Adeno-associated Virus Gene Therapy

Adeno-associated Virus Gene Therapy

Due to an increase in the aptitude for gene therapy, the advances in biotechnology, and increased incidences of genetic disorders and their corresponding understanding of them, the Adeno-Associated Virus (AAV) Gene Therapy Market is growing at an unprecedented pace. AAV vectors happen to be one of the most promising delivery vehicles for gene therapies due to their low immunogenicity, bi-determination ability to infect cells, and the ability to yield stable long-term gene expression in the host These vectors have been used in the recent past for the treatment of many genetic illnesses, like neurological, ophthalmological and musculoskeletal diseases.

The Adeno-associated Virus Gene Therapy Market is expected to grow at a strong CAGR of 39% during the forecast period owing to the increasing prevalence of neurological diseases. Neurological disorders such as Parkinson's disease, Alzheimer's disease, amyotrophic lateral sclerosis (ALS), and spinal muscular atrophy (SMA) are becoming increasingly common, creating a pressing need for effective treatments. The surge in the incidences of neurological disorders is one of the key drivers of the market. For instance, the WHO estimates that around 50 million people suffer from epilepsy globally, with nearly 10 million new cases diagnosed each year. Similarly, Alzheimer's Disease International reports that over 55 million people worldwide are living with dementia, and this number is projected to nearly double every 20 years, reaching 78 million by 2030 and 139 million by 2050.

• By application, the market is segmented into neurological disorders, musculoskeletal disorders, ophthalmologic disorders, and others. The musculoskeletal disorders segment held a significant share of the market in 2023. Musculoskeletal disorders (MSDs) consist of a wide range of conditions that affect the muscles, bones, joints, and connective tissues. According to the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), MSDs are prevalent and pose a substantial burden on healthcare systems worldwide. Worldwide, MSDs are widespread, and the United States is not an exception since approximately 50% of the adult populace suffer from MSD-related ailments that result in high health implications and productivity losses. This has, in turn, been attributed to the increase in its market share. Rising research and development activities are one of the major factors driving the market’s growth. For instance, in January 2024, GeneCo announced positive results from their Phase II clinical trial of AAV-based gene therapy for Duchenne muscular dystrophy (DMD), showing improvements in muscle strength and function.
• Based on end user, the market is segmented into hospital, clinics, and others. Hospitals held a dominant share of the market in 2023. The hospital segment plays a crucial role in the AAV (Adeno-Associated Virus) gene therapy market, serving as the primary setting for the administration of these advanced treatments. Hospitals are integral in the adoption, implementation, and delivery of AAV gene therapies due to their comprehensive medical infrastructure, specialized staff, and ability to manage complex patient care. Moreover, the initiation of several clinical trials in hospital is also driving the market growth. For instance, in Johns Hopkins Hospital commenced a large-scale clinical trial for AAV gene therapy in treating inherited retinal diseases, aiming to halt or reverse vision loss in patients.
• For a better understanding of the market adoption of the adeno-associated virus gene therapy industry, the market is analyzed based on its worldwide presence in the countries such as North America (U.S., Canada, Rest of North America), Europe (Germany, U.K., France, Spain, Italy, Rest of Europe), Asia-Pacific (China, Japan, India, Rest of Asia-Pacific), Rest of World. North America held a dominant share of the market in 2023. The North America AAV (Adeno-Associated Virus) gene therapy market is experiencing significant growth, driven by advancements in genetic research, increasing investment in biopharmaceutical companies, and a supportive regulatory environment. The market's expansion is propelled by the promise of AAV gene therapies to treat a variety of genetic disorders, including rare and previously untreatable conditions. Moreover, venture capitals firms, pharmaceutical companies, and government grants are providing the necessary financial support for clinical trials and commercialization efforts. For instance, in March 2024, RegenexBio announced the expansion of their gene therapy pipeline by initiating a Phase I/II clinical trial for an AAV-based treatment for Duchenne muscular dystrophy. This trial aims to assess the safety and efficacy of the therapy in slowing disease progression.
• Some of the major players operating in the market include Spark Therapeutics, Inc.; Novartis AG; Pfizer Inc.; UniQure N.V.; bluebird bio, Inc.; AveXis, Inc.; Ultragenyx Pharmaceutical Inc.; Freeline Therapeutics Holdings plc; LogicBio Therapeutics, Inc.; and MeiraGTx Holdings plc.