Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases Gene therapy

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Patents Grantslast 30 patents

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
Smart multidosing
- Patent number 12,293,815
- Issue date May 6, 2025
- OptimDosing LLC
- David Inwald
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
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
Modified RNAi agents
- Patent number 12,290,573
- Issue date May 6, 2025
- Alnylam Pharmaceuticals, Inc.
- Kallanthottathil G. Rajeev
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Gene editing to improve joint function
- Patent number 12,290,572
- Issue date May 6, 2025
- ORTHOBIO THERAPEUTICS, INC.
- Peter J. Millett
- 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
Gene therapy for haploinsufficiency
- Patent number 12,285,496
- Issue date Apr 29, 2025
- The Regents of the University of California
- Nadav Ahituv
- A01 - AGRICULTURE FORESTRY ANIMAL HUSBANDRY HUNTING TRAPPING FISHING
Information Patent Grant
Self-complementary adeno-associated virus vector and its use in tre...
- Patent number 12,285,497
- Issue date Apr 29, 2025
- Research Institute at Nationwide Children's Hospital
- Louise Rodino-Klapac
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
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
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
Reprogrammed T cell-like NK cells
- Patent number 12,280,092
- Issue date Apr 22, 2025
- NantBio, Inc.
- Kayvan Niazi
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Vaccines having an antigen and interleukin-21 as an adjuvant
- Patent number 12,280,108
- Issue date Apr 22, 2025
- The Trustees of the University of Pennsylvania
- David B. Weiner
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Composition for promoting extracellular vesicle production containi...
- Patent number 12,280,148
- Issue date Apr 22, 2025
- Exocalibre Therapeutics Co., Ltd.
- Tae-Hyoung Kim
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
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
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
Gene therapy methods for age-related diseases and conditions
- Patent number 12,281,154
- Issue date Apr 22, 2025
- President and Fellows of Harvard College
- Noah Davidsohn
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Methods and compositions for increasing protein expression and/or t...
- Patent number 12,281,314
- Issue date Apr 22, 2025
- Case Western Reserve University
- Jeffery M. Coller
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Ultrasound-guided rete testis injection/aspiration device
- Patent number 12,280,075
- Issue date Apr 22, 2025
- University of Pittsburgh of the Commonwealth System of Higher Education
- Kyle E. Orwig
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Lipid nanoparticle formulations for delivery of MRNA
- Patent number 12,280,117
- Issue date Apr 22, 2025
- Translate Bio, Inc.
- Frank DeRosa
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Composition for increasing the expression of a growth factor gene,...
- Patent number 12,280,155
- Issue date Apr 22, 2025
- G&P BIOSCIENCE CO., LTD.
- Seong-Hyun Ho
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
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
Truncated dysferlin for treatment of dysferlinopathy
- Patent number 12,281,145
- Issue date Apr 22, 2025
- The University of North Carolina at Chapel Hill
- Matthew Louis Hirsch
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Nucleic acid vaccines
- Patent number 12,274,743
- Issue date Apr 15, 2025
- ModernaTX, Inc.
- Giuseppe Ciaramella
- 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
Compositions and methods for enhanced gene expression in cone cells
- Patent number 12,275,959
- Issue date Apr 15, 2025
- Adverum Biotechnologies, Inc.
- Thomas W. Chalberg
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Method for utilizing engineered dendritic cells to induce gut-homin...
- Patent number 12,275,961
- Issue date Apr 15, 2025
- Loma Linda University
- Xiaolei Tang
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Genetically engineered cells
- Patent number 12,275,966
- Issue date Apr 15, 2025
- The Regents of the University of California
- Jennifer A. Doudna
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
High-transducing HSV vectors
- Patent number 12,275,949
- Issue date Apr 15, 2025
- Periphagen, Inc.
- David M. Krisky
- 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...

