characterised by an aspect of the 'active' part of the composition delivered

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A61K48/005

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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

Current Industry

A61K48/005characterised by an aspect of the 'active' part of the composition delivered

Industries

Patents Grantslast 30 patents

Information Patent Grant
Dry powder composition comprising long-chain RNA
- Patent number 12,318,486
- Issue date Jun 3, 2025
- CureVac SE
- Fabian Johannes Eber
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Feedback enabled synthetic genes, target seed match cassettes, and...
- Patent number 12,311,034
- Issue date May 27, 2025
- The University of North Carolina at Chapel Hill
- Steven James Gray
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Pharmaceutical compositions for the treatment of cancer
- Patent number 12,310,936
- Issue date May 27, 2025
- Seleq Oy
- Mikael Björnstedt
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Branched tail lipid compounds and compositions for intracellular de...
- Patent number 12,312,293
- Issue date May 27, 2025
- ModernaTX, Inc.
- Kerry E. Benenato
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
AAV capsid variants and uses thereof
- Patent number 12,296,025
- Issue date May 13, 2025
- VOYAGER THERAPEUTICS, INC.
- Mathieu Emmanuel Nonnenmacher
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Transcription regulatory elements and uses thereof
- Patent number 12,296,026
- Issue date May 13, 2025
- Astellas Gene Therapies, Inc.
- John T. Gray
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Optimized promoter sequences, intron-free expression constructs and...
- Patent number 12,290,574
- Issue date May 6, 2025
- SPARK THERAPEUTICS, INC.
- Xavier Anguela
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Heterologous targeting peptide grafted AAVs
- Patent number 12,291,719
- Issue date May 6, 2025
- UNIVERSITY OF MASSACHUSETTS
- Miguel Sena Esteves
- 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
Genome editing methods and constructs
- Patent number 12,285,495
- Issue date Apr 29, 2025
- FONDAZIONE TELETHON ETS
- Alberto Auricchio
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Riboswitch modulated gene therapy for retinal diseases
- Patent number 12,286,631
- Issue date Apr 29, 2025
- The Medical College of Wisconsin, Inc.
- Daniel M. Lipinski
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
5′-cap-trinucleotide- or higher oligonucleotide compounds and their...
- Patent number 12,281,138
- Issue date Apr 22, 2025
- BIONTECH RNA PHARMACEUTICALS GMBH
- Andreas Kuhn
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Frataxin expression constructs having engineered promoters and meth...
- Patent number 12,281,321
- Issue date Apr 22, 2025
- VOYAGER THERAPEUTICS, INC.
- Holger Patzke
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Recombinant adeno-associated viruses encoding serpin peptides and u...
- Patent number 12,274,757
- Issue date Apr 15, 2025
- University of Florida Research Foundation, Incorporated
- Alexandra Rose Lucas
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Methods of reducing FUS/TLS- or TDP-43-mediated neuronal cytotoxici...
- Patent number 12,275,764
- Issue date Apr 15, 2025
- Brandeis University
- Greg Petsko
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
AAV-mediated delivery of therapeutic antibodies to the inner ear
- Patent number 12,275,960
- Issue date Apr 15, 2025
- Akouos, Inc.
- Emmanuel John Simons
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Methods of treating and diagnosing epilepsy
- Patent number 12,274,756
- Issue date Apr 15, 2025
- Yale University
- Angelique Bordey
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Compositions and methods comprising engineered chimeric antigen rec...
- Patent number 12,269,860
- Issue date Apr 8, 2025
- AUTOLUS LIMITED
- Martin Pulé
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
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
Induction of osteogenesis by delivering BMP encoding RNA
- Patent number 12,263,229
- Issue date Apr 1, 2025
- ethris GmbH
- Elizabeth Balmayor
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Gene therapy for limb-girdle muscular dystrophy type 2C
- Patent number 12,263,230
- Issue date Apr 1, 2025
- Research Institute at Nationwide Children's Hospital
- Louise R. Rodino-Klapac
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Adeno-associated virus virions for treatment of epilepsy
- Patent number 12,263,231
- Issue date Apr 1, 2025
- Jichi Medical University
- Shin-ichi Muramatsu
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Composition for regulating production of proteins
- Patent number 12,263,232
- Issue date Apr 1, 2025
- Wyvern Pharmaceuticals Inc.
- Jill L. Thompson
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Systems, methods and compositions for sequence manipulation with op...
- Patent number 12,258,595
- Issue date Mar 25, 2025
- The Broad Institute, Inc.
- Silvana Konermann
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Adeno-associated virus vector delivery of a fragment of micro-dystr...
- Patent number 12,257,317
- Issue date Mar 25, 2025
- Newcastle University
- Hanns Lochmuller
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Optimized mini-dystrophin genes and expression cassettes and their use
- Patent number 12,257,321
- Issue date Mar 25, 2025
- The University of North Carolina at Chapel Hill
- Xiao Xiao
- A01 - AGRICULTURE FORESTRY ANIMAL HUSBANDRY HUNTING TRAPPING FISHING
Information Patent Grant
Optimised coding sequence and promoter
- Patent number 12,257,319
- Issue date Mar 25, 2025
- UCL BUSINESS LTD
- Amit Nathwani
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Methods and compositions for therapeutic protein delivery
- Patent number 12,258,597
- Issue date Mar 25, 2025
- REGENERON PHARMACEUTICALS, INC.
- Andrew Baik
- C07 - ORGANIC CHEMISTRY
Information Patent Grant
Polynucleotide compositions, related formulations, and methods of u...
- Patent number 12,257,318
- Issue date Mar 25, 2025
- ReCode Therapeutics, Inc.
- Mirko Hennig
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE

