Manipulation of the nucleic acid to modify its expression pattern

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A61K48/0066

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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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A61K48/0066Manipulation of the nucleic acid to modify its expression pattern

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Patents Grantslast 30 patents

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
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
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
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
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
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
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
Guide RNA that targets a mutant human inosine monophosphate deyhydr...
- Patent number 12,274,758
- Issue date Apr 15, 2025
- EMENDOBIO INC.
- Lior Izhar
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Compositions and methods for modulating Myc target protein 1
- Patent number 12,263,233
- Issue date Apr 1, 2025
- Washington University
- Kyunghee Choi
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Optimized CLN1 genes and expression cassettes and their use
- Patent number 12,257,322
- Issue date Mar 25, 2025
- The University of North Carolina at Chapel Hill
- Steven Gray
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Granulocyte colony-stimulating factor (GCSF) gene therapy for treat...
- Patent number 12,246,074
- Issue date Mar 11, 2025
- CHS Pharma Inc.
- Jang-Yen Wu
- C07 - ORGANIC CHEMISTRY
Information Patent Grant
Modified ligand-gated ion channels and methods of use
- Patent number 12,247,064
- Issue date Mar 11, 2025
- Howard Hughes Medical Institute
- Scott Sternson
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Adeno-associated virus factor VIII vectors
- Patent number 12,239,692
- Issue date Mar 4, 2025
- Biomarin Pharmaceutical Inc.
- Peter Cameron Colosi
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Hepatitis B vaccines and uses of the same
- Patent number 12,234,261
- Issue date Feb 25, 2025
- PRECIGEN, INC.
- Douglas E. Brough
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Therapeutic applications of CPF1-based genome editing
- Patent number 12,214,056
- Issue date Feb 4, 2025
- Duke University
- Charles A. Gersbach
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Pharmaceutical composition for prevention or treatment of heart fai...
- Patent number 12,215,362
- Issue date Feb 4, 2025
- BETHPHAGEN INC.
- Tae Hwan Kwak
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Engineering the RRM2 subunit of ribonucleotide reductase to resist...
- Patent number 12,208,133
- Issue date Jan 28, 2025
- University of Washington
- Michael Regnier
- 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
Materials and methods for treatment of hereditary haemochromatosis
- Patent number 12,186,406
- Issue date Jan 7, 2025
- CRISPR THERAPEUTICS AG
- Roman Lvovitch Bogorad
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Recombinogenic nucleic acid strands in situ
- Patent number 12,178,884
- Issue date Dec 31, 2024
- SNIPR TECHNOLOGIES LIMITED
- Jasper Clube
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Cells lacking B2M surface expression and methods for allogeneic adm...
- Patent number 12,180,263
- Issue date Dec 31, 2024
- President and Fellows of Harvard College
- Chad A. Cowan
- 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
Guide RNA that targets a mutant human guanylate cyclase 2A allele
- Patent number 12,168,062
- Issue date Dec 17, 2024
- EMENDOBIO INC.
- Lior Izhar
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Chimeric protein and related genic transfer technology
- Patent number 12,138,319
- Issue date Nov 12, 2024
- Lucio Pastore
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Development of optimized recombinant expression construct
- Patent number 12,134,776
- Issue date Nov 5, 2024
- G&P BIOSCIENCE CO., LTD.
- Seong Hyun Ho
- 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
Coronavirus vaccine
- Patent number 12,133,899
- Issue date Nov 5, 2024
- BIONTECH SE
- Ugur Sahin
- B82 - NANO-TECHNOLOGY
Information Patent Grant
Polynucleotides encoding JAGGED1 for the treatment of Alagille synd...
- Patent number 12,128,113
- Issue date Oct 29, 2024
- ModernaTX, Inc.
- Kerry Benenato
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Materials and methods for treatment of usher syndrome type 2A and/o...
- Patent number 12,122,998
- Issue date Oct 22, 2024
- CRISPR THERAPEUTICS AG
- Mariacarmela Allocca
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...