Patents Applicationslast 30 patents

Information Patent Application
RNA for In vivo Transfection with Increased Expression
- Publication number 20250144234
- Publication date May 8, 2025
- Capstan Therapeutics Inc.
- Daiki Matsuda
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
Materials and Methods for Treatment of Human Genetic Diseases Inclu...
- Publication number 20250146004
- Publication date May 8, 2025
- VERTEX PHARMACEUTICALS INCORPORATED
- Matthew Hebden Porteus
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
MODULAR BETARETROVIRUS RECEPTOR ATTACHMENT AND CELL ENTRY PROTEINS
- Publication number 20250144238
- Publication date May 8, 2025
- The Trustees of Boston College
- Welkin E. Johnson
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
DISRUPTION OF THE SECRETION OF INSULIN-LIKE GROWTH FACTOR 2 FOR CAN...
- Publication number 20250144242
- Publication date May 8, 2025
- THE HONG KONG UNIVERSITY OF SCIENCE AND TECHNOLOGY
- Yusong GUO
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
AAV CAPSIDS FOR IMPROVED HEART TRANSDUCTION AND DETARGETING OF LIVER
- Publication number 20250144243
- Publication date May 8, 2025
- The Trustees of the University of Pennsylvania
- Jacob Hoffman
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
NOVEL GENE DELIVERY AGENTS
- Publication number 20250144233
- Publication date May 8, 2025
- TIBA BIOTECH LLC
- Poulami Talukder
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
Deamidation Depleted Adeno-Associated Virus Product
- Publication number 20250145969
- Publication date May 8, 2025
- Oxford Biomedica (US) LLC
- Alex Meola
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHODS AND COMPOSITIONS FOR DUAL GLYCAN BINDING AAV VECTORS
- Publication number 20250145670
- Publication date May 8, 2025
- The University of North Carolina at Chapel Hill
- Aravind ASOKAN
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPOSITIONS AND METHODS FOR MODIFYING A HUMAN DYSTROPHIN GENE
- Publication number 20250144246
- Publication date May 8, 2025
- MAMMOTH BIOSCIENCES, INC.
- Lucas Benjamin HARRINGTON
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
FORMULATIONS TO ENHANCE THE DELIVERY OF GENE THERAPY VECTORS
- Publication number 20250144247
- Publication date May 8, 2025
- University of Iowa Research Foundation
- Ashley Cooney
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHODS USING SURFACE-EXPRESSIBLE ACTIVATABLE EPITOPES TO LOCALIZE...
- Publication number 20250144249
- Publication date May 8, 2025
- EARLI Inc.
- Alex HARWIG
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
PROGRAMMABLE LIPID NANOPARTICLE DELIVERY VIA CORONA PROTEIN ENGINEE...
- Publication number 20250144232
- Publication date May 8, 2025
- Massachusetts Institute of Technology
- Omar Abudayyeh
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
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
METHODS OF TARGETING REPETITIVE RNA IN HUNTINGTON'S DISEASE
- Publication number 20250144241
- Publication date May 8, 2025
- The Regents of the University of California
- Eugene Yeo
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
NON-IMMUNOGENIC CIRCULAR, NON-VIRAL DNA VECTORS
- Publication number 20250146016
- Publication date May 8, 2025
- Rampart Bioscience, Inc.
- Jeffrey S. Bartlett
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHODS FOR IN VIVO DELIVERY AND DETECTION OF CLUSTERED REGULARLY I...
- Publication number 20250145975
- Publication date May 8, 2025
- University of Kentucky Research Foundation
- Xiaoyue Yang
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
MESSENGER RNA ENCODING CAS9 FOR USE IN GENOME-EDITING SYSTEMS
- Publication number 20250145976
- Publication date May 8, 2025
- CRISPR Therapeutics AG
- Christian Cobaugh
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
MULTI-FUNCTIONAL COMBINATION OUTLINE TOOL
- Publication number 20250145995
- Publication date May 8, 2025
- GUO HONG LIANG
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHODS OF REPEAT DOSING AND ADMINISTRATION OF LIPID PARTICLES OR V...
- Publication number 20250144235
- Publication date May 8, 2025
- Sana Biotechnology, Inc.
- Suvi JAIN
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
NITROGEN-BRANCHED NON-LINEAR PEGYLATED LIPID AND APPLICATION THEREOF
- Publication number 20250144236
- Publication date May 8, 2025
- XIAMEN SINOPEG BIOTECH CO., LTD.
- Wengui WENG
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
AGENTS, COMPOSITIONS AND METHODS FOR ENHANCED DELIVERY OF NUCLEIC A...
- Publication number 20250144237
- Publication date May 8, 2025
- Cila Therapeutics Inc.
- Safia K. Rizvi
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
TREATMENT OF ACQUIRED FOCAL EPILEPSY
- Publication number 20250144244
- Publication date May 8, 2025
- UCL Business LTD
- Matthew Charles WALKER
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
GENE THERAPY FOR ARRHYTHMOGENIC CARDIOMYOPATHY
- Publication number 20250144245
- Publication date May 8, 2025
- Ginkgo BioWorks, Inc.
- John Graef
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHODS AND SYSTEMS FOR IMPROVED NUCLEIC ACID DELIVERY VIA ULTRASOUND
- Publication number 20250144248
- Publication date May 8, 2025
- SonoThera, Inc.
- Steven B. FEINSTEIN
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
Methods and Compositions for RNA-Directed Target DNA Modification a...
- Publication number 20250146024
- Publication date May 8, 2025
- The Regents of the University of California
- Jennifer A. Doudna
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
RECOMBINANT OPTIMIZED MECP2 CASSETTES AND METHODS FOR TREATING RETT...
- Publication number 20250136651
- Publication date May 1, 2025
- THE UNIVERSITY COURT OF THE UNIVERSITY OF EDINBURGH
- Stuart Robert Cobb
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
ANTI-VEGF ANTIBODY CONSTRUCTS AND RELATED METHODS FOR TREATING VEST...
- Publication number 20250136673
- Publication date May 1, 2025
- Akouos, Inc.
- Emmanuel John Simons
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPOSITIONS AND METHODS FOR TREATING SIALIDOSIS
- Publication number 20250135033
- Publication date May 1, 2025
- University of Massachusetts
- Heather Gray-Edwards
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPOSITIONS FOR EDITING MECP2 TRANSCRIPTS AND METHODS THEREOF
- Publication number 20250135036
- Publication date May 1, 2025
- WAVE LIFE SCIENCES LTD.
- Christopher Michael Acker
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...

Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases Gene therapy

Industry

Parent Industries

Current Industry

Sub Industries

Patents Grantslast 30 patents

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

Smart multidosing

Heterologous targeting peptide grafted AAVs

Modified RNAi agents

Gene editing to improve joint function

Genome editing methods and constructs

Gene therapy for haploinsufficiency

Self-complementary adeno-associated virus vector and its use in tre...

Means and methods for AAV gene therapy in humans

Riboswitch modulated gene therapy for retinal diseases

Reprogrammed T cell-like NK cells

Vaccines having an antigen and interleukin-21 as an adjuvant

Composition for promoting extracellular vesicle production containi...

Frataxin expression constructs having engineered promoters and meth...

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

Gene therapy methods for age-related diseases and conditions

Methods and compositions for increasing protein expression and/or t...

Ultrasound-guided rete testis injection/aspiration device

Lipid nanoparticle formulations for delivery of MRNA

Composition for increasing the expression of a growth factor gene,...

Recombinant adenoviruses carrying transgenes

Truncated dysferlin for treatment of dysferlinopathy

Nucleic acid vaccines

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

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

Compositions and methods for enhanced gene expression in cone cells

Method for utilizing engineered dendritic cells to induce gut-homin...

Genetically engineered cells

High-transducing HSV vectors

AAV-mediated delivery of therapeutic antibodies to the inner ear

Patents Applicationslast 30 patents

RNA for In vivo Transfection with Increased Expression

Materials and Methods for Treatment of Human Genetic Diseases Inclu...

MODULAR BETARETROVIRUS RECEPTOR ATTACHMENT AND CELL ENTRY PROTEINS

DISRUPTION OF THE SECRETION OF INSULIN-LIKE GROWTH FACTOR 2 FOR CAN...

AAV CAPSIDS FOR IMPROVED HEART TRANSDUCTION AND DETARGETING OF LIVER

NOVEL GENE DELIVERY AGENTS

Deamidation Depleted Adeno-Associated Virus Product

METHODS AND COMPOSITIONS FOR DUAL GLYCAN BINDING AAV VECTORS

COMPOSITIONS AND METHODS FOR MODIFYING A HUMAN DYSTROPHIN GENE

FORMULATIONS TO ENHANCE THE DELIVERY OF GENE THERAPY VECTORS

METHODS USING SURFACE-EXPRESSIBLE ACTIVATABLE EPITOPES TO LOCALIZE...

PROGRAMMABLE LIPID NANOPARTICLE DELIVERY VIA CORONA PROTEIN ENGINEE...

GENE THERAPY FOR THE TREATMENT OF WILSON'S DISEASE

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

METHODS OF TARGETING REPETITIVE RNA IN HUNTINGTON'S DISEASE

NON-IMMUNOGENIC CIRCULAR, NON-VIRAL DNA VECTORS

METHODS FOR IN VIVO DELIVERY AND DETECTION OF CLUSTERED REGULARLY I...

MESSENGER RNA ENCODING CAS9 FOR USE IN GENOME-EDITING SYSTEMS

MULTI-FUNCTIONAL COMBINATION OUTLINE TOOL

METHODS OF REPEAT DOSING AND ADMINISTRATION OF LIPID PARTICLES OR V...

NITROGEN-BRANCHED NON-LINEAR PEGYLATED LIPID AND APPLICATION THEREOF

AGENTS, COMPOSITIONS AND METHODS FOR ENHANCED DELIVERY OF NUCLEIC A...

TREATMENT OF ACQUIRED FOCAL EPILEPSY

GENE THERAPY FOR ARRHYTHMOGENIC CARDIOMYOPATHY

METHODS AND SYSTEMS FOR IMPROVED NUCLEIC ACID DELIVERY VIA ULTRASOUND

Methods and Compositions for RNA-Directed Target DNA Modification a...

RECOMBINANT OPTIMIZED MECP2 CASSETTES AND METHODS FOR TREATING RETT...

ANTI-VEGF ANTIBODY CONSTRUCTS AND RELATED METHODS FOR TREATING VEST...

COMPOSITIONS AND METHODS FOR TREATING SIALIDOSIS

COMPOSITIONS FOR EDITING MECP2 TRANSCRIPTS AND METHODS THEREOF