Drug intelligence / Profile preview

RTx-015

Development stage
Unknown
Lead developer
Ray Therapeutics
Modality
AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
Administration
Intravitreal
01

Overview

RTx-015 is an investigational optogenetic gene therapy developed by Ray Therapeutics for the treatment of inherited retinal degenerative diseases. It utilizes an adeno-associated virus (AAV.7m8) vector to deliver a gene encoding a light-sensitive opsin protein to retinal ganglion cells (RGCs) via intravitreal injection. The goal is to restore visual function in patients with advanced retinitis pigmentosa and potentially other blinding conditions by enabling surviving inner retinal neurons to respond to light after photoreceptor loss. The therapy is currently being evaluated in Phase 1 clinical trials for retinitis pigmentosa and choroideremia[2][3][6][9].

02

Targets

OPN4 (Melanopsin)

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