Leber Congenital Amaurosis Market

The global Leber Congenital Amaurosis Market is valued at USD 1.3 Billion in 2024 and is projected to reach a value of USD 2.14 Billion by 2035 at a CAGR (Compound Annual Growth Rate) of 4.65% between 2025 and 2035. The global Leber Congenital Amaurosis market is primarily driven by factors such as the rising prevalence of chronic conditions like diabetes and gastrointestinal disorders, an increasing number of emerging players, and the growing use of infusion pumps in home care settings, which boost demand.

Key Highlights

• Based on the Treatment Type, the Gene Therapy category accounted for significant market share of 38.7% in Leber Congenital Amaurosis market industry in 2024
• In 2024, by Target Gene, RPE65 dominated the Leber Congenital Amaurosis market with significant market share of 33.8%
• Based on End User, Hospitals dominated the market with market share of 41.6% in 2024
• North America dominated the Leber Congenital Amaurosis market industry with 40.5% Leber Congenital Amaurosis market share in 2024
• Asia Pacific region is anticipated to grow at the highest CAGR of 18.9% during the forecast period in Leber Congenital Amaurosis market industry

Treatment Type Overview

​The Treatment Type segment is divided into Gene Therapy, Pharmaceutical Drugs, Retinal Prosthesis, Assistive Devices, Others. The Gene Therapy segment held the significant Leber Congenital Amaurosis market size in 2024.

The Gene Therapy led the Leber Congenital Amaurosis market industry in 2024, capturing over significant revenue. By injecting a functional form of the damaged gene, gene therapy seeks to treat the genetic flaw causing Leber Congenital Amaurosis. Gene therapy offers a one-time fix with enduring results. While novel delivery techniques like suprachoroidal and subretinal injections are increasing the targeting of retinal cells, research is stepping up to improve the safety and effectiveness of viral vectors. The potential of gene therapy has been further confirmed by the approval of voretigene neparvovec for the treatment of RPE65-mediated LCA. If regulatory approval is granted, several gene therapy candidates in late-stage trials could soon enter the Leber Congenital Amaurosis market, solidifying gene therapy as the preferred treatment option.

Target Gene Overview

​The Target Gene segment is divided into RPE65, GUCY2D, AIPL1, RPGRIP1, CEP290, Other Genes. The RPE65 segment held the dominant share in 2024, accounting for significant Leber Congenital Amaurosis industry share of 33.8%.

RPE65 held the largest market share in the global Leber Congenital Amaurosis industry in 2024. RPE65 mutations represent a significant portion of all LCA cases, and therapies targeting RPE65 have shown promising results in restoring vision. Luxturna became the first FDA-approved gene therapy for an inherited retinal disease by targeting RPE65, offering long-lasting benefits with a single treatment. This success has established RPE65 as a key gene target. New therapies, such as ADVM-022, are also exploring the use of the AAV vector platform to deliver the RPE65 gene. Ongoing research is focused on improving vector engineering and refining the targeting of retinal cells. Given the progress made, RPE65 is expected to remain a leading gene target as more therapies enter clinical trials and commercialization, reinforcing its dominance in the Leber Congenital Amaurosis market.

End User Overview

​The End User segment is divided into Hospitals, Specialized Eye Clinics, Ophthalmology Research Centers, Home Care Settings, Others. The Hospitals segment held the dominant share in 2024, accounting for significant Leber Congenital Amaurosis industry share of 41.6%.

Hospitals held the largest market share in the global Leber Congenital Amaurosis industry in 2024. The LCA requires coordinated management across multiple specialties, and hospitals are ideally suited to meet these needs. They offer on-site retinal specialists, genetic testing, gene therapy administration, low vision aids, and vision rehabilitation programs, all in one location. This integrated approach ensures effective long-term monitoring and follow-up care. As new therapies emerge, hospitals will play a critical role in large-scale clinical trials as treatment centers. With expertise across various departments, hospitals also assist patients through complex procedures and stay informed about the rapidly evolving treatment landscape. Moving forward, hospitals are well-positioned to adopt advanced therapies, such as gene therapies, and lead initiatives like data registries for treated patients, solidifying their role as the primary healthcare setting for LCA management.

Regional Overview

In 2024, the Leber Congenital Amaurosis market size of the North America is USD XX Billion.

North America Leber Congenital Amaurosis industry report is driven by many key market players based in the U.S. and Canada, providing a competitive advantage in terms of Leber Congenital Amaurosis industry presence and market access. North America hosts some of the largest pharmaceutical companies, which heavily invest in research and development. Consequently, groundbreaking gene therapies and treatments for LCA are often first introduced in this region.

North Americas advanced healthcare infrastructure and high healthcare spending have facilitated broader patient access to expensive therapies, prompting companies to prioritize this Leber Congenital Amaurosis market for the commercialization of new treatments. The export of LCA therapies from North America to other developed markets is also significant. While patent expirations in the coming years may present challenges, North American firms maintain an edge with a robust pipeline of new drugs. Overall, the regions innovation ecosystem and commercialization environment solidify its position as a dominant force in the global Leber Congenital Amaurosis market.

Asia Pacific Leber Congenital Amaurosis Market Analysis

The Asia Pacific Leber Congenital Amaurosis market is expected to grow at a robust CAGR during the forecast period. The Asia Pacific region, particularly China and India, is expected to experience the fastest growth during the forecast period. The increasing medical needs of a growing patient population, coupled with rising healthcare investments, present significant opportunities. This has led several multinational companies to target the Asia Pacific region through partnerships, joint ventures, and investments.

The reduction of import duties and the development of local manufacturing capabilities are anticipated to improve drug accessibility and reduce prices over time. China, in particular, has been working to create an efficient regulatory framework to attract investments in advanced therapies. With its large untapped market potential, the Leber Congenital Amaurosis market in Asia Pacific is expected to grow rapidly, outpacing mature Western markets.

