Adeno-Associated Virus (AAV) Vectors in Gene Therapy - Pipeline Insight, 2025

This “Adeno-Associated Virus (AAV) Vectors in Gene Therapy - Pipeline Insight, 2025” report provides comprehensive insights about 180+ companies and 250+ 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

AAV Vectors in Gene Therapy: Understanding

AAV Vectors in Gene Therapy: Overview

Adeno-Associated Virus (AAV) vectors are widely regarded as one of the most effective delivery systems in gene therapy due to their ability to accurately deliver therapeutic genes into specific target cells while triggering minimal immune response. Naturally occurring and non-pathogenic in humans, AAVs are considered safe since they have not been associated with any human diseases. This safety profile makes them especially well-suited for in vivo gene delivery, where long-term or stable gene expression is required. AAV vectors can persist in cells, helping sustain gene activity over time.

Different AAV serotypes offer unique tissue-targeting properties, allowing therapies to be tailored for specific medical needs. For instance, AAV9 is capable of crossing the blood-brain barrier, making it an ideal candidate for treating central nervous system disorders, while AAV2 has proven effective in targeting eye tissues. This adaptability allows AAV vectors to be used in treating various genetic diseases, including conditions like hemophilia, spinal muscular atrophy (SMA), and inherited retinal disorders. Groundbreaking AAV-based treatments such as Luxturna and Zolgensma have received regulatory approval for rare genetic conditions, marking significant advancements in the field of gene therapy.

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.

“AAV Vectors in Gene Therapy - Pipeline Insight, 2025" report outlays comprehensive insights of present scenario and growth prospects across the mechanism of action. A detailed picture of the AAV Vectors in Gene Therapy pipeline landscape is provided which includes the disease overview and AAV Vectors in Gene Therapy treatment guidelines. The assessment part of the report embraces, in depth 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, 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 AAV Vectors in Gene Therapy R&D. The therapies under development are focused on novel approaches to treat/improve AAV Vectors in Gene Therapy.

AAV Vectors in Gene Therapy Emerging Drugs Chapters

This segment of the AAV Vectors in Gene Therapy report encloses its detailed analysis of various drugs in different stages of clinical development, including Phase III, II, 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.

AAV Vectors in Gene Therapy Emerging Drugs

DTX401: Ultragenyx Pharmaceutical

DTX401 is an investigational AAV8 gene therapy designed to deliver stable expression and activity of G6Pase-α under control of the native promoter. DTX401 is administered as a single intravenous infusion and has been shown in preclinical studies to improve G6Pase-α activity and reduce hepatic glycogen levels, a well-described biomarker of disease progression. In a Phase I/II clinical study, all nine patients showed a clinical response, with significant reductions in the need for cornstarch and improvements in glucose control and other metabolic parameters compared to baseline. The drug is currently being evaluated in Phase III clinical trial to treat patients with Glycogen storage disease type I.

SRP-9003: Sarepta Therapeutics

SRP-9003 is in development for the treatment of LGMD2E (also known as beta-sarcoglycanopathy and LGMDR4), a devastating monogenic neuromuscular disease caused by a lack of beta-sarcoglycan (beta-SG) proteins. SRP-9003 is a gene therapy construct that transduces skeletal and cardiac muscle, delivering a gene that codes for the full-length beta-SG protein, the absence of which is the sole cause of the progressive degeneration and a shortened lifespan characterized by the disease.

GNT-0003: Genethon

GNT-0003 combines normal copies of the UGT1A1 gene coding for the bilirubin metabolizing enzyme with an AAV vector. The gene therapy, administered intravenously, was designed by Genethon’s Immunology and Gene Therapy of Liver Diseases team, led by Dr. Giuseppe Ronzitti. GNT0003 is a genetically modified recombinant (r) viral vector composed of the AAV8 viral capsid carrying the UGT1A1 transgene which aims to correct the dysfunction of the mutated gene by achieving durable expression of a functional copy of the affected gene. The drug is currently in Phase II stage of development for the treatment of Crigler-Najjar syndrome.

