Invest in intelligence that delivers

US Retinal Specialists Highlight the Greatest Opportunity for Gene Therapies

Research conducted by Spherix Global Insights reveals Retinal Specialists likely to adopt gene therapies.

Exton, PA., March 19, 2024 – Gene therapies have emerged as a ray of hope, particularly in the realm of eye diseases. The focus on the eye has provided researchers with a promising pathway for delivering genetic material with precision and safety. Unlike other organs, the eye’s compact size and accessibility have made it an ideal application for gene therapies, offering targeted treatment with minimal systemic impact. 

As advancements continue, a plethora of gene therapies for retinal disorders are on the horizon. From rare hereditary conditions to widespread degenerative diseases, trials from companies like AbbVie-RegenX Bio, Adverum Biotechnologies, 4D-Molecular Technology, Johnson and Johnson, and MieraGTx are now moving into phase 3 clinical trials.  

Spherix Global Insights collaborated with 77 retinal specialists to understand their perspectives on adopting gene therapies, specifically where specialists were and were not willing to use gene therapy across diseases and patient types. The study revealed a clear trend: the greater the unmet need, the stronger the desire for an approved gene therapy. Specialists are particularly eager for treatments for blinding inherited retinal diseases like retinitis pigmentosa and Stargardt disease, where conventional treatments are lacking. However, another pressing concern emerged—the need for solutions in geographic atrophy (GA). 

Unlike other conditions, GA presents a unique challenge. Despite the availability of Apellis’ Syfovre and Iveric/Astellas’ Izervay, specialists expressed dissatisfaction with current therapies, highlighting the vulnerability of these approaches. Interestingly, diseases like neovascular age-related macular degeneration (nAMD) and diabetic retinopathy with and without macular edema (DR/DME), which have long-standing and well-established treatment options, did not evoke as strong a desire for gene therapies among retinal specialists. 

Furthermore, the retinal specialist survey explored their expectations regarding the degree of improvement they would deem necessary in gene therapy clinical trials. Given the current emphasis on maintaining and slowing vision loss in GA management, ophthalmologists are extending these standards to their expectations for gene therapy trial outcomes. The top drivers for gene therapy in GA include maintaining vision and the prevention of disease progression. This contrasts with conditions like nAMD and DR/DME, where vision improvement remains the primary focus, according to existing care standards. 

Overall, the evolving landscape of ocular care and the pivotal role of gene therapies in reshaping treatment paradigms indicate significant shifts afoot in the market. While retinal specialists express eagerness for gene therapies, dissatisfaction towards current treatments like Apellis’ Syfovre and Iveric/Astellas’ Izervay underscores the need for innovation. 

Spherix Global Insights will continue to track market shifts and stay updated on emerging treatments. As new therapies emerge and current treatments expand, Spherix will continue to offer valuable intelligence to improve patient outcomes. 

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. 

To learn more about Spherix Global Insights, visit or connect through LinkedIn. 

For more details on Spherix’s primary market research reports and interactive dashboard offerings, visit or register here: 

Spherix Global Insights Contact  

Blaine Cloud, Ph.D., Ophthalmology Franchise Head 

NOTICE: All company, brand or product names in this press release are trademarks of their respective holders. The findings and opinions expressed within are based on Spherix Global Insight’s analysis and do not imply a relationship with or endorsement. 

Sign up for alerts, market insights and exclusive content in your inbox.

Spherix is excited to announce our expansion into an additional, rapidly evolving specialty market:


Throughout 2024, we will be publishing over
10 groundbreaking studies covering a wide range of indications within classical hematology.