Based on the provided market data, Vantage Market Research offers customizations in the reports to meet the specific needs of clients.

Leber Congenital Amaurosis Market Trends

• Advancements in Gene Therapy: Innovations in gene therapy, particularly AAV-based treatments, are revolutionizing LCA care with potential one-time curative options
• Focus on Precision Medicine: Personalized treatments based on genetic profiles are improving patient outcomes
• Orphan Drug Designations: Regulatory incentives like market exclusivity and tax benefits are driving investment in LCA therapies
• Collaborations and Partnerships: Increased partnerships among companies, research institutions, and advocacy groups are accelerating drug development
• Improved Diagnostics: Advanced tools like next-generation sequencing are enabling early and accurate LCA diagnosis
• Expanding Pipeline: A growing number of therapies in clinical trials reflect heightened interest in addressing unmet needs
• Patient Advocacy: Awareness campaigns are boosting early diagnosis and participation in clinical trials

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Market Dynamics

Market Drivers for Leber Congenital Amaurosis Market

Rising prevalence of inherited retinal diseases

The prevalence of inherited retinal diseases is rising globally, with Leber congenital amaurosis (LCA), a form of retinal dystrophy, experiencing a steady increase in cases each year. Estimates suggest that 2-3 out of every 100,000 live births are diagnosed with LCA, translating to approximately 2,800 to 4,200 new cases annually, given the global birth rate of around 140 million. This growth is partly due to the increasing age of prospective parents. Modern medical advancements have enabled many to delay parenthood into their late 30s or early 40s. However, advanced parental age is a known risk factor for inherited genetic disorders, including LCA, as the likelihood of genetic mutations increases. With more individuals opting for later parenthood due to social and economic factors, the risk of passing on genetic retinal conditions has risen. Additionally, improved diagnostic capabilities have contributed to the apparent rise in LCA cases. Advanced genetic testing tools now allow for quicker and more accurate identification of rare retinal disorders, including in newborns. Previously, conditions like LCA often went undiagnosed due to limited expertise and diagnostic methods. Enhanced awareness through patient advocacy groups has also encouraged families to undergo genetic screening, leading to better disease detection and tracking worldwide.

Market Restraints for Leber Congenital Amaurosis market

High Cost of Treatment

The global Leber Congenital Amaurosis market faces a significant challenge due to the high cost of treatment. As a rare genetic disorder causing severe visual impairment from birth, LCA currently lacks approved drug therapies, and available options, such as gene therapies, come with hefty price tags. For instance, voretigene neparvovec, the first FDA-approved gene therapy for LCA, is priced at $850,000 per treatment. These exorbitant costs impose a heavy financial burden on patients, healthcare systems, and governments. Without adequate insurance coverage or financial support, many patients are unable to afford such treatments, limiting access and constraining the market potential for new therapies. To ensure wider accessibility, companies developing advanced therapies must address the challenge of balancing innovation with affordability.

Market Opportunities for Leber Congenital Amaurosis market

Advancements in Gene Therapies

The rise of gene therapies presents a transformative opportunity for the Leber Congenital Amaurosis market. As LCA is caused by specific genetic mutations, gene therapy offers a promising, potentially curative approach. Several companies are advancing gene therapies, with encouraging results from clinical trials. Voretigene neparvovec marked a milestone as the first approved gene therapy in 2021. Other candidates in late-stage development include AVROBIOs AVR-RD-01, MeiraGTx’s AAV-CNGB3, and AGTCs XLRP Therapy. The success of these initial therapies is expected to validate the gene therapy approach, attract further investment, and encourage the development of additional treatments targeting various genetic causes of LCA. Continued innovation in this field could establish gene therapy as a standard of care, significantly improving outcomes for LCA patients worldwide.

Competitive Landscape

The Leber Congenital Amaurosis market industry report is characterized by the presence of key players focusing on innovative therapies and advanced genetic treatments. Companies are investing in research and development to address the underlying genetic mutations associated with LCA, with significant emphasis on gene therapy and retinal implants. Strategic collaborations, partnerships, and acquisitions are driving advancements in treatment options. Additionally, increasing regulatory approvals and orphan drug designations are fostering market growth, while competition intensifies as new entrants and established players strive to expand their portfolios and enhance patient outcomes in this rare disease segment.

The key players in the global Leber Congenital Amaurosis market report include - Pfizer among others.

Recent Market Developments

Reserpine Shows Promise as Potential Treatment for Leber Congenital Amaurosis Type 10 (LCA10), According to National Eye Institute Researchers

• In 2023, researchers from the National Eye Institute discovered that Reserpine, an FDA-approved drug originally used to treat high blood pressure, shows promise for treating Leber congenital amaurosis type 10 (LCA10). In laboratory models, Reserpine helped preserve light-sensitive photoreceptors damaged by LCA10, a genetic disorder caused by CEP290 mutations

Editas Medicines EDIT-101 Demonstrates Significant Visual Improvements in Phase 1/2 Trial for Leber Congenital Amaurosis Type 10 (LCA10)

• In November 2022, Editas Medicine presented clinical data from the Phase 1/2 BRILLIANCE trial of EDIT-101, a CRISPR/Cas9-based therapy for LCA10, a rare retinal disorder. The trial revealed that three out of 14 patients treated with EDIT-101 showed significant improvements in best corrected visual acuity (BCVA) and other measures of visual function. EDIT-101 targets the IVS26 CEP290 mutation, which affects approximately 1,500 patients in the U.S. and currently lacks an effective treatment

The global Leber Congenital Amaurosis market can be categorized as Treatment Type, Target Gene, End User and Region.