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R100

Development stage: Preclinical
Lead developer: 4D Molecular Therapeutics
Modality: AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies, Gene Silencing → Gene Therapies, Gene Editing → Gene Therapies
Administration: Intravitreal

Overview

R100 is a proprietary adeno-associated virus (AAV) vector developed by 4D Molecular Therapeutics using its Therapeutic Vector Evolution platform. It is engineered for intravitreal delivery to the retina, designed to efficiently penetrate the internal limiting membrane barrier and transduce retinal cells. This enables robust expression of therapeutic transgenes for the treatment of various ophthalmic diseases. Astellas Gene Therapies has licensed the R100 vector technology from 4DMT to develop its own genetic payloads for rare monogenic ophthalmic diseases.

Other names: R100 vectorR-100 vectorR 100 vectorintravitreal retinotropic R100 vectorAAV2.R100AAV-2.R100AAV 2.R100

Targets

HSPG (Basement membrane-specific heparan sulfate proteoglycan core protein (perlecan))

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