The AAV Vectors in Gene Therapy Pipeline Insight 2023 report by DelveInsight offers a thorough global overview of AAV Vectors in Gene Therapy treatments, including those already in the market, as well as those in different phases of clinical development. Prominent pharmaceutical firms are dedicated to progressing the AAV Vectors in Gene Therapy pipeline landscape and fostering the potential growth of AAV Vectors in Gene Therapy therapeutic advancements.

 

Key Takeaways from the AAV Vectors in Gene Therapy Pipeline Report

• DelveInsight’s AAV Vectors in Gene Therapy pipeline report depicts a robust space with 70+ active players working to develop 235+ pipeline therapies for AAV Vectors in Gene Therapy treatment.
• The leading companies working in the AAV Vectors in Gene Therapy 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, 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, Spark 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, and others.
• Promising AAV Vectors in Gene Therapy Pipeline Therapies in the various stages of development include SB-525 (PF-07055480), Valoctocogene Roxaparvovec, AAV – CNGB3, AAV2/5-RPGR, BMN 307, GC301, AAV2hAQP1, and others.
• On March 2023, MeiraGTx UK II Ltd announced a study of Phase 1 & 2 Clinical Trials for AAV-CNGB3. A clinical trial of AAV – CNGB3 retinal gene therapy for patients with achromatopsia.
• On June 2023, Pfizer announced a study of Phase 2 Clinical Trials for SB-525 (PF-07055480). 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).
• On July 2023, BioMarin Pharmaceuticals announced a study of Phase 1 & 2 Clinical Trials for Dose 1 of BMN 331. This is a Phase 1/2, single-arm, open-label, dose-escalation and dose-expansion study of BMN 331 for the treatment of hereditary angioedema (HAE) due to C1 Esterase Inhibitor (C1-INH) protein deficiency. The study drug BMN 331is identified as AAV5 hSERPING1, an adeno-associated virus (AAV5)-based gene therapy vector that expresses wild-type human C1 Esterase Inhibitor (hC1-INH), under the control of a liver-selective promoter, and is being developed for the treatment of HAE with C1-INH deficiency.

 

Request a sample and discover the recent advances in AAV Vectors in Gene Therapy Treatment Drugs @ AAV Vectors in Gene Therapy Pipeline Report

 

The AAV Vectors in Gene Therapy pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage AAV Vectors in Gene Therapy drugs, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the AAV Vectors in Gene Therapy clinical trial landscape.

 

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.

 

Find out more about AAV Vectors in Gene Therapy Treatment Drugs @ Drugs for AAV Vectors in Gene Therapy Treatment

 

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

The AAV Vectors in Gene Therapy pipeline report proffers an integral view of the AAV Vectors in Gene Therapy emerging novel therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration.

 

Learn more about the emerging AAV Vectors in Gene Therapy Pipeline Therapies @ AAV Vectors in Gene Therapy Clinical Trials Assessment

 

Scope of the AAV Vectors in Gene Therapy Pipeline Report

• Coverage- Global
• Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
• Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III
• 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, 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, Spark 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, and others.
• Pipeline Therapies- SB-525 (PF-07055480), Valoctocogene Roxaparvovec, AAV – CNGB3, AAV2/5-RPGR, BMN 307, GC301, AAV2hAQP1, and others.

 

Dive deep into rich insights for new drugs for AAV Vectors in Gene Therapy Treatment, Visit @ AAV Vectors in Gene Therapy Market Drivers and Barriers, and Future Perspective

 

Table of Content

1. Introduction
2. Executive Summary
3. AAV vectors in gene therapy: Overview
4. Pipeline Therapeutics
5. 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

 

For further information on the AAV Vectors in Gene Therapy Pipeline therapeutics, reach out to AAV Vectors in Gene Therapy Unmet Needs and Analyst Views

 

About Us

DelveInsight is a leading healthcare-focused market research and consulting firm that provides clients with high-quality market intelligence and analysis to support informed business decisions. With a team of experienced industry experts and a deep understanding of the life sciences and healthcare sectors, we offer customized research solutions and insights to clients across the globe. Connect with us to get high-quality, accurate, and real-time intelligence to stay ahead of the growth curve.

Media Contact
Company Name: DelveInsight Business Research LLP
Contact Person: Yash Bhardwaj
Email: Send Email
Phone: 9193216187
Address:304 S. Jones Blvd #2432
City: Las Vegas
State: NV
Country: United States
Website: https://www.delveinsight.com/consulting