AAV Vectors in Gene Therapy Pipeline Drugs 2023 (Updated), FDA Approvals, Clinical Trials, MOA, ROA | Companies- BioMarin Pharmaceutical, Gensight Biologics, PTC therapeutics, Ultragenyx Pharma

(Albany, United States) As per DelveInsight's assessment, globally, the AAV Vectors in Gene Therapy pipeline constitutes 70+ key companies continuously working towards developing 235+ AAV Vectors in Gene Therapy treatment therapies, analysis of Clinical Trials, Therapies, Mechanism of Action, Route of Administration, and Developments analysis DelveInsight.

The AAV Vectors in Gene Therapy Pipeline report embraces in-depth commercial and clinical assessment of the pipeline products from the pre-clinical developmental phase to the marketed phase. The AAV Vectors in Gene Therapy pipeline report also covers a detailed description of the drug, including the mechanism of action of the drug, clinical studies, AAV Vectors in Gene Therapy NDA approvals (if any), and product development activities comprising the technology, collaborations, mergers acquisition, funding, designations, and other product-related details.

To explore more information on the latest breakthroughs in the AAV Vectors in Gene Therapy Pipeline treatment landscape of the report, click here @ https://www.delveinsight.com/report-store/adeno-associated-virus-aav-vectors-in-gene-therapy-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr

Key Takeaways from the AAV Vectors in Gene Therapy Pipeline Report
• DelveInsight's AAV Vectors in Gene Therapy Pipeline analysis depicts a robust space with 70+ active players working to develop 235+ pipeline treatment therapies.
• The leading AAV Vectors in Gene Therapy Companies are working in the market include BioMarin Pharmaceutical, Gensight Biologics, PTC therapeutics, Ultragenyx Pharmaceutical, MeiraGTx, Pfizer, Biogen, uniQure, Pfizer, Ultragenyx Pharmaceutical, REGENXBIO, Biogen, Spark therapeuics (Roche), Sarepta therapeutics, Neurophth Therapeutics, LYSOGENE, Gyroscope Therapeutics, Nanoscope Therapeutics, Homology medicines, Ultragenyx Pharmaceutical, Passage Bio, Freeline therapeutics, Astellas Pharma, Aspa therapeutics, Adrenas Therapeutics, ESTEVE, Sio Gene Therapies, Amicus therapeutics, 4D Molecular therapeutics, Taysha Gene Therapies, Atsena Therapeutics, BioMarin Pharmaceutical, Abeona Therapeutics, REGENXBIO, uniQure, Taysha Gene Therapies, Asklepios BioPharmaceutical, Sarepta Therapeutics, Abeona Therapeutics, Forge Biologics, Sangamo therapeutics, Bayer, LogicBio therapeutics, Solid Biosciences, Applied Genetic Technologies, Spark therapeuics (Roche), Eli lilly and company, Lexeo Therapeutics, Gensight Biologics, Alcyone Lifesciences, MeiraGTx, Audentes therapeutics, Vivet Therapeutics, Takeda, Astellas Gene Therapies, Homology Medicines, Adverum Biotechnologies, Libella Gene Therapeutics, Rocket Pharmaceuticals, Brain Neurotherapy Bio, Inc., Ocugen, Asklepios BioPharmaceutical, Neurogene, Jaguar Gene Therapy, LLC, Axovia Therapeutics, Coave therapeutics, Novartis/ Arctos medical, Novartis/Vedere Bio, REGENXBIO Inc., StrideBio, Abeona therapeutics, Sanofi/ Sirion therapeutics, Capsida Biotherapeutics, Kriya therapeutics, Neurophth, Poseida Therapeutics, BridgeBio Pharma, DiNAQOR/ Biomarin, ViGeneron, AskBio/Selecta Biosciences, Prevail therapeutics, Intas Pharmaceuticals, Tenaya Therapeutics, Scout Bio therapeutics, Ocugen, Oyster Point pharmaceuticals, Cyprium Therapeutics, Solid Biosciences, Amicus Therapeutics, Coave therapeutics, AvantiBio, Voyager Therapeutics/ Pfizer, Aruvant Sciences, Alcyone Lifesciences, Gene Therapy Research Institution Co., Ltd., and others.
• Emerging AAV Vectors in Gene Therapy Pipeline Therapies in the various stages of development include injection of scAAV2-P1ND4v2 1.18x10e9 vg (Low), SB-525 (PF-07055480), Injection of GS001, Valoctocogene Roxaparvovec, tgAAG76 (rAAV 2/2.hRPE65p.hRPE65), AAV - CNGB3, GNT0003, and others
• On March 2023, Genethon has announced drug named GNT0003 in the market. This is a Phase 1/2, multinational, open-label, study to evaluate the safety and efficacy of an intravenous infusion of GNT0003 in patients with Crigler-Najjar aged ≥10 years and requiring phototherapy. Patients will received a single administration of GNT0003 and will be followed for safety and efficacy of approximately 60 months (5 years):
• On March 2023, MeiraGTx UK II Ltd has announced drug named AAV - CNGB3 in the market by the phase 1 & 2. A clinical trial of AAV - CNGB3 retinal gene therapy for patients with Achromatopsia.
• On January 2023, BioMarin Pharmaceutical has announced drug named Valoctocogene Roxaparvovec in the market by the phase 1 & 2. This study is being conducted by BioMarin Pharmaceutical Inc. as an open label, single dose study to determine the safety of valoctocogene roxaparvovec (an Adenovirus-Associated Virus (AAV) based gene therapy vector) in severe Hemophilia A patients with pre-existing antibodies against AAV5.
• On May 2023, Zhang Lei, MD, Institute of Hematology & Blood Diseases Hospital has announced drug named Injection of GS001 in the market. IHBDH-GTHA-2020 is an open- label, non- randomized study to evaluate the safety, tolerability and kinetics of a single intravenous infusion of GS001 in hemophilia A subjects with
• On June 2023, Pfizer has announced drug named SB-525 (PF-07055480) in the market by the phase 2. The purpose of the study is to evaluate the safety, tolerability and time-course profile of FVIII activity after dosing with SB-525 (PF-07055480).

