Gene therapy in ophthalmology involves the use of therapeutic genes to treat various ocular diseases and disorders. Gene therapy provides a novel approach for the treatment of inherited retinal diseases by introducing a functional copy of the defective gene directly into the retinal cells. The major ocular diseases currently targeted through gene therapy include age-related macular degeneration, Leber’s congenital amaurosis, choroideremia, and retinitis pigmentosa. Gene therapy offers several advantages over traditional treatment options as it has the potential to cure genetic disorders at their root cause by compensating for mutated genes.
The global Gene Therapy in Ophthalmology market is estimated to be valued at US$ 621.9 million in 2030 and is expected to exhibit a CAGR of 16.4% over the forecast period 2020-2030.
Key Takeaways
Key players operating in the gene therapy in ophthalmology are Spark Therapeutics, MeiraGTx, Oxford BioMedica, REGENXBIO, and Adverum Biotechnologies.
The key opportunities in the market include a large patient pool of inherited retinal diseases, rising R&D investments by pharmaceutical companies, and growing funding from private and public organizations to support clinical trials. Advancements in viral vector engineering and gene editing technologies such as CRISPR have enabled more targeted and efficient delivery of therapeutic genes, thus revolutionizing ocular gene therapy.
Market Drivers:
The major market driver is the large underpenetrated market for inherited retinal diseases. It is estimated that over 350 million people worldwide are affected by some form of retinal disease or disorder. Gene therapy offers a potentially curative treatment option for these inherited retinal conditions unlike the existing symptomatic treatment landscape. Rising awareness about the benefits of gene therapy and growing focus on development of personalized treatment regimens will further propel the demand over the forecast period. Furthermore, the recent approval of voretigene neparvovec in 2018 accelerated the clinical development of other gene therapy candidates, thus driving market growth.
Challenges in the Gene Therapy in Ophthalmology Industry
The gene therapy in ophthalmology industry faces various challenges currently. Developing effective and safe gene therapies is technically extremely challenging given the complexity of gene editing. Further clinical trials need to thoroughly establish long term safety and efficacy of these therapies. Limited understanding of disease pathogenesis poses hurdles in development of targeted therapies. High costs involved in R&D and manufacturing also pose challenges. Regulatory approvals are another challenge area with rigorous safety testing protocols that need to be followed. Finally, patient acceptance and uptake also needs to be improved with better education and outreach regarding benefits of these therapies.
Current Challenges in the Gene Therapy in Ophthalmology Industry
The key current challenges in the gene therapy for ophthalmology industry include developing therapies for more complex retinal diseases, obtaining long term safety and efficacy data from clinical trials, bringing down manufacturing costs and making therapies affordable and accessible to more patients globally. While some therapies have been approved for rare retinal disorders, developing effective one-time treatments for more prevalent retinal diseases like age-related macular degeneration still remains a challenge.
SWOT Analysis
Strength: Emerging as a promising treatment option for inherited retinal disorders. Novel approach of a single administration for lifetime treatment offers significant advantages over periodic injections.
Weakness: High research and manufacturing costs. Long term safety and efficacy data still needed from ongoing clinical trials.
Opportunity: Large patient population of retinal diseases represents a major market potential. Focus on gene therapies for more prevalent conditions can address major unmet needs.
Threats: Stringent regulatory approvals. Challenges of establishing infrastructure for manufacturing and delivery at scale. Competition from newer drug therapies.
The United States currently represents the largest market for gene therapy in ophthalmology in terms of value, given presence of major players and high healthcare spending. The Asia Pacific region offers potential for fastest growth led by China, India and Japan on back of huge patient populations and improving access and affordability.
Europe is another major regional market focusing on clinical research and development activities for retinal gene therapies. Countries like the UK, Germany, France are actively working on development of newer pipeline candidates. Increasing collaboration between academia and industry is helping build regional expertise.
What Are The Key Data Covered In This Gene Therapy in Ophthalmology Market Report?
:- Market CAGR throughout the predicted period
:- Comprehensive information on the aspects that will drive the Gene Therapy in Ophthalmology’s growth between 2024 and 2031.
:- Accurate calculation of the size of the Gene Therapy in Ophthalmology and its contribution to the market, with emphasis on the parent market
:- Realistic forecasts of future trends and changes in consumer behaviour
:- Gene Therapy in Ophthalmology Industry Growth in North America, APAC, Europe, South America, the Middle East, and Africa
:- A complete examination of the market’s competitive landscape, as well as extensive information on vendors
:- Detailed examination of the factors that will impede the expansion of Gene Therapy in Ophthalmology vendors
*Note:
1. Source: Coherent Market Insights, Public sources, Desk research
2. We have leveraged AI tools to mine information and compile it