The Aav Vectors in Gene Therapy Pipeline Insight 2023 report by DelveInsight offers a thorough global overview of AAV Vectors in Gene Therapy treatments, including those already in the market, as well as those in different phases of clinical development. Prominent pharmaceutical firms are dedicated to progressing the AAV Vectors in Gene Therapy pipeline landscape and fostering the potential growth of AAV Vectors in Gene Therapy therapeutic advancements.

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.
On March 2023, MeiraGTx UK II Ltd announced a study of Phase 1 & 2 Clinical Trials for AAV-CNGB3. A clinical trial of AAV – CNGB3 retinal gene therapy for patients with achromatopsia.
On June 2023, Pfizer announced a study of Phase 2 Clinical Trials for SB-525 (PF-07055480). The purpose of the study is to evaluate the safety, tolerability and time-course profile of FVIII activity after dosing with SB-525 (PF-07055480).
On July 2023, BioMarin Pharmaceuticals announced a study of Phase 1 & 2 Clinical Trials for Dose 1 of BMN 331. This is a Phase 1/2, single-arm, open-label, dose-escalation and dose-expansion study of BMN 331 for the treatment of hereditary angioedema (HAE) due to C1 Esterase Inhibitor (C1-INH) protein deficiency. The study drug BMN 331is identified as AAV5 hSERPING1, an adeno-associated virus (AAV5)-based gene therapy vector that expresses wild-type human C1 Esterase Inhibitor (hC1-INH), under the control of a liver-selective promoter, and is being developed for the treatment of HAE with C1-INH deficiency.

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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 effective vehicle and can maintain long-term gene and protein expression following a single injection of the vector.

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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

The AAV Vectors in Gene Therapy pipeline report proffers an integral view of the AAV Vectors in Gene Therapy emerging novel therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration.

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Scope of the AAV Vectors in Gene Therapy Pipeline Report

Coverage- Global
Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
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.

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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

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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