CA3161901A1 - Novel cellular delivery methods - Google Patents

Novel cellular delivery methods Download PDF

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Publication number
CA3161901A1
CA3161901A1 CA3161901A CA3161901A CA3161901A1 CA 3161901 A1 CA3161901 A1 CA 3161901A1 CA 3161901 A CA3161901 A CA 3161901A CA 3161901 A CA3161901 A CA 3161901A CA 3161901 A1 CA3161901 A1 CA 3161901A1
Authority
CA
Canada
Prior art keywords
cpp
peptide
seq
molecule
interest
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
Application number
CA3161901A
Other languages
English (en)
French (fr)
Inventor
Shane Stone
Clinton Hall
Anja STIRNWEISS
Paula CUNNINGHAM
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Pyc Therapeutics Ltd
Original Assignee
Individual
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
Priority claimed from AU2019904882A external-priority patent/AU2019904882A0/en
Application filed by Individual filed Critical Individual
Publication of CA3161901A1 publication Critical patent/CA3161901A1/en
Pending legal-status Critical Current

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/001—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof by chemical synthesis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/30—Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
    • A61K47/42—Proteins; Polypeptides; Degradation products thereof; Derivatives thereof, e.g. albumin, gelatin or zein
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/62—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
    • A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • 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
    • A61K9/0048—Eye, e.g. artificial tears
    • A61K9/0051—Ocular inserts or implants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K19/00—Hybrid peptides, i.e. peptides covalently bound to nucleic acids, or non-covalently bound protein-protein complexes
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • 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
    • G01N33/53—Immunoassay; Biospecific binding assay; Materials therefor
    • 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
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • 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
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • C07K14/4723—Cationic antimicrobial peptides, e.g. defensins
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/10—Fusion polypeptide containing a localisation/targetting motif containing a tag for extracellular membrane crossing, e.g. TAT or VP22

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Ophthalmology & Optometry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Immunology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Neurology (AREA)
  • Plant Pathology (AREA)
  • Inorganic Chemistry (AREA)
  • Toxicology (AREA)
  • Urology & Nephrology (AREA)
  • Hematology (AREA)
  • Analytical Chemistry (AREA)
CA3161901A 2019-12-20 2020-12-18 Novel cellular delivery methods Pending CA3161901A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
AU2019904882A AU2019904882A0 (en) 2019-12-20 Novel Cellular Delivery Methods
AU2019904882 2019-12-20
PCT/AU2020/051397 WO2021119756A1 (en) 2019-12-20 2020-12-18 Novel cellular delivery methods

Publications (1)

Publication Number Publication Date
CA3161901A1 true CA3161901A1 (en) 2021-06-24

Family

ID=76476454

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3161901A Pending CA3161901A1 (en) 2019-12-20 2020-12-18 Novel cellular delivery methods

Country Status (10)

Country Link
US (1) US12698307B2 (enExample)
EP (1) EP4077354A4 (enExample)
JP (1) JP7684971B2 (enExample)
KR (1) KR20220117914A (enExample)
CN (1) CN115190884B (enExample)
AU (1) AU2020406670B2 (enExample)
BR (1) BR112022012057A2 (enExample)
CA (1) CA3161901A1 (enExample)
IL (1) IL294050A (enExample)
WO (1) WO2021119756A1 (enExample)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019165183A1 (en) 2018-02-22 2019-08-29 Entrada Therapeutics, Inc. Compositions and methods for treating mitochondrial neurogastrointestinal encephalopathy
CA3161901A1 (en) 2019-12-20 2021-06-24 Shane Stone Novel cellular delivery methods
US12338437B2 (en) 2020-05-11 2025-06-24 Stoke Therapeutics, Inc. OPA1 antisense oligomers for treatment of conditions and diseases
EP4469576A4 (en) * 2022-01-27 2026-04-15 Univ Alberta Antisense therapeutic products conjugate to pre-MIARN splicing modulating peptides for the treatment of diseases
WO2024073042A1 (en) * 2022-09-30 2024-04-04 Entrada Therapeutics, Inc. Ocular delivery of therapeutic agents
WO2024254639A1 (en) * 2023-06-16 2024-12-19 PYC Therapeutics Limited Compositions and methods for treatment of kidney disease
WO2025238644A1 (en) * 2024-05-16 2025-11-20 Skip Therapeutics Ltd. Compositions and methods for treating retinal diseases
WO2025240993A1 (en) * 2024-05-24 2025-11-27 PYC Therapeutics Limited Improved cell penetrating peptides and related compositions and methods

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6566330B1 (en) 1996-10-22 2003-05-20 Medical University Of South Carolina Foundation Research Development Positively charged non-natural amino acids, methods of making and using thereof in peptides
US6551795B1 (en) 1998-02-18 2003-04-22 Genome Therapeutics Corporation Nucleic acid and amino acid sequences relating to pseudomonas aeruginosa for diagnostics and therapeutics
JP2004520803A (ja) 2000-04-21 2004-07-15 コリクサ コーポレイション 尋常性挫瘡の治療および診断のための組成物および方法
US7214786B2 (en) * 2000-12-14 2007-05-08 Kovalic David K Nucleic acid molecules and other molecules associated with plants and uses thereof for plant improvement
US20060107345A1 (en) * 2003-09-30 2006-05-18 Nickolai Alexandrov Sequence-determined DNA fragments and corresponding polypeptides encoded thereby
US20160340683A1 (en) 2007-06-07 2016-11-24 Her Majesty The Queen In Right Of Canada As Represented By The Minister Of Agriculture And Agr Nanocarrier based plant transfection and transduction
NZ704191A (en) * 2010-06-14 2016-05-27 Hoffmann La Roche Cell-penetrating peptides and uses therof
KR102046830B1 (ko) * 2011-05-13 2019-11-20 코벤하븐스 유니버시테트 허혈성 뇌 손상에 대한 효과적인 신경보호제이며 통증의 치료를 위한 ｐｓｄ-95의 고-친화성, 이량체 저해제
AU2013316679B2 (en) 2012-09-13 2017-12-14 Les Hopitaux Universitaires De Geneve Cell penetrating peptides
US10316061B2 (en) * 2014-10-02 2019-06-11 Temple University Of The Commonwealth System Of Higher Education Synthesis of cell penetrating peptides for drug delivery and stem cell applications
EP3630131B1 (en) * 2017-06-02 2022-08-31 Arizona Board of Regents on behalf of Arizona State University A method to create personalized canine cancer vaccines
US20210087238A1 (en) * 2017-07-28 2021-03-25 Phylogica Limited Cell penetrating peptides and related compositions and methods
CA3161901A1 (en) 2019-12-20 2021-06-24 Shane Stone Novel cellular delivery methods
WO2025043278A1 (en) 2023-08-25 2025-03-06 PYC Therapeutics Limited Gene-mediated conditions

Also Published As

Publication number Publication date
CN115190884A (zh) 2022-10-14
JP2023511501A (ja) 2023-03-20
US20230048338A1 (en) 2023-02-16
JP7684971B2 (ja) 2025-05-28
EP4077354A1 (en) 2022-10-26
AU2020406670A1 (en) 2022-07-14
IL294050A (en) 2022-08-01
WO2021119756A1 (en) 2021-06-24
KR20220117914A (ko) 2022-08-24
BR112022012057A2 (pt) 2022-08-30
US12698307B2 (en) 2026-08-04
AU2020406670B2 (en) 2026-03-05
CN115190884B (zh) 2026-01-13
EP4077354A4 (en) 2024-01-24

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