GENETHON

Acid-alpha glucosidase variants and uses thereof

N° IL319481A

ACID-α GLUCOSIDASE VARIANTS AND USES THEREOF

N° JP2025060967A

HYBRID REGULATORY ELEMENTS

N° US2025127930A1

METHODS FOR AAV VECTOR RE-ADMINISTRATION

N° WO2025133247A1

PREDICTION OF MULTIPLE AAV PROPERTIES BY DEEP LEARNING

N° WO2025104321A1

FLUIDIC SYSTEM FOR PRODUCING EXTRACELLULAR VESICLES AND RELATED METHOD

N° US2024409869A1

HYBRID PROMOTERS FOR MUSCLE EXPRESSION

N° JP2024159800A

ACID-ALPHA GLUCOSIDASE VARIANTS AND USES THEREOF

N° AU2024205629A1

NOVEL ADENO-ASSOCIATED VIRUS (AAV) VARIANTS AND USES THEREOF FOR GENE THERAPY

N° JP2024149508A

NOVEL MIDIDYSTROPHINS

N° WO2025017169A1

NOVEL OPTIMIZED UTROPHIN MICRO-GENES

N° WO2025017168A1

N-TERMINAL TRUNCATED GLYCOGEN DEBRANCHING ENZYMES FOR THE TREATMENT OF GLYCOGEN STORAGE DISEASE III

N° WO2025003477A1

Hybrid Recombinant Adeno-Associated Virus Serotype Between AAV9 and AAVrh74 with Reduced Liver Tropism

N° US2024317814A1

LENTIVIRAL VECTOR FOR DOPAMINE SYNTHESIS

N° WO2024251679A1

MACHINE LEARNING METHOD FOR PHENOTYPE PREDICTION

N° WO2024218163A1

HYBRID REGULATORY ELEMENTS

N° JP2024079819A

COMPOSITIONS AND METHODS FOR INCREASING OR ENHANCING TRANSDUCTION OF GENE THERAPY VECTORS FOR REMOVING OR REDUCING IMMUNOGLOBULINS

N° JP2024075765A

INDUCTION OF IMMUNE TOLERANCE BY AAV VECTOR COMPRISING THE COMBINATION OF A LIVER DETARGETED CAPSID AND A TANDEM LIVER-MUSCLE SPECIFIC PROMOTER

N° WO2024188913A1

MINI-GDE FOR TREATMENT OF GLYCOGEN STORAGE DISEASE III

N° JP2024054398A

LSD1 INHIBITOR AND PRMT6 INHIBITOR FOR USE IN THE TREATMENT OF A DISEASE ASSOCIATED WITH GAIN-OF-FUNCTION OF ANDROGEN RECEPTOR (AR) AND/OR WITH OVEREXPRESSION OF AN AR COACTIVATOR

N° AU2023409849A1

TISSUE IMAGING METHODS AND SYSTEMS

N° WO2024133537A1

LSD1 INHIBITOR AND PRMT6 INHIBITOR FOR USE IN THE TREATMENT OF A DISEASE ASSOCIATED WITH GAIN-OF-FUNCTION OF ANDROGEN RECEPTOR (AR) AND/OR WITH OVEREXPRESSION OF AN AR COACTIVATOR

