Adeno-associated Virus Gene Therapy Market Size to Reach USD 107.2 Billion by 2035, Impelled by Advancements in Personalized Medicine.

The 7 major adeno-associated virus gene therapy market reached a value of USD 2.7 Billion in 2024. Looking forward, IMARC Group expects the 7MM to reach USD 107.2 Billion by 2035, exhibiting a growth rate (CAGR) of 40.03% during 2025-2035. The market is driven by ongoing developments in the fields of genetics and biotechnology, such as using capsid engineering to increase vector tropism and altering AAV transduction efficiency by enhancing the transgene cassette.

Advances in Early Detection and Diagnostic Technologies: Driving the Adeno-associated Virus Gene Therapy Market

The rapid evolution of early detection and diagnostic technologies has been a key driver in the expansion of the adeno-associated virus (AAV) gene therapy market. AAV-based gene therapies have gained significant traction due to their ability to deliver precise genetic modifications with long-term therapeutic benefits. However, the success of these therapies heavily relies on the ability to accurately diagnose genetic disorders at an early stage. Advances in polymerase chain reaction (PCR), next-generation sequencing, and bioinformatics have revolutionized the early detection of genetic mutations, enabling timely interventions and personalized treatment strategies. Improved diagnostic tools have also facilitated better patient stratification, ensuring that AAV-based therapies are administered to the right candidates. Liquid biopsies, for example, provide non-invasive methods to detect genetic abnormalities with high sensitivity and specificity. Additionally, breakthroughs in biomarkers and molecular diagnostics have enhanced the ability to monitor disease progression and treatment efficacy in real time. These advancements correct patient outcomes and increase the adoption of gene therapies in clinical settings. Regulatory agencies have recognized the importance of robust diagnostic frameworks in supporting gene therapy approvals. The integration of artificial intelligence and machine learning in diagnostics has further accelerated the identification of eligible patients, optimizing clinical trial designs and expediting market growth.

Request a PDF Sample Report: https://www.imarcgroup.com/adeno-associated-virus-gene-therapy-market/requestsample

Development of Novel Therapies and Pharmacological Treatments: Contributing to Market Expansion

The extension of novel therapies and pharmacological medications has significantly contributed to the expansion of the adeno-associated virus (AAV) gene therapy industry. Advances in biotechnology, molecular medicine, and genetic engineering have enabled the creation of innovative AAV vectors with enhanced efficiency, specificity, and safety. These improvements have broadened the therapeutic landscape, allowing for the treatment of previously incurable diseases such as spinal muscular atrophy (SMA), hemophilia, and retinal dystrophies. Pharmaceutical companies and research institutions are actively investing in the development of next-generation AAV therapies that address challenges such as immune responses, vector toxicity, and gene expression control. Strategies like capsid engineering, immune modulation, and optimized gene delivery techniques have led to improved clinical outcomes and expanded the potential of AAV-based treatments. Furthermore, combination therapies, where AAV gene therapy is paired with pharmacological agents, have shown promising results in enhancing treatment efficacy and durability. The regulatory landscape has also evolved to accommodate these advancements, with accelerated approvals and breakthrough therapy designations fostering market growth. The increasing number of clinical trials and positive regulatory feedback have strengthened investor confidence, leading to greater funding and commercialization efforts. As scientific innovation continues, the integration of novel pharmacological treatments with AAV gene therapy is expected to drive market expansion, offering new hope for patients with rare and complex genetic disorders. The ongoing R&D efforts in this field will likely lead to more effective, safer, and accessible gene therapies, further solidifying AAV’s role in the future of precision medicine.

Buy Full Report: https://www.imarcgroup.com/checkout?id=6765&method=809

Marketed Therapies in Adeno-associated Virus Gene Therapy Market

Luxturna (voretigene neparvovec-rzyl): Spark Therapeutics, Inc.

Luxturna (voretigene neparvovec-rzyl) provides a manuscript of the RPE65 gene to retinal pigment epithelial (RPE) cells in the eye applying an adeno-associated virus (AAV) vector. This efficiently replaces a faulty RPE65 gene, which causes vision impairment in patients with hereditary retinal dystrophy caused by biallelic RPE65 mutations. It enables RPE cells to manufacture the protein required to restart the visual cycle and enhance vision.

Zolgensma (onasemnogene abeparvovec): Novartis Gene Therapies, Inc.

