Gene Therapy in Ophthalmology Market is segmented By Indication (Inherited Retinal Diseases, Acquired Retinal Diseases), By Vector Type (Adeno-associated Virus (AAV), Len ....
Market Size in USD Bn
CAGR25.2%
Study Period | 2024 - 2031 |
Base Year of Estimation | 2023 |
CAGR | 25.2% |
Market Concentration | High |
Major Players | Johnson & Johnson Innovative Medicine, MeiraGTx, Beacon Therapeutics, Nanoscope Therapeutics, GenSight Biologics and Among Others. |
The gene therapy in ophthalmology market is estimated to be valued at USD 1.2 Bn in 2024 and is expected to reach USD 5.8 Bn by 2031, growing at a compound annual growth rate (CAGR) of 25.2% from 2024 to 2031. There is a rising trend seen in gene therapy to treat ophthalmic disorders. Manufacturers are involved in research and development of novel gene therapy products for ophthalmic conditions like age-related macular degeneration which has high prevalence rate.
Market Driver - Rising Prevalence of Genetic Ocular Disorders Leading to Increased Demand for Effective Treatments
Genetic factors contribute significantly to the development of various ophthalmic conditions including retinitis pigmentosa, age-related macular degeneration (AMD), and glaucoma. Researchers have found that mutations in as many as 250 genes can result in retinal degeneration alone. The worldwide prevalence of such inherited retinal diseases (IRDs) is estimated to range from 1 in 2,000–3,000 persons.
Recent epidemiological studies further suggest that the burden of genetic eye disorders is rising continuously mainly due to increasing life expectancy globally. The growing patient pools facing debilitating vision loss due to genetic causes have generated a strong need for groundbreaking treatments. Conventional options such as medication and surgery lack efficacy in arresting disease progression driven by genetic defects.
Gene therapies, however, offer the promise of long-term vision restoration or preservation by precisely targeting the underlying genetic roots of ocular disorders. This potential for gene therapies to provide a curative outcome through a single administration has spurred significant interest from biopharmaceutical firms. It has also encouraged greater patient enrollment in ongoing clinical trials evaluating candidate therapies for conditions like Leber congenital amaurosis and AMD.
Market Driver - Advances in Gene Editing Technologies Enhancing the Development of Novel Therapies
Over the past decade, revolutionary technologies for gene manipulation have enabled enormous progress in gene therapy research and product development. Notable among these are CRISPR-Cas9, zinc finger nucleases, and TALE nucleases which allow swift, accurate editing of DNA sequences. CRISPR in particular has revolutionized the field through its simplicity, versatility and cost-effectiveness. It has accelerated discovery and preclinical research by facilitating large-scale screening and targeted mutagenesis investigations.
Building on these platforms, scientists are gaining deeper insights into disease-causing gene mutations and the complex molecular mechanisms underlying retinal degeneration. This has led to the creation of several proof-of-concept animal models that emulate human genetic eye diseases. Such models have been instrumental in demonstrating proof-of-principle for novel therapeutic strategies like knocking-out defective genes, inserting functional gene copies and gene supplementation through viral vector delivery.
In tandem, the availability of advanced characterization tools is facilitating the selection of therapeutic candidates with ideal dosage, duration and tissue targeting attributes. Collectively, the accelerating pace of technological evolution is empowering scientists to translate ambitious gene editing concepts into promising therapeutics with attributes like one-time administration and life-long efficacy against previously untreatable genetic eye conditions.
Market Challenge - High Costs Associated with Gene Therapy Treatments Limiting Accessibility
Gene therapy treatments for various ophthalmic diseases have shown tremendous potential in clinical trials. However, one of the major challenges continues to be the high costs associated with developing and delivering such treatments. Developing an effective gene therapy typically requires years of rigorous research and testing. This clinical development process is highly expensive and risky.
Additionally, manufacturing gene therapies is a complex process that often involves customized viral vectors and careful dosing for each patient's specific needs. All of these factors contribute to the high price tags of many gene therapies currently available or in the pipeline.
For example, voretigene neparvovec, the first approved gene therapy for an inherited retinal disease, has a one-time list price of $850,000 per treatment. While it has shown promising results for patients, such high costs limit patient access and insurer reimbursement for this novel treatment approach.
As gene therapies for ophthalmic conditions continue to advance, bringing down the costs associated with development as well as administration will be crucial to maximize the utilization and benefits of this technology across broader patient populations.
Market Opportunity - Expansion into Emerging Markets with Unmet Medical Needs
One significant opportunity for the gene therapy market in ophthalmology lies in expanding into emerging markets across Asia, Latin America, Middle East, and Africa. Many populations in these regions suffer from a high prevalence of blinding ophthalmic diseases but have lacked access to advanced treatment options due to various economic and infrastructure barriers.
Gene therapies have the potential to transform the management of genetically-driven retinal disorders that currently have limited treatment options in these markets. International drug developers and domestic pharma companies can capitalize on this unmet need by conducting clinical trials and regulatory submissions strategically tailored for emerging markets. Partnering with local healthcare providers and patient advocacy groups will help facilitate patient identification and treatment delivery.
A successful emerging market expansion strategy could allow gene therapy companies to access larger patient pools and grow revenues in a meaningful way. This would also help fulfill the goal of making such revolutionary therapies globally accessible.
