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Beacon reports early success in XLRP gene therapy trial

Published 12/06/2024, 07:08 AM

LONDON - Syncona Ltd, a prominent life science investor, announced today that its portfolio company Beacon Therapeutics has shared promising three-month data from the Phase II DAWN trial of laru-zova (AGTC-501) in treating X-linked retinitis pigmentosa (XLRP). The interim findings were presented at the FLORetina-ICOOR Meeting in Florence, Italy, marking a significant milestone for Beacon.

The DAWN study, which focuses on patients previously treated with a different gene therapy, aims to evaluate the safety, efficacy, and tolerability of laru-zova in the untreated eye. The data indicates that laru-zova has been well-tolerated by all participants with no treatment emergent adverse events reported. Additionally, early improvements in low luminance visual acuity (LLVA), a key indicator of visual function, have been observed.

XLRP is a severe and progressive inherited retinal disease that predominantly affects boys and young men, often leading to blindness by middle age. Currently, there are no available treatments for this condition. Laru-zova, which expresses the full-length RPGR protein, is expected to address the complete range of photoreceptor damage caused by XLRP.

Chris Hollowood, CEO of Syncona Investment Management Limited, expressed optimism about the potential of laru-zova based on these interim results, which build on previous positive data from the Phase II SKYLINE trial. The ongoing Phase II/III VISTA trial of laru-zova is a pivotal study for the company, with a data readout anticipated in 2026.

Beacon Therapeutics is also actively enrolling patients for the VISTA trial as it advances laru-zova's development. The company, founded in 2023, focuses on ophthalmic gene therapies for various retinal diseases that can lead to blindness. Supported by Syncona and other investors, Beacon aims to deliver transformational treatments for conditions with high unmet needs.

This update is based on a press release statement and reflects the company's current progress in clinical trials without speculating on future outcomes or broader industry impacts.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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