Zolgensma (onasemnogene abeparvovec) is an adeno-associated virus (AAV) vector-based gene therapy used for dealing the pediatric patients below the age of two with spinal muscular atrophy (SMA) and bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. Zolgensma is the first gene treatment for SMA that exchanges the function of the missing or inactive SMN1 gene by delivering a new, functional SMN gene to the body’s cells in a single infusion. The therapy uses an adeno-associated virus vector to carry the gene.

Glybera (alipogene tiparvovec): uniQure

Glybera (alipogene tiparvovec) uses an adeno-associated virus vector to transfer a copy of the human lipoprotein lipase (LPL) gene directly into muscle cells. The medicine effectively replaces the faulty LPL gene in individuals with lipoprotein lipase deficiency, allowing them to produce the enzyme required to break down fats in the blood, thus decreasing triglyceride levels and preventing pancreatitis. This is delivered with a single injection into the leg muscle.

Emerging Therapies in Adeno-associated Virus Gene Therapy Market

BBM-H901: Shanghai Belief-Delivery BioMed Co., Ltd

BBM-H901, manufactured by Shanghai Belief-Delivery BioMed Co., Ltd., is an adeno-associated virus (AAV) gene therapy that works by inserting a copy of the human factor IX (FIX) gene into the liver cells of hemophilia B patients. The medicine causes the liver to manufacture the missing clotting factor IX, reducing bleeding episodes and restoring normal blood clotting ability. Essentially, it works by a technique of gene substitution to correct the genetic abnormality that causes hemophilia B.

Valoctocogene Roxaparvovec: BioMarin Pharmaceutical

Valoctocogene Roxaparvovec, a gene therapy developed by BioMarin Pharmaceutical, uses an adeno-associated virus (AAV) serotype 5 vector to deliver a functional copy of the human Factor VIII (FVIII) gene into the liver cells of hemophilia A patients. The medicine allows the body to generate its own FVIII protein, thereby correcting the bleeding problem and restoring normal clotting function. This is accomplished using a liver-specific promoter, which ensures that the gene is largely expressed in hepatocytes, where FVIII is naturally generated.

Detailed list of emerging therapies in Adeno-associated Virus Gene Therapy is provided in the final report…

Leading Companies in the Adeno-associated Virus Gene Therapy Market:

The market research report by IMARC involves a detailed study of the competitive landscape in the market. Across the global adeno-associated virus gene therapy market, some of the leading companies are at the forefront of platforms to enhance the management of adeno-associated virus gene therapy. Some of the leading players include Spark Therapeutics, Inc., Novartis Gene Therapies, Inc., and uniQure. These companies are driving innovation in the adeno-associated virus gene therapy market through diagnostic tools, continuous research, and expanding their product offerings.

In December 2024, Novartis reported encouraging topline findings from the Phase III STEER study. This pivotal research evaluated the safety and efficacy of OAV101 IT, an experimental intrathecal onasemnogene abeparvovec (OAV101 IT), in treatment-naïve patients with spinal muscular atrophy type 2, aged two to less than 18 years. The patients were able to sit but had never walked independently. The efficacy and safety of OAV101 IT were compared to sham control, which is a process that mimics the administration of an investigational medicine but fails to deliver any active treatment.

Request for customization: https://www.imarcgroup.com/request?type=report&id=6765&flag=E

Key Players in Adeno-associated Virus Gene Therapy Market:

The key players in the Adeno-associated Virus Gene Therapy market who are in several duration of developing unique therapies are Spark Therapeutics, Inc., Novartis Gene Therapies, Inc., Rocket Pharmaceuticals Inc., Forge Biologics, Inc, REGENXBIO Inc., uniQure, Shanghai Belief-Delivery BioMed Co., Ltd, BioMarin Pharmaceutical, and Others.

Regional Analysis:

The major markets for adeno-associated virus gene therapy include the United States, Germany, France, the United Kingdom, Italy, Spain, and Japan. According to estimates by IMARC, the United States has the largest patient pool for adeno-associated virus gene therapy while also demonstrating the largest market for its treatment. This can be recognized by the rising incidences of genetic disorders, including spinal muscular atrophy, hemophilia, and retinal diseases, which have increased the demand for effective gene therapies.

Moreover, the supportive regulatory framework provided by the U.S. Food and Drug Administration (FDA) has played an important role in market expansion. Programs such as Breakthrough Therapy Designation, Fast Track, and Orphan Drug Designation have streamlined approval processes, allowing innovative AAV-based treatments to reach patients faster. Additionally, the FDA’s growing acceptance of gene therapies has encouraged more companies to enter the market.

