REGENXBIO INC. logo

REGENXBIO INC.

41 Marks
Corp.
First Filed:May 6, 2011Latest Filed:Dec 16, 2025Address:9804 Medical Center Drive, Rockville, MD 20850, US

Portfolio Overview

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4

Top Classes

Class 42
Software, IT Services & Scientific Research
26(63%)
Class 5
Pharmaceuticals
13(32%)
Class 40
Material Treatment
1(2%)

TTAB Proceedings

Proceedings
4
As Plaintiff
4(100%)
As Defendant
0(0%)
Extensions of Time
5

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Trademark Activity Timeline(2011 – 2025)

Trademark Portfolio

45 results

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Filed
Registered
Last Activity
REYSTORDYS
REYSTORDYS
Trademark
Word
PENDING
Owner:
Serial:99550037
Filed:Dec 16, 2025
Classes:5
Description: Biological preparations for medical use in the treatment of genetic diseases and disorders, and muscle disorders, namely, Duchenne muscular dystrophy; Pharmaceutical preparations, namely, recombinant adeno-associated viral (rAAV) vectors for use in gene therapy; Pharmaceutical preparations for the treatment of genetic diseases and disorders, and muscle disorders, namely, Duchenne muscular dystrophy; Clinical medical reagents for use in gene therapy; Therapeutic pharmaceutical compositions that include recombinant adeno-associated viral (rAAV) vectors to provide a platform for specific expression of biologics with therapeutic effect in gene therapy; Pharmaceutical preparations containing adeno-associated viral (AAV) vectors and nucleic acids for a system delivering genes to biological cells for use in the treatment of genetic diseases and disorders, namely, Duchenne muscular dystrophy; Gene therapy preparation for medical use containing genetic materials for sustained protein expression, namely, microdystrophin expression
ECTRUDYS
ECTRUDYS
Trademark
Word
ABANDONED
Owner:
Serial:99172024
Filed:May 6, 2025
Classes:5
Description: Biological preparations for medical use in the treatment of genetic diseases and disorders, and muscle disorders, namely, Duchenne muscular dystrophy; Pharmaceutical preparations, namely, recombinant adeno-associated viral (rAAV) vectors for use in gene therapy; Pharmaceutical preparations for the treatment of genetic diseases and disorders, and muscle disorders, namely, Duchenne muscular dystrophy; Clinical medical reagents for use in gene therapy; Therapeutic pharmaceutical compositions that include recombinant adeno-associated viral (rAAV) vectors to provide a platform for specific expression of biologics with therapeutic effect in gene therapy; Pharmaceutical preparations containing adeno-associated viral (AAV) vectors and nucleic acids for a system delivering genes to biological cells for use in the treatment of genetic diseases and disorders, namely, Duchenne muscular dystrophy; Gene therapy preparation for medical use containing genetic materials for sustained protein expression, namely, microdystrophin expression
NAVGENDYS
NAVGENDYS
Trademark
Word
ABANDONED
Owner:
Serial:99171990
Filed:May 6, 2025
Classes:5
Description: Biological preparations for medical use in the treatment of genetic diseases and disorders, and muscle disorders, namely, Duchenne muscular dystrophy; Pharmaceutical preparations, namely, recombinant adeno-associated viral (rAAV) vectors for use in gene therapy; Pharmaceutical preparations for the treatment of genetic diseases and disorders, and muscle disorders, namely, Duchenne muscular dystrophy; Clinical medical reagents for use in gene therapy; Therapeutic pharmaceutical compositions that include recombinant adeno-associated viral (rAAV) vectors to provide a platform for specific expression of biologics with therapeutic effect in gene therapy; Pharmaceutical preparations containing adeno-associated viral (AAV) vectors and nucleic acids for a system delivering genes to biological cells for use in the treatment of genetic diseases and disorders, namely, Duchenne muscular dystrophy; Gene therapy preparation for medical use containing genetic materials for sustained protein expression, namely, microdystrophin expression
NAVGENVYS
NAVGENVYS
Trademark
Word
PENDING
Owner:
Serial:99172007
Filed:May 6, 2025
Classes:5
