(MENAFN- GetNews)
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
Introduction
Executive Summary
AAV vectors in gene therapy: Overview
Pipeline Therapeutics
Therapeutic Assessment
Late Stage Products (Preregistration)
Valoctocogene roxaparvovec: BioMarin Pharmaceutical
Drug profiles in the detailed report.....
Late Stage Products (Phase III)
DTX401: Ultragenyx Pharmaceutical
Drug profiles in the detailed report.....
Mid Stage Products (Phase II)
GT 005: Gyroscope Therapeutics
Drug profiles in the detailed report.....
Early stage products (Phase I/II)
Drug profiles in the detailed report.....
Early Stage Products (Phase I)
HMI-203: Homology Medicine
Drug profiles in the detailed report.....
Preclinical stage products
AXV101: Axovia Therapeutics
Drug profiles in the detailed report.....
Discovery stage products
DINA-002: DiNAQOR
Drug profiles in the detailed report.....
Inactive Products
AAV vectors in gene therapy Key Companies
AAV vectors in gene therapy Key Products
AAV vectors in gene therapy- Unmet Needs
AAV vectors in gene therapy- Market Drivers and Barriers
AAV vectors in gene therapy- Future Perspectives and Conclusion
AAV vectors in gene therapy Analyst Views
AAV vectors in gene therapy Key Companies
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.
MENAFN06052024003238003268ID1108180416