SG11201810162PA - Polynucleotides encoding citrin for the treatment of citrullinemia type 2 - Google Patents
Polynucleotides encoding citrin for the treatment of citrullinemia type 2Info
- Publication number
- SG11201810162PA SG11201810162PA SG11201810162PA SG11201810162PA SG11201810162PA SG 11201810162P A SG11201810162P A SG 11201810162PA SG 11201810162P A SG11201810162P A SG 11201810162PA SG 11201810162P A SG11201810162P A SG 11201810162PA SG 11201810162P A SG11201810162P A SG 11201810162PA
- Authority
- SG
- Singapore
- Prior art keywords
- cambridge
- modernatx
- massachusetts
- square
- citrin
- Prior art date
Links
Classifications
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- 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
-
- 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
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
-
- 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
- A61K9/513—Organic macromolecular compounds; Dendrimers
- A61K9/5146—Organic macromolecular compounds; Dendrimers obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyethylene glycol, polyamines, polyanhydrides
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P13/00—Drugs for disorders of the urinary 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
Abstract
INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property MD 1101111 0 111010101111101011111 0 011111111111111111111111111111111111111111111111 Organization International Bureau (10) International Publication Number (43) International Publication Date .....0\"\"\"1 WO 2017/201349 Al 23 November 2017 (23.11.2017) WIPO I PCT (51) International Patent Classification: SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, TR, A61K 38/17 (2006.01) A61P 3/00 (2006.01) TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW. C07K 14/47 (2006.01) A61K 9/127 (2006.01) (84) Designated States (unless otherwise indicated, for every (21) International Application Number: kind of regional protection available): ARIPO (BW, GH, PCT/US2017/033421 GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, (22) International Filing Date: UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, TJ, 18 May 2017 (18.05.2017) TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, (25) Filing Language: English MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, (26) Publication Language: English TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG). (30) Priority Data: 62/338,479 18 May 2016 (18.05.2016) US Published: — with international search report (Art. 21(3)) (71) Applicant: MODERNATX, INC. [US/US]; 200 Technol- — with sequence listing part of description (Rule 5.2(a)) ogy Square, Cambridge, Massachusetts 02139 (US). (72) Inventors: MARTINI, Paolo; c/o Modernatx, Inc., 200 Technology Square, Cambridge, Massachusetts 02139 (US). HOGE, Stephen; c/o Modernatx, Inc., 200 Tech- nology Square, Cambridge, Massachusetts 02139 (US). BENENATO, Kerry; c/o Modernatx, Inc., 200 Tech- nology Square, Cambridge, Massachusetts 02139 (US). PRESNYAK, Vladimir; c/o Modernatx, Inc., 200 Tech- nology Square, Cambridge, Massachusetts 02139 (US). MCFADYEN, lain; c/o Modernatx, Inc., 200 Technolo- Square, Cambridge, Massachusetts 02139 (US). KU- gy MARASINGHE, Ellalahewage Sathyajith; c/o Moder- natx, Inc., 200 Technology Square, Cambridge, Massachu- setts 02139 (US). CAO, Jinsong; c/o Modernatx, Inc., 200 Technology Square, Cambridge, Massachusetts 02139 (US). GUEY, Lin Tung; c/o Modernatx, Inc., 200 Technol- ogy Square, Cambridge, Massachusetts 02139 (US). SAB- NIS, Staci; c/o Modernatx, Inc., 200 Technology Square, Cambridge, Massachusetts 02139 (US). (74) Agent: NANNENGA-COMBS, Bonnie W. Sterne, et al.; Kessler, Goldstein & Fox P.L.L.C, 1100 New York Avenue, NW, Washington, District of Columbia 20005 (US). (81) Designated States (unless otherwise indicated, for every kind of national available): AE, AG, AL, AM, protection AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KH, KN, KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, 1-1 PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, SC, C:r 71- (54) Title: POLYNUCLEOTIDES ENCODING CITRIN FOR THE TREATMENT OF CITRULLINEMIA TYPE 2 M 1-1 (57) : The invention relates to mRNA therapy for the treatment of Citrullinemia Type 2 (\"CTLN2\"). mRNAs for use in the invention, when administered in vivo, encode human Citrin, isoforms thereof, functional fragments thereof, and fusion proteins ei ---- comprising Citrin. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells IN and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of Citrin © expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of biomarkers associated with deficient f'1 Citrin activity in subjects, namely ammonia and/or triglycerides. C
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662338479P | 2016-05-18 | 2016-05-18 | |
| PCT/US2017/033421 WO2017201349A1 (en) | 2016-05-18 | 2017-05-18 | Polynucleotides encoding citrin for the treatment of citrullinemia type 2 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| SG11201810162PA true SG11201810162PA (en) | 2018-12-28 |
Family
ID=59009788
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| SG11201810162PA SG11201810162PA (en) | 2016-05-18 | 2017-05-18 | Polynucleotides encoding citrin for the treatment of citrullinemia type 2 |
Country Status (9)
| Country | Link |
|---|---|
| US (2) | US10993918B2 (en) |
| EP (1) | EP3458081A1 (en) |
| JP (2) | JP7210287B2 (en) |
| AU (2) | AU2017268396B2 (en) |
| CA (1) | CA3024625A1 (en) |
| MA (1) | MA45036A (en) |
| MY (1) | MY201498A (en) |
| SG (1) | SG11201810162PA (en) |
| WO (1) | WO2017201349A1 (en) |
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-
2017
- 2017-05-18 SG SG11201810162PA patent/SG11201810162PA/en unknown
- 2017-05-18 EP EP17728001.3A patent/EP3458081A1/en active Pending
- 2017-05-18 CA CA3024625A patent/CA3024625A1/en active Pending
- 2017-05-18 AU AU2017268396A patent/AU2017268396B2/en active Active
- 2017-05-18 MY MYPI2018001974A patent/MY201498A/en unknown
- 2017-05-18 MA MA045036A patent/MA45036A/en unknown
- 2017-05-18 JP JP2018560776A patent/JP7210287B2/en active Active
- 2017-05-18 US US16/302,360 patent/US10993918B2/en active Active
- 2017-05-18 WO PCT/US2017/033421 patent/WO2017201349A1/en unknown
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2021
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2023
- 2023-01-11 JP JP2023002608A patent/JP2023055716A/en active Pending
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Also Published As
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| US20190175517A1 (en) | 2019-06-13 |
| JP2023055716A (en) | 2023-04-18 |
| MA45036A (en) | 2019-03-27 |
| AU2023216799A1 (en) | 2023-09-07 |
| CA3024625A1 (en) | 2017-11-23 |
| WO2017201349A1 (en) | 2017-11-23 |
| US20220071915A1 (en) | 2022-03-10 |
| US10993918B2 (en) | 2021-05-04 |
| JP7210287B2 (en) | 2023-01-23 |
| AU2017268396B2 (en) | 2023-05-18 |
| AU2017268396A1 (en) | 2018-12-06 |
| JP2019519511A (en) | 2019-07-11 |
| MY201498A (en) | 2024-02-27 |
| EP3458081A1 (en) | 2019-03-27 |
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