Description: Biological preparations for medical use in the treatment of genetic diseases and disorders, and muscle disorders, namely, Duchenne muscular dystrophy; Pharmaceutical preparations, namely, recombinant adeno-associated viral (rAAV) vectors for use in gene therapy; Pharmaceutical preparations for the treatment of genetic diseases and disorders, and muscle disorders, namely, Duchenne muscular dystrophy; Clinical medical reagents for use in gene therapy; Therapeutic pharmaceutical compositions that include recombinant adeno-associated viral (rAAV) vectors to provide a platform for specific expression of biologics with therapeutic effect in gene therapy; Pharmaceutical preparations containing adeno-associated viral (AAV) vectors and nucleic acids for a system delivering genes to biological cells for use in the treatment of genetic diseases and disorders, namely, Duchenne muscular dystrophy; Gene therapy preparation for medical use containing genetic materials for sustained protein expression, namely, microdystrophin expression
GEMPSIIVO
GEMPSIIVO
Trademark
Word
PENDING
Owner:
Serial:98196371
Filed:Sep 25, 2023
Classes:5
Description: Biological preparations for medical use in the treatment of genetic diseases and disorders, and central nervous system (CNS) disorders, namely, neurodegenerative disorders; pharmaceutical preparations, namely, recombinant adeno-associated viral (rAAV) vectors for use in gene therapy; Pharmaceutical preparations for the treatment of genetic diseases and disorders and central nervous system (CNS) disorders, namely, neurodegenerative disorders; Pharmaceutical preparations for use in gene therapy; Clinical medical reagents for use in gene therapy; Pharmaceutical preparations for the treatment of genetic disorders and neurodegenerative disorders; Therapeutic pharmaceutical compositions that include recombinant adeno-associated viral (rAAV) vectors to provide a platform for specific expression of biologics with therapeutic effect in gene therapy; Pharmaceutical preparations containing adeno-associated viral (AAV) vectors and nucleic acids for a system delivering genes to biological cells for use in the treatment of diseases, sicknesses, and disorders, namely, lysosomal storage disorders; gene therapy preparation for medical use containing genetic materials for sustained protein expression, namely, lysosomal enzyme expression
IDSYFUL
IDSYFUL
Trademark
Word
PENDING
Owner:
Serial:98196277
Filed:Sep 25, 2023
Classes:5
Description: Biological preparations for medical use in the treatment of genetic diseases and disorders, and central nervous system (CNS) disorders, namely, neurodegenerative disorders; pharmaceutical preparations, namely, recombinant adeno-associated viral (rAAV) vectors for use in gene therapy; Pharmaceutical preparations for the treatment of genetic diseases and disorders and central nervous system (CNS) disorders, namely, neurodegenerative disorders; Pharmaceutical preparations for use in gene therapy; Clinical medical reagents for use in gene therapy; Pharmaceutical preparations for the treatment of genetic disorders and neurodegenerative disorders; Therapeutic pharmaceutical compositions that include recombinant adeno-associated viral (rAAV) vectors to provide a platform for specific expression of biologics with therapeutic effect in gene therapy; Pharmaceutical preparations containing adeno-associated viral (AAV) vectors and nucleic acids for a system delivering genes to biological cells for use in the treatment of diseases, sicknesses, and disorders, namely, lysosomal storage disorders; gene therapy preparation for medical use containing genetic materials for sustained protein expression, namely, lysosomal enzyme expression
XARMAPSII
XARMAPSII
Trademark
Word
PENDING
Owner:
Serial:98196234
Filed:Sep 25, 2023
Classes:5
Description: Biological preparations for medical use in the treatment of genetic diseases and disorders, and central nervous system (CNS) disorders, namely, neurodegenerative disorders; pharmaceutical preparations, namely, recombinant adeno-associated viral (rAAV) vectors for use in gene therapy; Pharmaceutical preparations for the treatment of genetic diseases and disorders and central nervous system (CNS) disorders, namely, neurodegenerative disorders; Pharmaceutical preparations for use in gene therapy; Clinical medical reagents for use in gene therapy; Pharmaceutical preparations for the treatment of genetic disorders and neurodegenerative disorders; Therapeutic pharmaceutical compositions that include recombinant adeno-associated viral (rAAV) vectors to provide a platform for specific expression of biologics with therapeutic effect in gene therapy; Pharmaceutical preparations containing adeno-associated viral (AAV) vectors and nucleic acids for a system delivering genes to biological cells for use in the treatment of diseases, sicknesses, and disorders, namely, lysosomal storage disorders; gene therapy preparation for medical use containing genetic materials for sustained protein expression, namely, lysosomal enzyme expression
