The Business Research Company’s report on the Leber Congenital Amaurosis Market provides insights into the global market size, growth rate, regional distribution, competitive landscape, key segments, emerging trends, and strategic opportunities.
#How have key drivers contributed to the rapid growth of the leber congenital amaurosis market?
The increasing prevalence of inherited retinal diseases is expected to propel the growth of the leber congenital amaurosis (LCA) market. Inherited retinal diseases (IRDs) refer to genetic disorders affecting the retina, the light-sensitive tissue at the back of the eye responsible for vision. The increase in inherited retinal diseases is attributed to improving genetic testing, awareness and diagnosis, and longer life expectancy. Advancements in genetic testing have made it easier to diagnose inherited retinal diseases, leading to the identification of more cases. Leber congenital amaurosis helps advance the understanding of inherited retinal diseases by revealing genetic mutations and disease mechanisms that contribute to developing targeted therapies and treatments. For instance, in January 2025, according to the UK Parliament’s House of Commons Library, a UK-based government administration, approximately 25,000 people in the UK were affected by inherited retinal diseases in 2024, an increase of 2% yearly. Therefore, the increasing prevalence of inherited retinal diseases drives the leber congenital amaurosis market.
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How has the leber congenital amaurosis market size evolved, and what are the latest forecasts for its expansion?
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 6.0%. The growth in the historic period can be attributed to rise in awareness of rare eye disorders, increased government funding, improved diagnostic techniques, expansion of ophthalmic research centers, patient advocacy initiatives.
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 can be attributed to growing investment in gene therapy, increasing prevalence of inherited retinal diseases, regulatory approvals for novel treatments, expanding clinical trials, and rising healthcare expenditure. Major trends in the forecast period include emergence of CRISPR-based therapies, increasing collaboration between biotech firms, development of personalized medicine, expansion of teleophthalmology, technological advancements in retinal imaging.
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Which major companies dominate the leber congenital amaurosis market?
Major companies operating 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.
What trends will shape the future of the lead-based pigments market?
Major companies operating in the leber congenital amaurosis market are focusing on developing innovative solutions, such as RNA-based treatments to improve vision restoration and disease management. RNA-based treatments use RNA molecules to treat diseases by targeting genetic processes. These include mRNA vaccines, siRNA therapies, antisense oligonucleotides, and RNA aptamers. For instance, in January 2025, Laboratoires Théa S.A.S., a France-based pharmaceutical company, launched Sepul Bio, a dedicated business unit aimed at advancing RNA therapies for inherited retinal diseases, explicitly targeting sepofarsen for leber congenital amaurosis 10 (LCA10) and ultevursen for USH2A-associated retinitis pigmentosa. With the successful dosing of the first patient in the LUNA Phase 2b clinical trial for ultevursen, Sepul Bio is poised to make significant strides in addressing these rare genetic disorders.
Which region dominates the leber congenital amaurosis market, and what factors contribute to its leadership?
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 the leber congenital amaurosis market report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East, Africa.
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How is the leber congenital amaurosis market segmented, and which segment holds the largest share?
The leber congenital amaurosis market covered in this report is segmented –
1) By Treatment Type: Gene Therapy, Pharmaceutical Drugs, Retinal Prosthesis, Assistive Devices, Other Treatments
2) By Age Of Onset: Congenital Leber Congenital Amaurosis (LCA) (From Birth), Early Childhood Leber Congenital Amaurosis (LCA) (1-5 Years), Late-Onset Leber Congenital Amaurosis (LCA) (6 Years And Above)
3) By Distribution Channel:, Direct Tender, Retail Sales
4) By End User: Hospitals, Specialized Eye Clinics, Ophthalmology Research Centers, Home Care Settings, Other End Users
Subsegments:
1) By Gene Therapy: RPE65 Gene Replacement Therapy, CRISPR-Based Gene Editing, AAV (Adeno-Associated Virus) Vector Therapy, Emerging Experimental Gene Therapies
2) By Pharmaceutical Drugs: Retinoid-Based Therapies, Neuroprotective Agents, Anti-Inflammatory Drugs, Antioxidant Therapies
3) By Retinal Prosthesis: Implantable Retinal Chips, Artificial Retinal Stimulation Devices, Bionic Eye Technology
4) By Assistive Devices: Low Vision Aids, Screen Readers and Braille Devices, Mobility Aids for Vision Impairment
5) By Other Treatments: Stem Cell Therapy, Optogenetics-Based Treatments, Visual Rehabilitation Programs, Nutritional and Dietary Interventions
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How is the leber congenital amaurosis market defined, and what are its core characteristics?
Leber congenital amaurosis (LCA) is a genetic eye disorder that causes severe vision loss or blindness from infancy due to mutations affecting the retina’s ability to detect light. It is characterized by poor visual response, involuntary eye movements (nystagmus), and extreme farsightedness resulting from disrupted photoreceptor function and progressive retinal degeneration.
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