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
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
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
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
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
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
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
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...
Information Patent Grant
Base editing of ANGPTL3 and methods of using same for treatment of...
- Patent number 12,115,230
- Issue date Oct 15, 2024
- Verve Therapeutics, Inc.
- Alexandra Chadwick
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...

Patents Applicationslast 30 patents

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
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
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
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...
Information Patent Application
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
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...
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
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...
Information Patent Application
MODIFIED NUCLEOSIDES, NUCLEOTIDES, AND NUCLEIC ACIDS, AND USES THEREOF
- Publication number 20250090692
- Publication date Mar 20, 2025
- ModernaTX, Inc.
- Jason P. SCHRUM
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
TREATMENT OF INTERVERTEBRAL DISC DEGENERATION
- Publication number 20250090635
- Publication date Mar 20, 2025
- Kolon TissueGene, Inc.
- Moon Jong Noh
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
ADENO-ASSOCIATED VIRUS COMPOSITIONS FOR RESTORING PAH GENE FUNCTION...
- Publication number 20250090691
- Publication date Mar 20, 2025
- HOMOLOGY MEDICINES, INC.
- Jason Boke WRIGHT
- 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
GENE EDITING OF CAR-T CELLS FOR THE TREATMENT OF T CELL MALIGNANCIE...
- Publication number 20250082785
- Publication date Mar 13, 2025
- Washington University
- John F. DiPersio
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
MODIFIED POLYNUCLEOTIDES FOR THE PRODUCTION OF ONCOLOGY-RELATED PRO...
- Publication number 20250074962
- Publication date Mar 6, 2025
- ModernaTX, Inc.
- Antonin DE FOUGEROLLES
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPOSITIONS AND METHODS FOR DELIVERY OF AGENTS TO INNER EAR
- Publication number 20250073354
- Publication date Mar 6, 2025
- University of Rochester
- Maiken Nedergaard
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
NUCLEIC ACID EXPRESSION PLATFORM WITH INCREASED EXPRESSION EFFICIENCY
- Publication number 20250064985
- Publication date Feb 27, 2025
- SML BIOPHARM CO., LTD.
- Jae Hwan Nam
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
OPTIMIZED POLYNUCLEOTIDES FOR PROTEIN EXPRESSION
- Publication number 20250064986
- Publication date Feb 27, 2025
- PRECISION BIOSCIENCES, INC.
- Jason Richard Harris
- 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
Compositions and Methods for Treatment of Ocular Disease Associated...
- Publication number 20250057987
- Publication date Feb 20, 2025
- 4D MOLECULAR THERAPEUTICS INC.
- Christian BURNS
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
Guide RNA That Targets A Mutant Human Guanylate Cyclase 2A Allele
- Publication number 20250057988
- Publication date Feb 20, 2025
- EmendoBio Inc.
- Lior Izhar
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
GENE THERAPY FOR LAMIN A-ASSOCIATED DEFICIENCIES
- Publication number 20250049949
- Publication date Feb 13, 2025
- The Trustees of the University of Pennsylvania
- Christian Hinderer
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
ANTI-TFR:ACID SPHINGOMYELINASE FOR TREATMENT OF ACID SPHINGOMYELINA...
- Publication number 20250049896
- Publication date Feb 13, 2025
- Regeneron Pharmaceuticals, Inc.
- Maria Praggastis
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
SCALABLE AND HIGH-PURITY CELL-FREE SYNTHESIS OF CLOSED-ENDED DNA VE...
- Publication number 20250049961
- Publication date Feb 13, 2025
- GENERATION BIO CO.
- Russell Monds
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
ENGINEERED MUSCLE TARGETING COMPOSITIONS
- Publication number 20250051800
- Publication date Feb 13, 2025
- THE BROAD INSTITUTE, INC.
- Pardis SABETI
- 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

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

Modified ligand-gated ion channels and methods of use

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

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

Immunostimulating vector system

Coronavirus vaccine

Development of optimized recombinant expression construct

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

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

Base editing of ANGPTL3 and methods of using same for treatment of...

Patents Applicationslast 30 patents

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

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

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

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

SYSTEMIC ADMINISTRATION OF CIRCULAR RNA POLYNUCLEOTIDES ENCODING MU...

COMPOSITION AND METHODS FOR MODULATION OF ELOVL2

METHODS AND COMPOSITIONS FOR THE TREATMENT OF RARE DISEASES

MODIFIED NUCLEOSIDES, NUCLEOTIDES, AND NUCLEIC ACIDS, AND USES THEREOF

TREATMENT OF INTERVERTEBRAL DISC DEGENERATION

ADENO-ASSOCIATED VIRUS COMPOSITIONS FOR RESTORING PAH GENE FUNCTION...

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

GENE EDITING OF CAR-T CELLS FOR THE TREATMENT OF T CELL MALIGNANCIE...

MODIFIED POLYNUCLEOTIDES FOR THE PRODUCTION OF ONCOLOGY-RELATED PRO...

COMPOSITIONS AND METHODS FOR DELIVERY OF AGENTS TO INNER EAR

NUCLEIC ACID EXPRESSION PLATFORM WITH INCREASED EXPRESSION EFFICIENCY

OPTIMIZED POLYNUCLEOTIDES FOR PROTEIN EXPRESSION

GENE THERAPY FOR CILIOPATHIES

Compositions and Methods for Treatment of Ocular Disease Associated...

Guide RNA That Targets A Mutant Human Guanylate Cyclase 2A Allele

GENE THERAPY FOR LAMIN A-ASSOCIATED DEFICIENCIES

ANTI-TFR:ACID SPHINGOMYELINASE FOR TREATMENT OF ACID SPHINGOMYELINA...

SCALABLE AND HIGH-PURITY CELL-FREE SYNTHESIS OF CLOSED-ENDED DNA VE...

ENGINEERED MUSCLE TARGETING COMPOSITIONS