AAV Vectors in Gene Therapy Overview
Out of the several viral vectors that have been used to date for delivering the genes of interest, the Adeno-associated viral (AAV) vector appears to be the safest and effective vehicle and can maintain long-term gene and protein expression following a single injection of the vector.

For further information, refer to the detailed AAV Vectors in Gene Therapy Unmet Needs, click here for AAV Vectors in Gene Therapy Ongoing Clinical Trial Analysis @ https://www.delveinsight.com/sample-request/adeno-associated-virus-aav-vectors-in-gene-therapy-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr

AAV Vectors in Gene Therapy Emerging Drugs Profile
• GS010: Gensight Biologics
• Valoctocogene roxaparvovec: BioMarin Pharmaceutical
• DTX401: Ultragenyx Pharmaceutical
• AAV5-RPGR: MeiraGTx
• Timrepigene emparvovec: Biogen
• AMT 061: UniQure
• RGX-314: REGENXBIO
• SPK-8011: Spark Therapeutics
• NFS-01: Neurophth
• GT 005: Gyroscope Therapeutics

AAV Vectors in Gene Therapy Pipeline Therapeutics Assessment
There are approx. 70+ AAV Vectors in Gene Therapy companies which are developing the therapies for AAV vectors in gene therapy. The AAV Vectors in Gene Therapy companies which have their AAV vectors in gene therapy drug candidates in the most advanced stage, i.e. Preregistration include, BioMarin Pharmaceutical.