N° WO2024134525A1

Acid-alpha glucosidase variants and uses thereof

N° AU2023258449A1

HYBRID AAV VECTOR ENHANCING TRANSGENE EXPRESSION IN THE LIVER

N° EP4602055A1

HYBRID AAV VECTOR ENHANCING TRANSGENE EXPRESSION IN THE LIVER

N° WO2024079249A1

IMMUNOTHERAPY OF SKELETAL MYOPATHIES USING ANTI-FAP CAR-T CELLS

N° WO2024074727A1

IMMUNOTHERAPY OF SKELETAL MYOPATHIES USING ANTI-FAP CAR-T CELLS

N° EP4598566A1

Immunotherapy of skeletal muscle disease using anti-FAP CAR-T cells

N° CN120112308A

GALECTIN-3, A BIOMARKER OF LYSOSOMAL DAMAGE IN MUSCULAR DYSTROPHY

N° EP4516927A1

TREHALOSE-BASED COMBINED THERAPY FOR LYSOSOMAL DAMAGE

N° EP4516286A1

TREATMENT OF HYPERBILIRUBINEMIA

N° US2023414724A1

N-TERMINAL TRUNCATED GDE FOR THE TREATMENT OF GLYCOGEN STORAGE DISEASE III

N° WO2023237731A1

N-TERMINAL TRUNCATED GDE FOR THE TREATMENT OF GLYCOGEN STORAGE DISEASE III

N° EP4536816A1

PEPTIDE-MODIFIED AAV CAPSID WITH ENHANCED MUSCLE TRANSDUCTION EFFICIENCY

N° EP4536686A1

PEPTIDE-MODIFIED AAV CAPSID WITH ENHANCED MUSCLE TRANSDUCTION EFFICIENCY

N° WO2023237748A1

Peptide-modified AAV capsid with enhanced muscle transduction efficiency

N° CN119403823A

N-terminally truncated GDE for treatment of glycogen storage disease III

N° CN119343446A

METHODS FOR DIAGNOSING OR MONITORING MUSCULAR GLYCOGENOSIS

N° EP4474821A1

Acid-alpha glucosidase variants and uses thereof

N° IL302354A

USE OF BAZEDOXIFENE FOR INCREASING MUSCLE SURVIVAL

N° EP4493187A1

USE OF BAZEDOXIFENE FOR INCREASING MUSCLE SURVIVAL

N° WO2023175010A1

NOVEL POLYNUCLEOTIDES ENCODING A HUMAN FKRP PROTEIN

N° US2023321277A1

NUOVI INIBITORI DI REGOLATORI EPIGENETICI

N° IT202200026595A1

Acid-alpha glucosidase variants and uses thereof

N° IL299325A

FUKUTIN RELATED PROTEIN GENE TRANSFER INCREASE USING MODIFIED ITR SEQUENCES

N° WO2023111106A1

GAMMA-SARCOGLYCAN GENE TRANSFER INCREASE USING MODIFIED ITR SEQUENCES

N° WO2023111104A1

CALPAIN-3 GENE TRANSFER INCREASE USING MODIFIED ITR SEQUENCES

N° WO2023111107A1

ALPHA-SARCOGLYCAN GENE TRANSFER INCREASE USING MODIFIED ITR SEQUENCES

N° WO2023111102A1

ACID-ALPHA GLUCOSIDASE VARIANTS AND USES THEREOF

N° US2024043867A1

TREATMENT OF GLYCOGEN STORAGE DISEASE III

N° US2023190965A1

STABLE PSEUDOTYPED LENTIVIRAL PARTICLES AND USES THEREOF

N° US2023203537A1

HYBRID PROMOTERS FOR GENE EXPRESSION IN MUSCLES AND IN THE CNS

N° WO2023012313A1

HYBRID PROMOTERS FOR GENE EXPRESSION IN MUSCLES AND IN THE CNS

N° EP4381077A1

Hybrid promoters for gene expression in muscle and CNS

N° CN117897492A

HYBRID PROMOTERS FOR GENE EXPRESSION IN MUSCLES AND IN THE CNS

N° CA3226119A1

HYBRID PROMOTERS FOR GENE EXPRESSION IN MUSCLES AND IN THE CNS

N° US2024350670A1

ACID-ALPHA GLUCOSIDASE VARIANTS AND USES THEREOF

N° US2022389399A1

COMPOSITIONS AND METHODS FOR INCREASING OR ENHANCING TRANSDUCTION OF GENE THERAPY VECTORS AND FOR REMOVING OR REDUCING IMMUNOGLOBULINS

N° US2023142731A1

METHOD FOR REDUCING THE EXPRESSION OF NKCC1 IN A SUBJECT

N° US2022380807A1

Combination of a proteasome inhibitor and a hdac inhibitor and its use for the treatment of genetic diseases linked to a protein conformational disorder

