Adeno-Associated Virus (AAV) Vectors in Gene Therapy Pipeline, Clinical Trials, FDA Approvals, and Companies 2024 (Updated)

Adeno-Associated Virus (AAV) Vectors in Gene Therapy Pipeline, Clinical Trials, FDA Approvals, and Companies 2024 (Updated)

DelveInsight’s, “AAV Vectors in Gene Therapy Pipeline Insight, 2024″ report provides comprehensive insights about 70+ companies and 235+ pipeline drugs in AAV vectors in gene therapy pipeline landscape. It covers the 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 AAV 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, 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, 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.
  • January 2024:- Spark Therapeutics Inc.- A Phase 1/2 Safety Study in Subjects With CHM (Choroideremia) Gene Mutations Using an Adeno-Associated Virus Serotype 2 Vector to Deliver the Normal Human CHM Gene [AAV2-hCHM] to the Retina. This clinical study evaluates the safety and tolerability of AAV2-hCHM in participants with Choroideremia gene mutations. The primary objective is to evaluate the safety and tolerability of subretinal administration of AAV2-hCHM, in an inter-subject group dose escalation in individuals with choroideremia, based on a comprehensive clinical monitoring plan. The secondary objectives are to define the dose of AAV2-hCHM required to achieve stable, or improved, visual function/functional vision and to assess development of immune responses to adeno-associated virus vector, serotype 2 (AAV2) and Rab escort protein 1 (REP-1).

 

Request a sample and discover the recent advances in AAV vectors in gene therapy @ AAV Vectors in Gene Therapy Pipeline Report

 

Adeno-Associated Virus (AAV) Vectors in Gene Therapy Overview

Adeno-Associated Virus (AAV) vectors have emerged as a cornerstone in the field of gene therapy, revolutionizing the potential for treating a wide range of genetic and acquired diseases. These vectors, derived from naturally occurring non-pathogenic viruses, have gained prominence due to their safety profile, low immunogenicity, and ability to deliver therapeutic genes into target cells with remarkable precision.

 

AAV vectors in Gene Therapy Emerging Drugs

  • 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

 

Learn more about the AAV vectors for gene therapy in clinical trials @ AAV Vectors for Gene Therapy Drugs

 

AAV Vectors in Gene Therapy: Therapeutic Assessment

There are approx. 70+ key Adeno-Associated Virus (AAV) Vectors in Gene Therapy companies developing Adeno-Associated Virus (AAV) Vectors in Gene Therapy therapies. The Adeno-Associated Virus (AAV) Vectors in Gene Therapy companies that have their AAV vectors in gene therapy drug candidates in the most advanced stage, i.e. Preregistration include BioMarin Pharmaceutical.

 

DelveInsight’s Adeno-Associated Vector Report covers around 235+ products under different phases of clinical development like

  • Late stage products (Phase III)
  • Mid-stage products (Phase II)
  • Early-stage product (Phase I) along with the details of
  • Pre-clinical and Discovery stage candidates
  • Discontinued & Inactive candidates
  • Route of Administration

 

Discover more about AAV vectors in gene therapy in development @ AAV Vectors in Gene Therapy Clinical Trials

 

Adeno-Associated Vector Companies

BioMarin Pharmaceutical, Gensight Biologics, PTC therapeutics, Ultragenyx Pharmaceutical, MeiraGTx, Pfizer, Biogen, uniQure, 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, and others

 

Adeno-Associated Vector pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

  • Intracerebral
  • Intracerebroventricular
  • Intracisternal
  • Intramuscular
  • Intraocular
  • Intrapancreatic
  • Intrastriatal
  • Intrathecal
  • Intravenous
  • Intravitreous
  • Introvitinreous
  • Oral
  • Parenteral
  • Retinal
  • Intra-arterial
  • Molecule Type

 

Adeno-Associated Vector Products have been categorized under various Molecule types such as

  • Monoclonal Antibody
  • Peptides
  • Polymer
  • Small molecule
  • Gene therapy
  • Product Type

 

To know more about AAV vectors in gene therapy, visit @ AAV Vector Technology Segmentation

 

Scope of the Adeno-Associated Virus (AAV) 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.

 

For further information on the AAV vectors in gene therapy pipeline therapeutics, reach out @ AAV Vector Gene Therapy Products Development

 

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

 

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: 09650213330
Address:304 S. Jones Blvd #2432
City: Las Vegas
State: NV
Country: United States
Website: https://www.delveinsight.com/