Adeno-Associated Virus (AAV) Vectors in Gene Therapy Clinical Trials and Studies 2025: EMA, PDMA, FDA Approvals, Mechanism of Action, ROA, NDA, IND, and Companies

DelveInsight's, "Adeno-Associated Virus (AAV) Vectors in Gene Therapy Pipeline Insight, 2025" report provides comprehensive insights about 180+ companies and 200+ pipeline drugs in Adeno-Associated Virus (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.

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Key Takeaways from the Adeno-Associated Virus (AAV) Vectors in Gene Therapy Pipeline Report

* In March 2025, CSL Behring announced a study drug is identified as AAV5-hFIXco-Padua (AMT- 061). AMT-061 is a recombinant adeno-associated viral vector of serotype 5 (AAV5) containing the Padua variant of a codon-optimized human FIX complementary deoxyribonucleic acid (cDNA) under the control of a liver-specific promoter. The pharmaceutical form of AMT-061 is a solution for intravenous infusion administered at a dose of 2 x 10^13 gc/kg.
* In March 2025, UniQure Biopharma B.V. announced a second study of AMT-130 in patients with early manifest HD and is designed as part of an integrated two-study phase I/II program under a single data safety monitoring board (DSMB) with staggered enrollment based upon continued demonstration of safety of AMT-130 administration. Cohort 3 participants will receive either high or low dose (1:1 randomization). Participants enrolled in Cohort 3 will also receive an immunosuppression regimen consisting of dexamethasone, sirolimus, and rituximab.
* In March 2025, Pfizer announced a study to establish baseline prospective efficacy data of current FIX prophylaxis replacement therapy in the usual care setting of hemophilia B subjects, who are negative for nAb to AAV-Spark100, prior to the Phase 3 gene therapy study. To establish baseline prospective efficacy data of current FVIII prophylaxis replacement therapy in the usual care setting of hemophilia A subjects, who are negative for nAb to AAV6, prior to the Phase 3 gene therapy study.
* In March 2025, Adverum Biotechnologies, Inc. announced a phase 2, multi-center, randomized, double-masked, parallel group study is designed to evaluate the safety, tolerability, and efficacy of a single IVT injection of ADVM-022 at one of two doses (2 times 10^11 vg/eye [2E11] or 6 times 10^10 vg/eye [6E10]) accompanied by one of four prophylactic corticosteroid treatment regimens.
* DelveInsight's Adeno-Associated Virus (AAV) Vectors in Gene Therapy pipeline report depicts a robust space with 180+ active players working to develop 200+ pipeline therapies for Adeno-Associated Virus (AAV) Vectors in Gene Therapy treatment.
* The leading Adeno-Associated Virus (AAV) Vectors in Gene Therapy Companies such as Belief BioMed, Gensight Biologics, Johnson & Johnson Innovative Medicine/MeiraGTx, Passage Bio, InnoVec Biotherapeutics, Ultragenyx Pharmaceutical, MeiraGTx, Biogen, REGENXBIO, Beacon Therapeutics, Genethon, 4D Molecular Therapeutics, Adverum Biotechnologies, Rocket Pharmaceuticals, Innostellar Biotherapeutics, Aspa Therapeutics, iECURE, AviadoBio, Sarepta Therapeutics, Ray Therapeutics, Genascence Corporation, Exegenesis Bio, DiNAQOR, Tenaya Therapeutics, InnoVec Biotherapeutics, Jaguar Gene Therapy, LLC, Neurophth and others.
* Promising Adeno-Associated Virus (AAV) Vectors in Gene Therapy Pipeline Therapies such as Valoctocogene Roxaparvovec, AAV - CNGB3, SB-525 (PF-07055480), AAV2/5-RPGR, BMN 307, GC301, rAAV-Olig001-ASPA , and others.