Patents Applicationslast 30 patents

Information Patent Application
USE OF CHEMICAL EPIGENETIC MODIFIERS TO MODULATE GENE EXPRESSION
- Publication number 20250177566
- Publication date Jun 5, 2025
- The University of North Carolina at Chapel Hill
- Nathaniel A. Hathaway
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPOSITIONS AND METHODS FOR MODULATING ALPHA-SYNUCLEIN EXPRESSION
- Publication number 20250177568
- Publication date Jun 5, 2025
- The Board of Trustees of the Leland Stanford Junior University
- C. Alejandra Morato Torres
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHOD OF INCREASING THE FUNCTION OF AN AAV VECTOR
- Publication number 20250179525
- Publication date Jun 5, 2025
- The Trustees of the University of Pennsylvania
- Luk VANDENBERGHE
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
MODIFIED CRISPR-BASED GENE EDITING SYSTEM AND METHODS OF USE
- Publication number 20250177567
- Publication date Jun 5, 2025
- GenEditBio Limited
- Zongli ZHENG
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPOSITION FOR USE IN THE TREATMENT OF FABRY DISEASE
- Publication number 20250177572
- Publication date Jun 5, 2025
- Takeda Pharmaceutical Company Limited
- Rizwana Islam
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
CRISPR/SpCas9 VARIANT AND METHODS FOR ENHANCED CORRECTION OF DUCHEN...
- Publication number 20250179450
- Publication date Jun 5, 2025
- The Board of Regents of the University of Texas System
- Yu ZHANG
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHODS OF CYTOTOXIC GENE THERAPY TO TREAT TUMORS
- Publication number 20250177494
- Publication date Jun 5, 2025
- CANDEL THERAPEUTICS, INC.
- Estuardo Aguilar-Cordova
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
THERAPEUTIC USE OF P75NTR NEUROTROPHIN BINDING PROTEIN
- Publication number 20250177485
- Publication date Jun 5, 2025
- Levicept LTD
- Simon Westbrook
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
SEQUENCE OF ACTIVATED PROTEIN C
- Publication number 20250177571
- Publication date Jun 5, 2025
- Jichi Medical University
- Tsukasa OHMORI
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
RECOMBINANT ADENO-ASSOCIATED VIRUSES FOR CNS TROPIC DELIVERY
- Publication number 20250179518
- Publication date Jun 5, 2025
- Regenxbio Inc.
- Olivier Danos
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
Adeno-Associated Viral (AAV)-Mediated Sphingosine-1-Phosphate Lyase...
- Publication number 20250177563
- Publication date Jun 5, 2025
- The Regents of the University of California
- Julie D. Saba
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
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
COMPOSITIONS FOR THE MODIFICATION OF THE HUMAN NRAS AND KRAS GENES
- Publication number 20250177569
- Publication date Jun 5, 2025
- MAMMOTH BIOSCIENCES, INC.
- Subhadra JAYARAMAN RUKMINI
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
MULTIPLEXED REPRESSION OF IMMUNOSUPPRESSIVE GENES
- Publication number 20250177570
- Publication date Jun 5, 2025
- Yale University
- Sidi Chen
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
NON-INTEGRATING VIRAL DELIVERY SYSTEM AND METHODS RELATED THERETO
- Publication number 20250179150
- Publication date Jun 5, 2025
- American Gene Technologies International Inc.
- Charles David Pauza
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHODS AND MATERIALS FOR INHIBITING/PREVENTING THE TRAFFICKING OF...
- Publication number 20250177565
- Publication date Jun 5, 2025
