DelveInsight’s, “AAV vectors in gene therapy Pipeline Insight 2023″ report provides comprehensive insights about 70+ companies and 235+ pipeline drugs in AAV vectors in gene therapy pipeline landscape. It covers the Adeno-Associated Virus (AAV) Vectors in Gene Therapy pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

 

Key Takeaways from the Adeno-Associated Virus Vectors in Gene Therapy Pipeline Report

• DelveInsight’s Adeno-Associated Virus Vectors in Gene Therapy pipeline report depicts a robust space with 70+ active players working to develop 235+ pipeline therapies for Adeno-Associated Virus Vectors in Gene Therapy treatment.
• The leading companies working in the Adeno-Associated Virus 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, and others.
• Promising Adeno-Associated Virus Vectors in Gene Therapy Pipeline Therapies in the various stages of development include Valoctocogene Roxaparvovec, AAV – CNGB3, SB-525 (PF-07055480), AAV2/5-RPGR, BMN 307, GC301, rAAV-Olig001-ASPA, and others.
• September 2023: Pfizer announced a study of phase 3 clinical trials for PF-07055480 (giroctocogene fitelparovec): Recombinant AAV2/6 Human Factor VIII Gene Therapy. C3731003 is a pivotal Phase 3 study to evaluate the clinical efficacy and safety of a single IV infusion of PF-07055480 / giroctocogene fitelparvovec (Recombinant AAV2/6 Human Factor VIII Gene Therapy) in adult male participants with moderately severe or severe hemophilia A (FVIII:C≤1%) for the study duration of 5 years. The study will enroll eligible participants who have been followed on routine prophylaxis with FVIII products in the Lead-In study C0371004.
• September 2023: Aspa Therapeutics announced a study of phase 1 & 2 clinical trials for AAV9 BBP-812. The main objective of this trial is to evaluate the safety, tolerability, and pharmacodynamic activity of BBP-812, an investigational AAV9-based gene therapy, in pediatric participants with Canavan disease.

 

Request a sample and discover the recent advances in Adeno-Associated Virus Vectors in Gene Therapy Treatment Drugs @ Adeno-Associated Virus Vectors in Gene Therapy Pipeline Report

 

 

In the Adeno-Associated Virus Vectors in Gene Therapy pipeline report, detailed description of the drug is given which includes mechanism of action of the drug, Adeno-Associated Virus Vectors in Gene Therapy clinical trials studies, NDA approvals (if any), and product development activities comprising the technology, AAV vectors in gene therapy collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

 

Adeno-Associated Virus 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. AAV vectors are the leading viral vectors for gene delivery to treat a variety of human diseases.

 

Find out more about Adeno-Associated Virus Vectors in Gene Therapy Therapeutics Assessment @ Adeno-Associated Virus Vectors in Gene Therapy Preclinical and Discovery Stage Products

 

Adeno-Associated Virus 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

 

Adeno-Associated Virus Vectors in Gene Therapy Pipeline Therapeutics Assessment

There are approx. 70+ key companies which are developing the Adeno-Associated Virus Vectors in Gene Therapy therapies. The Adeno-Associated Virus 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.

 

Learn more about the emerging Adeno-Associated Virus Vectors in Gene Therapy Pipeline Therapies @ Adeno-Associated Virus Vectors in Gene Therapy Clinical Trials Assessment

 

Scope of the Adeno-Associated Virus Vectors in Gene Therapy Pipeline Report

• Coverage- Global
• Adeno-Associated Virus Vectors in Gene Therapy Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
• Adeno-Associated Virus Vectors in Gene Therapy Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III
• Adeno-Associated Virus 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, and others.
• Adeno-Associated Virus Vectors in Gene Therapy Pipeline Therapies- Valoctocogene Roxaparvovec, AAV – CNGB3, SB-525 (PF-07055480), AAV2/5-RPGR, BMN 307, GC301, rAAV-Olig001-ASPA, and others.

 

Dive deep into rich insights for new drugs for Adeno-Associated Virus Vectors in Gene Therapy treatment, Visit @ Adeno-Associated Virus 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 Adeno-Associated Virus Vectors in Gene Therapy pipeline therapeutics, reach out to Adeno-Associated Virus 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/ci-tracking