Spherix Global Insights

May 01, 2024

Gene Therapies in Degenerative Retinal Disease Hold Potential to Capture Significant Portion of Market

Pending trial results supporting prescribers’ efficacy and safety demands, gene therapies are poised to capture upwards of one-fifth of the AMD, GA and DME markets, according to Spherix Global Insights. 

EXTON, PA., May 1, 2024 — In the landscape of healthcare innovation, retinal disorders, including both inherited and degenerative diseases, have become a leading focus area for gene therapy investigation. With gene therapies representing the forefront of personalized medicine, and retinal disorders at the edge of the frontier, Spherix Global Insights conducted a study with practicing retinal specialists (n=77) to assess the current landscape and what would be required for integrating these pioneering therapies into future practice.   

While development potential and interest for this novel treatment approach is high for more rare inherited conditions such as retinitis pigmentosa and Stargardt’s, Spherix’s study zeroed in on highly prevalent degenerative disorders such as geographic atrophy (GA), age-related macular degeneration (AMD), and diabetic macular edema (DME). These conditions profoundly impact central vision later in life, thus emphasizing the critical need for interventions capable of arresting disease progression and safeguarding visual function. 

Results of the Spherix study indicate the vast majority of retinal specialists agree gene therapy will play a significant role in the future of treating retinal diseases. Moreover, gene therapies were ranked highest in terms of their attractiveness for a treatment of degenerative retinal diseases, outpacing topical eye drops, local injectable treatments (e.g., anti-VEGF), and other ongoing systemic treatments. Indeed, despite only modest levels of familiarity with late-stage clinical investigative gene therapies, there remains a nexus of innovation and opportunity, as retinal specialists project that gene therapies may be able to capture upwards of 20-25% share of patients across the global AMD, GA, and DME markets. Capturing this patient potential will require gene therapy manufacturers to meet specialists’ requisite efficacy benefits and safety demands.   

Retinal specialists highlight that the method of administration for gene therapies may significantly influence interest in one therapy over another. Nearly 90% of physicians concur that when a gene therapy is administered via subretinal injection, specialized training provided by manufacturers will be essential. They anticipate that the absence of such training may present a barrier to adoption.  Other factors that could inhibit or aid in uptake include the efficacy profile, active trial comparators, the safety profile, and established durability of gene therapies.  

The study unveiled a landscape brimming with potential, promising benefits for both physicians and patients alike, as gene therapies are estimated by prescribers to hold the potential for claiming a significant portion of the large global market for retinal disorders. With pioneering companies such as 4-D Molecular Therapeutics, REGENXBIO, Adverum Biotechnologies, Johnson & Johnson, and others leading the charge in gene therapy development, the stage is set for innovation to illuminate the path toward a brighter future for patients worldwide.  

Spherix’s recent gene therapy study answers key business questions regarding the future potential of gene therapies, including indications and endpoints of utmost interest to retinal specialists, their attitudes regarding the application timing and location for gene therapy, and the educational requirements they foresee needing from manufacturers. 

Special Topix™ is an independent service that includes access to a report or series of reports based on current events or topics of interest in specialty markets covered by Spherix. 

About Spherix Global Insights 

Spherix Global Insights is a leading provider of market research, business intelligence and advisory services to the global life sciences industry. The company’s unique service offerings are powered by deep therapeutic knowledge, the Spherix Network specialty physician panel, and commercially relevant analyses to enable strategic decision-making by our valued customers.  

A trusted advisor and industry thought leader, Spherix Global Insights provides specialized market expertise in six (6) focused therapeutic areas including: dermatology, gastroenterology, nephrology, neurology, rheumatology, and ophthalmology.  

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Spherix Global Insights Contacts 

Blaine Cloud, Ophthalmology Franchise Head 

[email protected]  

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