Besides this, advancements in diagnostic technologies have improved early detection, enabling timely treatment interventions. Expanding healthcare infrastructure, increasing reimbursement policies, and growing patient awareness about gene therapy benefits are further driving market growth.

Recent Developments in Adeno-associated Virus Gene Therapy Market:

• In March 2024, Forge Biologics stated that the Medicines and Healthcare Products Regulatory Agency (MHRA), the UK’s healthcare regulatory body, had allowed Innovation Passport designation to the company’s novel AAV gene therapy program, FBX-101, allowing it to enter the Innovative Licensing and Access Pathway (ILAP). FBX-101 was developed to treat Krabbe disease, a rare neurological disease which is generally fatal in untreated patients by second year of age.
• In February 2024, BioMarin Pharmaceutical Inc. introduced that new data on ROCTAVIAN (valoctocogene roxaparvovec-rvox) will be presented at the 2024 European Association for Haemophilia and Allied Disorders (EAHAD) Congress. A Phase 2 open-label study of ROCTAVIAN found that median factor VIII (FVIII) activity at year seven remained in the mild hemophilia range (10.3 IU/dL per chromogenic assay), and the mean annualized bleeding rate for treated bleeds over the full follow-up period decreased by 96% from baseline for an adult population with severe hemophilia A in the 6x1013 vg/kg dose cohort.

Key information covered in the report.

Base Year: 2024

Historical Period: 2019-2024

Market Forecast: 2025-2035

Countries Covered

• United States
• Germany
• France
• United Kingdom
• Italy
• Spain
• Japan

Analysis Covered Across Each Country

• Historical, current, and future epidemiology scenario
• Historical, current, and future performance of the adeno-associated virus gene therapy market
• Historical, current, and future performance of various therapeutic categories in the market
• Sales of various drugs across the adeno-associated virus gene therapy market
• Reimbursement scenario in the market
• In-market and pipeline drugs

Competitive Landscape:

This report offers a comprehensive analysis of current adeno-associated virus gene therapy marketed drugs and late-stage pipeline drugs.

Ask Our Expert & Browse Full Report with TOC: https://www.imarcgroup.com/adeno-associated-virus-gene-therapy-market/toc

In-Market Drugs

• Drug Overview
• Mechanism of Action
• Regulatory Status
• Clinical Trial Results
• Drug Uptake and Market Performance

Late-Stage Pipeline Drugs

• Drug Overview
• Mechanism of Action
• Regulatory Status
• Clinical Trial Results
• Drug Uptake and Market Performance

IMARC Group Offer Other Reports:

Head and Neck Squamous Cell Carcinoma Market: The 7 major head and neck squamous cell carcinoma markets reached a value of US$ 1.0 Billion in 2023. Looking forward, IMARC Group expects the 7MM to reach US$ 2.4 Billion by 2034, exhibiting a growth rate (CAGR) of 8.16% during 2024-2034.

Fibromyalgia Market: The 7 major fibromyalgia markets reached a value of US$ 2.2 Billion in 2023. Looking forward, IMARC Group expects the 7MM to reach US$ 3.0 Billion by 2034, exhibiting a growth rate (CAGR) of 2.69% during 2024-2034.

Erectile Dysfunction Market: The 7 major erectile dysfunction markets reached a value of US$ 1.9 Billion in 2023. Looking forward, IMARC Group expects the 7MM to reach US$ 3.9 Billion by 2034, exhibiting a growth rate (CAGR) of 6.53% during 2024-2034.

Krabbe Disease Market: The 7 major Krabbe disease markets reached a value of US$ 2.5 Billion in 2023, and projected the 7MM to reach US$ 4.7 Billion by 2034, exhibiting a growth rate (CAGR) of 5.81% during 2024-2034.

Cardiomyopathy Market: The 7 major cardiomyopathy markets are expected to exhibit a CAGR of 6.07% during 2024-2034.

Hemophilia A Market: The 7 major hemophilia A markets reached a value of US$ 10,288.7 Million in 2023, and projected the 7MM to reach US$ 28,447.2 Million by 2034, exhibiting a growth rate (CAGR) of 9.69% during 2024-2034.

Contact US

IMARC Group
134 N 4th St. Brooklyn, NY 11249, USA
Email: Sales@imarcgroup.com
Tel No:(D) +91 120 433 0800
Phone Number: - +1 631 791 1145, +91-120-433-0800