AAV Vectors in Gene Therapy Pipeline Report (Updated) | BioMarin Pharmaceutical, Gensight Biologics, PTC therapeutics, Ultragenyx Pharmaceutical, MeiraGTx, Pfizer, Biogen, and others.

AAV Vectors in Gene Therapy Pipeline Report (Updated) | BioMarin Pharmaceutical, Gensight Biologics, PTC therapeutics, Ultragenyx Pharmaceutical, MeiraGTx, Pfizer, Biogen, and others.

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 the gene therapy pipeline landscape. It covers the AAV Vectors in Gene Therapy pipeline drug profiles, including clinical and nonclinical stage products. It also covers the AAV Vectors in Gene Therapy pipeline 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 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.
  • November 2023: The National Human Genome Research Institute announced a study of Phase 1 & 2 clinical trials for AAV9-GLB1 and Sirolimus. A Phase 1/2 Study of Intravenous Gene Transfer with an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis.
  • November 2023: BioMarin Pharmaceuticals announced a study of Phase 3 clinical trials for Valoctocogene Roxaparvovec. This Phase III clinical study will assess the impact of BMN 270 (compared to FVIII prophylaxis) on the number of bleeding episodes irrespective of exogenous FVIII replacement treatment in the efficacy evaluation period (EEP) (from Week 5 post-BMN 270 infusion (Study Day 33) or the end of FVIII prophylaxis plus the washout period (3 days for products of standard half-life or plasma-derived and 5 days for products of extended half-life), whichever is later, to last visit by the data cut-off for the 2-year analysis, hereafter referred to as “Post FVIII Prophylaxis to Last Visit”).

 

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 most 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

There are approx. 70+ key companies developing therapies for AAV vectors in gene therapy. The 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 Pipeline 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

 

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 Pipeline Products have been categorized under various Molecule types such as

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

 

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
  • AAV Vectors in Gene Therapy Pipeline Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
  • AAV Vectors in Gene Therapy Pipeline 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

 

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