characterised by an aspect of the delivery route

Industry

CPC
A61K48/0075

This industry / category may be too specific. Please go to a parent level for more data

Parent Industries

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

A61K48/0075characterised by an aspect of the delivery route

Industries

Please log in for detailed analytics

Patents Grantslast 30 patents

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
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
Intrathecal delivery of recombinant adeno-associated virus 9
- Patent number 12,208,144
- Issue date Jan 28, 2025
- Nationwide Children's Hospital
- Brian K. Kaspar
- 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
Isoform Nell-1 peptide
- Patent number 12,186,367
- Issue date Jan 7, 2025
- The Regents of the University of California
- Kang Ting
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Gene therapy for recessive dystrophic epidermolysis bullosa using g...
- Patent number 12,173,314
- Issue date Dec 24, 2024
- The Board of Trustees of the Leland Stanford Junior University
- Zurab Siprashvili
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Collagen-mimetic peptide mediated delivery of nucleic acid carriers...
- Patent number 12,171,809
- Issue date Dec 24, 2024
- University of Delaware
- Kristi Kiick
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Expression vector for cholesterol 24-hydrolase in therapy of polygl...
- Patent number 12,138,298
- Issue date Nov 12, 2024
- BRAINVECTIS
- Sandro Alves
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Methods for treating or alleviating a bone-loss related disease or...
- Patent number 12,133,896
- Issue date Nov 5, 2024
- Kaohsiung Medical University
- Chau-Zen Wang
- 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
Gene therapy delivery of Parkin mutants having increased activity t...
- Patent number 12,133,897
- Issue date Nov 5, 2024
- NysnoBio GT Neurology, LLC
- Jennifer Johnston
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Lipid nanoparticle compositions and methods for mRNA delivery
- Patent number 12,121,592
- Issue date Oct 22, 2024
- Translate Bio, Inc.
- Frank DeRosa
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Methods and materials for GALGT2 gene therapy
- Patent number 12,121,595
- Issue date Oct 22, 2024
- Research Institute at Nationwide Children's Hospital
- Paul Taylor Martin
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Methods and compositions for nucleic acid and protein payload delivery
- Patent number 12,123,012
- Issue date Oct 22, 2024
- LIGANDAL, INC.
- Andre Ronald Watson
- B82 - NANO-TECHNOLOGY
Information Patent Grant
Gene therapy for recessive dystrophic epidermolysis bullosa using g...
- Patent number 12,110,504
- Issue date Oct 8, 2024
- The Board of Trustees of the Leland Stanford Junior University
- Zurab Siprashvili
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Satiation peptide administration
- Patent number 12,109,251
- Issue date Oct 8, 2024
- University of Florida Research Foundation, Incorporated
- Sergei Zolotukhin
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Methods for expressing proteins in axons
- Patent number 12,103,952
- Issue date Oct 1, 2024
- Cornell University
- Samie R. Jaffrey
- 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
Methods and compositions for treatment of ocular disorders and blin...
- Patent number 12,097,267
- Issue date Sep 24, 2024
- The Trustees of the University of Pennsylvania
- Jeannette Bennicelli
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
System and method for controlling oxygen delivery to implanted cells
- Patent number 12,090,300
- Issue date Sep 17, 2024
- Giner, Inc.
- Simon G. Stone
- C25 - ELECTROLYTIC OR ELECTROPHORETIC PROCESSES APPARATUS THEREFOR
Information Patent Grant
Rabies vaccine
- Patent number 12,083,190
- Issue date Sep 10, 2024
- CureVac SE
- Margit Schnee
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
System for gas treatment of a cell implant
- Patent number 12,070,383
- Issue date Aug 27, 2024
- Giner, Inc.
- Linda Tempelman
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Compositions and methods for treating non-age-associated hearing im...
- Patent number 12,071,627
- Issue date Aug 27, 2024
- Akouos, Inc.
- Emmanuel John Simons
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Injection of single-stranded or self-complementary adeno-associated...
- Patent number 12,070,510
- Issue date Aug 27, 2024
- Emory University
- Anthony Donsante
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Systems and methods for left ventricular unloading in biologic ther...
- Patent number 12,064,120
- Issue date Aug 20, 2024
- Abiomed, Inc.
- Roger J Hajjar
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
System and method of performing treatment along a lumen network
- Patent number 12,059,214
- Issue date Aug 13, 2024
- Covidien LP
- William S. Krimsky
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Delivery of NRF2 as therapy for protection against reactive oxygen...
- Patent number 12,060,395
- Issue date Aug 13, 2024
- University of Florida Research Foundation, Incorporated
- Cristhian J. Ildefonso
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Gene therapy for neurodegenerative disorders
- Patent number 12,049,626
- Issue date Jul 30, 2024
- Prevail Therapeutics, Inc.
- Asa Abeliovich
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Methods and compositions for inhibiting oxidative stress
- Patent number 12,042,546
- Issue date Jul 23, 2024
- President and Fellows of Harvard College
- Constance L. Cepko
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
AAV vectors
- Patent number 12,043,848
- Issue date Jul 23, 2024
- Vigeneron GmbH
- Stylianos Michalakis
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE

