Astellas has launched a phase 1/2 clinical trial of ASP2767, an investigational gene therapy designed to protect retinal nerve cells in patients with optic neuropathy due to open-angle glaucoma.

The first-in-human study will evaluate the safety, tolerability, and efficacy of ASP2767, which uses a viral vector to deliver genes that cause retinal nerve cells to produce proteins intended to provide neuroprotection. Unlike established glaucoma therapies that primarily target intraocular pressure, ASP2767 is being investigated as a potential approach to slowing or preventing vision loss associated with optic nerve damage.

The study will proceed in 2 phases. In the open-label phase 1 portion, participants will receive a single injection of ASP2767 in 1 eye, with doses increased in successive cohorts to determine the highest dose that can be safely administered. Participants will also receive prophylactic treatment to reduce the risk of inflammation.

Phase 2 will randomly assign participants to the selected phase 1 dose, a lower dose, or a sham-injection group. The second phase will be double-masked and sham-controlled. Investigators will assess safety and visual outcomes.

Participants in both phases will be followed for approximately 1 year after treatment. GP