Drug intelligence / Profile preview

GS030-DP + GS030-MD

Development stage
Unknown
Lead developer
GenSight Biologics
Modality
AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
Administration
Intravitreal, Device-based (ocular/visual Stimulation)
01

Overview

GS030-DP + GS030-MD is a combination investigational therapy developed for late-stage **retinitis pigmentosa**. It uses **GS030-DP**, a gene therapy delivered via a single intravitreal injection of an adeno-associated virus (AAV2) vector encoding the light-sensitive protein ChrimsonR-tdT into retinal ganglion cells, making them responsive to light and bypassing degenerated photoreceptors. Restoration of vision then requires **GS030-MD**, a wearable optronic visual stimulation device (light-stimulating goggles) that encodes the visual scene and projects specific wavelengths and intensity of light onto the treated retina to activate the newly light-sensitive cells. This optogenetic approach aims to restore functional vision in advanced retinitis pigmentosa, regardless of the underlying genetic mutation. GS030 has orphan drug designation in the US and Europe and is undergoing evaluation in the multicenter, open-label, dose-escalation PIONEER phase I/II trial[1][2][3][5][6][7].

Brand names
GS030GS-030GS 030
Other names
GS030 combined therapyGS-030 combined therapyGS 030 combined therapy

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