Leber Congenital Amaurosis Market is Estimated to Witness High Growth Owing to Increasing R&D Activities in Gene Therapy

Leber congenital amaurosis (LCA) is a group of inherited retinal diseases caused due to mutations in at least 18 different genes. It is the most common cause of inherited childhood blindness. The signs and symptoms of LCA usually appear within the first year of life and may include poor vision, nystagmus, and photophobia. People with LCA have a wide range of vision loss, from mild to seeing only light and shadows to total blindness.

The global LCA market is estimated to be valued at US$ 1.23 billion in 2024 and is expected to exhibit a CAGR of 4.5% over the forecast period 2024 to 2031.

Key Takeaways

Key players: Key players operating in the LCA market are Spark Therapeutics, Inc., MeiraGTx Limited, Sensorion, Adverum Biotechnologies, Inc., Acucela Inc., Ocugen, Inc, REGENXBIO Inc.

Key opportunities: Leber Congenital Amaurosis Market Size is increasing R&D investments for gene therapy and availability of therapies in late-stage clinical trials offer major growth opportunities in the LCA market. In addition, rising awareness regarding inherited retinal diseases and government support are supporting market growth.

Global expansion: Major players are focusing on conducting clinical trials in different geographies and expanding their product portfolio to strengthen their geographic presence globally. Furthermore, collaborations and acquisitions are helping companies to penetrate developing markets.

Market drivers: One key driver for the LCA market growth is increasing R&D activities in the field of gene therapy. Many pharmaceutical companies and research institutes are engaged in developing newer gene therapy treatments for curing LCA. For instance, Spark Therapeutics' Luxturna is the first FDA approved gene therapy for treatment of LCA. Such approvals and advancements in therapy development are driving the market growth.

PEST Analysis

Political: The government policies related to healthcare and ease of doing business in pharmaceutical industry impact the market. Regulations related to drug approvals and pricing influence the demand.

Economic: The per capital healthcare expenditure and economic growth determine the spending capability of consumers on expensive treatment options. Recession can impact the discretionary spending on such therapies.

Social: Growing awareness through patient advocacy groups, social media influence, and lifestyle changes drive the adoption. Social stigma related to genetic disorders is reducing which also propels the market growth.

Technological: Advancements in gene therapy, stem cells therapy and developmental of new drug molecules widen the treatment avenues. 3D printing, AI, and wearable technologies aid in faster diagnosis and treatment monitoring. Collaboration between academics and private players accelerate the R&D.

Geographical concentration

North America holds the major share of the global Leber Congenital Amaurosis market in terms of value, owing to the high healthcare spending, availability of advanced treatment options, and favorable reimbursement policies. The United States dominates the regional market due to presence of key market players and streamlined regulatory framework.

Fastest growing region

Asia Pacific exhibits the fastest growth during the forecast period due to rising healthcare awareness, increasing spending on specialty diseases, expanding private healthcare infrastructure, and growing penetration of medical insurance. Rapid economic development of countries such as China and India augments the regional market growth through enhanced accessibility and affordability of treatments. Improving patient identification and diagnostic rates additionally stimulates the APAC Leber Congenital Amaurosis market.

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