WO2024050039A3 - Methods and compositions for hydrodynamic gene delivery - Google Patents

Methods and compositions for hydrodynamic gene delivery Download PDF

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Publication number
WO2024050039A3
WO2024050039A3 PCT/US2023/031751 US2023031751W WO2024050039A3 WO 2024050039 A3 WO2024050039 A3 WO 2024050039A3 US 2023031751 W US2023031751 W US 2023031751W WO 2024050039 A3 WO2024050039 A3 WO 2024050039A3
Authority
WO
WIPO (PCT)
Prior art keywords
methods
hydrodynamic
delivery
gene delivery
hydrodynamic injection
Prior art date
Application number
PCT/US2023/031751
Other languages
French (fr)
Other versions
WO2024050039A2 (en
Inventor
Robert Kruse
Original Assignee
Hydrogene Therapeutics, Inc.
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
Application filed by Hydrogene Therapeutics, Inc. filed Critical Hydrogene Therapeutics, Inc.
Publication of WO2024050039A2 publication Critical patent/WO2024050039A2/en
Publication of WO2024050039A3 publication Critical patent/WO2024050039A3/en

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Classifications

    • 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
    • A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

The present disclosure relates to methods of pressure-guided hydrodynamic injection, into tissues such as kidney, pancreas, liver, common bile duct, etc., in certain embodiments for delivery of nucleic acids or viral vectors. This includes optimal pressures that mediate gene delivery and expression into the subject during such hydrodynamic injection. Methods of disentangling individual differences to achieve target pressure during the hydrodynamic procedure are described, while also avoiding tissue injury, ruptures, tears, decreasing the toxicity of hydrodynamic injection, and other considerations relevant to therapeutic delivery.
PCT/US2023/031751 2022-08-31 2023-08-31 Methods and compositions for hydrodynamic gene delivery WO2024050039A2 (en)

Applications Claiming Priority (14)

Application Number Priority Date Filing Date Title
US202263374070P 2022-08-31 2022-08-31
US202263374228P 2022-08-31 2022-08-31
US202263374058P 2022-08-31 2022-08-31
US202263374234P 2022-08-31 2022-08-31
US202263374073P 2022-08-31 2022-08-31
US202263374231P 2022-08-31 2022-08-31
US202263374216P 2022-08-31 2022-08-31
US63/374,234 2022-08-31
US63/374,231 2022-08-31
US63/374,216 2022-08-31
US63/374,058 2022-08-31
US63/374,228 2022-08-31
US63/374,073 2022-08-31
US63/374,070 2022-08-31

Publications (2)

Publication Number Publication Date
WO2024050039A2 WO2024050039A2 (en) 2024-03-07
WO2024050039A3 true WO2024050039A3 (en) 2024-04-11

Family

ID=90098626

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2023/031751 WO2024050039A2 (en) 2022-08-31 2023-08-31 Methods and compositions for hydrodynamic gene delivery

Country Status (1)

Country Link
WO (1) WO2024050039A2 (en)

Citations (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20080097384A1 (en) * 2004-03-25 2008-04-24 Andrew Pacey Gene Therapy
US20080306403A1 (en) * 2005-02-28 2008-12-11 Jan Malm Method and Device for Determining the Hydrodynamics of the Cerebrospinal Fluid System
US20150072328A1 (en) * 2012-11-06 2015-03-12 Acist Medical Systems, Inc. Simulated contrast injection medium
US20160213896A1 (en) * 2013-08-30 2016-07-28 Indiana University Research & Technology Corporation Hydrodynamic method and apparatus for delivering fluids to kidney tissues
US20170015994A1 (en) * 2014-02-24 2017-01-19 Massachusetts Institute Of Technology Methods for in vivo genome editing
US20170216408A1 (en) * 2015-10-30 2017-08-03 Spark Therapeutics, Inc. CpG REDUCED FACTOR VIII VARIANTS, COMPOSITIONS AND METHODS AND USES FOR TREATMENT OF HEMOSTASIS DISORDERS
US20200345867A1 (en) * 2019-04-30 2020-11-05 The Johns Hopkins University Organ directed gene delivery
WO2022204535A1 (en) * 2021-03-25 2022-09-29 The Johns Hopkins University Hydrodynamic gene delivery

Patent Citations (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20080097384A1 (en) * 2004-03-25 2008-04-24 Andrew Pacey Gene Therapy
US20080306403A1 (en) * 2005-02-28 2008-12-11 Jan Malm Method and Device for Determining the Hydrodynamics of the Cerebrospinal Fluid System
US20150072328A1 (en) * 2012-11-06 2015-03-12 Acist Medical Systems, Inc. Simulated contrast injection medium
US20160213896A1 (en) * 2013-08-30 2016-07-28 Indiana University Research & Technology Corporation Hydrodynamic method and apparatus for delivering fluids to kidney tissues
US20170015994A1 (en) * 2014-02-24 2017-01-19 Massachusetts Institute Of Technology Methods for in vivo genome editing
US20170216408A1 (en) * 2015-10-30 2017-08-03 Spark Therapeutics, Inc. CpG REDUCED FACTOR VIII VARIANTS, COMPOSITIONS AND METHODS AND USES FOR TREATMENT OF HEMOSTASIS DISORDERS
US20200345867A1 (en) * 2019-04-30 2020-11-05 The Johns Hopkins University Organ directed gene delivery
WO2022204535A1 (en) * 2021-03-25 2022-09-29 The Johns Hopkins University Hydrodynamic gene delivery

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
YUTING HUANG, ROBERT L. KRUSE, HUI DING, MOHAMAD I. ITANI, JONATHAN MORRISON, ZACK Z. WANG, FLORIN M. SELARU, VIVEK KUMBHARI: "Parameters of biliary hydrodynamic injection during endoscopic retrograde cholangio-pancreatography in pigs for applications in gene delivery", PLOS ONE, PUBLIC LIBRARY OF SCIENCE, US, vol. 16, no. 4, 28 April 2021 (2021-04-28), US , pages e0249931, XP093159546, ISSN: 1932-6203, DOI: 10.1371/journal.pone.0249931 *

Also Published As

Publication number Publication date
WO2024050039A2 (en) 2024-03-07

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