Leber Congenital Amaurosis Market Report 2025

The leber congenital amaurosis market size has grown strongly in recent years. It will grow from $1.01 billion in 2024 to $1.07 billion in 2025 at a compound annual growth rate (CAGR) of 6%. The growth observed during the historical period can be attributed to factors such as increased awareness of rare eye diseases, greater government funding, enhanced diagnostic methods, the expansion of ophthalmic research centers, and patient advocacy efforts.

The leber congenital amaurosis market size is expected to see strong growth in the next few years. It will grow to $1.34 billion in 2029 at a compound annual growth rate (CAGR) of 5.9%. The growth in the forecast period is expected to be driven by higher investments in gene therapy, the rising prevalence of inherited retinal disorders, regulatory approvals for new treatments, the expansion of clinical trials, and an increase in healthcare spending. Key trends for the forecast period include the rise of CRISPR-based therapies, greater collaboration between biotechnology companies, the development of personalized medicine, the growth of teleophthalmology, and advancements in retinal imaging technologies.

The rising prevalence of inherited retinal diseases is expected to drive the growth of the leber congenital amaurosis (LCA) market. Inherited retinal diseases (IRDs) are genetic disorders that affect the retina, the light-sensitive tissue at the back of the eye responsible for vision. The increase in these diseases can be attributed to advancements in genetic testing, greater awareness, better diagnosis, and longer life expectancy. Progress in genetic testing has made it easier to diagnose IRDs, resulting in more cases being identified. Leber congenital amaurosis has helped enhance the understanding of inherited retinal diseases by revealing genetic mutations and disease mechanisms, contributing to the development of targeted therapies and treatments. For example, according to the UK Parliament's House of Commons Library in January 2025, approximately 25,000 people in the UK were affected by inherited retinal diseases in 2024, marking a 2% annual increase. As a result, the growing prevalence of inherited retinal diseases is driving the growth of the leber congenital amaurosis market.

Companies in the leber congenital amaurosis market are increasingly focusing on developing RNA-based treatments to improve vision restoration and disease management. RNA-based therapies utilize RNA molecules to target genetic processes and treat diseases. These therapies include mRNA vaccines, siRNA therapies, antisense oligonucleotides, and RNA aptamers. For instance, in January 2025, Laboratoires Théa S.A.S., a French pharmaceutical company, launched Sepul Bio, a business unit dedicated to advancing RNA therapies for inherited retinal diseases. Sepul Bio is specifically targeting sepofarsen for leber congenital amaurosis 10 (LCA10) and ultevursen for USH2A-associated retinitis pigmentosa. Following the successful dosing of the first patient in the LUNA Phase 2b clinical trial for ultevursen, Sepul Bio is poised to make significant progress in addressing these rare genetic disorders.

In October 2024, Ocuphire Pharma Inc., a US-based biopharmaceutical company, acquired Opus Genetics Inc. for an undisclosed amount. This acquisition aims to create a leading clinical-stage biotechnology company focused on developing gene therapies for inherited retinal diseases (IRDs). Opus Genetics Inc. is a US-based company that specializes in developing gene therapies for IRDs.

Major players in the leber congenital amaurosis market are Sanofi S.A., Novartis AG, Astellas Pharma Inc., Invitae Corporation, Novelion Therapeutics Inc., Andelyn Biosciences Inc., CD Genomics Inc., Editas Medicine Inc., MeiraGTx Holdings plc, Sangamo Therapeutics Inc., LKC Technologies Inc., Aldeyra Therapeutics Inc., Asper Biogene OÜ, Allergan Inc., ProQR Therapeutics N.V., GenSight Biologics S.A., Ocugen Inc., IVERIC bio Inc. (formerly Ophthotech Corporation), Atsena Therapeutics Inc., HuidaGene Therapeutics Co. Ltd.

North America was the largest region in the leber congenital amaurosis market in 2024. Asia-Pacific is expected to be the fastest-growing region in the forecast period. The regions covered in leber congenital amaurosis report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East and Africa. The countries covered in the leber congenital amaurosis market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA, Canada, Italy, Spain.

Leber congenital amaurosis (LCA) is an inherited eye disorder that leads to significant vision impairment or blindness from a young age, caused by genetic mutations that hinder the retina’s ability to process light. The condition is marked by weak visual responses, involuntary eye movements (nystagmus), and severe farsightedness due to malfunctioning photoreceptors and gradual retinal degeneration.

Treatment options for LCA primarily include gene therapy, medication, retinal prosthetics, assistive technologies, and other interventions. Gene therapy is a technique that uses genetic material to treat or prevent disorders such as LCA by fixing defective genes. LCA is classified based on the age of onset, including congenital LCA (present at birth), early childhood LCA (1-5 years old), and late-onset LCA (after 6 years). Medications for LCA are distributed through various means such as direct tenders and retail sales, and are used in hospitals, specialized eye clinics, ophthalmology research centers, and home care environments.

The leber congenital amaurosis market research report is one of a series of new reports that provides leber congenital amaurosis market statistics, including the leber congenital amaurosis industry global market size, regional shares, competitors with the leber congenital amaurosis market share, detailed leber congenital amaurosis market segments, market trends, and opportunities, and any further data you may need to thrive in the leber congenital amaurosis industry. This leber congenital amaurosis market research report delivers a complete perspective of everything you need, with an in-depth analysis of the current and future scenarios of the industry.

The leber congenital amaurosis (LCA) market consists of revenues earned by entities by providing services such as clinical trial services, healthcare and patient support services, vision rehabilitation services, and diagnostic testing services. The market value includes the value of related goods sold by the service provider or included within the service offering. The leber congenital amaurosis (LCA) market also includes sales of retinal implants, diagnostic testing kits, assistive devices, and gene therapies. Values in this market are ‘factory gate’ values, that is the value of goods sold by the manufacturers or creators of the goods, whether to other entities (including downstream manufacturers, wholesalers, distributors and retailers) or directly to end customers. The value of goods in this market includes related services sold by the creators of the goods.

The market value is defined as the revenues that enterprises gain from the sale of goods and/or services within the specified market and geography through sales, grants, or donations in terms of the currency (in USD, unless otherwise specified).

The revenues for a specified geography are consumption values that are revenues generated by organizations in the specified geography within the market, irrespective of where they are produced. It does not include revenues from resales along the supply chain, either further along the supply chain or as part of other products.

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