Gene Therapy For Rare Disease Market Size Share Growth and Report 2024

Market Overview:

The global gene therapy for rare disease market is projected to grow at a high CAGR from 2024 to 2031. Rare diseases, affecting a small fraction of the population, are often chronic and life-threatening. In 2022, around 470 million individuals globally were diagnosed with various rare diseases. According to the World Health Organization (WHO), a disease is classified as rare or orphan if it affects fewer than 65 individuals per 100,000 population.

Most rare diseases are caused by genetic defects and lack approved therapies. Only 5% of rare diseases have approved treatments, with about 30% of patients dying within the first five years of life. Gene therapy, which targets and repairs defective genes, is considered the ultimate treatment for rare genetic diseases, aiming to improve long-term quality of life. The market is driven by factors such as the rising prevalence of congenital rare diseases and increased investments in gene therapy development.

Market Trends:

There has been a significant surge in the prioritization and development of gene therapies for rare diseases. The lack of standardized treatment regimens and effective existing treatments positions gene therapy as a revolutionary approach for curing these conditions. Major pharmaceutical and biopharmaceutical companies are heavily investing in developing advanced gene therapies, which, upon approval, are expected to transform market dynamics.

For instance, a cross-sectional web-based survey published in Sage Journals on June 10, 2022, predicted that by 2036, gene therapy would become the standard treatment for rare diseases, with CRISPR-Cas9 being the most successful gene-editing approach. Companies like Pfizer Inc. are actively developing gene therapies, such as fidanacogene elaparvovec and giroctocogene fitelparvovec for hemophilia B and A, respectively, which are currently in phase 3 clinical trials. Similarly, Astellas Pharma Inc. and Novartis AG are advancing gene therapies for conditions like X-linked myotubular myopathy and spinal muscular atrophy (SMA).

The increasing prevalence of rare congenital abnormalities and rising awareness about gene therapy's importance are expected to drive market growth during the forecast period.
Despite its potential, gene therapy faces several challenges that may limit its adoption. The high cost of gene therapy, such as the $2.1 million one-time treatment cost of Zolgensma in 2023, is a significant barrier. Additionally, questions about the long-term efficacy of gene therapy, given its often single-administration nature, also pose a challenge.

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Market Segmentation:

The global gene therapy for rare disease market is segmented by vector type, delivery type, indication, age group, end user, and region. The viral vectors segment accounted for approximately 76.29% of the market share. Viral vectors are preferred due to their natural ability to efficiently deliver genetic material to target cells. Many approved and developing gene therapies utilize viral vectors.
For instance, a study published in January 2022 by the National Center for Biotechnology Information reported that viral vectors dominate gene therapy due to their high success rates. Adeno-associated virus-based vectors are particularly prominent, followed by adenovirus and HSV. Approved therapies like Zolgensma and Luxturna use adeno-associated viral vectors, while Abecma uses lentivirus vectors.

Geographical Analysis:

North America is expected to dominate the market, holding a 52.15% share, driven by substantial investments in gene therapy development and favorable reimbursement scenarios. The region, especially the U.S. and Canada, is at the forefront of healthcare innovation, with strong collaborations between pharmaceutical companies, academia, and research institutes.

For instance, Ori Biotech Ltd. received $100 million in funding on January 18, 2022, to develop a cell and gene therapy platform. Major companies like Bristol-Myers Squibb and Novartis generate significant revenue from the U.S., reflecting the region's market dominance. For example, Bristol-Myers Squibb generates 57% of Abecma's revenue from the U.S., and Novartis generates 32% of Zolgensma's revenue from the U.S.

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Competitive Landscape:

Key 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. These companies are focused on developing and commercializing innovative gene therapies to address the unmet needs of patients with rare genetic disorders.

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This release was published on openPR.