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AAV vectors in Gene Therapy Emerging Drugs

* BBM-H901: Belief BioMed

BBM-H901 is indicated for prophylactic treatment of bleeding in adults with hemophilia B. BBM-H901 is designed to restore the production of factor IX (FIX) the blood-clotting protein that is faulty or missing in people with hemophilia B by delivering a functional copy of the F9 gene to liver cells, which are the main producers of clotting factors in the body. Gene therapy components are delivered to liver cells by a modified, harmless adeno-associated virus (AAV) that works as a vehicle. The drug is currently being registered for the treatment of hemophilia B.

* GS010: Gensight Biologics

LUMEVOQ Registered (GS010; lenadogene nolparvovec) targets Leber Hereditary Optic Neuropathy (LHON) by leveraging a mitochondrial targeting sequence (MTS) proprietary technology platform, arising from research conducted at the Institut de la Vision in Paris, which, when associated with the gene of interest, allows the platform to specifically address defects inside the mitochondria using an AAV vector (Adeno-Associated Virus). The gene of interest is transferred into the cell to be expressed and produces the functional protein, which will then be shuttled to the mitochondria through specific nucleotidic sequences in order to restore the missing or deficient mitochondrial function. "LUMEVOQ" was accepted as the invented name for GS010 (lenadogene nolparvovec) by the European Medicines Agency (EMA) in October 2018. Currently the drug is in Phase III stage of its development for the treatment of Leber Hereditary Optic Neuropathy.

* AAV-CNGA3: Johnson & Johnson Innovative Medicine/ MeiraGTx

AAV-CNGA3, a gene therapy treatment designed to restore cone function, is delivered to the cone receptors at the back of the eye via subretinal injection. It was designed with a synthetic promoter associated with strong gene expression to account for the larger amount of protein needed to restore cone function in achromatopsia (ACHM) patients with a CNGA3 gene mutation. AAV-CNGA3 was granted Orphan Drug designation by the FDA and EMA, as well as Rare Pediatric Disease designation and Fast Track designation by the FDA, for the treatment of ACHM caused by mutations in the CNGA3 gene. According to company's pipeline the drug is being evaluated in a Phase II trial for the treatment of achromatopsia.

* PBGM01: Passage Bio

PBGM01 is an AAV-delivery gene therapy currently being developed for the treatment of infantile GM1, in which patients have mutations in the GLB1 gene causing little or no residual -gal enzyme activity and subsequent neurodegeneration. PBGM01 utilizes a next-generation AAVhu68 capsid administered through the cisterna magna to deliver a functional GLB1 gene encoding -gal to the brain and peripheral tissues. By reducing the accumulation of GM1 gangliosides, PBGM01 has the potential to reverse neuronal toxicity, thereby restoring developmental potential. In preclinical models, PBGM01 has demonstrated broad brain distribution and high levels of expression of the -gal enzyme in both the CNS and critical peripheral organs, suggesting potential treatment for both the CNS and peripheral manifestations of GM1. The drug is currently being evaluated in Phase I/II stage of development to treat Gangliosidosis.

* IVB102: InnoVec Biotherapeutics

IVB102 is a drug for the treatment of X-linked retinoschisis (XLRS) developed based on a new vector independently developed by Innovecon. Preclinical data showed that the visual electrophysiological signals of model animals treated with IVB102 can be restored to a level comparable to that of wild-type animals, with the potential of being 'best in class'. The positive feedback received from the FDA on IVB102 proves the FDA's recognition of IVB102 and also reflects the urgent need for drugs from regulators and patients. Innovecon is rapidly advancing the clinical research of IVB102. The drug is currently being evaluated in a Phase I trial for the treatment of XLRS.

