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AHUMAN NECESSITIES A61Medical science A61KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases Gene therapy

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A61K48/0083characterised by an aspect of the administration regime

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Patents Grantslast 30 patents

Information Patent Grant
In vivo gene therapy delivery procedure and device
- Patent number 12,303,687
- Issue date May 20, 2025
- Wisconsin Alumni Research Foundation
- Susan Hagness
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Means and methods for AAV gene therapy in humans
- Patent number 12,285,451
- Issue date Apr 29, 2025
- Uniqure IP B.V.
- Bart Antonius Nijmeijer
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Recombinant adenoviruses carrying transgenes
- Patent number 12,280,123
- Issue date Apr 22, 2025
- EpicentRx, Inc.
- Christopher Larson
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Composition and methods for treatment of ornithine transcarbamylase...
- Patent number 12,268,754
- Issue date Apr 8, 2025
- Translate Bio, Inc.
- Kim Askew
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Allele-specific inactivation of mutant HTT via gene editing at codi...
- Patent number 12,268,755
- Issue date Apr 8, 2025
- UNIVERSITY OF MASSACHUSETTS
- Michael Harry Brodsky
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Sulfamidase (SGSH) variants, vectors, compositions and methods and...
- Patent number 12,221,635
- Issue date Feb 11, 2025
- The Childeren's Hospital of Philadelphia
- Beverly L. Davidson
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Compositions and methods for promoting homology directed repair
- Patent number 12,201,699
- Issue date Jan 21, 2025
- Editas Medicine, Inc.
- Cecilia Cotta-Ramusino
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Gene therapy for ciliopathies
- Patent number 12,171,794
- Issue date Dec 24, 2024
- UCL BUSINESS LTD
- Philip Beales
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Apparatus for efficient genetic modification of cells
- Patent number 12,139,697
- Issue date Nov 12, 2024
- The Charles Stark Draper Laboratory, Inc.
- Kenneth T. Kotz
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Immunostimulating vector system
- Patent number 12,133,898
- Issue date Nov 5, 2024
- Provecs Medical GmbH
- Frank Schnieders
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Methods for efficient delivery of therapeutic molecules in vitro an...
- Patent number 12,102,691
- Issue date Oct 1, 2024
- Massachusetts Eye and Ear Infirmary
- Zheng-Yi Chen
- C07 - ORGANIC CHEMISTRY
Information Patent Grant
Gene therapy of hemophilia a using viral vectors encoding recombina...
- Patent number 12,091,675
- Issue date Sep 17, 2024
- Takeda Pharmaceutical Company Limited
- Hanspeter Rottensteiner
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Transgene cassettes, AAV vectors and AAV viral vectors for the expr...
- Patent number 12,077,772
- Issue date Sep 3, 2024
- The Board of Regents of the University of Texas System
- Steven J. Gray
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Nucleic acid-polypeptide compositions and methods of inducing exon...
- Patent number 12,064,483
- Issue date Aug 20, 2024
- AVIDITY BIOSCIENCES, INC.
- Arthur A. Levin
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Treating metastatic cancer and model systems for metastatic disease
- Patent number 12,059,480
- Issue date Aug 13, 2024
- Memorial Sloan Kettering Cancer Center
- Karuna Ganesh
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Tunable endogenous protein degradation with heterobifunctional comp...
- Patent number 12,023,385
- Issue date Jul 2, 2024
- Dana-Farber Cancer Institute, Inc.
- Dennis Buckley
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Gene therapies for lysosomal disorders
- Patent number 11,999,974
- Issue date Jun 4, 2024
- Prevail Therapeutics, Inc.
- Asa Abeliovich
- G01 - MEASURING TESTING
Information Patent Grant
Use of bacterial voltage gated ion channels for human therapies
- Patent number 11,980,671
- Issue date May 14, 2024
- Duke University
- Nenad Bursac
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Immunisation of large mammals with low doses of RNA
- Patent number 11,913,001
- Issue date Feb 27, 2024
- GlaxoSmithKline Biologicals SA
- Andrew Geall
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Immunisation of large mammals with low doses of RNA
- Patent number 11,905,514
- Issue date Feb 20, 2024
- GlaxoSmithKline Biological S.A.
- Andrew Geall
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
AAV9-mediated gene therapy for treating mucopolysaccharidosis type I
- Patent number 11,890,329
- Issue date Feb 6, 2024
- The Trustees of the University of Pennsylvania
- Christian Hinderer
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Immunization of large mammals with low doses of RNA
- Patent number 11,891,608
- Issue date Feb 6, 2024
- GlaxoSmithKline Biologicals SA
- Andrew Geall
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Gene therapy for AADC deficiency
- Patent number 11,865,188
- Issue date Jan 9, 2024
- NATIONAL TAIWAN UNIVERSITY
- Mark Pykett
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Immunisation of large mammals with low doses of RNA
- Patent number 11,851,660
- Issue date Dec 26, 2023
- GlaxoSmithKline Biologicals SA
- Andrew Geall
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Immunisation of large mammals with low doses of RNA
- Patent number 11,845,925
- Issue date Dec 19, 2023
- GlaxoSmithKline Biologicals SA
- Andrew Geall
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
In vivo therapeutic delivery procedure and device
- Patent number 11,839,761
- Issue date Dec 12, 2023
- Wisconsin Alumni Research Foundation
- Susan Hagness
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Codon optimized GLA genes and uses thereof
- Patent number 11,802,278
- Issue date Oct 31, 2023
- David H. Kirn
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Method for the treatment of malignancies
- Patent number 11,801,312
- Issue date Oct 31, 2023
- University of South Florida
- Richard Heller
- C07 - ORGANIC CHEMISTRY
Information Patent Grant
Methods for selective kinase inhibition by endogenously produced an...
- Patent number 11,793,889
- Issue date Oct 24, 2023
- KINASE PHARMA INC.
- Bradley G Thompson
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Immunization of large mammals with low doses of RNA
- Patent number 11,773,395
- Issue date Oct 3, 2023
- GlaxoSmithKline Biologicals SA
- Andrew Geall
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...

