SG11201805320XA - Materials and methods for treatment of amyotrophic lateral sclerosis and/or frontal temporal lobular degeneration - Google Patents
Materials and methods for treatment of amyotrophic lateral sclerosis and/or frontal temporal lobular degenerationInfo
- Publication number
- SG11201805320XA SG11201805320XA SG11201805320XA SG11201805320XA SG11201805320XA SG 11201805320X A SG11201805320X A SG 11201805320XA SG 11201805320X A SG11201805320X A SG 11201805320XA SG 11201805320X A SG11201805320X A SG 11201805320XA SG 11201805320X A SG11201805320X A SG 11201805320XA
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- SG
- Singapore
- Prior art keywords
- international
- methods
- lateral sclerosis
- amyotrophic lateral
- materials
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- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/90—Stable introduction of foreign DNA into chromosome
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
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Abstract
INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau „ (10) International Publication Number (43) International Publication Date WO 2017/109757 A1 29 June 2017 (29.06.2017) WIPO I PCT (51) International Patent Classification: C12N15/113 (2010.01) A61K 48/00 (2006.01) C12N15/90 (2006.01) (21) International Application Number: PCT/IB2016/057958 (22) International Filing Date: 22 December 2016 (22.12.2016) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/387,424 23 December 2015 (23.12.2015) US 62/324,281 18 April 2016 (18.04.2016) US (71) Applicant: CRISPR THERAPEUTICS AG [CH/CH]; Aeschenvorstadt 36, 4051 Basel (CH). (72) Inventors: COWAN, Chad Albert; 675 West Kendall Street, Cambridge, MA 02142 (US). KABADI, Ami Meda; 675 West Kendall Street, Cambridge, MA 02142 (US). LUNDBERG, Ante Sven; 675 West Kendall Street, Cambridge, MA 02142 (US). (81) Designated States (unless otherwise indicated, for every kind of national protection available)'. AE, AG, AL, AM, 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, PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW. (84) Designated States (unless otherwise indicated, for every kind of regional protection available)'. ARIPO (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, TJ, 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, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG). Published: — with international search report (Art. 21(3)) — before the expiration of the time limit for amending the claims and to be republished in the event of receipt of amendments (Rule 48.2(h)) — with sequence listing part of description (Rule 5.2(a)) (54) Title: MATERIALS AND METHODS FOR TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS AND/OR FRONTAL TEMPORAL LOBULAR DEGENERATION C90RF IVT screen 180 i> i> o\ © 1181® IP! ip 'to 1 11 p i I: : H l!i illiilillililililliiliiii! £ ilp I iilililll Sill ifllll - s. ,§5p,5 5!111 s11!141!IIts!11111!I!14!I!!I!III!11!!!!11!If o CJ o (57) : The present application provides materials and methods for treating a patient with Amyotrophic Lateral Sclerosis (ALS) and/or Frontaltemporal Lobular Degeneration (FTLD), both ex vivo and in vivo. In addition, the present application provides materials and methods for editing to modulate the expression, function or activity of the C90RF72 gene in a cell by genome editing.
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201562387424P | 2015-12-23 | 2015-12-23 | |
US201662324281P | 2016-04-18 | 2016-04-18 | |
PCT/IB2016/057958 WO2017109757A1 (en) | 2015-12-23 | 2016-12-22 | Materials and methods for treatment of amyotrophic lateral sclerosis and/or frontal temporal lobular degeneration |
Publications (1)
Publication Number | Publication Date |
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SG11201805320XA true SG11201805320XA (en) | 2018-07-30 |
Family
ID=57956331
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
SG11201805320XA SG11201805320XA (en) | 2015-12-23 | 2016-12-22 | Materials and methods for treatment of amyotrophic lateral sclerosis and/or frontal temporal lobular degeneration |
Country Status (11)
Country | Link |
---|---|
US (1) | US20210260219A1 (en) |