98123490
98123490
Trademark
Design
REGISTERED
Owner:
Serial:98123490
Filed:Aug 9, 2023
Classes:5
Registration:7709746
Registered:Mar 4, 2025
Description: Biotechnological platform technology, namely, biological nanoparticles, recombinant viruses, and adenoviruses for gene delivery to biological cells that includes recombinant adeno-associated viral (rAAV) vectors to provide a platform for specific expression of biologics with therapeutic effect in gene therapy, RNA interference (RNAi), and cell therapy
REGENXBIO
REGENXBIO
Trademark
Word
REGISTERED
Owner:
Serial:98123485
Filed:Aug 9, 2023
Classes:5
Registration:7709745
Registered:Mar 4, 2025
Description: Biotechnological platform technology, namely, biological nanoparticles, recombinant viruses, and adenoviruses for gene delivery to biological cells that includes recombinant adeno-associated viral (rAAV) vectors to provide a platform for specific expression of biologics with therapeutic effect in gene therapy, RNA interference (RNAi), and cell therapy
REGENXBIO
REGENXBIO
Trademark
Combined
REGISTERED
Owner:
Serial:98123484
Filed:Aug 9, 2023
Classes:5
Registration:7709744
Registered:Mar 4, 2025
Description: Biotechnological platform technology, namely, biological nanoparticles, recombinant viruses, and adenoviruses for gene delivery to biological cells that includes recombinant adeno-associated viral (rAAV) vectors to provide a platform for specific expression of biologics with therapeutic effect in gene therapy, RNA interference (RNAi), and cell therapy
AFFINITY BEYOND
AFFINITY BEYOND
Service Mark
Combined
REGISTERED
Owner:
Serial:97672147
Filed:Nov 10, 2022
Classes:42
Registration:7357017
Registered:Apr 9, 2024
Description: Providing medical and scientific research information in the field of clinical trials; Medical and scientific research, namely, conducting clinical trials for others
AFFINITY BEYOND
AFFINITY BEYOND
Service Mark
Combined
REGISTERED
Owner:
Serial:97672155
Filed:Nov 10, 2022
Classes:42
Registration:7357018
Registered:Apr 9, 2024
Description: Providing medical and scientific research information in the field of clinical trials; Medical and scientific research, namely, conducting clinical trials for others
AFFINITY BEYOND
AFFINITY BEYOND
Service Mark
Word
REGISTERED
Owner:
Serial:97672137
Filed:Nov 10, 2022
Classes:42
Registration:7368612
Registered:Apr 23, 2024
Description: Providing medical and scientific research information in the field of clinical trials; Medical and scientific research, namely, conducting clinical trials for others
CAMPSIITE
CAMPSIITE
Service Mark
Word
REGISTERED
Owner:
Serial:97526610
Filed:Jul 29, 2022
Classes:42
Registration:7070144
Registered:May 30, 2023
Description: Providing medical and scientific research information in the field of clinical trials; Medical and scientific research, namely, conducting clinical trials for others
NAVXCELL
NAVXCELL
Trademark
Word
REGISTERED
Owner:
Serial:97436707
Filed:May 31, 2022
Classes:5
Registration:7134753
Registered:Aug 8, 2023
Description: Biotechnological platform technology, namely, cell lines for preparation of biological vectors, and recombinant viruses for gene delivery, that includes recombinant adeno-associated viral (rAAV) vectors to provide a platform for specific production of biologics with therapeutic effect in gene therapy, including gene therapy delivery of antibody biologics, enzymes and RNA interference (RNAi)
NAVXPRESS
NAVXPRESS
Service Mark
Word
REGISTERED
Owner:
Serial:97436697
Filed:May 31, 2022
Classes:40
Registration:7566017
Registered:Nov 12, 2024
Description: Custom manufacture of reagents for scientific and medical research and development; Custom manufacturing services, namely, production for others of clinical and medical reagents for use in gene therapy, for development of pharmaceutical preparations for use in gene therapy, and of therapeutic compositions that include recombinant adeno-associated viral (rAAV) vectors to provide a platform for specific expression of biologics with therapeutic effect in gene therapy, including gene therapy delivery of antibody biologics, enzymes and RNA interference (RNAi) | Custom design and development of reagents for scientific and medical research and development
AFFINITY
AFFINITY
Service Mark
Word
REGISTERED
Owner:
Serial:97125444
Filed:Nov 15, 2021
Classes:42