Patents Applicationslast 30 patents

Information Patent Application
GENE THERAPY FOR THE TREATMENT OF HT1
- Publication number 20250177564
- Publication date Jun 5, 2025
- LogicBio Therapeutics, Inc.
- Qiang Xiong
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHODS FOR INCREASING PLATELET COUNT BY INHIBITING BILIVERDIN IXBE...
- Publication number 20250170277
- Publication date May 29, 2025
- The Research Foundation for the State University of New York
- Wadie F. BAHOU
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND METHODS OF INDUCING EXON...
- Publication number 20250161344
- Publication date May 22, 2025
- Avidity Biosciences, Inc.
- Arthur A. LEVIN
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
COMPOSITIONS AND METHODS FOR IN VIVO NUCLEASE-MEDIATED TREATMENT OF...
- Publication number 20250161493
- Publication date May 22, 2025
- The Trustees of the University of Pennsylvania
- Lili Wang
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
CHIMERIC NEUROTROPIC AAV CAPSIDS
- Publication number 20250161486
- Publication date May 22, 2025
- The University of North Carolina at Chapel Hill
- Bin Xiao
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
METHODS FOR DECREASING PATHOLOGIC ALPHA-SYNUCLEIN USING AGENTS THAT...
- Publication number 20250161413
- Publication date May 22, 2025
- Dana-Farber Cancer Institute, Inc.
- Bruce M. Spiegelman
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
COMPOSITION AND METHODS FOR TREATMENT OF ORNITHINE TRANSCARBAMYLASE...
- Publication number 20250152739
- Publication date May 15, 2025
- Translate Bio, Inc.
- Kim Askew
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
GENE THERAPY FOR THE TREATMENT OF WILSON'S DISEASE
- Publication number 20250144239
- Publication date May 8, 2025
- LogicBio Therapeutics, Inc.
- Shengwen Zhang
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHODS FOR USE OF VIRAL VECTOR CONSTRUCTS FOR THE TREATMENT OF FAB...
- Publication number 20250144240
- Publication date May 8, 2025
- Sangamo Therapeutics, Inc.
- Cristobal PASSALACQUA
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
ADENO-ASSOCIATED VIRAL VECTOR FOR GLUT1 EXPRESSION AND USES THEREOF
- Publication number 20250135040
- Publication date May 1, 2025
- Spacecraft Seven, LLC
- Christopher Dean HERZOG
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHODS OF TREATING OCULAR NEOVASCULAR DISEASES USING AAV2 VARIANTS...
- Publication number 20250137011
- Publication date May 1, 2025
- Adverum Biotechnologies, Inc.
- Kelly Hanna
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
NOVEL IMMUNOSTIMULATING VECTOR SYSTEM
- Publication number 20250127931
- Publication date Apr 24, 2025
- Provecs Medical GmbH
- Frank Schnieders
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
LENTIVIRAL GENE THERAPY RESCUES FUNCTION IN A MOUSE MODEL OF USHER 1B
- Publication number 20250121093
- Publication date Apr 17, 2025
- University of Kansas
- Hinrich STAECKER
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
NANOPARTICLES FUNCTIONALIZED WITH GENE EDITING TOOLS AND RELATED ME...
- Publication number 20250101425
- Publication date Mar 27, 2025
- STEMGENICS, INC.
- Andranik Andrew Aprikyan
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
POLYNUCLEOTIDES ENCODING GLUCOSE-6-PHOSPHATASE FOR THE TREATMENT OF...
- Publication number 20250090684
- Publication date Mar 20, 2025
- ModernaTX, Inc.
- Husain Attarwala
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
METHODS OF EX VIVO DOSING AND ADMINISTRATION OF LIPID PARTICLES OR...
- Publication number 20250082777
- Publication date Mar 13, 2025
- Sana Biotechnology, Inc.
- Aaron Edward FOSTER
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
