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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

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