Registration:7263068
Registered:Jan 2, 2024
Description: Providing medical and scientific research information in the field of clinical trials; Medical and scientific research, namely, conducting clinical trials for others
AFFINITY DUCHENNE
AFFINITY DUCHENNE
Service Mark
Combined
REGISTERED
Owner:
Serial:97125435
Filed:Nov 15, 2021
Classes:42
Registration:7197150
Registered:Oct 17, 2023
Description: Providing medical and scientific research information in the field of clinical trials; medical and scientific research, namely, conducting clinical trials for others
AFFINITY-DUCHENNE
AFFINITY-DUCHENNE
Service Mark
Word
ABANDONED
Owner:
Serial:97125450
Filed:Nov 15, 2021
Classes:42
ELAAVATE
ELAAVATE
Service Mark
Word
ABANDONED
Owner:
Serial:90632317
Filed:Apr 8, 2021
Classes:42
GLIMPSE
GLIMPSE
Service Mark
Word
ABANDONED
Owner:
Serial:90530563
Filed:Feb 16, 2021
Classes:42
VISTA
VISTA
Service Mark
Word
ABANDONED
Owner:
Serial:90530555
Filed:Feb 16, 2021
Classes:42
AAVIATION
AAVIATION
Service Mark
Word
ABANDONED
Owner:
Serial:90432225
Filed:Dec 30, 2020
Classes:42
ASCENT
ASCENT
Service Mark
Word
REGISTERED
Owner:
Serial:90133732
Filed:Aug 24, 2020
Classes:42
Registration:8078650
Registered:Dec 23, 2025
Description: Medical and scientific research, namely, conducting clinical trials for others, all the above for gene therapy, namely for use in the treatment of wet age-related macular degeneration (AMD) using subretinal delivery
EMBARK
EMBARK
Service Mark
Word
ABANDONED
Owner:
Serial:90133768
Filed:Aug 24, 2020
Classes:42
NAAVIGATE
NAAVIGATE
Service Mark
Word
ABANDONED
Owner:
Serial:90133798
Filed:Aug 24, 2020
Classes:42
AAVIATE
AAVIATE
Service Mark
Word
REGISTERED
Owner:
Serial:88863049
Filed:Apr 7, 2020
Classes:42
Registration:6274208
Registered:Feb 16, 2021
Description: Providing medical and scientific research information in the field of clinical trials; Medical and scientific research, namely, conducting clinical trials for others
CAMPSITE
CAMPSITE
Service Mark
Word
ABANDONED
Owner:
Serial:88863066
Filed:Apr 7, 2020
Classes:42
ALTITUDE
ALTITUDE
Service Mark
Word
REGISTERED
Owner:
Serial:88500159
Filed:Jul 3, 2019
Classes:42
Registration:6631033
Registered:Feb 1, 2022
Description: Providing medical and scientific research information in the field of clinical trials; Medical and scientific research, namely, conducting clinical trials for others
ATMOSPHERE
ATMOSPHERE
Service Mark
Word
REGISTERED
Owner:
Serial:88500144
Filed:Jul 3, 2019
Classes:42
Registration:6323051
Registered:Apr 13, 2021
Description: Providing medical and scientific research information in the field of clinical trials in the field of gene therapy; Medical and scientific research, namely, conducting clinical trials for others in the field of gene therapy
AAVIATION
AAVIATION
Service Mark
Word
ABANDONED
Owner:
Serial:88085174
Filed:Aug 20, 2018
Classes:42
CAMPSITE
CAMPSITE
Service Mark
Word
ABANDONED
Owner:
Serial:87200042
Filed:Oct 11, 2016
Classes:42
AAVIATE
AAVIATE
Service Mark
Word
ABANDONED
Owner:
Serial:87174815
Filed:Sep 18, 2016
Classes:42
86397688
86397688
Service Mark
Design
REGISTERED
Owner:
Serial:86397688
Filed:Sep 17, 2014
Classes:42
Registration:4984046
Registered:Jun 21, 2016
Description: Custom development of biochemical reagents, namely, synthesis for others of clinical medical reagents for use in gene therapy, development of pharmaceutical preparations for use in gene therapy, and therapeutic compositions that include recombinant adeno-associated viral (rAAV) vectors to provide a platform for specific expression of biologics with therapeutic effect in gene therapy, RNA interference (RNAi) and cell therapy; Scientific and medical research and development; genetic engineering services, biotechnology research services, and research and development of new products in the field of gene therapeutics and research tools, including those based on recombinant adeno-associated viral vectors
REGENXBIO
REGENXBIO
Service Mark
Word
REGISTERED
Owner:
Serial:86397653
Filed:Sep 17, 2014
Classes:42
Registration:5018428
Registered:Aug 9, 2016
Description: Custom development of biochemical reagents, namely, synthesis for others of clinical medical reagents for use in gene therapy, development of pharmaceutical preparations for use in gene therapy, and therapeutic compositions that include recombinant adeno-associated viral (rAAV) vectors to provide a platform for specific expression of biologics with therapeutic effect in gene therapy, and RNA interference (RNAi); Scientific and medical research and development; genetic engineering services, biotechnology research services, and research and development of new products in the field of gene therapeutics and research tools, including those based on recombinant adeno-associated viral vectors
