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HUB-101

Development stage: Preclinical
Lead developer: Hubble Therapeutics
Modality: AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
Administration: Subretinal, Intravitreal

Overview

HUB-101 is an adeno-associated virus (AAV)-based gene therapy developed by Hubble Therapeutics. It is designed for the treatment of rare and severe eye disorders, specifically targeting Leber congenital amaurosis type 16 (LCA16) and other retinal disorders. The therapy works through gene transference to deliver a functional copy of the KCNJ13 gene, aiming to restore or replace KCNJ13 protein function in affected retinal cells. This approach addresses genetic defects that cause vision loss in these conditions. HUB-101 has received orphan drug designation for Leber congenital amaurosis and rare pediatric disease designation in the United States[1][2][3][4].

Other names: HUB 101HUB101HUB-101

Targets

KCNJ13 (Kir7.1)

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