REGENXBIO Unveils New Data on Diabetic Retinopathy Treatment

Innovative Advances in Diabetic Retinopathy Treatment

REGENXBIO Inc. (NASDAQ: RGNX) recently announced an exciting development regarding its Phase II ALTITUDE trial at a significant ophthalmology event. The company is set to present interim data highlighting the efficacy of surabgene lomparvovec, also known as ABBV-RGX-314, delivered via the suprachoroidal route for treating diabetic retinopathy (DR). This gene therapy, developed in partnership with AbbVie, represents a promising new approach to managing DR and wet age-related macular degeneration (wet AMD).

Presentation Details at the American Academy of Ophthalmology

The presentation, scheduled for a prime spot on October 17 at 4:46 p.m. ET, will feature groundbreaking results from two years of trials involving non-proliferative diabetic retinopathy. Renowned expert Dr. Charles C. Wykoff from Retina Consultants of Texas is set to lead the discussion during the session on late-breaking developments.

Highlighting the Potential of Sura-vec

Sura-vec involves the NAV^® AAV8 vector and is being explored as a one-time treatment option for chronic retinal conditions such as wet AMD and DR. This innovative therapy aims to tackle the underlying issues by counteracting vascular endothelial growth factor (VEGF), a key contributor to the formation of leaky blood vessels that can lead to vision loss.

About REGENXBIO Inc.

Founded in 2009, REGENXBIO has been at the forefront of advancing the field of AAV gene therapy. The company is dedicated to delivering transformative treatments for patients suffering from rare genetic disorders and eye diseases. Its impressive pipeline includes RGX-202 aimed at Duchenne muscular dystrophy, as well as treatments for mucopolysaccharidosis types I and II, both in collaboration with Nippon Shinyaku.

Impact of AAV Technology

REGENXBIO's innovative AAV platform has already provided life-changing therapies to thousands of patients, including those treated with Novartis' ZOLGENSMA^®. This gene therapy platform continues to showcase the potential to revolutionize the management of numerous health conditions by offering one-time treatments that focus on curing rather than managing symptoms.

Future Prospects and Commitments

As REGENXBIO continues to pioneer in gene therapy, the commitment to improving lives remains strong. The company's focus is not just on current projects but also on future innovations in gene therapy that can address unmet medical needs worldwide. Each step forward in research signifies a hope for better treatment options for patients dealing with debilitating health challenges.

Contact Information

If you need further assistance or information, please reach out to:

Dana Cormack, Corporate Communications

George E. MacDougall, Investor Relations

For more information, visit the official REGENXBIO website.

Frequently Asked Questions

What is the purpose of the ALTITUDE trial?

The ALTITUDE trial aims to evaluate the safety and effectiveness of surabgene lomparvovec for treating diabetic retinopathy through suprachoroidal delivery.

Who is presenting the data at the American Academy of Ophthalmology?

Dr. Charles C. Wykoff, a leading retina specialist, will present the interim data from the trial.

What conditions does surabgene lomparvovec target?

This investigational therapy targets wet AMD and diabetic retinopathy, focusing on treating the underlying causes of these conditions.

How does surabgene lomparvovec work?

Surabgene lomparvovec is designed to inhibit VEGF, preventing the growth of abnormal blood vessels and fluid leakage in the retina.

What is the significance of the upcoming presentation?

The presentation will highlight new findings that could shape future treatments for diabetes-related eye conditions, marking an important milestone in ophthalmic gene therapies.

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