Patents Applicationslast 30 patents

Information Patent Application
TREATING METASTATIC CANCER AND MODEL SYSTEMS FOR METASTATIC DISEASE
- Publication number 20250213731
- Publication date Jul 3, 2025
- Memorial Sloan-Kettering Cancer Center
- Karuna Ganesh
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
Targeted CRISPR Delivery Platforms
- Publication number 20250197889
- Publication date Jun 19, 2025
- University of Massachusetts
- Erik J. Sontheimer
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
POLYNUCLEOTIDES CONTAINING A STABILIZING TAIL REGION
- Publication number 20250186624
- Publication date Jun 12, 2025
- ModernaTX, Inc.
- Gabor BUTORA
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
EXPRESSION SYSTEM FOR THE TREATMENT OF CANCER
- Publication number 20250186623
- Publication date Jun 12, 2025
- FUNDACION PARA LA INVESTIGACION MEDICA APLICADA
- Jesus María Prieto Valtueña
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
RECOMBINOGENIC NUCLEIC ACID STRANDS IN SITU
- Publication number 20250186625
- Publication date Jun 12, 2025
- SNIPR Technologies Limited
- Jasper CLUBE
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
MATERIALS AND METHODS FOR THE TREATMENT OF EIF2B5 MUTATIONS AND DIS...
- Publication number 20250186621
- Publication date Jun 12, 2025
- Research Institute at Nationwide Children's Hospital
- Kevin Flanigan
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPOSITIONS USEFUL IN TREATMENT OF METACHROMATIC LEUKODYSTROPHY
- Publication number 20250177495
- Publication date Jun 5, 2025
- The Trustees of the University of Pennsylvania
- Juliette Hordeaux
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
Materials & Methods for Treatment of Macular Degeneration
- Publication number 20250177573
- Publication date Jun 5, 2025
- The University of Bristol
- Ying Kai Chan
- 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
GENE DELIVERY VECTOR FOR DELIVERING HUMAN VEGF RECEPTOR FUSION PROT...
- Publication number 20250170276
- Publication date May 29, 2025
- Shanghai Correge Pharmaceutical Technology Co., Ltd.
- Lihui CHEN
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
CpG REDUCED FACTOR VIII VARIANTS, COMPOSITIONS AND METHODS AND USES...
- Publication number 20250171521
- Publication date May 29, 2025
- Spark Therapeutics, Inc.
- Xavier ANGUELA
- 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
TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS
- Publication number 20250163114
- Publication date May 22, 2025
- Brandeis University
- Greg Petsko
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
BASE EDITING OF ANGPTL3 AND METHODS OF USING SAME FOR TREATMENT OF...
- Publication number 20250152746
- Publication date May 15, 2025
- Verve Therapeutics, Inc.
- Alexandra Chadwick
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHODS OF TREATING GAUCHER DISEASE AND GBA-PARKINSON’S DISEASE
- Publication number 20250152747
- Publication date May 15, 2025
- Genzyme Corporation
- Swathi AYLOO
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
NUCLEIC ACID COMPRISING OR CODING FOR A HISTONE STEM-LOOP AND A POL...
- Publication number 20250152745
- Publication date May 15, 2025
- CureVac SE
- Andreas THESS
- 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
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
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
SPLICE-SWITCHING OLIGONUCLEOTIDES AND METHODS OF USE
- Publication number 20250136995
- Publication date May 1, 2025
- DUKE UNIVERSITY
- Jennifer Freedman
- 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
MATERIALS AND METHODS FOR TREATMENT OF USHER SYNDROME TYPE 2A AND/O...
- Publication number 20250122497
- Publication date Apr 17, 2025
- CRISPR Therapeutics AG
- Mariacarmela ALLOCCA
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
NUCLEIC ACID ENCODING RRM2 SUBUNIT OF RIBONUCLEOTIDE REDUCTASE TO R...
- Publication number 20250121038
- Publication date Apr 17, 2025
- University of Washington
- Michael REGNIER
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
THERAPEUTIC APPLICATIONS OF CPF1-BASED GENOME EDITING
- Publication number 20250114482
- Publication date Apr 10, 2025
- DUKE UNIVERSITY
- Charles A. Gersbach
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPOSITIONS FOR TREATING NEUROLOGICAL DISEASE
- Publication number 20250109398
- Publication date Apr 3, 2025
- ASKLEPIOS BIOPHARMACEUTICAL, INC.
- Anna TRETIAKOVA
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
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NON-TOXIC HSV VECTORS FOR EFFICIENT GENE DELIVERY APPLICATIONS AND...
- Publication number 20250109411
- Publication date Apr 3, 2025
- University of Pittsburgh of the Commonwealth System of Higher Education
- Joseph C. Glorioso
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
CIRCULAR RNA VACCINES AND METHODS OF USE THEREOF
- Publication number 20250108134
- Publication date Apr 3, 2025
- Beijing Changping Laboratory
- Wensheng WEI
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
COMPOSITION AND METHODS FOR MODULATION OF ELOVL2
- Publication number 20250099553
- Publication date Mar 27, 2025
- YouHealth Biotech, Limited
- Kang ZHANG
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
SYSTEMIC ADMINISTRATION OF CIRCULAR RNA POLYNUCLEOTIDES ENCODING MU...
- Publication number 20250099621
- Publication date Mar 27, 2025
- Orna Therapeutics, Inc.
- Allen T. Horhota
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
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METHODS AND COMPOSITIONS FOR THE TREATMENT OF RARE DISEASES
- Publication number 20250099624
- Publication date Mar 27, 2025
- Sangamo Therapeutics, Inc.
- Michael C. HOLMES
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...

