Drug intelligence / Profile preview

IVB102

Development stage
Unknown
Lead developer
InnoVec Biotherapeutics
Modality
AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies, Gene Silencing → Gene Therapies, Gene Editing → Gene Therapies
Administration
Intravitreal
01

Overview

**IVB102** is an AAV-based gene therapy developed for the treatment of **X-linked retinoschisis (XLRS)**, a rare inherited retinal degenerative disorder primarily affecting young males and caused by mutations in the RS1 gene. IVB102 is designed to deliver a functional RS1 gene to retinal cells to restore normal retinoschisin protein expression and improve retinal integrity and function. It is administered via intravitreal injection and is based on a novel AAV vector platform independently developed by the company. IVB102 has received FDA Rare Pediatric Disease Designation for XLRS and is currently in early-phase (Phase 1) clinical trials[1][2][3][4][6][7][10].

02

Targets

RS1 (Retinoschisin)

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