Stargardt Disease Therapeutics Market Poised for Steady Growth Due to Advancements in Gene Therapy

Stargardt disease therapeutics market includes drugs for treating Stargardt disease, a genetic disorder of the retina that causes progressive vision loss. Stargardt disease results in a dysfunction or absence of the ABCA4 transport protein that transports all-trans-retinal from photoreceptor cells. This leads to accumulation of lipofuscin and subsequent retinal degeneration. Products in development target restoring ABCA4 transporter function through gene therapy or reducing toxic byproducts. Intravitreal gene therapy aims to deliver a normal copy of the ABCA4 gene to photoreceptor cells to restore its function. Other therapies involve inhibitors of retinoid isomerase RPE65 to reduce levels of toxic all-trans-retinal.

Global stargardt disease therapeutics market is estimated to be valued at US$ 213.5 Mn in 2023, and is expected to exhibit a CAGR of 31.7% during the forecast period (2023-2030).

Key Takeaways

Key players operating in the Stargardt disease therapeutics market are Kubota Pharmaceutical Holdings Co., Ltd., Stargazer Pharmaceuticals Inc., Iveric Bio, Inc., Sanofi S.A., Alkeus Pharmaceuticals Inc., Astellas Pharma Inc., CHABiotech CO., Ltd, ReVision Therapeutics, Inc. and Lin BioScience, Inc. Majority of these players have gene therapy candidates in clinical trials for treating Stargardt disease. Gene therapy offers advantages of a one-time treatment with long-lasting effects compared to alternatives. Technological advancements in gene delivery vectors like adeno-associated virus have enabled efficacious delivery of normal genes to target tissues.

Major opportunities lie in the approval and commercialization of first gene therapies for Stargardt disease. An effective treatment can address a huge unmet need. Biomarkers to better identify patients likely to respond are another area of active research. Efficient screening tools utilizing next-generation sequencing can help identify more Stargardt disease cases and enable early intervention.

Market Drivers

The key driver for the Stargardt Disease Therapeutics Market Growth is the lack of approved treatment options. Currently, only supportive measures like nutritional supplements and low-vision devices are available which do not halt disease progression. This huge unmet medical need has led to intense RD efforts on disease-modifying therapies. As gene therapy candidates show promise in clinical trials, the market is expected to grow sharply in the forecast period with potential first launches. Growing diagnosis rates aided by genetic testing and rising awareness will also bolster demand.