Manipulation of the nucleic acid to modify its expression pattern

Industry

Parent Industries

Current Industry

Patents Grantslast 30 patents

Pharmaceutical compositions for the treatment of cancer

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

Gene therapy for haploinsufficiency

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

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

Lipid nanoparticle formulations for delivery of MRNA

Recombinant adenoviruses carrying transgenes

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

Guide RNA that targets a mutant human inosine monophosphate deyhydr...

Compositions and methods for modulating Myc target protein 1

Optimized CLN1 genes and expression cassettes and their use

Granulocyte colony-stimulating factor (GCSF) gene therapy for treat...

Modified ligand-gated ion channels and methods of use

Adeno-associated virus factor VIII vectors

Hepatitis B vaccines and uses of the same

Therapeutic applications of CPF1-based genome editing

Pharmaceutical composition for prevention or treatment of heart fai...

Engineering the RRM2 subunit of ribonucleotide reductase to resist...

Compositions and methods for promoting homology directed repair

Materials and methods for treatment of hereditary haemochromatosis

Recombinogenic nucleic acid strands in situ

Cells lacking B2M surface expression and methods for allogeneic adm...

Gene therapy for ciliopathies

Guide RNA that targets a mutant human guanylate cyclase 2A allele

Chimeric protein and related genic transfer technology

Development of optimized recombinant expression construct

Immunostimulating vector system

Coronavirus vaccine

Polynucleotides encoding JAGGED1 for the treatment of Alagille synd...

Materials and methods for treatment of usher syndrome type 2A and/o...

Patents Applicationslast 30 patents

TREATING METASTATIC CANCER AND MODEL SYSTEMS FOR METASTATIC DISEASE

Targeted CRISPR Delivery Platforms

POLYNUCLEOTIDES CONTAINING A STABILIZING TAIL REGION

EXPRESSION SYSTEM FOR THE TREATMENT OF CANCER

RECOMBINOGENIC NUCLEIC ACID STRANDS IN SITU

MATERIALS AND METHODS FOR THE TREATMENT OF EIF2B5 MUTATIONS AND DIS...

COMPOSITIONS USEFUL IN TREATMENT OF METACHROMATIC LEUKODYSTROPHY

Materials & Methods for Treatment of Macular Degeneration

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

GENE DELIVERY VECTOR FOR DELIVERING HUMAN VEGF RECEPTOR FUSION PROT...

CpG REDUCED FACTOR VIII VARIANTS, COMPOSITIONS AND METHODS AND USES...

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

TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS

BASE EDITING OF ANGPTL3 AND METHODS OF USING SAME FOR TREATMENT OF...

METHODS OF TREATING GAUCHER DISEASE AND GBA-PARKINSON’S DISEASE

NUCLEIC ACID COMPRISING OR CODING FOR A HISTONE STEM-LOOP AND A POL...

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

NON-IMMUNOGENIC CIRCULAR, NON-VIRAL DNA VECTORS

GENE THERAPY FOR ARRHYTHMOGENIC CARDIOMYOPATHY

SPLICE-SWITCHING OLIGONUCLEOTIDES AND METHODS OF USE

NOVEL IMMUNOSTIMULATING VECTOR SYSTEM

MATERIALS AND METHODS FOR TREATMENT OF USHER SYNDROME TYPE 2A AND/O...

NUCLEIC ACID ENCODING RRM2 SUBUNIT OF RIBONUCLEOTIDE REDUCTASE TO R...

THERAPEUTIC APPLICATIONS OF CPF1-BASED GENOME EDITING

COMPOSITIONS FOR TREATING NEUROLOGICAL DISEASE

NON-TOXIC HSV VECTORS FOR EFFICIENT GENE DELIVERY APPLICATIONS AND...

CIRCULAR RNA VACCINES AND METHODS OF USE THEREOF

COMPOSITION AND METHODS FOR MODULATION OF ELOVL2

SYSTEMIC ADMINISTRATION OF CIRCULAR RNA POLYNUCLEOTIDES ENCODING MU...

METHODS AND COMPOSITIONS FOR THE TREATMENT OF RARE DISEASES