Gene therapy for rare disease involves the introduction of a functional gene into the patient’s cells to compensate for an abnormal or missing gene. Gene therapies help treat rare diseases caused due to a single genetic defect. There is a high unmet medical need to treat rare genetic diseases as many lack effective treatment options. The technology has gained popularity owing to increasing investments by key players in R&D initiatives to develop novel gene therapies.

The global gene therapy for rare disease market is estimated to be valued at US$ 8.9 billion in 2024 and is expected to exhibit a CAGR of 9.0% over the forecast period 2024 to 2030.

Key Takeaways
Key players operating in the gene therapy for rare disease market are Topcon Corporation, Agfa-Gevaert N.V., Sonomed Escalon, Carl Zeiss Meditec AG, EyePACS, VersaSuite, Heidelberg Engineering GmbH, ScImage, Inc., IBM Corporation, and Visbion Limited. These players are focusing on new product launches, collaborations and acquisitions to strengthen their market position.

Some of the major opportunities in the Gene Therapy For Rare Disease Market Demand include increasing number of clinical trials, high prevalence of rare genetic diseases, creation of gene therapy databases and growing investments by pharmaceutical companies and government bodies.

The market is also witnessing significant expansion in regions such as North America, Europe, Asia Pacific, Latin America, and Middle East and Africa. Key players are expanding their geographical footprint by entering untapped markets to enhance access to novel gene therapies.

Market drivers and restraints
Major drivers aiding growth of the gene therapy for rare disease market include rising approvals for novel gene therapies and increasing awareness about benefits of gene replacement. Regulatory bodies are fast-tracking approval process for drugs targeting rare disease to promote patient access. Approval of first few gene therapies has boosted confidence in the medical community about clinical efficacy and safety.

However, high costs associated with research, development and production of gene therapies continues to restrain market growth. Gene therapies pose technical challenges such as immune response against vector or transgene expression. Further, limitations in gene delivery are also hampering widespread adoption. Safety issues if genetic material integrates into wrong location can potentially trigger cancer. Stringent regulations also pose challenges for companies to navigate approval process smoothly.

Segment Analysis
The gene therapy for rare disease market is dominated by retinal disorder sub-segment currently holding around 30-35% market share. This is due to high prevalence of retinal disorders like retinitis pigmentosa and age-related macular degeneration. Furthermore, success of gene therapy products for retinal disorders like Luxturna by Spark Therapeutics has boosted the adoption of retinal gene therapy. Neurological disorder sub-segment is expected to emerge as fastest growing segment during forecast period attributed to ongoing clinical trials for treating severe neurodegenerative diseases.

Global Analysis

North America currently captures around 60% of global gene therapy for rare disease market revenues attributed to regulatory favourable environment, presence of key market players and higher healthcare spending. The region is expected to continue its dominance during forecast period. Europe is second largest regional market expected to grow at a CAGR of over 8% between 2024-2030. Asian countries especially China and India are fastest growing regional markets owing to improving healthcare infrastructure, increasing investments in life science research and rising healthcare expenditure. Growing expertise in genetic research and presence of large patient pool makes Asia Pacific an emerging and lucrative market for rare disease gene therapies market.

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