FACTOR VIII (FVIII) GENE THERAPY METHODS
- Publication number 20250073353
- Publication date Mar 6, 2025
- Spark Therapeutics, Inc.
- Xavier ANGUELA
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
AAV VECTORS ENCODING BASE EDITORS AND USES THEREOF
- Publication number 20250064981
- Publication date Feb 27, 2025
- THE BROAD INSTITUTE, INC.
- David R. Liu
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
GENE THERAPY FOR CILIOPATHIES
- Publication number 20250057899
- Publication date Feb 20, 2025
- UCL Business LTD
- Philip Beales
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
Co-Delivery of a Gene Editor Construct and a Donor Template
- Publication number 20250057980
- Publication date Feb 20, 2025
- TOME BIOSCIENCES, INC.
- Jonathan Douglas Finn
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
IMPROVED GENE THERAPY CONSTRUCTS FOR THE TREATMENT OF PROPIONIC ACI...
- Publication number 20250049952
- Publication date Feb 13, 2025
- The United States of America,as represented by the Secretary,Department of He...
- Charles P. Venditti
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHODS AND COMPOSITIONS FOR TREATING BAG-3 RELATED CARDIOMYOPATHY...
- Publication number 20250041453
- Publication date Feb 6, 2025
- University of Florida Research Foundation, Incorporated
- Barry John Byrne
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
ANTI-TFR:GAA AND ANTI-CD63:GAA INSERTION FOR TREATMENT OF POMPE DIS...
- Publication number 20250041455
- Publication date Feb 6, 2025
- Regeneron Pharmaceuticals, Inc.
- Andrew Baik
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
GENE THERAPY FOR TREATMENT OF MUCOPOLYSACCHARIDOSIS IIIA
- Publication number 20250041445
- Publication date Feb 6, 2025
- The Trustees of the University of Pennsylvania
- Juliette Hordeaux
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHODS AND COMPOSITIONS FOR TREATING MYBPC3 RELATED HYPERTROPHIC C...
- Publication number 20250041452
- Publication date Feb 6, 2025
- University of Florida Research Foundation, Incorporated
- Barry John Byrne
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
GENE THERAPY OF HEMOPHILIA A USING VIRAL VECTORS ENCODING RECOMBINA...
- Publication number 20250043310
- Publication date Feb 6, 2025
- Takeda Pharmaceutical Company Limited
- Hanspeter Rottensteiner
- A01 - AGRICULTURE FORESTRY ANIMAL HUSBANDRY HUNTING TRAPPING FISHING
Information Patent Application
CRISPR-MEDIATED TRANSGENE INSERTION IN NEONATAL CELLS
- Publication number 20250032642
- Publication date Jan 30, 2025
- Regeneron Pharmaceuticals, Inc.
- Andrew Baik
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
BAG3 COMPOSITIONS AND METHODS
- Publication number 20250001014
- Publication date Jan 2, 2025
- Temple University - Of The Commonwealth System of Higher Education
- Arthur M. FELDMAN
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
IMMUNISATION OF LARGE MAMMALS WITH LOW DOSES OF RNA
- Publication number 20240417740
- Publication date Dec 19, 2024
- GlaxoSmithKline Biologicals S.A.
- Andrew GEALL
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
ANELLOVECTORS FOR DELIVERY OF EFFECTORS TO THE CENTRAL NERVOUS SYSTEM
- Publication number 20240415978
- Publication date Dec 19, 2024
- FLAGSHIP PIONEERING INNOVATIONS V, INC.
- Roger Joseph Hajjar
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...

characterised by an aspect of the administration regime

Industry

Parent Industries

Current Industry

Patents Grantslast 30 patents

In vivo gene therapy delivery procedure and device

Means and methods for AAV gene therapy in humans

Recombinant adenoviruses carrying transgenes

Composition and methods for treatment of ornithine transcarbamylase...