- The Trustees of Dartmouth College
- Matthew C. HAVRDA
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
POLYOMAVIRAL GENE DELIVERY VECTOR PARTICLE COMPRISING A NUCLEIC ACI...
- Publication number 20250170272
- Publication date May 29, 2025
- AMARNA HOLDING B.V.
- Ferdy Rudolph Van Diemen
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHODS AND COMPOSITIONS FOR CAR T CELL THERAPY
- Publication number 20250171803
- Publication date May 29, 2025
- Endocyte, Inc.
- Philip Stewart LOW
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
GENE THERAPEUTICS FOR TREATING BONE DISORDERS
- Publication number 20250170274
- Publication date May 29, 2025
- University of Massachusetts
- Jae-Hyuck Shim
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHOD OF INCREASING THE FUNCTION OF AN AAV VECTOR
- Publication number 20250171801
- Publication date May 29, 2025
- The Trustees of the University of Pennsylvania
- Luk VANDENBERGHE
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
THERAPEUTIC FACTORS FOR THE TREATMENT OF POLYQ DISEASES
- Publication number 20250170273
- Publication date May 29, 2025
- UNIVERSITA' DEGLI STUDI DI PADOVA (95%)
- Graziano Martello
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHODS AND MEANS FOR THE PREVENTION AND/OR TREATMENT OF HEMOPHILIC...
- Publication number 20250170275
- Publication date May 29, 2025
- uniQure biopharma B.V.
- Ellen Broug-Holub
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND USES THEREOF
- Publication number 20250171544
- Publication date May 29, 2025
- Avidity Biosciences, Inc.
- Andrew John GEALL
- 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
VIRAL GENE THERAPY AS TREATMENT FOR CHOLESTEROL STORAGE DISEASE OR...
- Publication number 20250161383
- Publication date May 22, 2025
- The United States of America, As Represented by the Secretary, Department of...
- Charles P. Venditti
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
SYNTHETIC CANCER-SPECIFIC PROMOTERS
- Publication number 20250161496
- Publication date May 22, 2025
- EARLI Inc.
- Dariusz WODZIAK
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
THERAPEUTIC ADENO-ASSOCIATED VIRUS FOR TREATING POMPE DISEASE WITH...
- Publication number 20250163457
- Publication date May 22, 2025
- ASKLEPIOS BIOPHARMACEUTICAL, INC.
- Sam HOPKINS
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
ARTIFICIAL EXPRESSION CONSTRUCTS FOR MODULATING GENE EXPRESSION IN...
- Publication number 20250163458
- Publication date May 22, 2025
- Allen Institute
- Tanya Daigle
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
COMPOSITIONS AND METHODS FOR TREATING DISEASE
- Publication number 20250161484
- Publication date May 22, 2025
- Yale University
- Elias Quijano
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
MEDICINE FOR DISEASE CAUSED BY FRAME-SHIFT MUTATION
- Publication number 20250161489
- Publication date May 22, 2025
- NATIONAL UNIVERSITY CORPORATION TOKAI NATIONAL HIGHER EDUCATION AND RESEARCH...
- Koji Nishiguchi
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE

characterised by an aspect of the 'active' part of the composition delivered

Industry

Parent Industries

Current Industry

Patents Grantslast 30 patents

Dry powder composition comprising long-chain RNA

Feedback enabled synthetic genes, target seed match cassettes, and...

Pharmaceutical compositions for the treatment of cancer

Branched tail lipid compounds and compositions for intracellular de...

