J&J’s $130M gene therapy bet flunks rare retinal disease trial
J&J's $130M Gene Therapy Bet Flunks Rare Retinal Disease Trial
Johnson & Johnson's gene therapy treatment for X-linked retinitis pigmentosa (XLRP) failed to meet its primary endpoints in a Phase 3 clinical trial4.
The gene therapy, known as bota-vec, was acquired by J&J in a $130 million deal approximately a year ago2.
The failure was announced by the company on May 5, 20252.
This treatment was specifically targeting a rare genetic disease that leads to progressive vision loss4.
The therapy failed to improve visual navigation in patients with this rare eye disease53.
This failure follows a trend of other major pharmaceutical companies (including Vertex and Pfizer) experiencing setbacks with gene therapy treatments4.
The news comes as Opus Genetics reported more promising results with their OPGX-LCA5 gene therapy, which showed potential to restore meaningful vision in a different rare eye condition, according to 12-month Phase 1/2 results1.
Sources:
1. https://www.biospace.com/press-releases/opus-genetics-announces-presentation-of-opgx-lca5-gene-therapy-data-at-arvo-12-month-phase-1-2-results-support-potential-to-restore-to-meaningful-vision
2. https://firstwordpharma.com/story/5956636
3. https://www.ophthalmologytimes.com/view/j-j-gene-therapy-treatment-fails-primary-endpoints
4. https://endpts.com/jj-eye-disease-gene-therapy-bota-vec-fails-phase-3/