EP (1) | EP3394260B1 (en) |
JP (1) | JP6947729B2 (en) |
KR (1) | KR20180118111A (en) |
CN (1) | CN109312339B (en) |
AU (1) | AU2016376191A1 (en) |
BR (1) | BR112018012894A2 (en) |
CA (1) | CA3009308A1 (en) |
MX (1) | MX2018007840A (en) |
SG (1) | SG11201805320XA (en) |
WO (1) | WO2017109757A1 (en) |
Families Citing this family (56)
Publication number | Priority date | Publication date | Assignee | Title |
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WO2013066438A2 (en) | 2011-07-22 | 2013-05-10 | President And Fellows Of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
US9163284B2 (en) | 2013-08-09 | 2015-10-20 | President And Fellows Of Harvard College | Methods for identifying a target site of a Cas9 nuclease |
US9359599B2 (en) | 2013-08-22 | 2016-06-07 | President And Fellows Of Harvard College | Engineered transcription activator-like effector (TALE) domains and uses thereof |
US9228207B2 (en) | 2013-09-06 | 2016-01-05 | President And Fellows Of Harvard College | Switchable gRNAs comprising aptamers |
US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
US9322037B2 (en) | 2013-09-06 | 2016-04-26 | President And Fellows Of Harvard College | Cas9-FokI fusion proteins and uses thereof |
US20150165054A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting caspase-9 point mutations |
EP3750907A3 (en) | 2014-03-18 | 2021-04-28 | University of Massachusetts | Raav-based compositions and methods for treating amyotrophic lateral sclerosis |
US10077453B2 (en) | 2014-07-30 | 2018-09-18 | President And Fellows Of Harvard College | CAS9 proteins including ligand-dependent inteins |
RU2020115485A (en) | 2015-05-29 | 2020-06-11 | Регенерон Фармасьютикалс, Инк. | ANIMALS, DIFFERENT FROM HUMAN, WITH VIOLATION IN LOCUS C9ORF72 |
EP3365357B1 (en) | 2015-10-23 | 2024-02-14 | President and Fellows of Harvard College | Evolved cas9 proteins for gene editing |
EP3478841A1 (en) * | 2016-06-29 | 2019-05-08 | Crispr Therapeutics AG | Materials and methods for treatment of apolipoprotein c3 (apociii)-related disorders |
WO2018007871A1 (en) * | 2016-07-08 | 2018-01-11 | Crispr Therapeutics Ag | Materials and methods for treatment of transthyretin amyloidosis |
AU2017306676B2 (en) | 2016-08-03 | 2024-02-22 | President And Fellows Of Harvard College | Adenosine nucleobase editors and uses thereof |
AU2017308889B2 (en) | 2016-08-09 | 2023-11-09 | President And Fellows Of Harvard College | Programmable Cas9-recombinase fusion proteins and uses thereof |
US11542509B2 (en) | 2016-08-24 | 2023-01-03 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
WO2018064600A1 (en) * | 2016-09-30 | 2018-04-05 | Regeneron Pharmaceuticals, Inc. | Non-human animals having a hexanucleotide repeat expansion in a c9orf72 locus |
SG11201903089RA (en) | 2016-10-14 | 2019-05-30 | Harvard College | Aav delivery of nucleobase editors |
GB201619876D0 (en) | 2016-11-24 | 2017-01-11 | Cambridge Entpr Ltd | Controllable transcription |
WO2018119359A1 (en) | 2016-12-23 | 2018-06-28 | President And Fellows Of Harvard College | Editing of ccr5 receptor gene to protect against hiv infection |
EP3585895A1 (en) | 2017-02-22 | 2020-01-01 | CRISPR Therapeutics AG | Compositions and methods for gene editing |
WO2018165504A1 (en) | 2017-03-09 | 2018-09-13 | President And Fellows Of Harvard College | Suppression of pain by gene editing |
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- 2016-12-22 AU AU2016376191A patent/AU2016376191A1/en not_active Abandoned
- 2016-12-22 MX MX2018007840A patent/MX2018007840A/en unknown
- 2016-12-22 CN CN201680082299.7A patent/CN109312339B/en active Active
- 2016-12-22 EP EP16831921.8A patent/EP3394260B1/en active Active
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JP2018538002A (en) | 2018-12-27 |
CN109312339A (en) | 2019-02-05 |
BR112018012894A2 (en) | 2018-12-04 |
WO2017109757A1 (en) | 2017-06-29 |
EP3394260A1 (en) | 2018-10-31 |
KR20180118111A (en) | 2018-10-30 |
EP3394260B1 (en) | 2021-02-17 |
CA3009308A1 (en) | 2017-06-29 |
AU2016376191A1 (en) | 2018-07-12 |
MX2018007840A (en) | 2019-05-02 |
CN109312339B (en) | 2022-01-28 |
US20210260219A1 (en) | 2021-08-26 |
JP6947729B2 (en) | 2021-10-13 |
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