The gene therapy for rare disease market is estimated to be valued at US$ 193.56 Bn in 2023 and is expected to exhibit a CAGR of 11.% over the forecast period 2023 to 2030, as highlighted in a new report published by Coherent Market Insights.
Gene therapy involves altering genes within an individual’s cells and tissues to treat or cure disease. For rare diseases that can significantly impact quality of life and have no approved treatments, gene therapy offers hope as a potentially curative approach. Current gene therapies are focused on inherited retinal diseases, hemophilia, and certain inherited immunodeficiencies. Advances in vector development, gene editing tools, and targeted delivery are expanding the universe of diseases that may be amenable to gene-based treatments.
Increasing research funding and government initiatives: Government agencies and foundations worldwide are increasing funding for research on developing gene and cell therapies for rare diseases. This is expected to boost development of gene therapies and commercialization. Growing prevalence of rare genetic disorders: According to the National Institutes of Health, over 350 million people suffer from rare diseases worldwide. Growing prevalence of these conditions is anticipated to drive the demand for innovative gene therapy solutions over the forecast period.
The global gene therapy for rare disease market can be segmented based on vector type, therapeutic area, and end user. Based on vector type, the retroviral vector segment currently dominates the market. Retroviral vectors are considered the first generation of vectors for gene therapy and have been widely used in clinical trials. Their ability to efficiently and stably integrate into the host genome makes them well suited for the treatment of many monogenic diseases.
Political: Government initiatives to support research on rare diseases and reduce development costs through tax credits and incentives are positively impacting the market. However, strict regulations for gene therapy products delay market approval.
Economic: High healthcare costs associated with the treatment of rare diseases is driving demand for more targeted and efficacious therapies like gene therapy. However, the high development costs of these advanced therapies pose challenges.
Social: Growing rare disease patient population and support from advocacy groups are raising awareness and driving the adoption of new treatment technologies. However, social and ethical concerns associated with gene therapy limit its complete acceptance.
Technological: Advancements in vector engineering, gene editing, and manufacturing platforms are improving gene therapy safety, efficacy and reducing costs. Novel approaches like in vivo gene therapy also hold promise.
The Global Gene Therapy For Rare Disease Market Growth is expected to witness high, exhibiting CAGR of 11% over the forecast period, due to increasing research investments and advancements in vector technologies. The market size for 2023 is US$ 193.56 Bn. Regional analysis: North America currently dominates the market due to presence of major players, high healthcare expenditure, and supportive regulatory environment for clinical trials. Asia Pacific is expected to grow at the fastest pace due to rising incidence of rare diseases and increasing awareness.
Key players: Amgen Inc., Biomarin Pharmaceuticals, Bayer AG are some of the leading players operating in the gene therapy for rare disease market. Amgen has a strong rare disease portfolio and is conducting late stage trials for hemophilia gene therapy. Biomarin has commercialized several gene therapies for rare diseases and is developing more pipeline candidates. Bayer is co-developing gene therapies with partners and evaluates internal candidates.
Key players operating in the gene therapy for rare disease market are Amgen Inc., Biomarin Pharmaceuticals, Bayer AG. Amgen has a strong rare disease portfolio and is conducting late stage trials for hemophilia gene therapy. Biomarin has commercialized several gene therapies for rare diseases and is developing more pipeline candidates. Bayer is co-developing gene therapies with partners and evaluates internal candidates.
1. Source: Coherent Market Insights, Public sources, Desk research
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