REGENXBIO
REGENXBIO
Service Mark
Combined
REGISTERED
Owner:
Serial:86397777
Filed:Sep 17, 2014
Classes:42
Registration:5018429
Registered:Aug 9, 2016
Description: Custom development of biochemical reagents, namely, synthesis for others of clinical medical reagents for use in gene therapy, development of pharmaceutical preparations for use in gene therapy, and therapeutic compositions that include recombinant adeno-associated viral (rAAV) vectors to provide a platform for specific expression of biologics with therapeutic effect in gene therapy, and RNA interference (RNAi); Scientific and medical research and development; genetic engineering services, biotechnology research services, and research and development of new products in the field of gene therapeutics and research tools, including those based on recombinant adeno-associated viral vectors
NAV
NAV
Service Mark
Word
REGISTERED
Owner:
Serial:85314692
Filed:May 6, 2011
Classes:44
Registration:4824624
Registered:Oct 6, 2015
Description: Medical services in the field of biotechnology, namely, biological nanoparticles, recombinant viruses, and adenoviruses for gene delivery to biological cells; medical services in the field of biotechnology, namely, biological nanoparticles, recombinant viruses, and adenoviruses for gene delivery to biological cells; medical services related to genetic engineering; medical services in the field of gene therapeutics; and medical services based on recombinant adeno-associated viral vectors for others
NAV
NAV
Trademark
Word
REGISTERED
Owner:
Serial:85314870
Filed:May 6, 2011
Classes:5
Registration:4384443
Registered:Aug 13, 2013
Description: Biotechnological platform technology, namely, biological nanoparticles, recombinant viruses, and adenoviruses for gene delivery to biological cells that includes recombinant adeno-associated viral (rAAV) vectors to provide a platform for specific expression of biologics with therapeutic effect in gene therapy, RNA interference (RNAi), and cell therapy
NAV
NAV
Trademark
Word
CANCELLED
Owner:
Serial:85314838
Filed:May 6, 2011
Classes:5
Registration:4384442
Registered:Aug 13, 2013
NAV
NAV
Service Mark
Word
REGISTERED
Owner:
Serial:85314794
Filed:May 6, 2011
Classes:42
Registration:4181837
Registered:Jul 31, 2012
Description: Custom development of biochemical reagents, namely, synthesis for others of clinical medical reagents for use in gene therapy, development of pharmaceutical preparations for use in gene therapy, and therapeutic compositions that include recombinant adeno-associated viral (rAAV) vectors to provide a platform for specific expression of biologics with therapeutic effect in gene therapy, RNA interference (RNAi), and cell therapy
NAV
NAV
Service Mark
Word
REGISTERED
Owner:
Serial:85314740
Filed:May 6, 2011
Classes:42
Registration:5612076
Registered:Nov 20, 2018
Description: Scientific research and development in the field of biotechnology, namely, biological nanoparticles, recombinant viruses, and adenoviruses for gene delivery to biological cells; medical research and development in the field of biotechnology, namely, biological nanoparticles, recombinant viruses, and adenoviruses for gene delivery to biological cells; research related to genetic engineering; research and development of new biotechnology products in the field of gene therapeutics; and research tools based on recombinant adeno-associated viral vectors for others
98833529
98833529
Trademark
Design
Previous
PENDING
Owner:
Serial:98833529
Filed:Nov 1, 2024
Classes:5
Description: Biological preparations for medical use in the treatment of genetic diseases and disorders, and central nervous system (CNS) disorders, namely, neurodegenerative disorders; pharmaceutical preparations, namely, recombinant adeno-associated viral (rAAV) vectors for use in gene therapy; Pharmaceutical preparations for the treatment of genetic diseases and disorders and central nervous system (CNS) disorders, namely, neurodegenerative disorders; Pharmaceutical preparations for use in gene therapy; Clinical medical reagents for use in gene therapy; Pharmaceutical preparations for the treatment of genetic disorders and neurodegenerative disorders; Therapeutic pharmaceutical compositions that include recombinant adeno-associated viral (rAAV) vectors to provide a platform for specific expression of biologics with therapeutic effect in gene therapy; Pharmaceutical preparations containing adeno-associated viral (AAV) vectors and nucleic acids for a system delivering genes to biological cells for use in the treatment of diseases, sicknesses, and disorders, namely, lysosomal storage disorders; gene therapy preparation for medical use containing genetic materials for sustained protein expression, namely, lysosomal enzyme expression