AAV capsid variants and uses thereof

Transcription regulatory elements and uses thereof

Optimized promoter sequences, intron-free expression constructs and...

Heterologous targeting peptide grafted AAVs

Means and methods for AAV gene therapy in humans

Genome editing methods and constructs

Riboswitch modulated gene therapy for retinal diseases

5′-cap-trinucleotide- or higher oligonucleotide compounds and their...

Frataxin expression constructs having engineered promoters and meth...

Recombinant adeno-associated viruses encoding serpin peptides and u...

Methods of reducing FUS/TLS- or TDP-43-mediated neuronal cytotoxici...

AAV-mediated delivery of therapeutic antibodies to the inner ear

Methods of treating and diagnosing epilepsy

Compositions and methods comprising engineered chimeric antigen rec...

Composition and methods for treatment of ornithine transcarbamylase...

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

Induction of osteogenesis by delivering BMP encoding RNA

Gene therapy for limb-girdle muscular dystrophy type 2C

Adeno-associated virus virions for treatment of epilepsy

Composition for regulating production of proteins

Systems, methods and compositions for sequence manipulation with op...

Adeno-associated virus vector delivery of a fragment of micro-dystr...

Optimized mini-dystrophin genes and expression cassettes and their use

Optimised coding sequence and promoter

Methods and compositions for therapeutic protein delivery

Polynucleotide compositions, related formulations, and methods of u...

Patents Applicationslast 30 patents

USE OF CHEMICAL EPIGENETIC MODIFIERS TO MODULATE GENE EXPRESSION

COMPOSITIONS AND METHODS FOR MODULATING ALPHA-SYNUCLEIN EXPRESSION

METHOD OF INCREASING THE FUNCTION OF AN AAV VECTOR

MODIFIED CRISPR-BASED GENE EDITING SYSTEM AND METHODS OF USE

COMPOSITION FOR USE IN THE TREATMENT OF FABRY DISEASE

CRISPR/SpCas9 VARIANT AND METHODS FOR ENHANCED CORRECTION OF DUCHEN...

METHODS OF CYTOTOXIC GENE THERAPY TO TREAT TUMORS

THERAPEUTIC USE OF P75NTR NEUROTROPHIN BINDING PROTEIN

SEQUENCE OF ACTIVATED PROTEIN C

RECOMBINANT ADENO-ASSOCIATED VIRUSES FOR CNS TROPIC DELIVERY

Adeno-Associated Viral (AAV)-Mediated Sphingosine-1-Phosphate Lyase...

GENE THERAPY FOR THE TREATMENT OF HT1

COMPOSITIONS FOR THE MODIFICATION OF THE HUMAN NRAS AND KRAS GENES

MULTIPLEXED REPRESSION OF IMMUNOSUPPRESSIVE GENES

NON-INTEGRATING VIRAL DELIVERY SYSTEM AND METHODS RELATED THERETO

METHODS AND MATERIALS FOR INHIBITING/PREVENTING THE TRAFFICKING OF...

POLYOMAVIRAL GENE DELIVERY VECTOR PARTICLE COMPRISING A NUCLEIC ACI...

METHODS AND COMPOSITIONS FOR CAR T CELL THERAPY

GENE THERAPEUTICS FOR TREATING BONE DISORDERS

METHOD OF INCREASING THE FUNCTION OF AN AAV VECTOR

THERAPEUTIC FACTORS FOR THE TREATMENT OF POLYQ DISEASES

METHODS AND MEANS FOR THE PREVENTION AND/OR TREATMENT OF HEMOPHILIC...

NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND USES THEREOF

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

VIRAL GENE THERAPY AS TREATMENT FOR CHOLESTEROL STORAGE DISEASE OR...

SYNTHETIC CANCER-SPECIFIC PROMOTERS

THERAPEUTIC ADENO-ASSOCIATED VIRUS FOR TREATING POMPE DISEASE WITH...

ARTIFICIAL EXPRESSION CONSTRUCTS FOR MODULATING GENE EXPRESSION IN...

COMPOSITIONS AND METHODS FOR TREATING DISEASE

MEDICINE FOR DISEASE CAUSED BY FRAME-SHIFT MUTATION