Patents Applicationslast 30 patents

Information Patent Application
GENE THERAPY DELIVERY OF PARKIN MUTANTS HAVING INCREASED ACTIVITY T...
- Publication number 20250049958
- Publication date Feb 13, 2025
- NysnoBio GT Neurology, LLC
- Jennifer JOHNSTON
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
ADENO-ASSOCIATED VIRUS PARTICLES AND METHODS OF USE THEREOF
- Publication number 20250041450
- Publication date Feb 6, 2025
- Insmed Incorporated
- Brian KASPAR
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
USE OF ADENO-ASSOCIATED VIRAL VECTORS TO CORRECT GENE DEFECTS/ EXPR...
- Publication number 20250041457
- Publication date Feb 6, 2025
- Akouos, Inc.
- Emmanuel John Simons
- 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
SYNTHETIC RECEPTORS
- Publication number 20250032643
- Publication date Jan 30, 2025
- UCL Business LTD
- Dimitri KULLMANN
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
Compositions and Methods for Cell-Specific Expression of Target Genes
- Publication number 20250025575
- Publication date Jan 23, 2025
- The Trustees of Princeton University
- Carola Maturana
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHODS AND MATERIALS FOR GALGT2 GENE THERAPY
- Publication number 20250025578
- Publication date Jan 23, 2025
- Research Institute at Nationwide Children's Hospital
- Paul Taylor Martin
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
Compositions and Methods for Treatment of Diseases Associated with...
- Publication number 20250025580
- Publication date Jan 23, 2025
- INTELLIA THERAPEUTICS, INC.
- Sean Michael Burns
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
FUNCTIONAL AAV CAPSIDS FOR INTRAVITREAL ADMINISTRATION
- Publication number 20250026795
- Publication date Jan 23, 2025
- Shape Therapeutics Inc.
- Thomas PACKARD
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHOD FOR DECREASING DEGENERATION OF RETINAL GANGLION CELLS
- Publication number 20250018058
- Publication date Jan 16, 2025
- Icahn School of Medicine at Mount Sinai
- Bo CHEN
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
MODIFIED POLYNUCLEOTIDES FOR THE PRODUCTION OF BIOLOGICS AND PROTEI...
- Publication number 20250018062
- Publication date Jan 16, 2025
- Moderna TX, Inc.
- Antonin DE FOUGEROLLES
- Y02 - TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMA...
Information Patent Application
GENE THERAPY IN SUCCINIC SEMIALDEHYDE DEHYDROGENASE DEFICIENCY (SSA...
- Publication number 20250018061
- Publication date Jan 16, 2025
- The Children's Medical Center Corporation
- Alexander Rotenberg
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
POLYNUCLEOTIDE COMPOSITIONS, RELATED FORMULATIONS, AND METHODS OF U...
- Publication number 20250019409
- Publication date Jan 16, 2025
- ReCode Therapeutics, Inc.
- Mirko Hennig
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
Method of Treating Geographic Atrophy with a Gene Therapy Vector Ex...
- Publication number 20250018063
- Publication date Jan 16, 2025
- Adam Rogers
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
SYSTEMS AND METHODS FOR LEFT VENTRICULAR UNLOADING IN BIOLOGIC THER...
- Publication number 20250009357
- Publication date Jan 9, 2025
- ABIOMED, Inc.
- Roger J. Hajjar
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
GENE THERAPY FOR NEURODEGENERATIVE DISORDERS
- Publication number 20250011783
- Publication date Jan 9, 2025
