent/WO2024238828A2/en not_active Ceased
- 2024-05-16 EP EP24808122.6A patent/EP4712948A2/de active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| WO2024238828A2 (en) | 2024-11-21 |
| AU2024272060A1 (en) | 2026-01-08 |
| WO2024238828A3 (en) | 2024-12-26 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| STAA | Information on the status of an ep patent application or granted ep patent |
Free format text: STATUS: THE INTERNATIONAL PUBLICATION HAS BEEN MADE |
|
| PUAI | Public reference made under article 153(3) epc to a published international application that has entered the european phase |
Free format text: ORIGINAL CODE: 0009012 |
|
| STAA | Information on the status of an ep patent application or granted ep patent |
Free format text: STATUS: REQUEST FOR EXAMINATION WAS MADE |
|
| 17P | Request for examination filed |
Effective date: 20251216 |
|
| AK | Designated contracting states |
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