Syncona firm Beacon reports encouraging gene therapy trial results
By Josh White
Date: Wednesday 07 May 2025
(Sharecast News) - Syncona said in an update on Wednesday that its portfolio company Beacon Therapeutics has reported encouraging six-month interim results from its phase two 'DAWN' trial, marking a key milestone in the development of its gene therapy candidate for X-linked retinitis pigmentosa (XLRP).
The FTSE 250 company said the data, presented at the ARVO 2025 annual meeting, showed that patients treated with Beacon's lead asset, 'laru-zova', or laruparetigene zovaparvovec, experienced meaningful improvements in visual function.
Specifically, patients demonstrated early gains in low luminance visual acuity, with greater two- and three-line improvements in treated eyes compared to untreated fellow eyes after six months.
Sustained improvements in retinal sensitivity, measured by microperimetry, were also observed.
Laru-zova was generally well-tolerated, with ocular side effects that were mostly mild or moderate, related to surgical procedures and steroid use, and had largely resolved by the time of reporting.
No serious unexpected adverse reactions, retinal detachments, or cases of endophthalmitis were observed.
Syncona said the publication of the data represented a capital access milestone for Beacon, underscoring the therapeutic potential of the programme and advancing its path towards potential commercialisation.
"We are really pleased with the progress at Beacon - it is fantastic to see continued visual improvements in patients evaluated at the six-month time point in this data update," said Elisa Petris, Syncona Investment Management partner and board director at Beacon Therapeutics.
"We are optimistic as the company progresses enrollment for its phase two and three pivotal study and look forward to seeing data in this crucial programme.
"We see a really differentiated opportunity with this therapy to transform the lives of patients with a devastating retinal blinding condition."
At 1054 BST, shares in Syncona were flat at 90p.
Reporting by Josh White for Sharecast.com.
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