- Prevail Therapeutics, Inc.
- Asa ABELIOVICH
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
NOCICEPTOR-SPECIFIC GENE REGULATORY ELEMENTS FOR THE TREATMENT OF PAIN
- Publication number 20250009906
- Publication date Jan 9, 2025
- President and Fellows of Harvard College
- William Renthal
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
GENE THERAPY FOR GALACTOSEMIA
- Publication number 20250001008
- Publication date Jan 2, 2025
- BridgeBio Gene Therapy Research, Inc.
- Clayton BEARD
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
AAV CAPSID VARIANTS AND USES THEREOF
- Publication number 20250001012
- Publication date Jan 2, 2025
- Mathieu Emmanuel Nonnenmacher
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
AAV2-MEDIATED GENE DELIVERY OF SFASL AS A NEUROPROTECTIVE THERAPY I...
- Publication number 20250001015
- Publication date Jan 2, 2025
- University of Massachusetts
- Ann Marshak-Rothstein
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
RETGC Gene Therapy
- Publication number 20250002936
- Publication date Jan 2, 2025
- MEIRAGTX UK II LIMITED
- Anastasios Georgiadis
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
Methods and Compositions for Treating Conditions Involving the Eye
- Publication number 20250001004
- Publication date Jan 2, 2025
- Krystal Biotech, Inc.
- Suma Krishnan
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
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
Biomarkers For Lysosomal Storage Diseases
- Publication number 20240424137
- Publication date Dec 26, 2024
- Amicus Therapeutics, Inc.
- Jon Brudvig
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
COMPOSITIONS USEFUL IN TREATMENT OF CDKL5 DEFICIENCY DISORDER (CDD)
- Publication number 20240425882
- Publication date Dec 26, 2024
- The Trustees of the University of Pennsylvania
- James M. Wilson
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
COMPOSITIONS, SYSTEMS, AND METHODS RELATED TO TRANSGENIC HOOKWORMS...
- Publication number 20240424030
- Publication date Dec 26, 2024
- Washington University
- Makedonka Mitreva
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
CRISPR/CAS-RELATED METHODS AND COMPOSITIONS FOR KNOCKING OUT C5
- Publication number 20240415980
- Publication date Dec 19, 2024
- Regeneron Pharmaceuticals, Inc.
- Kishor Devalaraja-Narashimha
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
MESSENGER RNA THERAPY FOR TREATMENT OF ARTICULAR DISEASE
- Publication number 20240415958
- Publication date Dec 19, 2024
- Translate Bio, Inc.
- Frank DeRosa
- 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...
Information Patent Application
AAV-MEDIATED THERAPIES FOR VISION LOSS ASSOCIATED WITH FRIEDREICH’S...
- Publication number 20240415983
- Publication date Dec 19, 2024
- University of Florida Research Foundation, Incorporated
- Sanford L. Boye
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...

characterised by an aspect of the delivery route

Industry

Parent Industries

Current Industry

Patents Grantslast 30 patents

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

Therapeutic applications of CPF1-based genome editing

Intrathecal delivery of recombinant adeno-associated virus 9

Materials and methods for treatment of hereditary haemochromatosis

Isoform Nell-1 peptide

Gene therapy for recessive dystrophic epidermolysis bullosa using g...