Allele-specific inactivation of mutant HTT via gene editing at codi...

Sulfamidase (SGSH) variants, vectors, compositions and methods and...

Compositions and methods for promoting homology directed repair

Gene therapy for ciliopathies

Apparatus for efficient genetic modification of cells

Immunostimulating vector system

Methods for efficient delivery of therapeutic molecules in vitro an...

Gene therapy of hemophilia a using viral vectors encoding recombina...

Transgene cassettes, AAV vectors and AAV viral vectors for the expr...

Nucleic acid-polypeptide compositions and methods of inducing exon...

Treating metastatic cancer and model systems for metastatic disease

Tunable endogenous protein degradation with heterobifunctional comp...

Gene therapies for lysosomal disorders

Use of bacterial voltage gated ion channels for human therapies

Immunisation of large mammals with low doses of RNA

Immunisation of large mammals with low doses of RNA

AAV9-mediated gene therapy for treating mucopolysaccharidosis type I

Immunization of large mammals with low doses of RNA

Gene therapy for AADC deficiency

Immunisation of large mammals with low doses of RNA

Immunisation of large mammals with low doses of RNA

In vivo therapeutic delivery procedure and device

Codon optimized GLA genes and uses thereof

Method for the treatment of malignancies

Methods for selective kinase inhibition by endogenously produced an...

Immunization of large mammals with low doses of RNA

Patents Applicationslast 30 patents

GENE THERAPY FOR THE TREATMENT OF HT1

METHODS FOR INCREASING PLATELET COUNT BY INHIBITING BILIVERDIN IXBE...

NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND METHODS OF INDUCING EXON...

COMPOSITIONS AND METHODS FOR IN VIVO NUCLEASE-MEDIATED TREATMENT OF...

CHIMERIC NEUROTROPIC AAV CAPSIDS

METHODS FOR DECREASING PATHOLOGIC ALPHA-SYNUCLEIN USING AGENTS THAT...

COMPOSITION AND METHODS FOR TREATMENT OF ORNITHINE TRANSCARBAMYLASE...

GENE THERAPY FOR THE TREATMENT OF WILSON'S DISEASE

METHODS FOR USE OF VIRAL VECTOR CONSTRUCTS FOR THE TREATMENT OF FAB...

ADENO-ASSOCIATED VIRAL VECTOR FOR GLUT1 EXPRESSION AND USES THEREOF

METHODS OF TREATING OCULAR NEOVASCULAR DISEASES USING AAV2 VARIANTS...

NOVEL IMMUNOSTIMULATING VECTOR SYSTEM

LENTIVIRAL GENE THERAPY RESCUES FUNCTION IN A MOUSE MODEL OF USHER 1B

NANOPARTICLES FUNCTIONALIZED WITH GENE EDITING TOOLS AND RELATED ME...

POLYNUCLEOTIDES ENCODING GLUCOSE-6-PHOSPHATASE FOR THE TREATMENT OF...

METHODS OF EX VIVO DOSING AND ADMINISTRATION OF LIPID PARTICLES OR...

FACTOR VIII (FVIII) GENE THERAPY METHODS

AAV VECTORS ENCODING BASE EDITORS AND USES THEREOF

GENE THERAPY FOR CILIOPATHIES

Co-Delivery of a Gene Editor Construct and a Donor Template

IMPROVED GENE THERAPY CONSTRUCTS FOR THE TREATMENT OF PROPIONIC ACI...

METHODS AND COMPOSITIONS FOR TREATING BAG-3 RELATED CARDIOMYOPATHY...

ANTI-TFR:GAA AND ANTI-CD63:GAA INSERTION FOR TREATMENT OF POMPE DIS...

GENE THERAPY FOR TREATMENT OF MUCOPOLYSACCHARIDOSIS IIIA

METHODS AND COMPOSITIONS FOR TREATING MYBPC3 RELATED HYPERTROPHIC C...

GENE THERAPY OF HEMOPHILIA A USING VIRAL VECTORS ENCODING RECOMBINA...

CRISPR-MEDIATED TRANSGENE INSERTION IN NEONATAL CELLS

BAG3 COMPOSITIONS AND METHODS

IMMUNISATION OF LARGE MAMMALS WITH LOW DOSES OF RNA

ANELLOVECTORS FOR DELIVERY OF EFFECTORS TO THE CENTRAL NERVOUS SYSTEM