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AAV Vectors in Gene Therapy Drugs & Companies
• GNT0003: Genethon
• AAV - CNGB3: Janssen Research & Development LLC
• tgAAG76 (rAAV 2/2.hRPE65p.hRPE65): Targeted Genetics Corporation
• Valoctocogene Roxaparvovec: BioMarin Pharmaceutical
• SB-525 (PF-07055480): Pfizer

AAV Vectors in Gene Therapy Therapeutics Assessment
• Assessment by Stage and Product Type
• Assessment by Route of Administration
• Assessment by Stage and Route of Administration
• Assessment by Molecule Type
• Assessment by Stage and Molecule Type

Some of the Companies in the AAV Vectors in Gene Therapy Therapeutics Market include-
BioMarin Pharmaceutical, Gensight Biologics, PTC therapeutics, Ultragenyx Pharmaceutical, MeiraGTx, Pfizer, Biogen, uniQure, Pfizer, Ultragenyx Pharmaceutical, REGENXBIO, Biogen, Spark therapeuics (Roche), Sarepta therapeutics, Neurophth Therapeutics, LYSOGENE, Gyroscope Therapeutics, Nanoscope Therapeutics, Homology medicines, Ultragenyx Pharmaceutical, Passage Bio, Freeline therapeutics, Astellas Pharma, Aspa therapeutics, Adrenas Therapeutics, ESTEVE, Sio Gene Therapies, Amicus therapeutics, 4D Molecular therapeutics, Taysha Gene Therapies, Atsena Therapeutics, BioMarin Pharmaceutical, Abeona Therapeutics, REGENXBIO, uniQure, Taysha Gene Therapies, Asklepios BioPharmaceutical, Sarepta Therapeutics, Abeona Therapeutics, Forge Biologics, Sangamo therapeutics, Bayer, LogicBio therapeutics, Solid Biosciences, Applied Genetic Technologies, Spark therapeuics (Roche), Eli lilly and company, Lexeo Therapeutics, Gensight Biologics, Alcyone Lifesciences, MeiraGTx, Audentes therapeutics, Vivet Therapeutics, Takeda, Astellas Gene Therapies, Homology Medicines, Adverum Biotechnologies, Libella Gene Therapeutics, Rocket Pharmaceuticals, Brain Neurotherapy Bio, Inc., Ocugen, Asklepios BioPharmaceutical, Neurogene, Jaguar Gene Therapy, LLC, Axovia Therapeutics, Coave therapeutics, Novartis/ Arctos medical, Novartis/Vedere Bio, REGENXBIO Inc., StrideBio, Abeona therapeutics, Sanofi/ Sirion therapeutics, Capsida Biotherapeutics, Kriya therapeutics, Neurophth, Poseida Therapeutics, BridgeBio Pharma, DiNAQOR/ Biomarin, ViGeneron, AskBio/Selecta Biosciences, Prevail therapeutics, Intas Pharmaceuticals, Tenaya Therapeutics, Scout Bio therapeutics, Ocugen, Oyster Point pharmaceuticals, Cyprium Therapeutics, Solid Biosciences, Amicus Therapeutics, Coave therapeutics, AvantiBio, Voyager Therapeutics/ Pfizer, Aruvant Sciences, Alcyone Lifesciences, Gene Therapy Research Institution Co., Ltd., and others.

Dive deep into rich insights for drugs for AAV Vectors in Gene Therapy Pipeline, click here for AAV Vectors in Gene Therapy Unmet Needs and Analyst Views @ https://www.delveinsight.com/sample-request/adeno-associated-virus-aav-vectors-in-gene-therapy-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr

