Gene Therapy For Rare Disease Market Size, Share, Industry, Forecast and Outlook (2024-2031)

Global Gene Therapy For Rare Disease Market is segmented By Vector Type (Viral, Non-Viral) By Delivery Type (In-Vivo, Ex-Vivo) By Indication (Musculoskeletal, Neurological, Oncology, Metabolic, Blood-Disorders, Others) By Age Group (Child, Adults, Geriatrics) By End User (Hospitals & Clinics, Specialty Centers, Others) and By Region (North America, Europe, South America, Asia Pacific, Middle East, and Africa) – Share, Size, Outlook, and Opportunity Analysis, 2024-2031

Market Overview

The global gene therapy for rare disease market reached US$ YY million in 2023 and is expected to reach US$ YY million by 2031, growing at a CAGR of YY% during the forecast period 2024-2031. 

Rare diseases are the condtions that affects only a small proportion of population and are often chronic and life threatening. In 2022, there are approximately 470 million people suffering from one or the other type of rare disease. According to the World Health Organization, a disease can be termed as rare or orphan, when it affects not more than 65 people per 100,000 population. 

Majority of the rare diseases are caused by genetic defects to which approved therapies are not available. Only 5% of the rare disease have approved treatments and about 30% of the patients with rare diseases die in the first five years of lifetime.  In the patients with rare diseases caused by genetic defects, gene therapy is the ultimate treatment modality. This gene therapy aims at targeting and repairing the defective gene and improve the quality of life over long-term. Factors such as rise in prevalance of congential rare diseases, rising investments in gene therapy development are expected to boost the market growth.

Market Scope

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Market Dynamics: Drivers

Rising prioritization of gene therapy for rare diseases and increasing product development

In the recent years, there has been a notable surge in the prioritization of gene therapy for rare diseases, accompanied by a parallel increase in product devlopment efforts. Lack of standardized treatment regimen, lack of disease understanding, and no means to cure the disease with the existing treatment modalities, gene therapy is expected to bring revolution for curing these rare conditions in the upcoming future. Major pharmaeutical and biopharmaceutical companies have been investing in development of advanced gene therapy which once approved and enter into the market, may bring huge makeshift in market dynamics. 

For instance, on June 10, 2022 a cross sectional web-based survery published in Sage Journals said that in the next 15 years i.e., by 2036, gene therapy would be the standard option for treatment of rare diseases. The survey had also reported that CRISPR-Cas9 would be the most successful gene edting approach in replacing the defective genes.

Pfizer Inc. has been developing several gene therapies which are currently in different phases of clinical trials. For example, the company has been recruiting study subjects for phase 3 trials of its gene therapy fidanacogene elaparvovec for the treatment of hemophilia B, and has initiated phase 3 trials of giroctocogene fitelparvovec for the treatment of hemophilia A, which is expected to finish by 2028. Moreover, Pfizer has been developing gene therapies for other rare diseases such as duchenne muscular dystrophy (DMD) and transthyretin amyloidosis.

Astellas pharma inc. has been developing gene therapies for rare disorders such as x-linked myotubular myopathy, pompe disease etc. The company has received orphan drug designaation for its investigational gene therapy resamirigene bilparvovec (AT132) by the U.S. Food and Drug Administration. AT132 is designed for the treatment of x-linked myotubular myopathy which is currently in phase 3 clinical trials. 

Another major pharmaceutical company Novartis AG has been conducting phase 3 clinical trials for its gene therapy OAV101 for the treatment of spinal muscular atrophy (SMA), a rare form of muscular condition in children. The trail is being conducted across various locations globally and it anticipitated to finish by the end of 2024. Likewise, several pharma giants have been focused on introducing gene therapy for several rare conditons into the market. 

Moreover, rising prevalance of rare congential abnormalities, rising awareness regarding the importance of gene therapy are expected to boost the market growth in the forecast period.

Restraints

Although gene therapy has been revolutionizing rare disease therapeutics, several factors may restrain their adaptability. Gene therapy, unlike regular pharmacological treatment, is expensive, and affordability is often questioned when it is one of the options for disease treatment. For instance, the cost of Zolgensma is approximately $2.1 million in 2023 for one-time treatment. This cost factor is the major restraining parameter that limits the usage of gene therapy. Moreover, since the gene therapy is most often given for a single time for any disease, its efficacy after in long term is another questionable factor. 

Segment Analysis

The global gene therapy for rare disease market is segmented based on vector type, delivery type, indication, age group, end user and region.

