AAV Vectors in Gene Therapy - Pipeline Insight, 2025

The “AAV Vectors in Gene Therapy- Pipeline Insight, 2025” report provides comprehensive insights about 180+ companies and 200+ 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.

Geography Covered

• Global coverage

Adeno-Associated Virus (AAV) Vectors in Gene Therapy: Understanding

Adeno-Associated Virus (AAV) Vectors in Gene Therapy: Overview

Adeno-Associated Virus (AAV) vectors are a leading delivery system in gene therapy, valued for their ability to transfer therapeutic genes into target cells with high precision and minimal immune response. Non-pathogenic and naturally present in humans, AAVs are considered safe, especially since they have not been linked to disease in humans. This makes them suitable for delivering genes directly into the body (in vivo), particularly for treatments requiring long-term or stable gene expression, as AAV vectors can integrate into cells to maintain gene expression over extended periods.

Different AAV serotypes offer specific tissue-targeting capabilities, allowing for the customization of treatments. For example, AAV9 can cross the blood-brain barrier, making it suitable for central nervous system (CNS) disorders, while other serotypes, like AAV2, have shown success in ocular applications. This versatility enables AAV vectors to address a range of diseases, including genetic disorders like hemophilia, spinal muscular atrophy (SMA), and inherited retinal diseases. Notably, AAV-based therapies such as Luxturna and Zolgensma are approved by regulatory bodies for treating rare genetic disorders, setting important milestones for gene therapy's future.

Despite their promise, AAV vectors face significant production and scalability challenges. Producing AAV vectors at clinical scale while maintaining high purity and stability is complex and costly, which contributes to the high price of AAV-based treatments. This challenge has led to innovations in manufacturing, such as the use of baculovirus-insect cell systems, which can help address scalability. However, further improvements in manufacturing processes are essential to making AAV gene therapies more accessible.

There are also safety considerations associated with AAV-based gene therapy. High doses of AAV have been linked to liver toxicity in some studies, raising questions about optimal dosing strategies. Additionally, a portion of the population has pre-existing immunity to AAV due to natural exposure, which can reduce the efficacy of AAV-mediated treatments. To overcome these issues, researchers are exploring next-generation AAV capsid designs to lower immune responses and improve tissue specificity.

Looking forward, the field is advancing rapidly, with efforts focused on optimizing AAV vectors for safety, efficacy, and specificity. Dual AAV vector systems are being developed to expand the types of genetic material that AAVs can carry, as their payload capacity is limited to about 4.7 kb. Additionally, AAVs are being paired with CRISPR-Cas9 technology for precise gene editing, enhancing the potential of AAV in treating a wider array of genetic disorders. With these innovations, AAV vectors continue to be at the forefront of gene therapy, promising new solutions for previously untreatable genetic diseases.

"Adeno-Associated Virus (AAV) Vectors in Gene Therapy- Pipeline Insight, 2025" report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Adeno-Associated Virus (AAV) Vectors in Gene Therapy pipeline landscape is provided which includes the disease overview and Adeno-Associated Virus (AAV) Vectors in Gene Therapy treatment guidelines. The assessment part of the report embraces, in depth Adeno-Associated Virus (AAV) Vectors in Gene Therapy commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Adeno-Associated Virus (AAV) Vectors in Gene Therapy collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

The companies and academics are working to assess challenges and seek opportunities that could influence Adeno-Associated Virus (AAV) Vectors in Gene Therapy R&D. The therapies under development are focused on novel approaches to treat/improve Adeno-Associated Virus (AAV) Vectors in Gene Therapy.

Adeno-Associated Virus (AAV) Vectors in Gene Therapy Emerging Drugs Chapters

This segment of the Adeno-Associated Virus (AAV) Vectors in Gene Therapy report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, II, II/III I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Adeno-Associated Virus (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® (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 β-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 β-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 β-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.

Adeno-Associated Virus (AAV) Vectors in Gene Therapy: Therapeutic Assessment

This segment of the report provides insights about the different Adeno-Associated Virus (AAV) Vectors in Gene Therapy drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Adeno-Associated Virus (AAV) Vectors in Gene Therapy

There are approx. 180+ key companies which are developing the therapies for Adeno-Associated Virus (AAV) Vectors in Gene Therapy. The companies which have their Adeno-Associated Virus (AAV) Vectors in Gene Therapy drug candidates in the most advanced stage, i.e. Registration include, Belief BioMed

Phases

The report covers around 200+ 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 Virus (AAV) Vectors in Gene Therapy 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

Molecule Type

Products have been categorized under various Molecule types such as

• Monoclonal antibody
• Small molecule
• Peptide

Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Adeno-Associated Virus (AAV) Vectors in Gene Therapy: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Adeno-Associated Virus (AAV) Vectors in Gene Therapy therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Adeno-Associated Virus (AAV) Vectors in Gene Therapy drugs.

Adeno-Associated Virus (AAV) Vectors in Gene Therapy Report Insights

• Adeno-Associated Virus (AAV) Vectors in Gene Therapy Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Adeno-Associated Virus (AAV) Vectors in Gene Therapy Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing Adeno-Associated Virus (AAV) Vectors in Gene Therapy drugs?
• How many Adeno-Associated Virus (AAV) Vectors in Gene Therapy drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Adeno-Associated Virus (AAV) Vectors in Gene Therapy?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Adeno-Associated Virus (AAV) Vectors in Gene Therapy therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Adeno-Associated Virus (AAV) Vectors in Gene Therapy and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• 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

Key Products

• BBM-H901
• GS010
• AAV-CNGA3
• PBGM01
• IVB102
• DTX401
• AAV5-RPGR
• BIIB112
• RGX-314
• AGTC-501
• GNT-0003
• 4D-150
• ADVM-022
• RP-A501
• LX102
• BBP-812
• ECUR-506
• AVB-101
• SRP-6004
• RTX 015
• GNSC-001
• Research Program: Wet Age-related macular degeneration
• DiNA-001
• TN-201
• IVB103
• JAG101
• NFS- 04

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