Novartis has been a pioneer in developing gene therapy for ophthalmology indications. In 2017, it received approval for Luxturna (voretigene neparvovec), the first ever gene therapy approved in the US for an inherited retinal disease. Luxturna is designed for patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. It helped restore vision in patients who would have otherwise experienced progressive vision loss. Luxturna’s approval demonstrated the promise of gene therapy for treating inherited eye conditions. It gave Novartis first-mover advantage and established its leadership in this therapeutic area.
Spark Therapeutics followed a similar path to Novartis. In 2018, it received FDA approval for Luxturma (voretigene neparvovec-rzyl), a gene therapy for patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. This validated the efficacy and safety of gene therapy for retinal conditions. It allowed Spark to grab market share. By 2026, Luxturma is projected to achieve $550 million in global sales according to analysts at GlobalData.
Both companies’ successes reinforced gene therapy as a viable treatment paradigm for ophthalmic conditions like inherited retina diseases. They established clinical proof-of-concept which de-risked the field and attracted more players. Over 15 biotechs are now developing various gene therapies for retinal degenerations, glaucoma, dry eye etc.
Insights, By Vector Type: Clinical Potential of AAV Drives its Dominance
In terms of vector type, adeno-associated virus (AAV) segment is estimated to hold 64.5% share of the market in 2024, due to its excellent clinical potential for ocular gene therapy. AAV vectors have emerged as the leading vector of choice owing to their non-pathogenic nature and ability to efficiently transduce retinal cells without adverse effects. They also have the advantage of long-term transgene expression in post-mitotic cells like photoreceptors.
Moreover, AAV serotypes like AAV2 demonstrate tropism for different retinal cell types making them suitable for targeting specific retinal disorders. Several AAV-based gene therapies are in late stages of clinical trials showing encouraging safety and efficacy data for indications like LCA and wet AMD.
The scalability of AAV vector production and longer stability also offer economic and logistical benefits. Their proven ability to safely deliver therapeutic genes makes AAV the most widely used and commercially viable vector system currently for retinal gene therapy.
Insights, By End User: Need for Advanced Care Drives Hospital Segment
In terms of end user, hospitals contribute the highest share of the market owing to the need for advanced infrastructure and multidisciplinary care required for gene therapy procedures. Gene therapy for ocular disorders is a complex treatment modality requiring specialized equipment, trained professionals, and biosafety facilities for vector production and administration.
Hospitals with ophthalmology and research departments are currently the main centers undertaking clinical trials as well as approved treatments for retinal gene therapies. They have the necessary setup for conducting vector production, as well as multidisciplinary teams of surgeons, doctors, and technicians for treatment delivery.
The in-patient hospital setting also allows for postoperative care and monitoring of patients. As gene therapy demand increases with commercialization, hospitals will play a central role in its delivery utilizing their advanced facilities and wider network to improve patient access to these novel sight-saving therapies. Ambulatory surgery centers and specialized eye clinics may also contribute to the hospital segment growth over the long term.
The major players operating in the gene therapy in ophthalmology market include Johnson & Johnson Innovative Medicine, MeiraGTx, Beacon Therapeutics, Nanoscope Therapeutics, GenSight Biologics, 4D Molecular Therapeutics, Coave Therapeutics, Bionic Sight, Neuropht Therapeutics, Adverum Biotechnologies, EyeVensys, Exegenesis Bio, Atsena Therapeutics, Ocugen, AbbVie, REGENXBIO, Skyline Therapeutics, Huida Gene Therapeutics, Opus Genetics, Spark Therapeutics, Novartis AG, Adverum Biotechnologies, REGENXBIO Inc., and AGTC.
Gene Therapy in Ophthalmology Market
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How big is the gene therapy in ophthalmology market?
The gene therapy in ophthalmology market is estimated to be valued at USD 1.2 Bn in 2024 and is expected to reach USD 5.8 Bn by 2031.
What are the key factors hampering the growth of the gene therapy in ophthalmology market?
High costs associated with gene therapy treatments limiting accessibility and regulatory hurdles and lengthy approval processes slowing market entry are the major factors hampering the growth of the gene therapy in ophthalmology market.
What are the major factors driving the gene therapy in ophthalmology market growth?
The rising prevalence of genetic ocular disorders leading to increased demand for effective treatments and advances in gene editing technologies enhancing the development of novel therapies are the major factors driving the gene therapy in ophthalmology market.
Which is the leading indication in the gene therapy in ophthalmology market?
The leading indication segment is inherited retinal diseases.
Which are the major players operating in the gene therapy in ophthalmology market?
Johnson & Johnson Innovative Medicine, MeiraGTx, Beacon Therapeutics, Nanoscope Therapeutics, GenSight Biologics, 4D Molecular Therapeutics, Coave Therapeutics, Bionic Sight, Neuropht Therapeutics, Adverum Biotechnologies, EyeVensys, Exegenesis Bio, Atsena Therapeutics, Ocugen, AbbVie, REGENXBIO, Skyline Therapeutics, Huida Gene Therapeutics, Opus Genetics, Spark Therapeutics, Novartis AG, Adverum Biotechnologies, REGENXBIO Inc., and AGTC are the major players.
What will be the CAGR of the gene therapy in ophthalmology market?
The CAGR of the gene therapy in ophthalmology market is projected to be 25.2% from 2024-2031.