The viral vectors in the vector type segment accounted for approximately 76.29% of the global gene therapy for rare disease market share

Vectors used in the gene therapy are the carriers or vehicles that deliver the therapeutic genetic meaterials to the site of repair. There are notably two types of vectors used in gene therapy which include viral type and non-viral type. Viral type vectors are the most commonly used carriers in gene therapy due to their natural ability to efficiently infect and deliver genetic material into the target cells. Majority of the gene therapies that are currently in the market and are being developed are based on viral vectors to deliver the therapeutic gene. Moreover, many scientific studies conducted to test the efficiency of different vectors have recommended viral vectors for gene delivery. 

For instance, in January 2022, an investigational study published in the National Center for Biotechnology Information has reported that the viral vectors are the most widely used vectors in gene therapy. The study evaluated the 13 commercially available gene therapies and more than 284 active clinical trials, and has concluded than gene therapies based on viral vectors have the high success rate for regulatory approval. In these active clinical trials, Adeno-associated virus based vectors have domiated with 48.2% share followed by adenovirus and HSV. 

On March 7, 2023 according to a study published in MDPI, viral vectors provide considerable potential for treating chronic disorders such SCID, muscular dystrophy, hemophilia, β-thalassemia, and SCD. The article has also stated that, preclinical and clinical studies have demonstrated the safety, efficacy and tolarability of viral vectors used for gene therapies. 

Many gene therapies that are currently approved for treatment of rare conditions are based on viral vectors. For example, Zolgensma by Novartis AG used for spinal muscular atrophy patients is a based on adeno-associated viral vector, Luxturna developed by Spark Therapeutics, Inc. is a also based on adeno-associated viral vector. Abecma developed by Bristol-Myers Squibb Company for the treatment of refractory multiple myeloma is based on lentivirus vector etc. 

Geographical Analysis 

North America is expected to dominate the market with 52.15% share in the Global gene therapy for rare disease Market

North America is expected to dominate the market due to highest investment by major market players in the development and launch of gene therapy. Moreover, the favourable reimbursement scenario for gene therapies in the region is expected to contribute to the higher sales volume in the region. The North American countries especially U.S. and Canada are majorly focused on brining novelty in the healthcare industry, with many pharmaceutical and biopharmaceutical companies, academia, research institutes working in collaboration for pioneering these innovations. 

For instance, on January 18, 2022, Ori Biotech Ltd. A U.S. based company had received $100 million funding for development and launch of its cell and gene therapy platform. This platform aims at assisiting the gene therapy developers and manufacturers for production in clinical levels and transition it to commercial levels. 

Moreover, the major market leaders in the cell and gene therapy have stronghold presence in the region due to its favourable and stringent regulatory frameworks and high scope for investments. These companies focus on commericialization of their gene therapies in the high-income North American countries and generate major portion of revenue from the region which is a major contributing factor for region’s dominance in the market

For instance, Bristol-Myers Squibb Company. Generates 57% of the revenue for Abecma gene therapy from the U.S. only, where as Novartis AG generates 32% of the Zolgensma revenue from the U.S. Sarepta Therapeutics, Inc. has generated more than 90% of the revenue from the U.S. for its gene therapy product elevidys used for duchenne muscular dystrophy.

Competitive Landscape

The major players in the gene therapy for rare disease market include Novartis AG, bluebird bio, Inc., Sarepta Therapeutics, Inc., Spark Therapeutics, Inc., BioMarin Pharmaceutical, Inc., Vertex Pharmaceuticals Incorporated, Bristol-Myers Squibb Company., Orchard Therapeutics plc., and Krystal Biotech, Inc. among others. 

COVID-19 Impact Analysis

The COVID-19 pandemic has significantly impacted the global market for rare disease gene therapy, leading to workforce shortages and reduced research and development activities in many laboratories developing gene therapies. The pandemic has significantly disrupted the cell and gene therapy industry, causing significant complexities in material supply, manufacturing, and logistics processes.

For instance, on August 6, 2022 an article published by Thermo Fisher Scientific, stated that the COVID-19 pandemic shifted resources from cell and gene therapy programs to SARS-CoV-2 treatments, causing a shortage of resources and impacting complex processes. This led to a decline in non-COVID-19 research and increased manufacturing challenges in standardization and scalability.

Why Purchase the Report?

• To visualize the global gene therapy for rare disease market segmentation based on vector type, delivery type, indication, age group, end user and region as well as understand key commercial assets and players.
• Identify commercial opportunities by analyzing trends and co-development
• Excel data sheet with numerous data points of gene therapy for rare disease market-level with all segments.
• PDF report consists of a comprehensive analysis after exhaustive qualitative interviews and an in-depth study.
• Product mapping available as excel consisting of key products of all the major players.

The global gene therapy for rare disease market report would provide approximately 75 tables, 72 figures, and 187 Pages.

Target Audience 2024

• Manufacturers/ Buyers
• Industry Investors/Investment Bankers
• Research Professionals
• Emerging Companies