Collagen-mimetic peptide mediated delivery of nucleic acid carriers...

Expression vector for cholesterol 24-hydrolase in therapy of polygl...

Methods for treating or alleviating a bone-loss related disease or...

Immunostimulating vector system

Gene therapy delivery of Parkin mutants having increased activity t...

Lipid nanoparticle compositions and methods for mRNA delivery

Methods and materials for GALGT2 gene therapy

Methods and compositions for nucleic acid and protein payload delivery

Gene therapy for recessive dystrophic epidermolysis bullosa using g...

Satiation peptide administration

Methods for expressing proteins in axons

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

Methods and compositions for treatment of ocular disorders and blin...

System and method for controlling oxygen delivery to implanted cells

Rabies vaccine

System for gas treatment of a cell implant

Compositions and methods for treating non-age-associated hearing im...

Injection of single-stranded or self-complementary adeno-associated...

Systems and methods for left ventricular unloading in biologic ther...

System and method of performing treatment along a lumen network

Delivery of NRF2 as therapy for protection against reactive oxygen...

Gene therapy for neurodegenerative disorders

Methods and compositions for inhibiting oxidative stress

AAV vectors

Patents Applicationslast 30 patents

GENE THERAPY DELIVERY OF PARKIN MUTANTS HAVING INCREASED ACTIVITY T...

ADENO-ASSOCIATED VIRUS PARTICLES AND METHODS OF USE THEREOF

USE OF ADENO-ASSOCIATED VIRAL VECTORS TO CORRECT GENE DEFECTS/ EXPR...

GENE THERAPY FOR TREATMENT OF MUCOPOLYSACCHARIDOSIS IIIA

SYNTHETIC RECEPTORS

Compositions and Methods for Cell-Specific Expression of Target Genes

METHODS AND MATERIALS FOR GALGT2 GENE THERAPY

Compositions and Methods for Treatment of Diseases Associated with...

FUNCTIONAL AAV CAPSIDS FOR INTRAVITREAL ADMINISTRATION

METHOD FOR DECREASING DEGENERATION OF RETINAL GANGLION CELLS

MODIFIED POLYNUCLEOTIDES FOR THE PRODUCTION OF BIOLOGICS AND PROTEI...

GENE THERAPY IN SUCCINIC SEMIALDEHYDE DEHYDROGENASE DEFICIENCY (SSA...

POLYNUCLEOTIDE COMPOSITIONS, RELATED FORMULATIONS, AND METHODS OF U...

Method of Treating Geographic Atrophy with a Gene Therapy Vector Ex...

SYSTEMS AND METHODS FOR LEFT VENTRICULAR UNLOADING IN BIOLOGIC THER...

GENE THERAPY FOR NEURODEGENERATIVE DISORDERS

NOCICEPTOR-SPECIFIC GENE REGULATORY ELEMENTS FOR THE TREATMENT OF PAIN

GENE THERAPY FOR GALACTOSEMIA

AAV CAPSID VARIANTS AND USES THEREOF

AAV2-MEDIATED GENE DELIVERY OF SFASL AS A NEUROPROTECTIVE THERAPY I...

RETGC Gene Therapy

Methods and Compositions for Treating Conditions Involving the Eye

BAG3 COMPOSITIONS AND METHODS

Biomarkers For Lysosomal Storage Diseases

COMPOSITIONS USEFUL IN TREATMENT OF CDKL5 DEFICIENCY DISORDER (CDD)

COMPOSITIONS, SYSTEMS, AND METHODS RELATED TO TRANSGENIC HOOKWORMS...

CRISPR/CAS-RELATED METHODS AND COMPOSITIONS FOR KNOCKING OUT C5

MESSENGER RNA THERAPY FOR TREATMENT OF ARTICULAR DISEASE

ANELLOVECTORS FOR DELIVERY OF EFFECTORS TO THE CENTRAL NERVOUS SYSTEM

AAV-MEDIATED THERAPIES FOR VISION LOSS ASSOCIATED WITH FRIEDREICH’S...