Scope of the AAV Vectors in Gene Therapy Pipeline Report
• Coverage- Global
• AAV Vectors in Gene Therapy Companies- BioMarin Pharmaceutical, Gensight Biologics, PTC therapeutics, Ultragenyx Pharmaceutical, MeiraGTx, Pfizer, Biogen, uniQure, Pfizer, Ultragenyx Pharmaceutical, REGENXBIO, Biogen, Spark therapeuics (Roche), Sarepta therapeutics, Neurophth Therapeutics, LYSOGENE, Gyroscope Therapeutics, Nanoscope Therapeutics, Homology medicines, Ultragenyx Pharmaceutical, Passage Bio, Freeline therapeutics, Astellas Pharma, Aspa therapeutics, Adrenas Therapeutics, ESTEVE, Sio Gene Therapies, Amicus therapeutics, 4D Molecular therapeutics, Taysha Gene Therapies, Atsena Therapeutics, BioMarin Pharmaceutical, Abeona Therapeutics, REGENXBIO, uniQure, Taysha Gene Therapies, Asklepios BioPharmaceutical, Sarepta Therapeutics, Abeona Therapeutics, Forge Biologics, Sangamo therapeutics, Bayer, LogicBio therapeutics, Solid Biosciences, Applied Genetic Technologies, Spark therapeuics (Roche), Eli lilly and company, Lexeo Therapeutics, Gensight Biologics, Alcyone Lifesciences, MeiraGTx, Audentes therapeutics, Vivet Therapeutics, Takeda, Astellas Gene Therapies, Homology Medicines, Adverum Biotechnologies, Libella Gene Therapeutics, Rocket Pharmaceuticals, Brain Neurotherapy Bio, Inc., Ocugen, Asklepios BioPharmaceutical, Neurogene, Jaguar Gene Therapy, LLC, Axovia Therapeutics, Coave therapeutics, Novartis/ Arctos medical, Novartis/Vedere Bio, REGENXBIO Inc., StrideBio, Abeona therapeutics, Sanofi/ Sirion therapeutics, Capsida Biotherapeutics, Kriya therapeutics, Neurophth, Poseida Therapeutics, BridgeBio Pharma, DiNAQOR/ Biomarin, ViGeneron, AskBio/Selecta Biosciences, Prevail therapeutics, Intas Pharmaceuticals, Tenaya Therapeutics, Scout Bio therapeutics, Ocugen, Oyster Point pharmaceuticals, Cyprium Therapeutics, Solid Biosciences, Amicus Therapeutics, Coave therapeutics, AvantiBio, Voyager Therapeutics/ Pfizer, Aruvant Sciences, Alcyone Lifesciences, Gene Therapy Research Institution Co., Ltd., and others.
• AAV Vectors in Gene Therapy Therapies- injection of scAAV2-P1ND4v2 1.18x10e9 vg (Low), SB-525 (PF-07055480), Injection of GS001, Valoctocogene Roxaparvovec, tgAAG76 (rAAV 2/2.hRPE65p.hRPE65), AAV - CNGB3, GNT0003, and others
• AAV Vectors in Gene Therapy Segmentation: Product Type, Molecule Type, Route of Administration

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Table of Content
1. Introduction
2. AAV Vectors in Gene Therapy Executive Summary
3. AAV vectors in gene therapy: Overview
4. AAV Vectors in Gene Therapy Pipeline Therapeutics
5. AAV Vectors in Gene Therapy Therapeutic Assessment
6. Late Stage Products (Preregistration)
7. Valoctocogene roxaparvovec: BioMarin Pharmaceutical
8. Drug profiles in the detailed report…..
9. Late Stage Products (Phase III)
10. DTX401: Ultragenyx Pharmaceutical
11. Drug profiles in the detailed report…..
12. Mid Stage Products (Phase II)
13. GT 005: Gyroscope Therapeutics
14. Drug profiles in the detailed report…..
15. Early stage products (Phase I/II)
16. Drug profiles in the detailed report…..
17. Early Stage Products (Phase I)
18. HMI-203: Homology Medicine
19. Drug profiles in the detailed report…..
20. Preclinical stage products
21. AXV101: Axovia Therapeutics
22. Drug profiles in the detailed report…..
23. Discovery stage products
24. DINA-002: DiNAQOR
25. Drug profiles in the detailed report…..
26. Inactive Products
27. AAV vectors in gene therapy Key Companies
28. AAV vectors in gene therapy Key Products
29. AAV vectors in gene therapy- Unmet Needs
30. AAV vectors in gene therapy- Market Drivers and Barriers
31. AAV vectors in gene therapy- Future Perspectives and Conclusion
32. AAV vectors in gene therapy Analyst Views
33. AAV vectors in gene therapy Key Companies
34. Appendix

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