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BB-201

Development stage: Preclinical
Lead developer: Benitec Biopharma
Modality: Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics, Gene Editing → Gene Therapies, Gene Addition/Replacement → Gene Therapies
Administration: Intravitreal, Subretinal

Overview

BB-201 is a human therapeutic program developed by Benitec Biopharma Limited. It utilizes proprietary DNA-directed RNA interference (ddRNAi) technology for gene silencing, primarily aimed at treating wet age-related macular degeneration.

Targets

PABPN1 (Polyadenylate-binding nuclear protein 1)

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