Drug intelligence / Profile preview

DCV1-0401

Development stage
Unknown
Lead developer
Tokyo Medical Center
Modality
AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies, Gene Silencing → Gene Therapies, Gene Editing → Gene Therapies
Administration
Ophthalmic
01

Overview

DCV1-0401 is an investigational adeno-associated virus (AAV) vector-based gene therapy designed for the treatment of retinitis pigmentosa (RP) associated with mutations in the *EYS* (Eyes Shut Homolog) gene. The *EYS* gene is a critical component for maintaining the structural stability of the photoreceptor outer segments, and its mutation is a leading cause of autosomal recessive RP, particularly in East Asian populations. Due to the large size of the *EYS* cDNA, DCV1-0401 utilizes a specialized approach, such as a minigene or truncated version, to fit within the AAV vector's capacity. Administered via subretinal injection, the therapy aims to restore EYS protein function, thereby preserving photoreceptor cells and slowing the progression of vision loss. The development is led by the National Hospital Organization Tokyo Medical Center with funding and support from the Japan Agency for Medical Research and Development (AMED).

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