NAVSUNLI
NAVSUNLI
Trademark
Combined
Previous
PENDING
Owner:
Serial:98833523
Filed:Nov 1, 2024
Classes:5
Description: Biological preparations for medical use in the treatment of genetic diseases and disorders, and central nervous system (CNS) disorders, namely, neurodegenerative disorders; pharmaceutical preparations, namely, recombinant adeno-associated viral (rAAV) vectors for use in gene therapy; Pharmaceutical preparations for the treatment of genetic diseases and disorders and central nervous system (CNS) disorders, namely, neurodegenerative disorders; Pharmaceutical preparations for use in gene therapy; Clinical medical reagents for use in gene therapy; Pharmaceutical preparations for the treatment of genetic disorders and neurodegenerative disorders; Therapeutic pharmaceutical compositions that include recombinant adeno-associated viral (rAAV) vectors to provide a platform for specific expression of biologics with therapeutic effect in gene therapy; Pharmaceutical preparations containing adeno-associated viral (AAV) vectors and nucleic acids for a system delivering genes to biological cells for use in the treatment of diseases, sicknesses, and disorders, namely, lysosomal storage disorders; gene therapy preparation for medical use containing genetic materials for sustained protein expression, namely, lysosomal enzyme expression
NAVSUNLI
NAVSUNLI
Trademark
Combined
Previous
PENDING
Owner:
Serial:98833538
Filed:Nov 1, 2024
Classes:5
Description: Biological preparations for medical use in the treatment of genetic diseases and disorders, and central nervous system (CNS) disorders, namely, neurodegenerative disorders; pharmaceutical preparations, namely, recombinant adeno-associated viral (rAAV) vectors for use in gene therapy; Pharmaceutical preparations for the treatment of genetic diseases and disorders and central nervous system (CNS) disorders, namely, neurodegenerative disorders; Pharmaceutical preparations for use in gene therapy; Clinical medical reagents for use in gene therapy; Pharmaceutical preparations for the treatment of genetic disorders and neurodegenerative disorders; Therapeutic pharmaceutical compositions that include recombinant adeno-associated viral (rAAV) vectors to provide a platform for specific expression of biologics with therapeutic effect in gene therapy; Pharmaceutical preparations containing adeno-associated viral (AAV) vectors and nucleic acids for a system delivering genes to biological cells for use in the treatment of diseases, sicknesses, and disorders, namely, lysosomal storage disorders; gene therapy preparation for medical use containing genetic materials for sustained protein expression, namely, lysosomal enzyme expression
NAVSUNLI
NAVSUNLI
Trademark
Word
Previous
PENDING
Owner:
Serial:98196308
Filed:Sep 25, 2023
Classes:5
Description: Biological preparations for medical use in the treatment of genetic diseases and disorders, and central nervous system (CNS) disorders, namely, neurodegenerative disorders; pharmaceutical preparations, namely, recombinant adeno-associated viral (rAAV) vectors for use in gene therapy; Pharmaceutical preparations for the treatment of genetic diseases and disorders and central nervous system (CNS) disorders, namely, neurodegenerative disorders; Pharmaceutical preparations for use in gene therapy; Clinical medical reagents for use in gene therapy; Pharmaceutical preparations for the treatment of genetic disorders and neurodegenerative disorders; Therapeutic pharmaceutical compositions that include recombinant adeno-associated viral (rAAV) vectors to provide a platform for specific expression of biologics with therapeutic effect in gene therapy; Pharmaceutical preparations containing adeno-associated viral (AAV) vectors and nucleic acids for a system delivering genes to biological cells for use in the treatment of diseases, sicknesses, and disorders, namely, lysosomal storage disorders; gene therapy preparation for medical use containing genetic materials for sustained protein expression, namely, lysosomal enzyme expression