Leber Congenital Amaurosis Market to Reach USD 2.14 Billion by 2035

According to analysts at Vantage Market Research, the Global Leber Congenital Amaurosis Market size is worth USD 1.3 Billion in 2024 and is projected to reach USD 2.14 Billion by 2035, growing at a CAGR (Compound Annual Growth Rate) of 4.65% from 2024 to 2035. Key trends of market include Emerging CRISPR-based treatments, increased focus on retinal gene therapies, partnerships for clinical trials, advancements in diagnostic tools, and rising adoption of personalized medicine.

Market Overview

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. 

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 AVROBIO's AVR-RD-01, MeiraGTx’s AAV-CNGB3, and AGTC's 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. 

Editas Medicine's 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

Key Takeaways from the Report

• North America dominated the market with 40.5% of market share in 2024.  The region has hosts some of the largest pharmaceutical companies, which heavily invest in research and development
• Based on the Treatment Type, the Gene Therapy category accounted for significant market share of 38.7% in 2024. The segment is driven by injecting a functional form of the damaged gene, gene therapy seeks to treat the genetic flaw causing Leber Congenital Amaurosis
• In 2024, by Target Gene, RPE65 dominated the market with significant market share of 33.8%. RPE65 mutations represent a significant portion of all LCA cases, and therapies targeting RPE65 have shown promising results in restoring vision
• Based on End User, Hospitals dominated the market with market share of 41.6% in 2024. Hospitals offers on-site retinal specialists, genetic testing, gene therapy administration, low vision aids, and vision rehabilitation programs, all in one location

Top Companies

• Pfizer
• Novartis
• AbbVie
• Gilead Sciences
• Spark Therapeutics
• Biogen
• Avexis
• Horizon Therapeutics
• Aerie Pharmaceuticals
• Roche
• Regeneron Pharmaceuticals
• GenSight Biologics
• Orphan Biovitrum
• Apellis Pharmaceuticals
• Regenxbio

Report Coverage

Our market research reports provide comprehensive insights that are essential for strategic decision-making. We cover all key aspects of the market, including dynamics such as drivers, restraints, opportunities, and challenges, alongside the latest industry trends. Our analysis includes an in-depth technology roadmap, product life cycle evaluation, and PESTLE analysis, ensuring a thorough understanding of the market environment. We also assess GDP growth outlooks, examine regional market landscapes, and evaluate the impact of major events like the COVID-19 pandemic. Additionally, our reports feature a detailed competitive landscape, including company market shares and profiles, providing actionable intelligence to empower your business strategies.

Latest News

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