N° IL294212A

COMPOSITIONS AND METHODS FOR IMPROVING VIRAL VECTOR EFFICIENCY

N° US2023117384A1

Acid-alpha glucosidase variants and uses thereof

N° US2022275396A1

Production of recombinant viral vectors from plant hairy roots

N° IL292491A

USE OF BAZEDOXIFENE FOR INCREASING MUSCLE SURVIVAL

N° US2025205247A1

FUKUTIN RELATED PROTEIN GENE TRANSFER INCREASE USING MODIFIED ITR SEQUENCES

N° EP4198047A1

GAMMA-SARCOGLYCAN GENE TRANSFER INCREASE USING MODIFIED ITR SEQUENCES

N° EP4198134A1

CALPAIN-3 GENE TRANSFER INCREASE USING MODIFIED ITR SEQUENCES

N° EP4198048A1

ALPHA-SARCOGLYCAN GENE TRANSFER INCREASE USING MODIFIED ITR SEQUENCES

N° EP4198046A1

LYSOSOMAL ACID LIPASE VARIANTS AND USES THEREOF

N° CA3199661A1

LYSOSOMAL ACID LIPASE VARIANTS AND USES THEREOF

N° WO2022122883A1

Lysosomal acidic lipase variants and uses thereof

N° CN116917471A

LYSOSOMAL ACID LIPASE VARIANTS AND USES THEREOF

N° EP4259790A1

LYSOSOMAL ACID LIPASE VARIANTS AND USES THEREOF

N° US2024067942A1

NEW GENE THERAPY FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY

N° WO2022122733A1

Gene therapy of fibroblast growth factor 23 related hypophosphatemic diseases

N° IL287289A

Peptide-modified hybrid recombinant adeno-associated virus serotype between aav9 and aavrh74 with reduced liver tropism and increased muscle transduction

N° IL286926A

COMPOSITIONS AND METHODS FOR IMPROVING VIRAL VECTOR EFFICIENCY

N° JP2022002530A

ENHANCING UTROPHIN EXPRESSION IN CELL BY INDUCING MUTATIONS WITHIN UTROPHIN REGULATORY ELEMENTS AND THERAPEUTIC USE THEREOF

N° WO2022069598A1

Enhancing expression of anti-amyotrophin-associated protein in cell by inducing mutation within anti-amyotrophin-associated protein regulatory element and therapeutic uses thereof

N° CN116323941A

ENHANCING UTROPHIN EXPRESSION IN CELL BY INDUCING MUTATIONS WITHIN UTROPHIN REGULATORY ELEMENTS AND THERAPEUTIC USE THEREOF

N° EP4222263A1

Enhancing Utrophin Expression in Cell by Inducing Mutations Within Utrophin Regulatory Elements and Therapeutic Use Thereof

N° US2023272433A1

ENHANCING UTROPHIN EXPRESSION IN CELL BY INDUCING MUTATIONS WITHIN UTROPHIN REGULATORY ELEMENTS AND THERAPEUTIC USE THEREOF

N° CA3200563A1

Peptide-modified AAV capsid

N° CN116096431A

PEPTIDE-MODIFIED AAV CAPSID

N° EP4211231A1

PEPTIDE-MODIFIED AAV CAPSID

N° WO2022053630A1

Peptide-Modified AAV Capsid

N° US2024209030A1

PEPTIDE-MODIFIED AAV CAPSID

N° FI4211231T3

PEPTIDE-MODIFIED AAV CAPSID

N° EP4421179A2

Peptid-modificeret AVV-kapsid

N° DK4211231T3

PEPTIDE-MODIFIED AAV CAPSID

N° CA3193128A1

PEPTIDE-MODIFIED AAV CAPSID

N° PT4211231T

PEPTIDE-MODIFIED AAV CAPSID

N° PL4211231T3

CAPSIDE DE AAV MODIFICADA CON PEPTIDOS

N° ES2984259T3

CAPSÍDEO DE AAV MODIFICADO POR PEPTÍDEO

N° BR112023004176A2

SCALABLE LENTIVIRAL VECTOR PRODUCTION SYSTEM COMPATIBLE WITH INDUSTRIAL PHARMACEUTICAL APPLICATIONS

N° US2022235371A1

C-TERMINAL TRUNCATED GDE FOR THE TREATMENT OF GLYCOGEN STORAGE DISEASE III

N° WO2022043280A1

C-TERMINAL TRUNCATED GDE FOR THE TREATMENT OF GLYCOGEN STORAGE DISEASE III

N° EP4200410A1

C-TERMINAL TRUNCATED GDE FOR THE TREATMENT OF GLYCOGEN STORAGE DISEASE III

N° US2023313152A1

A METHOD FOR DETERMINING THE NATURE OF DNA IMPURITIES INSIDE VIRAL PARTICLES

N° EP4119682A1

A NOVEL MUSCLE-SPECIFIC PROMOTER

N° WO2022008711A1