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Beacon’s gene therapy for vision loss condition meets main trial goal

By Thomson Reuters Sep 21, 2026 | 7:05 AM

Sept 21 (Reuters) – Beacon Therapeutics said on Monday its experimental gene therapy improved the ability to read in low-light conditions in ​a late-stage trial on people with ‌a form of inherited eye disease that can cause progressive vision loss.

Here are some details:

• The therapy, laru-zova, was tested as a treatment for X-linked retinitis pigmentosa (XLRP), ‌a ​disease that predominantly affects boys ⁠and young men, beginning ⁠with night blindness and followed by a narrowing of the peripheral field of vision.

• The 12-month trial evaluated 85 male subjects aged ​12 to 48.

• It met its main goal, with a significant proportion of those receiving ⁠the therapy improving their ⁠ability to read at least 15 ​additional letters in low-light conditions, while no participants in ​the untreated control group achieved this improvement.

• ‌XLRP affects about 4 in 100,000 males across the U.S. and Europe, according to National Institutes of Health data. There are currently no ⁠approved treatments for the condition.

• “We are now the only company with a study that is read out positively ⁠on its ‌primary endpoint within a pivotal phase ⁠three study, and so that’s giving ​us ‌a chronologic advantage,” said CEO Lance ​Baldo.

• The ⁠privately held company said it will hold discussions with global regulatory authorities on a marketing application submission based on these trial results.

(Reporting by Christy Santhosh and Kamal Choudhury in Bengaluru; Editing by ​Vijay Kishore)