RP-A501: Rocket Pharmaceuticals

RP-A501 is Rocket’s investigational gene therapy product for the treatment of Danon disease and the first gene therapy for a cardiovascular condition to demonstrate safety and efficacy in clinical studies. Danon disease is caused by mutations in the LAMP2 gene. RP-A501 consists of a recombinant adeno-associated serotype 9 (AAV9) capsid containing a full-length, wild-type version of the human LAMP2B transgene (AAV9.LAMP2B) which, when inserted into heart cells harboring mutations in the endogenous LAMP2 gene, has the potential to fully restore cardiac function at its root. RP-A501 represents a single dose treatment and is administered as an intravenous infusion. In preclinical and clinical studies, AAV9.LAMP2B has been shown to target cardiac cells (cardiomyocytes) and deliver the functional LAMP2B gene to heart tissue, which ultimately leads to improved cardiac structure and function in patients.

HG202: HuidaGene Therapeutics

HG202 is a novel CRISPR/Cas13 RNA-targeting therapy packaged in a single adeno-associated viral (AAV) vector independently-developed by HuidaGene's proprietary HG-PRECISE platform to target and knockdown the expression of VEGF-A mRNA within the local retina. This HG202 therapy is designed to treat nAMD patients who have developed resistance to anti-VEGF treatment as well as patients who are responsive to current standard anti-VEGF therapies. HG202 efficiently inhibits choroidal neovascularization better than existing therapies such as Aflibercept antibodies in head-to-head preclinical comparison studies. HG202 is currently being evaluated in a Phase I trial for the treatment of neovascular Age-related Macular Degeneration (nAMD).

RTX 015: Ray Therapeutics

RTX 015, also known as RAY-001, is a gene therapy that utilizes an adeno-associated virus (AAV) vector. It is being developed by Ray Therapeutics specifically for the treatment of retinitis pigmentosa, a degenerative eye disease that leads to vision loss. The therapy employs optogenetics, which involves the introduction of a gene that encodes a light-sensitive protein into retinal cells, thereby enabling these cells to respond to light and potentially restore vision in individuals with damaged photoreceptors.

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.

ZM-01: Zhongmou Therapeutics

ZM-01 is a novel gene therapy developed by Zhongmou Therapeutics, specifically designed for treating retinitis pigmentosa (RP), a hereditary retinal disease that leads to progressive vision loss and can result in legal blindness. This therapy utilizes an adeno-associated virus (AAV) vector to deliver genetic material aimed at addressing the underlying causes of RP, regardless of the specific mutations involved.

Further product details are provided in the report…….

AAV Vectors in Gene Therapy: Therapeutic Assessment

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

Major Players in AAV Vectors in Gene Therapy

There are approx. 180+ key companies which are developing the therapies for AAV Vectors in Gene Therapy. The companies which have their AAV Vectors in Gene Therapy drug candidates in the most advanced stage, i.e. Phase III include Ultragenyx Pharmaceutical.

Phases

The report covers around 250+ 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

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

• Intra-articular
• Intraocular
• Intrathecal
• Intravenous
• Oral
• Parenteral
• Subcutaneous
• Topical
• Transdermal

Molecule Type

Products have been categorized under various Molecule types such as

• Oligonucleotide
• Peptide
• Small molecule

Product Type

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

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 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 AAV Vectors in Gene Therapy drugs.

AAV Vectors in Gene Therapy Report Insights

• AAV Vectors in Gene Therapy Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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 AAV Vectors in Gene Therapy drugs?
• How many 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 AAV Vectors in Gene Therapy?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the 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 AAV Vectors in Gene Therapy and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• GenSight Biologics
• Ultragenyx Pharmaceutical
• MeiraGTx
• Nanoscope Therapeutics
• REGENXBIO
• Astellas Gene therapy
• GeneCradle Therapeutics
• Asklepios BioPharmaceutical
• MeiraGTx
• 4D molecular therapeutics
• Adverum Biotechnologies
• Rocket Pharmaceuticals
• Innostellar Biotherapeutics
• Passage Bio
• Aspa therapeutics
• Solid Biosciences
• Lantu Biopharma
• Ascidian Therapeutics
• Decibel Therapeutics
• Sio Gene Therapies

Key Products

• GS010
• DTX401
• AAV5-RPGR
• MCO-010
• RGX-121
• AT132
• GC101
• AAV2-GDNF
• AAV-AQP1
• 4D-150
• ADVM-022
• RP-A501
• LX102
• PBFT02
• BBP-812
• SGT-003
• Vesemnogene lantuparvovec
• ACDN-01
• DB-OTO
• AAV9-GLB1

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