The Adeno-Associated Virus (AAV) Vectors in Gene Therapy Pipeline Report Provides Insights into

* The report provides detailed insights about companies that are developing therapies for the treatment of Adeno-Associated Virus (AAV) Vectors in Gene Therapy with aggregate therapies developed by each company for the same.
* It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Adeno-Associated Virus (AAV) Vectors in Gene Therapy Treatment.
* Adeno-Associated Virus (AAV) Vectors in Gene Therapy Companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
* Adeno-Associated Virus (AAV) Vectors in Gene Therapy Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.
* Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Adeno-Associated Virus (AAV) Vectors in Gene Therapy market

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

Belief BioMed, Gensight Biologics, Johnson & Johnson Innovative Medicine/MeiraGTx, Passage Bio, InnoVec Biotherapeutics, Ultragenyx Pharmaceutical, MeiraGTx, Biogen, REGENXBIO, Beacon Therapeutics, Genethon, 4D Molecular Therapeutics, Adverum Biotechnologies, Rocket Pharmaceuticals, Innostellar Biotherapeutics, Aspa Therapeutics, iECURE, AviadoBio, Sarepta Therapeutics, Ray Therapeutics, Genascence Corporation, Exegenesis Bio, DiNAQOR, Tenaya Therapeutics, InnoVec Biotherapeutics, Jaguar Gene Therapy, LLC, Neurophth 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

* Intravenous
* Subcutaneous
* Oral
* Intramuscular

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

* Monoclonal antibody
* Small molecule
* Peptide

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Scope of the Adeno-Associated Virus (AAV) Vectors in Gene Therapy Pipeline Report

* Coverage- Global
* Adeno-Associated Virus (AAV) Vectors in Gene Therapy Companies- Belief BioMed, Gensight Biologics, Johnson & Johnson Innovative Medicine/MeiraGTx, Passage Bio, InnoVec Biotherapeutics, Ultragenyx Pharmaceutical, MeiraGTx, Biogen, REGENXBIO, Beacon Therapeutics, Genethon, 4D Molecular Therapeutics, Adverum Biotechnologies, Rocket Pharmaceuticals, Innostellar Biotherapeutics, Aspa Therapeutics, iECURE, AviadoBio, Sarepta Therapeutics, Ray Therapeutics, Genascence Corporation, Exegenesis Bio, DiNAQOR, Tenaya Therapeutics, InnoVec Biotherapeutics, Jaguar Gene Therapy, LLC, Neurophth and others.
* Adeno-Associated Virus (AAV) 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.
* Adeno-Associated Virus (AAV) Vectors in Gene Therapy Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
* Adeno-Associated Virus (AAV) Vectors in Gene Therapy Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III

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Table of Content

* Introduction
* Executive Summary
* Adeno-Associated Virus (AAV) Vectors in Gene Therapy: Overview
* Pipeline Therapeutics
* Therapeutic Assessment
* Adeno-Associated Virus (AAV) Vectors in Gene Therapy- DelveInsight's Analytical Perspective
* Late Stage Products (Registration)
* BBM-H901: Belief BioMed
* Drug profiles in the detailed report.....
* Mid Stage Products (Phase II)
* AAV-CNGA3: Johnson & Johnson Innovative Medicine/ MeiraGTx
* Drug profiles in the detailed report.....
* Early Stage Products (Phase I)
* IVB102: InnoVec Biotherapeutics
* Drug profiles in the detailed report.....
* Preclinical and Discovery Stage Products
* Drug Name: Company Name
* Drug profiles in the detailed report.....
* Inactive Products
* Adeno-Associated Virus (AAV) Vectors in Gene Therapy Key Companies
* Adeno-Associated Virus (AAV) Vectors in Gene Therapy Key Products
* Adeno-Associated Virus (AAV) Vectors in Gene Therapy- Unmet Needs
* Adeno-Associated Virus (AAV) Vectors in Gene Therapy- Market Drivers and Barriers
* Adeno-Associated Virus (AAV) Vectors in Gene Therapy- Future Perspectives and Conclusion
* Adeno-Associated Virus (AAV) Vectors in Gene Therapy Analyst Views
* Adeno-Associated Virus (AAV) Vectors in Gene Therapy Key Companies
* Appendix

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